Trial Outcomes & Findings for Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor (NCT NCT00851721)
NCT ID: NCT00851721
Last Updated: 2021-05-19
Results Overview
Participants were Randomized to Receive 1 of the 2 Following Treatment Regimens: 1.On-Demand: FEIBA NF dose \& dosing interval as prescribed by treating physician 2.Prophylaxis: 85 ± 15 U/kg of FEIBA NF every other day during 12-month prophylactic period Annualized rate of bleeding episodes was calculated as: (Number of bleeding episodes/observed treatment period in days) \* 365.25
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
52 participants
Primary outcome timeframe
12 months ± 14 days
Results posted on
2021-05-19
Participant Flow
Enrollment was conducted in Europe, North America, Asia-Pacific, and South America at 17 clinical sites beginning in March 2009.
52 participants were enrolled. Sixteen participants discontinued, (ten were screen failures and six were withdrawn before randomization (2 sponsor's decision- inhibitors, 3 withdrew consent, and 1 due to investigator decision to have participant on prophylaxis). Therefore 36 participants were randomized.
Participant milestones
| Measure |
On-demand Arm
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : Standard FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Overall Study
STARTED
|
19
|
17
|
|
Overall Study
COMPLETED
|
17
|
16
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
On-demand Arm
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : Standard FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Overall Study
planned surgery
|
1
|
0
|
|
Overall Study
Death
|
1
|
0
|
|
Overall Study
Adverse Event
|
0
|
1
|
Baseline Characteristics
Efficacy and Safety Study of Prophylactic Versus On-Demand Treatment With Feiba NF in Subjects With Hemophilia A or B and a High Titer Inhibitor
Baseline characteristics by cohort
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : Standard FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
Total
n=36 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
29.1 years
STANDARD_DEVIATION 15.2 • n=5 Participants
|
25.6 years
STANDARD_DEVIATION 15.4 • n=7 Participants
|
27.4 years
STANDARD_DEVIATION 15.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
36 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
Brazil
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
Ukraine
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Region of Enrollment
Romania
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
Croatia
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
Russian Federation
|
8 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Region of Enrollment
Bulgaria
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
Japan
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Region of Enrollment
New Zealand
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 months ± 14 daysPopulation: Efficacy Intent to Treat Analysis Dataset: Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.
Participants were Randomized to Receive 1 of the 2 Following Treatment Regimens: 1.On-Demand: FEIBA NF dose \& dosing interval as prescribed by treating physician 2.Prophylaxis: 85 ± 15 U/kg of FEIBA NF every other day during 12-month prophylactic period Annualized rate of bleeding episodes was calculated as: (Number of bleeding episodes/observed treatment period in days) \* 365.25
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Reduction in Annualized Bleeding Episode Rate (ABR) Among Participants Receiving Prophylactic Treatment as Compared to Those Treated On-demand
|
28.7 bleeds/year
Inter-Quartile Range 1.8 • Interval 17.7 to 50.0
|
7.9 bleeds/year
Inter-Quartile Range 1.8 • Interval 2.9 to 11.0
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Efficacy Intent to Treat Analysis Dataset: Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.
Spontaneous includes unknown/undermined etiology
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
Spontaneous
|
18.9 Bleeds per year
Interval 10.9 to 43.5
|
5.6 Bleeds per year
Interval 2.9 to 8.0
|
|
Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
Traumatic
|
4.7 Bleeds per year
Interval 1.9 to 10.7
|
2.5 Bleeds per year
Interval 0.0 to 3.1
|
|
Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
Joint
|
22.9 Bleeds per year
Interval 14.1 to 46.9
|
6.0 Bleeds per year
Interval 2.9 to 10.0
|
|
Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
Non-Joint
|
2.9 Bleeds per year
Interval 1.0 to 4.9
|
0.5 Bleeds per year
Interval 0.0 to 2.0
|
|
Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
Spontaneous Joint
|
16.6 Bleeds per year
Interval 9.9 to 40.8
|
4.5 Bleeds per year
Interval 2.9 to 8.0
|
|
Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
Spontaneous Non-Joint
|
1.0 Bleeds per year
Interval 1.0 to 2.9
|
0.0 Bleeds per year
Interval 0.0 to 1.0
|
|
Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
Traumatic Joint
|
4.0 Bleeds per year
Interval 1.0 to 7.1
|
1.0 Bleeds per year
Interval 0.0 to 3.1
|
|
Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
Traumatic Non-Joint
|
0.0 Bleeds per year
Interval 0.0 to 1.9
|
0.0 Bleeds per year
Interval 0.0 to 1.0
|
|
Annualized Bleeding Rate by Treatment Regimen, Bleeding Etiology, and Bleed Type
All Bleeding Etiologies, and Bleed Types
|
28.7 Bleeds per year
Interval 17.7 to 50.0
|
7.9 Bleeds per year
Interval 2.9 to 11.0
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Efficacy Intent to Treat Analysis Dataset
Annualized bleed rates were transformed using the square root of the number of bleeding episodes observed (X bleeds/year), X' = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using the t-test. The difference in mean transformed ABRs was used to perform statistical tests and generate p-values at a significance level of 5% Participants were Randomized to Receive 1 of the 2 Following Treatment Regimens: 1.On-Demand: FEIBA NF dose \& dosing interval as prescribed by treating physician 2.Prophylaxis: 85 ± 15 U/kg of FEIBA NF every other day during 12-month prophylactic period
Outcome measures
| Measure |
On-demand Arm
n=36 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type
Spontaneous Bleeds
|
2.2 (bleeds/year)^(1/2)
Standard Deviation 1.8
|
—
|
|
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type
Traumatic Bleeds
|
1.0 (bleeds/year)^(1/2)
Standard Deviation 1.2
|
—
|
|
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type
Joint Bleeds
|
2.4 (bleeds/year)^(1/2)
Standard Deviation 1.9
|
—
|
|
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type
Non-Joint Bleeds
|
0.8 (bleeds/year)^(1/2)
Standard Deviation 0.9
|
—
|
|
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type
Spontaneous Joint Bleeds
|
2.1 (bleeds/year)^(1/2)
Standard Deviation 1.8
|
—
|
|
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type
Spontaneous Non-Joint Bleeds
|
0.8 (bleeds/year)^(1/2)
Standard Deviation 0.8
|
—
|
|
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type
Traumatic Joint Bleeds
|
0.9 (bleeds/year)^(1/2)
Standard Deviation 1.2
|
—
|
|
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens by Bleeding Etiology, and Bleeding Type
Traumatic Non-Joint Bleeds
|
-0.0 (bleeds/year)^(1/2)
Standard Deviation 0.9
|
—
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Efficacy Intent to Treat Analysis Dataset: Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.
Target joints are ≥4 bleeds/6 months in any one of the following joints: ankles, knees, elbows, and hips; a target joint bleeding episode refers to an individual anatomical location.
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Annualized Bleeding Rate for New Target Joints
|
5.9 Bleeds per year
Interval 0.0 to 12.9
|
0.0 Bleeds per year
Interval 0.0 to 4.1
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Efficacy Intent to Treat Analysis Dataset: Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.
Annualized bleed rates (ABRs) were transformed using the square root of the number of bleeding episodes observed (X bleeds/year), X' = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using a two-sample, two-sided t-test. The difference in mean transformed ABRs was used to perform statistical tests and generate p-values at a significance level of 5%
Outcome measures
| Measure |
On-demand Arm
n=36 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Differences in Mean Transformed Annualized Bleeding Rate Between On-Demand and Prophylaxis Treatment Regimens: New Target Joints
|
1.6 (bleeds/year)^(1/2)
Standard Deviation 2.2
|
—
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Efficacy Intent to Treat Analysis Dataset: Dataset consists of data from all randomized participants. Dataset includes those who discontinued FEIBA NF but did not withdraw informed consent and were willing to continue providing data. For these participants, only the data collected before FEIBA NF discontinuation is included.
Target Joints are defined as ≥4 bleeds/6 months in any one of the following joints: ankles, knees, elbows and hips
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Number of New Target Joints
|
23 new target joints
|
7 new target joints
|
SECONDARY outcome
Timeframe: Throughout the study period, 12 months ± 14 daysPopulation: Additional evaluations for bleeding episodes in the intent-to-treat analysis dataset: consists of all participants with at least 1 bleeding episode treated with investigational product.
Pain caused by a bleeding episode in adolescents and adults (≥12 years old) was measured at pre-infusion (pre-inf) and at 6 ± 0.5 hours (h) and 24 ± 1 h post-infusion (post-inf) (after the last infusion given to treat a bleeding episode) on the VAS pain scale in millimeters from 0 (no pain) to 100 (worst possible pain). For analysis purposes, if short acting analgesics (duration of activity approximately 6 ± 0.5 h) were used, pain was assigned the highest possible score (100). Pain assessment occurred after each infusion related to single bleeding episodes. In case participants required an additional infusion within 24h, pain was assessed 6 ± 0.5 h and 24 ±1 h following the subsequent infusion. Change in VAS scores at 6 ± 0.5 h and 24 ±1 h post-infusion were also compared relative to pre-infusion VAS scores (ie, (pre-infusion VAS score) - (post-infusion VAS score)).
Outcome measures
| Measure |
On-demand Arm
n=522 Bleeding episodes
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=158 Bleeding episodes
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old)
Pre-Infusion (N= 513, 130)
|
29.0 Scores on a scale
Inter-Quartile Range 43.0 • Interval 12.0 to 55.0
|
49.5 Scores on a scale
Inter-Quartile Range 46.9 • Interval 34.7 to 81.6
|
|
Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old)
6 ± 0.5 hours post-infusion (N= 522, 158)
|
10.0 Scores on a scale
Inter-Quartile Range 33.0 • Interval 4.0 to 37.0
|
33.8 Scores on a scale
Inter-Quartile Range 44.6 • Interval 8.0 to 52.6
|
|
Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old)
24 ± 1 hours post-infusion (N= 512,131)
|
3.0 Scores on a scale
Inter-Quartile Range 13.0 • Interval 1.0 to 14.0
|
6.2 Scores on a scale
Inter-Quartile Range 19.0 • Interval 1.0 to 20.0
|
|
Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old)
Change (Pre-Inf to 6h post-inf) (N= 509, 129)
|
10.0 Scores on a scale
Interval 4.0 to 21.8
|
19.4 Scores on a scale
Interval 5.0 to 41.6
|
|
Assessment of Objective Clinical Symptoms- Visual Analog Scale (VAS): Pain in Adolescents and Adults (≥12 Years Old)
Change (Pre-Inf to 24h post-inf) (N= 489, 102)
|
18.0 Scores on a scale
Interval 8.0 to 39.6
|
37.8 Scores on a scale
Interval 17.0 to 75.5
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Additional evaluations for bleeding episodes in the intent-to-treat analysis dataset: consists of all participants with at least 1 bleeding episode treated with investigational product.
Pain caused by a bleeding episode (BE) in pediatric participants (\<12 years old) was measured at pre-infusion (pre-inf) and at 6 ± 0.5 h and 24 ± 1 h post-infusion (post-inf) (after the last infusion given to treat a bleeding episode) using the children's VAS pain scale (a facial expression scale with one end marked as no pain and the opposite end marked as the worst possible pain). For analysis purposes, if short acting analgesics (duration of activity approximately 6 ± 0.5 h) were used, pain was assigned the highest possible score (worst possible pain). Scores on the children's VAS scale are presented as: -No Pain -Mild Pain -Moderate pain -Severe pain -Very severe pain
Outcome measures
| Measure |
On-demand Arm
n=77 Bleeding episodes (BEs)
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=9 Bleeding episodes (BEs)
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
Pre-Infusion (N= 54, 8) - Mild Pain
|
3 Bleeding episodes
|
1 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
Pre-Infusion (N= 54, 8) - No Pain
|
27 Bleeding episodes
|
3 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
Pre-Infusion (N= 54, 8) - Moderate Pain
|
12 Bleeding episodes
|
3 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
Pre-Infusion (N= 54, 8) - Severe Pain
|
10 Bleeding episodes
|
1 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
Pre-Infusion (N= 54, 8) - Very Severe Pain
|
2 Bleeding episodes
|
0 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
6 ± 0.5 h post-infusion (N= 74, 8) - No Pain
|
47 Bleeding episodes
|
3 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
± 0.5 h post-infusion (N= 74, 8) - Mild Pain
|
9 Bleeding episodes
|
3 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
± 0.5 h post-infusion (N= 74, 8) - Moderate Pain
|
15 Bleeding episodes
|
2 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
± 0.5 h post-infusion (N= 74, 8) - Severe Pain
|
3 Bleeding episodes
|
0 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
± 0.5 h post-infusion (N= 74, 8)- Very Severe Pain
|
0 Bleeding episodes
|
0 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
24 ± 1 h post-infusion (N= 77, 9) - No Pain
|
60 Bleeding episodes
|
7 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
24 ± 1 h post-infusion (N= 77, 9) - Mild Pain
|
11 Bleeding episodes
|
1 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
24 ± 1 h post-infusion (N= 77, 9) - Moderate Pain
|
6 Bleeding episodes
|
1 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
24 ± 1 h post-infusion (N= 77, 9) - Severe Pain
|
0 Bleeding episodes
|
0 Bleeding episodes
|
|
Assessment of Clinical Symptoms - Visual Analog Scale (VAS): Pain in Pediatrics (<12 Years Old)
24± 1h post-infusion (N = 77, 9) -Very Severe Pain
|
0 Bleeding episodes
|
0 Bleeding episodes
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Safety Analysis Set
ROM was measured using a goniometer for 3 key joints (ie, ankles, knees, and elbows) at screening, month 6, and termination (end of study visit)
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Flexion: Termination (n = 17,16)
|
110.0 degrees
Interval 90.0 to 140.0
|
125.0 degrees
Interval 75.0 to 145.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Pronation: Screening (n = 18,16)
|
64.0 degrees
Interval 55.0 to 90.0
|
55.0 degrees
Interval 22.5 to 77.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Supination: Termination (n= 18,16)
|
20.0 degrees
Interval 10.0 to 30.0
|
20.0 degrees
Interval 10.0 to 30.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Extension: Screening
|
15.0 degrees
Interval 0.0 to 45.0
|
10.0 degrees
Interval 0.0 to 25.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Extension: Month 6 (n = 18,16)
|
10.0 degrees
Interval 0.0 to 41.0
|
8.0 degrees
Interval 0.0 to 42.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Extension: Termination (n = 17,16)
|
20.0 degrees
Interval 0.0 to 41.0
|
15.5 degrees
Interval 0.0 to 40.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Flexion: Screening (n = 19,17)
|
125.0 degrees
Interval 90.0 to 140.0
|
115.0 degrees
Interval 80.0 to 140.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Flexion: Month 6 (n = 18,16)
|
130.0 degrees
Interval 90.0 to 140.0
|
125.0 degrees
Interval 72.5 to 143.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Pronation: Month 6 (n = 17,14)
|
80.0 degrees
Interval 60.0 to 90.0
|
67.5 degrees
Interval 35.0 to 80.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Pronation: Termination (n = 17,15)
|
68.0 degrees
Interval 60.0 to 90.0
|
65.0 degrees
Interval 30.0 to 80.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Supination: Screening (n = 18,16)
|
75.0 degrees
Interval 50.0 to 90.0
|
42.5 degrees
Interval 17.5 to 77.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Supination: Month 6 (n = 17,14)
|
80.0 degrees
Interval 50.0 to 90.0
|
60.0 degrees
Interval 35.0 to 80.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Elbow - Supination: Termination (n = 17,15)
|
70.0 degrees
Interval 50.0 to 90.0
|
70.0 degrees
Interval 35.0 to 80.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Extension: Screening (n = 19,17)
|
20.0 degrees
Interval 0.0 to 50.0
|
10.0 degrees
Interval 0.0 to 50.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Extension: Month 6 (n = 18,16)
|
17.5 degrees
Interval 0.0 to 50.0
|
10.0 degrees
Interval 0.0 to 42.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Extension: Termination (n = 18,16)
|
22.5 degrees
Interval 0.0 to 45.0
|
20.0 degrees
Interval 0.0 to 62.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Flexion: Screening (n = 19,17)
|
120.0 degrees
Interval 105.0 to 140.0
|
100.0 degrees
Interval 80.0 to 130.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Flexion: Month 6 (n = 18,16)
|
122.5 degrees
Interval 110.0 to 140.0
|
100.0 degrees
Interval 85.0 to 136.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Flexion: Termination (n = 18,16)
|
121.0 degrees
Interval 110.0 to 140.0
|
99.0 degrees
Interval 70.0 to 127.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Pronation: Screening (n = 18,16)
|
75.0 degrees
Interval 55.0 to 90.0
|
57.5 degrees
Interval 27.5 to 80.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Pronation: Month 6 (n = 17,14)
|
80.0 degrees
Interval 55.0 to 90.0
|
72.5 degrees
Interval 40.0 to 80.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Pronation: Termination (n = 18,15)
|
67.5 degrees
Interval 55.0 to 90.0
|
70.0 degrees
Interval 40.0 to 80.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Supination: Screening (n = 18,16)
|
75.0 degrees
Interval 45.0 to 90.0
|
52.5 degrees
Interval 20.0 to 80.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Supination: Month 6 (n = 17,14)
|
70.0 degrees
Interval 45.0 to 90.0
|
72.5 degrees
Interval 30.0 to 80.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Elbow - Supination: Termination (n = 18,15)
|
75.0 degrees
Interval 45.0 to 90.0
|
70.0 degrees
Interval 30.0 to 80.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Knee - Extension: Screening (n = 18,16)
|
15.0 degrees
Interval 5.0 to 90.0
|
1.5 degrees
Interval 0.0 to 55.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Knee - Extension: Month 6 (n = 17,14)
|
15.0 degrees
Interval 5.0 to 70.0
|
0.0 degrees
Interval 0.0 to 20.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Knee - Extension: Termination (n = 18,15)
|
15.0 degrees
Interval 5.0 to 90.0
|
0.0 degrees
Interval 0.0 to 70.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Knee - Flexion: Screening (n = 19,17)
|
100.0 degrees
Interval 80.0 to 130.0
|
90.0 degrees
Interval 65.0 to 115.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Knee - Flexion: Month 6 (n = 18,16)
|
112.5 degrees
Interval 80.0 to 130.0
|
97.5 degrees
Interval 65.0 to 125.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Knee - Flexion: Termination (n = 18,16)
|
105.0 degrees
Interval 60.0 to 130.0
|
92.5 degrees
Interval 70.0 to 125.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Knee - Extension: Screening (n = 18,16)
|
35.0 degrees
Interval 10.0 to 90.0
|
5.0 degrees
Interval 0.0 to 30.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Knee - Extension: Month 6 (n = 17,14)
|
20.0 degrees
Interval 10.0 to 90.0
|
10.0 degrees
Interval 2.0 to 30.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Knee - Extension: Termination (n = 18,15)
|
17.5 degrees
Interval 10.0 to 90.0
|
5.0 degrees
Interval 0.0 to 30.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Knee - Flexion: Screening (n = 19,17)
|
90.0 degrees
Interval 20.0 to 122.0
|
80.0 degrees
Interval 60.0 to 125.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Knee - Flexion: Month 6 (n = 18,16)
|
90.0 degrees
Interval 20.0 to 130.0
|
82.5 degrees
Interval 56.0 to 127.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Knee - Flexion: Termination (n = 18,16)
|
85.0 degrees
Interval 20.0 to 120.0
|
87.5 degrees
Interval 56.0 to 127.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Dorsiflexion: Screening (n = 18,16)
|
20.0 degrees
Interval 15.0 to 30.0
|
17.5 degrees
Interval 9.0 to 37.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Dorsiflexion: Month 6 (n = 17,14)
|
20.0 degrees
Interval 11.0 to 35.0
|
17.5 degrees
Interval 8.0 to 50.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Dorsiflexion: Termination (n = 17,15)
|
15.0 degrees
Interval 11.0 to 25.0
|
20.0 degrees
Interval 5.0 to 50.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Plantarflexion: Screening (n = 18,16)
|
34.5 degrees
Interval 30.0 to 40.0
|
30.0 degrees
Interval 10.0 to 35.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Plantarflexion: Month 6 (n = 17,14)
|
34.0 degrees
Interval 25.0 to 35.0
|
30.0 degrees
Interval 15.0 to 40.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Plantarflexion: Termination (n=17,15)
|
35.0 degrees
Interval 30.0 to 40.0
|
30.0 degrees
Interval 15.0 to 35.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Pronation: Screening (n = 19,17)
|
14.0 degrees
Interval 10.0 to 20.0
|
15.0 degrees
Interval 10.0 to 15.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Pronation: Month 6 (n = 18,15)
|
15.0 degrees
Interval 10.0 to 20.0
|
15.0 degrees
Interval 10.0 to 16.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Pronation: Termination (n = 18,16)
|
15.0 degrees
Interval 10.0 to 20.0
|
12.5 degrees
Interval 10.0 to 15.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Supination: Screening (n = 19,17)
|
25.0 degrees
Interval 10.0 to 30.0
|
15.0 degrees
Interval 10.0 to 27.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Supination: Month 6 (n = 18,15)
|
25.0 degrees
Interval 20.0 to 30.0
|
25.0 degrees
Interval 10.0 to 30.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Left Ankle - Supination: Termination (n = 18,16)
|
25.0 degrees
Interval 16.0 to 30.0
|
20.0 degrees
Interval 10.0 to 27.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Dorsiflexion: Screening (n = 18,16)
|
15.0 degrees
Interval 10.0 to 20.0
|
20.0 degrees
Interval 7.5 to 35.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Dorsiflexion: Month 6 (n = 17,14)
|
15.0 degrees
Interval 15.0 to 20.0
|
25.0 degrees
Interval 6.0 to 40.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Dorsiflexion: Termination (n= 17,15)
|
15.0 degrees
Interval 14.0 to 20.0
|
20.0 degrees
Interval 10.0 to 40.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Plantarflexion: Screening (n= 18,16)
|
35.0 degrees
Interval 30.0 to 40.0
|
20.0 degrees
Interval 9.0 to 39.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Plantarflexion: Month 6 (n= 17,14)
|
30.0 degrees
Interval 25.0 to 40.0
|
23.0 degrees
Interval 10.0 to 40.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Plantarflexion: Termination(n=17,15)
|
30.0 degrees
Interval 25.0 to 40.0
|
21.0 degrees
Interval 10.0 to 40.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Pronation: Screening (n = 19,17)
|
10.0 degrees
Interval 10.0 to 15.0
|
10.0 degrees
Interval 10.0 to 20.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Pronation: Month 6 (n= 18,15)
|
10.0 degrees
Interval 10.0 to 20.0
|
10.0 degrees
Interval 10.0 to 20.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Pronation: Termination (n= 18,16)
|
10.0 degrees
Interval 10.0 to 17.0
|
11.0 degrees
Interval 10.0 to 17.5
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Supination: Screening (n = 19,17)
|
17.0 degrees
Interval 10.0 to 30.0
|
20.0 degrees
Interval 10.0 to 30.0
|
|
Assessment of Clinical Symptoms - Range of Motion (ROM)
Right Ankle - Supination: Month 6 (n= 18,15)
|
20.0 degrees
Interval 10.0 to 30.0
|
20.0 degrees
Interval 10.0 to 30.0
|
SECONDARY outcome
Timeframe: 6 h ± 30 min post-infusionPopulation: Bleeding Episodes Analysis Set Consists of all participants who experienced a bleeding episode (BE)
Number of rAHF-PFM-treated bleeding episodes with an assessment of hemostasis (4-point ordinal scale): Excellent: Full pain relief \& bleeding cessation within \~6 hours of 1 infusion. Additional infusions may have been given to maintain hemostasis; Good: Definite pain relief and/or improvement in bleeding within \~6 hours after infusion. Possibly requires \>1 infusion for complete resolution; Fair: Probable or slight relief of pain \& slight improvement in bleeding within \~6 hours after infusion. Requires \>1 infusion for complete resolution; None: No improvement or condition worsens
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=14 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours
Rating Not Done (n = 3, 2)
|
3 bleeding episodes
|
2 bleeding episodes
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours
Not Available (n = 0, 1)
|
0 bleeding episodes
|
2 bleeding episodes
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours
Excellent (n = 16, 5)
|
109 bleeding episodes
|
16 bleeding episodes
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours
Good (n = 19, 14)
|
355 bleeding episodes
|
106 bleeding episodes
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours
Fair (n = 15, 6)
|
142 bleeding episodes
|
42 bleeding episodes
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 6 Hours
None (n = 6, 2)
|
14 bleeding episodes
|
5 bleeding episodes
|
SECONDARY outcome
Timeframe: 24 ± 1 h post-infusionPopulation: Bleeding Episodes Analysis Set Consists of all participants who experienced a bleeding episode (BE)
Number of rAHF-PFM-treated bleeding episodes with an assessment of hemostasis (4-point ordinal scale): Excellent: Full pain relief \& bleeding cessation within \~24 hours of 1 infusion. Additional infusions may have been given to maintain hemostasis; Good: Definite pain relief and/or improvement in bleeding within \~24 hours after infusion. Possibly requires \>1 infusion for complete resolution; Fair: Probable or slight relief of pain \& slight improvement in bleeding within \~24 hours after infusion. Requires \>1 infusion for complete resolution; None: No improvement or condition worsens
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=14 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours
Excellent (n = 16, 6)
|
282 bleeding episodes
|
42 bleeding episodes
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours
Good (n = 19, 14)
|
280 bleeding episodes
|
89 bleeding episodes
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours
Fair (n = 9, 3)
|
36 bleeding episodes
|
12 bleeding episodes
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours
None (n = 1,0)
|
1 bleeding episodes
|
0 bleeding episodes
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours
Rating Not Done (n = 8, 3)
|
24 bleeding episodes
|
28 bleeding episodes
|
|
Assessment of Hemostasis for Treatment of Bleeding Episodes- Overall Efficacy Rating at 24 Hours
Not Available (n = 0, 1)
|
0 bleeding episodes
|
2 bleeding episodes
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Additional Evaluations for Bleeding Episodes Analysis Set - Consists of all participants with at least 1 bleeding episode treated with investigational product, FEIBA NF.
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=14 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Total Weight Adjusted Dose to Control a Bleeding Episode
|
4049.7 Units/kg
Interval 2324.5 to 7408.4
|
1524.9 Units/kg
Interval 293.2 to 2883.5
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Additional Evaluations for Bleeding Episodes Analysis Set - Consists of all participants with at least 1 bleeding episode treated with investigational product, FEIBA NF.
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=14 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding
1 infusion
|
352 Bleeding Episodes (BEs)
|
98 Bleeding Episodes (BEs)
|
|
The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding
2 infusions
|
134 Bleeding Episodes (BEs)
|
41 Bleeding Episodes (BEs)
|
|
The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding
3 infusions
|
62 Bleeding Episodes (BEs)
|
13 Bleeding Episodes (BEs)
|
|
The Number of Bleeding Episode (BE) Which Required 1, 2, 3, or ≥4 Infusions to Control Bleeding
≥4 infusions
|
75 Bleeding Episodes (BEs)
|
21 Bleeding Episodes (BEs)
|
SECONDARY outcome
Timeframe: Screening visit, Month 3, Month 6, Month 9, and Termination visitPopulation: Safety Analysis Set Participants with Clinically Significant Laboratory Results
The normal reference range of values for aPTT is 22.8 - 31 seconds.
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results
Screening visit (n= 19, 16)
|
67.3 seconds
Interval 63.1 to 75.1
|
68.5 seconds
Interval 61.0 to 73.1
|
|
Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results
Month 3 (n= 14, 15)
|
68.0 seconds
Interval 62.7 to 76.2
|
62.7 seconds
Interval 57.5 to 67.2
|
|
Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results
Month 6 (n= 17, 15)
|
68.2 seconds
Interval 64.6 to 72.1
|
65.6 seconds
Interval 62.6 to 69.5
|
|
Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results
Month 9 (n= 17, 15)
|
70.0 seconds
Interval 64.6 to 77.9
|
68.1 seconds
Interval 58.1 to 73.9
|
|
Abnormal Activated Partial Thromboplastin Time (aPTT) Assay Results
Termination visit (n= 18, 16)
|
69.8 seconds
Interval 63.4 to 75.6
|
69.4 seconds
Interval 60.5 to 77.1
|
SECONDARY outcome
Timeframe: Screening visit, Month 3, Month 6, Month 9, and Termination visitPopulation: Safety Analysis Set Participants with Clinically Significant Laboratory Results
The normal reference range of values for D-dimers is \<500 ng/mL.
Outcome measures
| Measure |
On-demand Arm
n=9 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=9 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Abnormal D-Dimer Assay Results
Screening visit (n= 7, 6)
|
862.0 ng/mL
Interval 647.0 to 1035.0
|
833.5 ng/mL
Interval 644.0 to 1164.0
|
|
Abnormal D-Dimer Assay Results
Month 3 (n= 7, 8)
|
690.0 ng/mL
Interval 561.0 to 830.0
|
1021.5 ng/mL
Interval 730.0 to 1633.5
|
|
Abnormal D-Dimer Assay Results
Month 6 (n= 9, 8)
|
969.0 ng/mL
Interval 753.0 to 1103.0
|
1032.0 ng/mL
Interval 652.0 to 1442.5
|
|
Abnormal D-Dimer Assay Results
Month 9 (n= 6, 8)
|
731.5 ng/mL
Interval 592.0 to 1398.0
|
1121.0 ng/mL
Interval 942.5 to 1448.5
|
|
Abnormal D-Dimer Assay Results
Termination visit (n= 5, 9)
|
813.0 ng/mL
Interval 695.0 to 1386.0
|
1031.0 ng/mL
Interval 872.0 to 1258.0
|
SECONDARY outcome
Timeframe: Screening visit, Month 3, Month 6, Month 9, and Termination visitPopulation: Safety Analysis Set Participants with Clinically Significant Laboratory Results
The normal reference range of values for fibrinogen is 200-400 mg/dL.
Outcome measures
| Measure |
On-demand Arm
n=4 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=6 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Abnormal Fibrinogen Assay Results
Month 6 (n= 3, 1)
|
415.0 mg/dL
Interval 412.0 to 418.0
|
759.0 mg/dL
Interval 759.0 to 759.0
|
|
Abnormal Fibrinogen Assay Results
Month 9 (n= 1, 4)
|
508.0 mg/dL
Interval 508.0 to 508.0
|
326.5 mg/dL
Interval 183.5 to 635.5
|
|
Abnormal Fibrinogen Assay Results
Screening visit (n= 4, 6)
|
428.0 mg/dL
Interval 414.0 to 478.5
|
422.0 mg/dL
Interval 411.0 to 428.0
|
|
Abnormal Fibrinogen Assay Results
Month 3 (n= 0, 2)
|
NA mg/dL
There were no abnormal fibrinogen assay results for this arm/group at this time point
|
402.0 mg/dL
Interval 199.0 to 605.0
|
|
Abnormal Fibrinogen Assay Results
Termination visit (n= 2, 2)
|
456.5 mg/dL
Interval 425.0 to 488.0
|
522.5 mg/dL
Interval 414.0 to 631.0
|
SECONDARY outcome
Timeframe: Screening visit, Month 3, Month 6, Month 9, and Termination visitPopulation: Safety Analysis Set Participants with Clinically Significant Laboratory Results
The normal reference range of values for FDP is 0-5 ug/mL.
Outcome measures
| Measure |
On-demand Arm
n=3 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=2 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Abnormal Fibrin Degradation Products (FDP) Assay Results
Screening visit (n= 1, 2)
|
16.0 ug/mL
Interval 16.0 to 16.0
|
12.0 ug/mL
Interval 8.0 to 16.0
|
|
Abnormal Fibrin Degradation Products (FDP) Assay Results
Month 3 (n= 0, 2)
|
NA ug/mL
There were no abnormal fibrin degradation products assay results for this arm/group at this time point
|
8.0 ug/mL
Interval 8.0 to 8.0
|
|
Abnormal Fibrin Degradation Products (FDP) Assay Results
Month 6 (n= 1, 1)
|
8.0 ug/mL
Interval 8.0 to 8.0
|
8.0 ug/mL
Interval 8.0 to 8.0
|
|
Abnormal Fibrin Degradation Products (FDP) Assay Results
Month 9 (n= 1, 2)
|
8.0 ug/mL
Interval 8.0 to 8.0
|
8.0 ug/mL
Interval 8.0 to 8.0
|
|
Abnormal Fibrin Degradation Products (FDP) Assay Results
Termination visit (n= 3, 1)
|
8.0 ug/mL
Interval 8.0 to 8.0
|
8.0 ug/mL
Interval 8.0 to 8.0
|
SECONDARY outcome
Timeframe: Screening visit, Month 3, Month 6, Month 9, and Termination visitPopulation: Safety Analysis Set Participants with Clinically Significant Laboratory Results
The normal reference range of values for prothrombin fragment F 1.2 is 69-229 pmol/L.
Outcome measures
| Measure |
On-demand Arm
n=6 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=9 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Abnormal Prothrombin Fragment F 1.2 Assay Results
Screening visit (n= 2, 1)
|
284.0 pmol/L
Interval 278.0 to 290.0
|
308.0 pmol/L
Interval 308.0 to 308.0
|
|
Abnormal Prothrombin Fragment F 1.2 Assay Results
Month 3 (n= 6, 7)
|
336.5 pmol/L
Interval 267.0 to 414.0
|
579.0 pmol/L
Interval 354.0 to 972.0
|
|
Abnormal Prothrombin Fragment F 1.2 Assay Results
Month 6 (n= 3, 9)
|
448.0 pmol/L
Interval 272.0 to 714.0
|
430.0 pmol/L
Interval 366.0 to 504.0
|
|
Abnormal Prothrombin Fragment F 1.2 Assay Results
Month 9 (n= 4, 9)
|
382.0 pmol/L
Interval 313.0 to 405.5
|
415.0 pmol/L
Interval 272.0 to 472.0
|
|
Abnormal Prothrombin Fragment F 1.2 Assay Results
Termination visit (n= 4, 7)
|
252.5 pmol/L
Interval 250.0 to 265.0
|
643.0 pmol/L
Interval 376.0 to 1679.0
|
SECONDARY outcome
Timeframe: Screening visit, Month 3, Month 6, Month 9, and Termination visitPopulation: Safety Analysis Set Participants with Clinically Significant Laboratory Results
The normal reference range of values for PT is 9.7-12.3 sec.
Outcome measures
| Measure |
On-demand Arm
n=1 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=1 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Abnormal Prothrombin Time Assay Results
Termination visit (n= 0, 1)
|
NA seconds
There were no abnormal prothrombin time assay results for this arm/group at this time point
|
13.4 seconds
Interval 13.4 to 13.4
|
|
Abnormal Prothrombin Time Assay Results
Screening visit (n= 1, 1)
|
9.4 seconds
Interval 9.4 to 9.4
|
9.6 seconds
Interval 9.6 to 9.6
|
|
Abnormal Prothrombin Time Assay Results
Month 3 (n= 1, 0)
|
9.5 seconds
Interval 9.5 to 9.5
|
NA seconds
There were no abnormal prothrombin time assay results for this arm/group at this time point
|
|
Abnormal Prothrombin Time Assay Results
Month 6 (n= 0, 1)
|
NA seconds
There were no abnormal prothrombin time assay results for this arm/group at this time point
|
12.5 seconds
Interval 12.5 to 12.5
|
|
Abnormal Prothrombin Time Assay Results
Month 9 (n= 1, 1)
|
12.7 seconds
Interval 12.7 to 12.7
|
13.6 seconds
Interval 13.6 to 13.6
|
SECONDARY outcome
Timeframe: Screening visit, Month 3, Month 6, Month 9, and Termination visitPopulation: Safety Analysis Set Participants with Clinically Significant Laboratory Results
The normal reference range of values for TAT is 1-4.1 ug/L.
Outcome measures
| Measure |
On-demand Arm
n=4 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=7 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Abnormal Thrombin-Antithrombin III (TAT) Assay Results
Screening visit (n= 4, 1)
|
5.1 ug/L
Interval 4.4 to 5.9
|
5.6 ug/L
Interval 5.6 to 5.6
|
|
Abnormal Thrombin-Antithrombin III (TAT) Assay Results
Month 3 (n= 3, 7)
|
4.7 ug/L
Interval 4.5 to 5.9
|
12.3 ug/L
Interval 4.4 to 40.8
|
|
Abnormal Thrombin-Antithrombin III (TAT) Assay Results
Month 6 (n= 3, 6)
|
7.9 ug/L
Interval 6.3 to 10.5
|
5.6 ug/L
Interval 4.4 to 6.1
|
|
Abnormal Thrombin-Antithrombin III (TAT) Assay Results
Month 9 (n= 0, 3)
|
NA ug/L
There were no abnormal Thrombin-Antithrombin III assay results for this arm/group at this time point
|
6.0 ug/L
Interval 5.6 to 52.9
|
|
Abnormal Thrombin-Antithrombin III (TAT) Assay Results
Termination visit (n= 2, 5)
|
10.3 ug/L
Interval 8.0 to 12.5
|
5.6 ug/L
Interval 5.1 to 7.3
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Safety Analysis Set
-Hepatitis A Virus Antibody (HAV Ab) -Hepatitis B Virus Core Antibody (HBcAb) -Hepatitis B Virus Surface Antibody (HBsAb) -Hepatitis B Virus Surface Antigen (HBsAg) -Hepatitis C Virus (HCV)
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBcAb: Serology not available
|
1 participants
|
1 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBsAb: Screening Negative; Termination Positive
|
2 participants
|
5 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HCV: Serology not available
|
1 participants
|
1 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HAV Ab: Screening Negative; Termination Positive
|
1 participants
|
0 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HAV Ab: Screening Negative; Termination Negative
|
8 participants
|
11 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HAV Ab: Screening Positive; Termination Positive
|
8 participants
|
4 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HAV Ab: Screening Positive; Termination Negative
|
1 participants
|
1 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HAV Ab: Serology not available
|
1 participants
|
1 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBcAb: Screening Negative; Termination Positive
|
0 participants
|
1 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBcAb: Screening Negative; Termination Negative
|
8 participants
|
11 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBcAb: Screening Positive; Termination Positive
|
9 participants
|
4 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBcAb: Screening Positive; Termination Negative
|
1 participants
|
0 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBsAb: Screening Negative; Termination Negative
|
2 participants
|
2 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBsAb: Screening Positive; Termination Positive
|
13 participants
|
9 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBsAb: Screening Positive; Termination Negative
|
1 participants
|
0 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBsAb: Serology not available
|
1 participants
|
1 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBsAg: Screening Negative; Termination Positive
|
0 participants
|
0 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBsAg: Screening Negative; Termination Negative
|
18 participants
|
16 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HBsAg: Serology not available
|
1 participants
|
1 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HCV: Screening Negative; Termination Positive
|
0 participants
|
0 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HCV: Screening Negative; Termination Negative
|
6 participants
|
10 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HCV: Screening Positive; Termination Positive
|
12 participants
|
6 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Hepatitis A, Hepatitis B, and Hepatitis C
HCV: Screening Positive; Termination Negative
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Safety Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab)
HIV 1/2 Ab: Screen Negative; Termination Positive
|
0 participants
|
0 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab)
HIV 1/2 Ab: Screen Negative; Termination Negative
|
18 participants
|
16 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: HIV-1/2 Antibody (Ab)
HIV 1/2 Ab: : Serology not available
|
1 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Safety Analysis Set
Normal range (0 - 0.89 IV); High (\> 0.89 IV) - Parvovirus B19 IgG Antibody \[IV\] (Parvo IgG Ab)
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV]
Parvo IgG Ab: Screening High ; Termination Normal
|
3 participants
|
1 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV]
Parvo IgG Ab: Screening High ; Termination High
|
14 participants
|
13 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV]
Parvo IgG Ab: Screening Normal; Termination Normal
|
0 participants
|
2 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV]
Parvo IgG Ab: Screening Normal ; Termination High
|
1 participants
|
0 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgG Antibody [IV]
Parvo IgG Ab: Serology not available
|
1 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Safety Analysis Set
Normal range (0 - 0.89 IV); High (\> 0.89 IV) - Parvovirus B19 IgM Antibody \[IV\] (Parvo IgM Ab)
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV]
Parvo IgM Ab: Screening High ; Termination Normal
|
0 participants
|
0 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV]
Parvo IgM Ab: Screening High ; Termination High
|
1 participants
|
0 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV]
Parvo IgM Ab: Screening Normal; Termination Normal
|
17 participants
|
16 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV]
Parvo IgM Ab: Screening Normal ; Termination High
|
0 participants
|
0 participants
|
|
Viral Serology From Screening Visit and Study Termination Visit: Parvovirus B19 IgM Antibody [IV]
Parvo IgM Ab: Serology not available
|
1 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Safety Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Rate of Related Adverse Events (AEs) Per Year
|
0.000 Related AEs per year
Interval 0.0 to 0.0
|
0.000 Related AEs per year
Interval 0.0 to 0.979
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Safety Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Rate of Related Adverse Events (AEs) During or Within 1 Hour of Infusion Per Year
|
0.000 Related AEs within/during 1hr per year
Interval 0.0 to 0.0
|
0.000 Related AEs within/during 1hr per year
Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Safety Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=19 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Number of Related Thromboembolic Adverse Events (AEs)
|
0 Related thromboembolic AEs
|
0 Related thromboembolic AEs
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Safety Analysis Set - Hemophilia A study participants
Absolute Changes in Inhibitor Titer (or no change in low or high titer status): -Inhibitor Titer went from Low (≤5 BU) to Low (≤5 BU) -Inhibitor Titer went from Low (≤5 BU) to High (\>5 BU) -Inhibitor Titer went from High (\>5 BU) to Low (≤5 BU) -Inhibitor Titer went from High (\>5 BU) to High (\>5 BU)
Outcome measures
| Measure |
On-demand Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels
Screening to 6 Month- Low to High (N=2, 1)
|
12.9 Bethesda Units (BU)
Interval 4.9 to 20.8
|
5.3 Bethesda Units (BU)
Interval 5.3 to 5.3
|
|
Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels
Screening to 3 Month- Low to High (N=2, 3)
|
12.1 Bethesda Units (BU)
Interval 12.1 to 12.1
|
4.0 Bethesda Units (BU)
Interval 0.7 to 16.8
|
|
Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels
Screening to 3 Month- High to Low (N=1, 0)
|
2.0 Bethesda Units (BU)
Interval 2.0 to 2.0
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels
Screening to 6 Month- High to Low (N=1, 0)
|
3.4 Bethesda Units (BU)
Interval 3.4 to 3.4
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels
Screening to 9 Month- Low to High (N=2, 1)
|
5.0 Bethesda Units (BU)
Interval 4.3 to 5.6
|
3.7 Bethesda Units (BU)
Interval 3.7 to 3.7
|
|
Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels
Screening to 9 Month- High to Low (N=0, 2)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
2.1 Bethesda Units (BU)
Interval 0.9 to 3.2
|
|
Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels
Screening to Termination- Low to High (N=2, 1)
|
39.2 Bethesda Units (BU)
Interval 3.8 to 74.7
|
5.8 Bethesda Units (BU)
Interval 5.8 to 5.8
|
|
Absolute Changes in Inhibitor Titer of Hemophilia A Participants With Shifts in Factor VIII (FVIII) Inhibitor Titer Levels
Screening to Termination- High to Low (N=1, 2)
|
14.6 Bethesda Units (BU)
Interval 14.6 to 14.6
|
3.3 Bethesda Units (BU)
Interval 3.3 to 3.3
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Safety Analysis Set - Hemophilia B study participants
Absolute Changes in Inhibitor Titer (or no change in low or high titer status): -Inhibitor Titer went from Low (≤5 BU) to Low (≤5 BU) -Inhibitor Titer went from Low (≤5 BU) to High (\>5 BU) -Inhibitor Titer went from High (\>5 BU) to Low (≤5 BU) -Inhibitor Titer went from High (\>5 BU) to High (\>5 BU)
Outcome measures
| Measure |
On-demand Arm
n=1 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=1 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 3 Month- Low to Low (N=1, 0)
|
3.2 Bethesda Units (BU)
Interval 3.2 to 3.2
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 3 Month- Low to High (N=0, 0)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 3 Month- High to Low (N=0, 0)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 3 Month- High to High (N=1, 1)
|
0.3 Bethesda Units (BU)
Interval 0.3 to 0.3
|
10.7 Bethesda Units (BU)
Interval 10.7 to 10.7
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 6 Month- Low to Low (N=1, 0)
|
1.5 Bethesda Units (BU)
Interval 1.5 to 1.5
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 6 Month- Low to High (N=0, 0)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 6 Month- High to Low (N=0, 0)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 6 Month- High to High (N=1, 1)
|
1.7 Bethesda Units (BU)
Interval 1.7 to 1.7
|
23.2 Bethesda Units (BU)
Interval 23.2 to 23.2
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 9 Month- Low to Low (N=1, 0)
|
3.2 Bethesda Units (BU)
Interval 3.2 to 3.2
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 9 Month- Low to High (N=0, 0)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 9 Month- High to Low (N=0, 0)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to 9 Month- High to High (N=0, 1)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
37.0 Bethesda Units (BU)
Interval 37.0 to 37.0
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to Termination - Low to Low (N=0, 0)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to Termination- Low to High (N=0, 0)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to Termination- High to Low (N=0, 0)
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
NA Bethesda Units (BU)
There were no inhibitor titer shifts for this arm/group at this time point
|
|
Absolute Changes in Inhibitor Titer of Hemophilia B Participants With Shifts in Factor IX (FIX) Inhibitor Titer Levels
Screening to Termination - High to High (N=1, 1)
|
11.3 Bethesda Units (BU)
Interval 11.3 to 11.3
|
21.4 Bethesda Units (BU)
Interval 21.4 to 21.4
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Intent to Treat Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Pharmacoeconomics: Annual Days Lost Due to Bleeding (Work or School)
|
16.4 days
Standard Deviation 25.76 • Interval 0.0 to 20.0
|
8.8 days
Standard Deviation 14.42 • Interval 0.0 to 10.5
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Pharmacoeconomic Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=18 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Pharmacoeconomics: Annual Number of Hospitalizations for Bleeding
|
0.6 hospitalizations
Standard Deviation 1.15 • Interval 0.0 to 1.0
|
0.6 hospitalizations
Standard Deviation 2.03 • Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Pharmacoeconomic Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=18 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Pharmacoeconomics: Annual Number of Hospitalizations for Indwelling Line
|
0.1 hospitalizations
Standard Deviation 0.32 • Interval 0.0 to 0.0
|
0.1 hospitalizations
Standard Deviation 0.25 • Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Pharmacoeconomic Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=18 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Pharmacoeconomics: Annual Number of Emergency Room Visits
|
0.4 Emergency room visits
Standard Deviation 0.98 • Interval 0.0 to 0.0
|
0.4 Emergency room visits
Standard Deviation 1.09 • Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Pharmacoeconomic Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=18 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Pharmacoeconomics: Annual Number of Physician's Office Visits
|
2.2 Physician's office visits
Standard Deviation 4.15 • Interval 0.0 to 3.0
|
2.6 Physician's office visits
Standard Deviation 4.60 • Interval 0.0 to 4.0
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Pharmacoeconomic Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=18 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Pharmacoeconomics: Annual Total Length of Hospitalization for Bleeding
|
5.0 Days
Standard Deviation 9.48 • Interval 0.0 to 9.0
|
5.3 Days
Standard Deviation 17.00 • Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Pharmacoeconomic Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=18 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Pharmacoeconomics: Annual Total Length of Hospitalization for Indwelling Line
|
0.7 Days
Standard Deviation 2.61
|
0.7 Days
Standard Deviation 2.75
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Pharmacoeconomic Analysis Set
Outcome measures
| Measure |
On-demand Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Pharmacoeconomics: Annual Total Number of Days Lost (Work or School)
|
17.4 Days
Standard Deviation 25.42
|
15.4 Days
Standard Deviation 24.28
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: HRQoL Intent-to-Treat Analysis Dataset - comprised of all participants ≥14 years of age who were randomized, had any available assessments at any available study visits (baseline, 6- month, and 12-month) as defined in the protocol
EQ-5D is a participant answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The EQ-5D total score ranges from 0 (worst health state) to 1 (perfect health state) and 1 reflects the best outcome. EQ-5D Index scores based on EQ-5D questionnaire were calculated for participants ≥14 years of age, at screening, 6 months, and at termination visit. Changes in scores at 6 months and termination were also calculated. A relatively higher score represents better quality of life.
Outcome measures
| Measure |
On-demand Arm
n=16 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=12 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores
Screening (N= 16, 12)
|
0.627 Scores on a scale
Standard Deviation 0.2067
|
0.620 Scores on a scale
Standard Deviation 0.1841
|
|
Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores
6 Months (N= 15, 11)
|
0.621 Scores on a scale
Standard Deviation 0.1880
|
0.729 Scores on a scale
Standard Deviation 0.1392
|
|
Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores
Termination (N= 15, 10)
|
0.605 Scores on a scale
Standard Deviation 0.2146
|
0.700 Scores on a scale
Standard Deviation 0.1233
|
|
Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores
Change (Screening - Month 6) (N= 15, 11)
|
-0.006 Scores on a scale
Standard Deviation 0.2088
|
-0.096 Scores on a scale
Standard Deviation 0.2403
|
|
Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores
Change (Screening - Termination) (N= 15, 10)
|
0.010 Scores on a scale
Standard Deviation 0.2470
|
-0.075 Scores on a scale
Standard Deviation 0.2594
|
|
Health-Related Quality of Life (HRQoL): EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Index Scores
Change (Month 6 - Termination) (N= 15, 10)
|
0.016 Scores on a scale
Standard Deviation 0.1765
|
0.001 Scores on a scale
Standard Deviation 0.1493
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset for all participants ≥ 16 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
The Haem-A-QoL instrument has been developed and used in Hemophilia A patients. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports \& Leisure (S\&L), School \& Work (W\&S), Dealing with Hemophilia (Dealing), Family Planning (FP), Feeling, Relationships (R'ships), Treatment, View, and Outlook for the Future (Future). A Haem-A-QoL Total Score (Total) was also calculated. For the Haem-A-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. Haem-A-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated.
Outcome measures
| Measure |
On-demand Arm
n=15 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=12 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Dealing- Change(Screening - Termination)(N=14, 11)
|
10.1 Scores on a scale
Standard Deviation 27.19
|
-1.4 Scores on a scale
Standard Deviation 21.37
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
FP- Screening (N=9, 8)
|
16.7 Scores on a scale
Standard Deviation 20.49
|
34.9 Scores on a scale
Standard Deviation 31.31
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Feeling- Month 6 (N=14, 11)
|
33.9 Scores on a scale
Standard Deviation 28.77
|
29.0 Scores on a scale
Standard Deviation 17.29
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Future- Screening (N=14, 12)
|
46.8 Scores on a scale
Standard Deviation 28.46
|
48.6 Scores on a scale
Standard Deviation 23.32
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Future- Change(Screening - Termination)(N=13, 11)
|
-3.8 Scores on a scale
Standard Deviation 20.53
|
0.3 Scores on a scale
Standard Deviation 16.58
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
PH- Screening (N=15, 12)
|
64.0 Scores on a scale
Standard Deviation 22.46
|
66.4 Scores on a scale
Standard Deviation 24.21
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Treatment- Month 6 (N=14, 11)
|
30.4 Scores on a scale
Standard Deviation 19.82
|
40.9 Scores on a scale
Standard Deviation 16.96
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Treatment- Change (Screening - Month 6) (N=14, 11)
|
6.5 Scores on a scale
Standard Deviation 23.72
|
0.0 Scores on a scale
Standard Deviation 16.74
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Treatment-Change(Screening -Termination)(N=14, 11)
|
2.1 Scores on a scale
Standard Deviation 17.81
|
-1.7 Scores on a scale
Standard Deviation 23.86
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
View- Termination (N=14, 11)
|
47.9 Scores on a scale
Standard Deviation 22.59
|
37.7 Scores on a scale
Standard Deviation 21.26
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
W&S-Screening (N=13, 10)
|
43.8 Scores on a scale
Standard Deviation 25.90
|
57.7 Scores on a scale
Standard Deviation 17.87
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
W&S- Month 6 (N=13, 9)
|
31.7 Scores on a scale
Standard Deviation 26.82
|
37.5 Scores on a scale
Standard Deviation 20.73
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
W&S- Change (Month 6- Termination)(N=12, 9)
|
5.2 Scores on a scale
Standard Deviation 15.95
|
0.7 Scores on a scale
Standard Deviation 21.75
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Future- Termination (N=14, 11)
|
47.9 Scores on a scale
Standard Deviation 25.47
|
46.8 Scores on a scale
Standard Deviation 18.74
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Future- Change (Screening - Month 6) (N=13, 11)
|
4.0 Scores on a scale
Standard Deviation 26.84
|
1.1 Scores on a scale
Standard Deviation 24.34
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Future- Change (Month 6 - Termination)(N=14, 11)
|
-7.7 Scores on a scale
Standard Deviation 20.25
|
-0.8 Scores on a scale
Standard Deviation 16.30
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
PH- Month 6 (N=14, 11)
|
50.8 Scores on a scale
Standard Deviation 27.55
|
43.1 Scores on a scale
Standard Deviation 25.24
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
PH- Termination (N=14, 11)
|
56.1 Scores on a scale
Standard Deviation 24.98
|
42.3 Scores on a scale
Standard Deviation 18.86
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
PH- Change (Screening - Month 6) (N=14, 11)
|
14.6 Scores on a scale
Standard Deviation 21.70
|
21.1 Scores on a scale
Standard Deviation 28.77
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
PH- Change(Screening - Termination)(N=14, 11)
|
9.3 Scores on a scale
Standard Deviation 23.93
|
21.9 Scores on a scale
Standard Deviation 24.79
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
PH- Change (Month 6 - Termination)(N=14, 11)
|
-5.3 Scores on a scale
Standard Deviation 10.65
|
0.8 Scores on a scale
Standard Deviation 20.20
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
R'ships- Screening (N=15, 11)
|
28.3 Scores on a scale
Standard Deviation 36.57
|
26.9 Scores on a scale
Standard Deviation 28.83
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
R'ships- Month 6 (N=14, 11)
|
19.0 Scores on a scale
Standard Deviation 28.20
|
15.2 Scores on a scale
Standard Deviation 13.85
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
R'ships- Termination (N=14, 10)
|
17.9 Scores on a scale
Standard Deviation 22.13
|
15.0 Scores on a scale
Standard Deviation 17.48
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
R'ships- Change (Screening - Month 6) (N=14, 10)
|
7.7 Scores on a scale
Standard Deviation 34.04
|
10.4 Scores on a scale
Standard Deviation 27.45
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
R'ships- Change(Screening - Termination)(N=14, 10)
|
8.9 Scores on a scale
Standard Deviation 30.57
|
12.1 Scores on a scale
Standard Deviation 25.49
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
R'ships- Change (Month 6 - Termination)(N=14, 10)
|
1.2 Scores on a scale
Standard Deviation 21.40
|
1.7 Scores on a scale
Standard Deviation 16.57
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Dealing- Screening (N=15, 12)
|
35.0 Scores on a scale
Standard Deviation 19.72
|
26.0 Scores on a scale
Standard Deviation 15.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Dealing- Month 6 (N=14, 11)
|
25.0 Scores on a scale
Standard Deviation 16.67
|
18.9 Scores on a scale
Standard Deviation 21.11
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Dealing- Termination (N=14, 11)
|
25.6 Scores on a scale
Standard Deviation 25.21
|
23.7 Scores on a scale
Standard Deviation 22.15
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
S&L- Screening (N=12, 8)
|
69.3 Scores on a scale
Standard Deviation 17.11
|
83.1 Scores on a scale
Standard Deviation 14.13
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Dealing- Change (Screening - Month 6) (N=14, 11)
|
10.7 Scores on a scale
Standard Deviation 18.61
|
3.4 Scores on a scale
Standard Deviation 22.62
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
S&L- Month 6 (N=12, 8)
|
63.1 Scores on a scale
Standard Deviation 23.09
|
69.7 Scores on a scale
Standard Deviation 19.75
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Dealing- Change (Month 6 - Termination)(N=14, 11)
|
-0.6 Scores on a scale
Standard Deviation 16.81
|
-4.8 Scores on a scale
Standard Deviation 21.26
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
FP- Month 6 (N=12, 7)
|
20.5 Scores on a scale
Standard Deviation 26.71
|
14.3 Scores on a scale
Standard Deviation 11.25
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
FP- Termination (N=13, 8)
|
19.1 Scores on a scale
Standard Deviation 16.25
|
21.1 Scores on a scale
Standard Deviation 22.14
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
S&L- Termination (N=12, 8)
|
72.3 Scores on a scale
Standard Deviation 17.30
|
78.1 Scores on a scale
Standard Deviation 12.52
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
FP- Change (Screening - Month 6) (N=9, 7)
|
-3.7 Scores on a scale
Standard Deviation 26.94
|
14.9 Scores on a scale
Standard Deviation 28.73
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
FP- Change(Screening - Termination)(N=9, 7)
|
-2.5 Scores on a scale
Standard Deviation 25.57
|
13.1 Scores on a scale
Standard Deviation 20.26
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
S&L- Change (Screening - Month 6) (N=9, 5)
|
9.9 Scores on a scale
Standard Deviation 19.75
|
20.5 Scores on a scale
Standard Deviation 27.97
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
S&L- Change(Screening - Termination)(N=9, 5)
|
1.2 Scores on a scale
Standard Deviation 15.91
|
7.0 Scores on a scale
Standard Deviation 21.68
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
FP- Change (Month 6 - Termination)(N=11, 6)
|
-0.8 Scores on a scale
Standard Deviation 32.68
|
-8.3 Scores on a scale
Standard Deviation 28.41
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Feeling- Screening (N=15, 12)
|
39.6 Scores on a scale
Standard Deviation 28.22
|
48.2 Scores on a scale
Standard Deviation 29.02
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Feeling- Termination (N=14, 11)
|
30.8 Scores on a scale
Standard Deviation 28.53
|
26.1 Scores on a scale
Standard Deviation 20.69
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
S&L- Change (Month 6 - Termination)(N=11, 7)
|
-10.2 Scores on a scale
Standard Deviation 13.44
|
-2.5 Scores on a scale
Standard Deviation 15.61
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Feeling- Change (Screening - Month 6) (N=14, 11)
|
4.9 Scores on a scale
Standard Deviation 27.75
|
17.3 Scores on a scale
Standard Deviation 34.72
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Feeling- Change(Screening - Termination)(N=14, 11)
|
8.0 Scores on a scale
Standard Deviation 31.91
|
20.2 Scores on a scale
Standard Deviation 24.58
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Feeling- Change (Month 6 - Termination)(N=14, 11)
|
3.1 Scores on a scale
Standard Deviation 16.40
|
2.8 Scores on a scale
Standard Deviation 23.94
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Treatment- Screening (N=15, 12)
|
36.9 Scores on a scale
Standard Deviation 13.32
|
39.6 Scores on a scale
Standard Deviation 20.70
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Future- Month 6 (N=14, 11)
|
40.2 Scores on a scale
Standard Deviation 26.14
|
46.0 Scores on a scale
Standard Deviation 18.03
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Treatment- Termination (N=14, 11)
|
34.8 Scores on a scale
Standard Deviation 14.87
|
42.6 Scores on a scale
Standard Deviation 17.80
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Treatment- Change (Month 6- Termination)(N=14, 11)
|
-4.4 Scores on a scale
Standard Deviation 15.07
|
-1.7 Scores on a scale
Standard Deviation 18.70
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
View- Screening (N=15, 12)
|
52.3 Scores on a scale
Standard Deviation 24.49
|
55.6 Scores on a scale
Standard Deviation 11.97
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
View- Month 6 (N=14, 11)
|
44.3 Scores on a scale
Standard Deviation 27.66
|
40.0 Scores on a scale
Standard Deviation 13.23
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
View- Change (Screening - Month 6) (N=14, 11)
|
7.5 Scores on a scale
Standard Deviation 23.68
|
14.3 Scores on a scale
Standard Deviation 17.25
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
View- Change (Screening -Termination)(N=14, 11)
|
3.9 Scores on a scale
Standard Deviation 23.79
|
16.6 Scores on a scale
Standard Deviation 15.01
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
View- Change (Month 6- Termination)(N=14, 11)
|
-3.6 Scores on a scale
Standard Deviation 17.03
|
2.3 Scores on a scale
Standard Deviation 20.78
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
W&S- Termination (N=12, 9)
|
28.6 Scores on a scale
Standard Deviation 22.53
|
36.8 Scores on a scale
Standard Deviation 19.63
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
W&S- Change (Screening - Month 6) (N=12, 8)
|
10.9 Scores on a scale
Standard Deviation 30.98
|
25.3 Scores on a scale
Standard Deviation 25.60
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
W&S- Change (Screening -Termination)(N=12, 8)
|
16.1 Scores on a scale
Standard Deviation 33.86
|
25.3 Scores on a scale
Standard Deviation 19.01
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Total- Screening (N=14, 12)
|
44.0 Scores on a scale
Standard Deviation 15.50
|
49.2 Scores on a scale
Standard Deviation 15.43
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Total- Month 6 (N=14, 11)
|
37.4 Scores on a scale
Standard Deviation 19.37
|
38.1 Scores on a scale
Standard Deviation 11.34
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Total- Termination (N=14, 11)
|
40.2 Scores on a scale
Standard Deviation 17.55
|
38.7 Scores on a scale
Standard Deviation 14.21
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Total- Change (Screening - Month 6) (N=13, 11)
|
9.2 Scores on a scale
Standard Deviation 18.44
|
10.2 Scores on a scale
Standard Deviation 17.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Total- Change (Screening -Termination)(N=13, 11)
|
6.1 Scores on a scale
Standard Deviation 15.41
|
9.5 Scores on a scale
Standard Deviation 12.77
|
|
Hemophilia-specific Quality of Life Questionnaire for Adults (Haem-A-QoL) ≥ 16 Years Old
Total- Change (Month 6- Termination)(N=14, 11)
|
-2.7 Scores on a scale
Standard Deviation 9.21
|
-0.7 Scores on a scale
Standard Deviation 14.40
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - for all participants \<16 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports \& School (S\&S), Dealing with Hemophilia (Dealing), Family, Feeling, Relationships (R'ships), Treatment, View, Outlook for the Future (Future), Friends, Others, and Support. A Haemo-QoL Total Score (Total) was also calculated. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. Haemo-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated.
Outcome measures
| Measure |
On-demand Arm
n=4 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=5 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Dealing- Change(Screening - Termination)(N=4, 4)
|
4.5 Scores on a scale
Standard Deviation 7.92
|
13.4 Scores on a scale
Standard Deviation 5.36
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Family- Month 6 (N=4, 5)
|
50.3 Scores on a scale
Standard Deviation 27.65
|
49.1 Scores on a scale
Standard Deviation 10.27
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Family- Termination (N=4, 5)
|
45.8 Scores on a scale
Standard Deviation 31.03
|
39.8 Scores on a scale
Standard Deviation 13.77
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Feeling- Termination (N=4, 5)
|
33.6 Scores on a scale
Standard Deviation 36.07
|
27.8 Scores on a scale
Standard Deviation 16.97
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Friends- Screening (N=4, 5)
|
35.9 Scores on a scale
Standard Deviation 20.65
|
52.1 Scores on a scale
Standard Deviation 17.18
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Future- Month 6 (N=2, 2)
|
40.6 Scores on a scale
Standard Deviation 13.26
|
37.5 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Others- Change (Month 6 - Termination)(N=4, 5)
|
14.6 Scores on a scale
Standard Deviation 19.98
|
-1.7 Scores on a scale
Standard Deviation 10.87
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
PH- Change (Month 6 - Termination)(N=4, 5)
|
12.5 Scores on a scale
Standard Deviation 22.87
|
7.7 Scores on a scale
Standard Deviation 22.25
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Support- Change (Screening -Termination)(N=4, 4
|
-4.7 Scores on a scale
Standard Deviation 13.86
|
3.1 Scores on a scale
Standard Deviation 15.73
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Support- Change (Month 6- Termination)(N=4, 4)
|
-14.1 Scores on a scale
Standard Deviation 21.27
|
-6.3 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Treatment- Screening (N=4, 5)
|
29.0 Scores on a scale
Standard Deviation 13.23
|
64.2 Scores on a scale
Standard Deviation 22.60
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
View-Month 6 (N=4, 5)
|
46.3 Scores on a scale
Standard Deviation 24.45
|
36.4 Scores on a scale
Standard Deviation 22.04
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Total- Change (Month 6- Termination)(N=4, 5)
|
7.4 Scores on a scale
Standard Deviation 8.91
|
2.8 Scores on a scale
Standard Deviation 7.06
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Dealing- Screening (N=4, 4)
|
25.9 Scores on a scale
Standard Deviation 11.80
|
39.3 Scores on a scale
Standard Deviation 17.00
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Dealing- Month 6 (N=4, 4)
|
25.6 Scores on a scale
Standard Deviation 19.31
|
36.6 Scores on a scale
Standard Deviation 16.07
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Dealing- Termination (N=4, 5)
|
21.4 Scores on a scale
Standard Deviation 16.24
|
28.6 Scores on a scale
Standard Deviation 13.36
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Dealing- Change (Screening - Month 6) (N=4, 4)
|
0.3 Scores on a scale
Standard Deviation 8.61
|
2.7 Scores on a scale
Standard Deviation 7.92
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Dealing- Change (Month 6 - Termination)(N=4, 4)
|
4.2 Scores on a scale
Standard Deviation 11.85
|
10.7 Scores on a scale
Standard Deviation 7.72
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Family- Screening (N=4, 5)
|
55.3 Scores on a scale
Standard Deviation 19.05
|
50.9 Scores on a scale
Standard Deviation 8.72
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Family- Change (Screening - Month 6) (N=4, 5)
|
5.0 Scores on a scale
Standard Deviation 9.26
|
1.7 Scores on a scale
Standard Deviation 15.65
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Family- Change(Screening - Termination)(N=4, 5)
|
9.5 Scores on a scale
Standard Deviation 15.52
|
11.1 Scores on a scale
Standard Deviation 19.22
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Family- Change (Month 6 - Termination)(N=4, 5)
|
4.5 Scores on a scale
Standard Deviation 12.16
|
9.3 Scores on a scale
Standard Deviation 5.24
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Feeling- Screening (N=4, 5)
|
44.1 Scores on a scale
Standard Deviation 14.25
|
38.2 Scores on a scale
Standard Deviation 2.77
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Feeling- Month 6 (N=4, 5)
|
50.3 Scores on a scale
Standard Deviation 27.02
|
37.5 Scores on a scale
Standard Deviation 22.21
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Feeling- Change (Screening - Month 6) (N=4, 5)
|
-6.3 Scores on a scale
Standard Deviation 24.27
|
0.7 Scores on a scale
Standard Deviation 24.02
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Feeling- Change(Screening - Termination)(N=4, 5)
|
10.5 Scores on a scale
Standard Deviation 27.92
|
10.5 Scores on a scale
Standard Deviation 19.45
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Feeling- Change (Month 6 - Termination)(N=4, 5)
|
16.7 Scores on a scale
Standard Deviation 13.85
|
9.7 Scores on a scale
Standard Deviation 20.64
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Friends- Month 6 (N=4, 5)
|
26.6 Scores on a scale
Standard Deviation 17.95
|
36.3 Scores on a scale
Standard Deviation 29.78
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Friends- Termination (N=4, 5)
|
34.4 Scores on a scale
Standard Deviation 14.88
|
66.3 Scores on a scale
Standard Deviation 20.06
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Friends- Change (Screening - Month 6) (N=4, 5)
|
9.4 Scores on a scale
Standard Deviation 27.72
|
15.8 Scores on a scale
Standard Deviation 21.88
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Friends- Change(Screening - Termination)(N=4, 5)
|
1.6 Scores on a scale
Standard Deviation 17.95
|
-14.2 Scores on a scale
Standard Deviation 28.31
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Friends- Change (Month 6 - Termination)(N=4, 5)
|
-7.8 Scores on a scale
Standard Deviation 22.46
|
-30.0 Scores on a scale
Standard Deviation 31.68
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Future- Screening (N=2, 2)
|
53.1 Scores on a scale
Standard Deviation 13.26
|
43.8 Scores on a scale
Standard Deviation 17.68
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Future- Termination (N=2, 2)
|
31.3 Scores on a scale
Standard Deviation 0.00
|
43.8 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Future- Change (Screening - Month 6) (N=2, 2)
|
12.5 Scores on a scale
Standard Deviation 0.00
|
6.3 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Future- Change(Screening - Termination)(N=2, 2)
|
21.9 Scores on a scale
Standard Deviation 13.26
|
0.0 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Future- Change (Month 6 - Termination)(N=2, 2)
|
9.4 Scores on a scale
Standard Deviation 13.26
|
-6.3 Scores on a scale
Standard Deviation 0.00
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Others- Screening (N=4, 5)
|
27.1 Scores on a scale
Standard Deviation 34.44
|
29.2 Scores on a scale
Standard Deviation 16.67
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Others- Month 6 (N=4, 5)
|
42.7 Scores on a scale
Standard Deviation 39.14
|
24.2 Scores on a scale
Standard Deviation 15.70
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Others- Termination (N=4, 5)
|
28.1 Scores on a scale
Standard Deviation 32.87
|
25.8 Scores on a scale
Standard Deviation 13.63
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Others- Change (Screening - Month 6) (N=4, 5)
|
-15.6 Scores on a scale
Standard Deviation 16.80
|
5.0 Scores on a scale
Standard Deviation 20.28
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Others- Change(Screening - Termination)(N=4, 5)
|
-1.0 Scores on a scale
Standard Deviation 15.36
|
3.3 Scores on a scale
Standard Deviation 15.98
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
PH- Screening (N=4, 5)
|
58.0 Scores on a scale
Standard Deviation 11.06
|
52.1 Scores on a scale
Standard Deviation 23.23
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
PH- Month 6 (N=4, 5)
|
53.6 Scores on a scale
Standard Deviation 13.98
|
48.0 Scores on a scale
Standard Deviation 17.23
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
PH- Termination (N=4, 5)
|
41.1 Scores on a scale
Standard Deviation 11.85
|
40.4 Scores on a scale
Standard Deviation 17.02
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
PH- Change (Screening - Month 6) (N=4, 5)
|
4.5 Scores on a scale
Standard Deviation 12.16
|
4.1 Scores on a scale
Standard Deviation 32.71
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
PH- Change(Screening - Termination)(N=4, 5)
|
17.0 Scores on a scale
Standard Deviation 17.10
|
11.8 Scores on a scale
Standard Deviation 29.60
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
R'ships- Screening (N=2, 2)
|
12.5 Scores on a scale
Standard Deviation 17.68
|
43.8 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
R'ships- Month 6 (N=2, 2)
|
25.0 Scores on a scale
Standard Deviation 35.36
|
43.8 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
R'ships- Termination (N=2, 2)
|
0.0 Scores on a scale
Standard Deviation 0.00
|
37.5 Scores on a scale
Standard Deviation 17.68
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
R'ships- Change (Screening - Month 6) (N=2, 2)
|
-12.5 Scores on a scale
Standard Deviation 17.68
|
0.0 Scores on a scale
Standard Deviation 0.00
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
R'ships-Change(Screening -Termination)(N=2, 2)
|
12.5 Scores on a scale
Standard Deviation 17.68
|
6.3 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
R'ships- Change (Month 6- Termination)(N=2, 2)
|
25.0 Scores on a scale
Standard Deviation 35.36
|
6.3 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
S&S- Screening (N=4, 5)
|
67.1 Scores on a scale
Standard Deviation 10.68
|
59.1 Scores on a scale
Standard Deviation 23.73
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
S&S- Month 6 (N=4, 5)
|
62.5 Scores on a scale
Standard Deviation 7.74
|
56.5 Scores on a scale
Standard Deviation 9.07
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
S&S- Termination (N=4, 5)
|
55.6 Scores on a scale
Standard Deviation 17.16
|
57.2 Scores on a scale
Standard Deviation 17.29
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
S&S- Change (Screening - Month 6) (N=4, 5)
|
4.6 Scores on a scale
Standard Deviation 6.89
|
2.6 Scores on a scale
Standard Deviation 16.32
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
S&S- Change (Screening -Termination)(N=4, 5)
|
11.5 Scores on a scale
Standard Deviation 12.02
|
1.9 Scores on a scale
Standard Deviation 10.22
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
S&S- Change (Month 6- Termination)(N=4, 5)
|
6.9 Scores on a scale
Standard Deviation 18.07
|
-0.8 Scores on a scale
Standard Deviation 8.94
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Support-Screening (N=4, 4)
|
28.1 Scores on a scale
Standard Deviation 25.77
|
51.6 Scores on a scale
Standard Deviation 9.38
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Support-Month 6 (N=4, 4)
|
18.8 Scores on a scale
Standard Deviation 29.32
|
42.2 Scores on a scale
Standard Deviation 17.95
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Support- Termination (N=4, 5)
|
32.8 Scores on a scale
Standard Deviation 32.02
|
48.8 Scores on a scale
Standard Deviation 20.44
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Support- Change (Screening - Month 6) (N=4, 4)
|
9.4 Scores on a scale
Standard Deviation 24.21
|
9.4 Scores on a scale
Standard Deviation 10.83
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Treatment- Month 6 (N=4, 5)
|
21.9 Scores on a scale
Standard Deviation 6.90
|
54.8 Scores on a scale
Standard Deviation 14.53
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Treatment- Termination (N=4, 5)
|
17.1 Scores on a scale
Standard Deviation 22.12
|
41.0 Scores on a scale
Standard Deviation 13.34
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Treatment- Change (Screening - Month 6) (N=4, 5)
|
7.1 Scores on a scale
Standard Deviation 10.10
|
9.4 Scores on a scale
Standard Deviation 13.06
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Treatment- Change(Screening -Termination)(N=4, 5)
|
11.9 Scores on a scale
Standard Deviation 17.16
|
23.2 Scores on a scale
Standard Deviation 16.94
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Treatment- Change (Month 6- Termination)(N=4, 5)
|
4.8 Scores on a scale
Standard Deviation 22.47
|
13.8 Scores on a scale
Standard Deviation 11.14
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
View-Screening (N=4, 5)
|
47.4 Scores on a scale
Standard Deviation 18.25
|
36.7 Scores on a scale
Standard Deviation 16.94
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
View- Termination (N=4, 5)
|
32.8 Scores on a scale
Standard Deviation 35.68
|
30.8 Scores on a scale
Standard Deviation 12.85
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
View- Change (Screening - Month 6) (N=4, 5)
|
1.1 Scores on a scale
Standard Deviation 6.38
|
0.3 Scores on a scale
Standard Deviation 7.63
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
View- Change (Screening -Termination)(N=4, 5)
|
14.7 Scores on a scale
Standard Deviation 18.62
|
5.9 Scores on a scale
Standard Deviation 10.08
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
View- Change (Month 6- Termination)(N=4, 5)
|
13.5 Scores on a scale
Standard Deviation 13.02
|
5.6 Scores on a scale
Standard Deviation 16.70
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Total-Screening (N=4, 5)
|
43.7 Scores on a scale
Standard Deviation 13.67
|
48.1 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Total-Month 6 (N=4, 5)
|
42.0 Scores on a scale
Standard Deviation 13.84
|
42.3 Scores on a scale
Standard Deviation 11.08
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Total- Termination (N=4, 5)
|
34.5 Scores on a scale
Standard Deviation 21.83
|
39.5 Scores on a scale
Standard Deviation 8.52
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Total- Change (Screening - Month 6) (N=4, 5)
|
1.7 Scores on a scale
Standard Deviation 1.19
|
5.7 Scores on a scale
Standard Deviation 14.97
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Parent's Evaluation
Total- Change (Screening -Termination)(N=4, 5)
|
9.1 Scores on a scale
Standard Deviation 8.57
|
8.6 Scores on a scale
Standard Deviation 8.72
|
SECONDARY outcome
Timeframe: 12 months ± 14 daysPopulation: Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - comprised of all participants \<16 years old, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: Physical Health (PH), Sports \& School (S\&S), Dealing with Hemophilia (Dealing), Family, Feeling, Relationships (R'ships), Treatment, View, Outlook for the Future (Future), Friends, Others, and Support. A Haemo-QoL Total Score (Total) was also calculated. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. Haemo-QoL scores at screening, 6 months, and at termination visit were collected. Changes in scores at 6 months and termination were also calculated.
Outcome measures
| Measure |
On-demand Arm
n=4 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=5 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Family- Termination (N=4, 5)
|
38.3 Scores on a scale
Standard Deviation 21.25
|
32.5 Scores on a scale
Standard Deviation 21.09
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Feeling- Month 6 (N=4, 5)
|
41.4 Scores on a scale
Standard Deviation 43.03
|
22.0 Scores on a scale
Standard Deviation 18.68
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Feeling- Termination (N=4, 5)
|
34.4 Scores on a scale
Standard Deviation 39.48
|
19.2 Scores on a scale
Standard Deviation 15.39
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Friends- Change (Screening - Month 6) (N=4, 5)
|
6.3 Scores on a scale
Standard Deviation 25.52
|
16.3 Scores on a scale
Standard Deviation 24.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Others- Termination (N=4, 5)
|
18.8 Scores on a scale
Standard Deviation 21.11
|
13.3 Scores on a scale
Standard Deviation 12.64
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Others- Change (Screening - Month 6) (N=4, 5)
|
9.4 Scores on a scale
Standard Deviation 8.59
|
10.0 Scores on a scale
Standard Deviation 25.79
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Others- Change(Screening - Termination)(N=4, 5)
|
24.0 Scores on a scale
Standard Deviation 14.18
|
13.3 Scores on a scale
Standard Deviation 23.09
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Others- Change (Month 6 - Termination)(N=4, 5)
|
14.6 Scores on a scale
Standard Deviation 19.39
|
3.3 Scores on a scale
Standard Deviation 16.24
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
PH- Change (Month 6 - Termination)(N=4, 5)
|
8.0 Scores on a scale
Standard Deviation 12.16
|
8.2 Scores on a scale
Standard Deviation 20.66
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
R'ships- Termination (N=2, 2)
|
6.3 Scores on a scale
Standard Deviation 8.84
|
18.8 Scores on a scale
Standard Deviation 26.52
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
S&S- Screening (N=4, 5)
|
53.3 Scores on a scale
Standard Deviation 14.74
|
51.3 Scores on a scale
Standard Deviation 19.82
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Support- Termination (N=4, 5)
|
17.2 Scores on a scale
Standard Deviation 17.21
|
65.0 Scores on a scale
Standard Deviation 15.69
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Treatment- Termination (N=4, 5)
|
35.9 Scores on a scale
Standard Deviation 26.10
|
44.2 Scores on a scale
Standard Deviation 27.10
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Treatment- Change (Screening - Month 6) (N=4, 5)
|
-3.2 Scores on a scale
Standard Deviation 4.43
|
12.9 Scores on a scale
Standard Deviation 21.22
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Treatment- Change(Screening -Termination)(N=4, 5)
|
-8.6 Scores on a scale
Standard Deviation 13.86
|
25.4 Scores on a scale
Standard Deviation 28.00
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Treatment- Change (Month 6- Termination)(N=4, 5)
|
-5.3 Scores on a scale
Standard Deviation 11.25
|
12.6 Scores on a scale
Standard Deviation 15.53
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
View-Screening (N=4, 5)
|
46.0 Scores on a scale
Standard Deviation 19.33
|
31.9 Scores on a scale
Standard Deviation 14.36
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
View- Termination (N=4, 5)
|
32.5 Scores on a scale
Standard Deviation 32.75
|
32.2 Scores on a scale
Standard Deviation 8.60
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
View- Change (Screening - Month 6) (N=4, 5)
|
6.7 Scores on a scale
Standard Deviation 14.96
|
-0.5 Scores on a scale
Standard Deviation 18.94
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
View- Change (Month 6- Termination)(N=4, 5)
|
6.8 Scores on a scale
Standard Deviation 8.67
|
0.2 Scores on a scale
Standard Deviation 13.48
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Total-Screening (N=4, 5)
|
44.9 Scores on a scale
Standard Deviation 12.28
|
45.1 Scores on a scale
Standard Deviation 9.16
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Total- Change (Screening - Month 6) (N=4, 5)
|
7.5 Scores on a scale
Standard Deviation 7.21
|
7.4 Scores on a scale
Standard Deviation 16.57
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Total- Change (Screening -Termination)(N=4, 5)
|
12.7 Scores on a scale
Standard Deviation 8.69
|
9.8 Scores on a scale
Standard Deviation 12.35
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Total- Change (Month 6- Termination)(N=4, 5)
|
5.2 Scores on a scale
Standard Deviation 3.55
|
2.5 Scores on a scale
Standard Deviation 9.46
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
R'ships- Screening (N=2, 2)
|
6.3 Scores on a scale
Standard Deviation 8.84
|
43.8 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
R'ships- Month 6 (N=2, 2)
|
18.8 Scores on a scale
Standard Deviation 26.52
|
43.8 Scores on a scale
Standard Deviation 8.84
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
R'ships- Change (Screening - Month 6) (N=2, 2)
|
-12.5 Scores on a scale
Standard Deviation 17.68
|
0.0 Scores on a scale
Standard Deviation 0.00
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
R'ships-Change(Screening -Termination)(N=2, 2)
|
0.0 Scores on a scale
Standard Deviation 0.00
|
25.0 Scores on a scale
Standard Deviation 17.68
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
R'ships- Change (Month 6- Termination)(N=2, 2)
|
12.5 Scores on a scale
Standard Deviation 17.68
|
25.0 Scores on a scale
Standard Deviation 17.68
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
S&S- Month 6 (N=4, 5)
|
46.8 Scores on a scale
Standard Deviation 13.42
|
54.2 Scores on a scale
Standard Deviation 5.53
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Dealing- Screening (N=4, 4)
|
33.0 Scores on a scale
Standard Deviation 17.10
|
44.6 Scores on a scale
Standard Deviation 20.72
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Dealing- Month 6 (N=4, 4)
|
21.4 Scores on a scale
Standard Deviation 2.92
|
37.5 Scores on a scale
Standard Deviation 13.20
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Dealing- Termination (N=4, 5)
|
25.0 Scores on a scale
Standard Deviation 9.22
|
32.9 Scores on a scale
Standard Deviation 12.73
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Dealing- Change (Screening - Month 6) (N=4, 4)
|
11.6 Scores on a scale
Standard Deviation 15.26
|
7.1 Scores on a scale
Standard Deviation 8.75
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Dealing- Change(Screening - Termination)(N=4, 4)
|
8.0 Scores on a scale
Standard Deviation 15.26
|
14.3 Scores on a scale
Standard Deviation 12.37
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Dealing- Change (Month 6 - Termination)(N=4, 4)
|
-3.6 Scores on a scale
Standard Deviation 6.52
|
7.1 Scores on a scale
Standard Deviation 6.52
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Family- Screening (N=4, 5)
|
50.5 Scores on a scale
Standard Deviation 23.94
|
35.6 Scores on a scale
Standard Deviation 22.05
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Family- Month 6 (N=4, 5)
|
43.4 Scores on a scale
Standard Deviation 28.78
|
36.4 Scores on a scale
Standard Deviation 23.06
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Family- Change (Screening - Month 6) (N=4, 5)
|
7.0 Scores on a scale
Standard Deviation 20.31
|
-0.7 Scores on a scale
Standard Deviation 16.38
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Family- Change(Screening - Termination)(N=4, 5)
|
12.2 Scores on a scale
Standard Deviation 25.72
|
3.1 Scores on a scale
Standard Deviation 15.51
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Family- Change (Month 6 - Termination)(N=4, 5)
|
5.2 Scores on a scale
Standard Deviation 13.66
|
3.9 Scores on a scale
Standard Deviation 12.20
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Feeling- Screening (N=4, 5)
|
54.8 Scores on a scale
Standard Deviation 31.91
|
34.9 Scores on a scale
Standard Deviation 12.89
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Feeling- Change (Screening - Month 6) (N=4, 5)
|
13.4 Scores on a scale
Standard Deviation 16.91
|
12.9 Scores on a scale
Standard Deviation 27.77
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Feeling- Change(Screening - Termination)(N=4, 5)
|
20.4 Scores on a scale
Standard Deviation 11.77
|
15.7 Scores on a scale
Standard Deviation 23.20
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Feeling- Change (Month 6 - Termination)(N=4, 5)
|
7.0 Scores on a scale
Standard Deviation 7.79
|
2.8 Scores on a scale
Standard Deviation 15.26
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Friends- Screening (N=4, 5)
|
34.4 Scores on a scale
Standard Deviation 25.26
|
58.8 Scores on a scale
Standard Deviation 20.54
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Friends- Month 6 (N=4, 5)
|
28.1 Scores on a scale
Standard Deviation 15.73
|
42.5 Scores on a scale
Standard Deviation 29.78
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Friends- Termination (N=4, 5)
|
32.8 Scores on a scale
Standard Deviation 12.88
|
56.3 Scores on a scale
Standard Deviation 27.24
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Friends- Change(Screening - Termination)(N=4, 5)
|
1.6 Scores on a scale
Standard Deviation 28.58
|
2.5 Scores on a scale
Standard Deviation 22.79
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Friends- Change (Month 6 - Termination)(N=4, 5)
|
-4.7 Scores on a scale
Standard Deviation 17.95
|
-13.8 Scores on a scale
Standard Deviation 20.44
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Future- Screening (N=2, 2)
|
40.6 Scores on a scale
Standard Deviation 13.26
|
43.8 Scores on a scale
Standard Deviation 17.68
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Future- Month 6 (N=2, 2)
|
43.8 Scores on a scale
Standard Deviation 17.68
|
43.8 Scores on a scale
Standard Deviation 17.68
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
S&S- Termination (N=4, 5)
|
33.2 Scores on a scale
Standard Deviation 5.64
|
49.8 Scores on a scale
Standard Deviation 18.49
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
S&S- Change (Screening - Month 6) (N=4, 5)
|
6.5 Scores on a scale
Standard Deviation 11.42
|
-2.8 Scores on a scale
Standard Deviation 18.53
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
S&S- Change (Screening -Termination)(N=4, 5)
|
20.1 Scores on a scale
Standard Deviation 14.74
|
1.5 Scores on a scale
Standard Deviation 18.74
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
S&S- Change (Month 6- Termination)(N=4, 5)
|
13.5 Scores on a scale
Standard Deviation 9.10
|
4.4 Scores on a scale
Standard Deviation 15.19
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Future- Termination (N=2, 2)
|
46.9 Scores on a scale
Standard Deviation 22.10
|
28.1 Scores on a scale
Standard Deviation 22.10
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Support-Screening (N=4, 4)
|
32.8 Scores on a scale
Standard Deviation 3.13
|
62.5 Scores on a scale
Standard Deviation 31.04
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Support-Month 6 (N=4, 4)
|
28.1 Scores on a scale
Standard Deviation 10.83
|
56.3 Scores on a scale
Standard Deviation 31.87
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Support- Change (Screening - Month 6) (N=4, 4)
|
4.7 Scores on a scale
Standard Deviation 12.88
|
6.3 Scores on a scale
Standard Deviation 31.46
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Support- Change (Screening -Termination)(N=4, 4
|
15.6 Scores on a scale
Standard Deviation 18.75
|
-3.1 Scores on a scale
Standard Deviation 18.75
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Future- Change (Screening - Month 6) (N=2, 2)
|
-3.1 Scores on a scale
Standard Deviation 4.42
|
0.0 Scores on a scale
Standard Deviation 0.00
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Support- Change (Month 6- Termination)(N=4, 4)
|
10.9 Scores on a scale
Standard Deviation 10.67
|
-9.4 Scores on a scale
Standard Deviation 25.77
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Treatment- Screening (N=4, 5)
|
27.3 Scores on a scale
Standard Deviation 15.38
|
69.6 Scores on a scale
Standard Deviation 25.63
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Future- Change(Screening - Termination)(N=2, 2)
|
-6.3 Scores on a scale
Standard Deviation 8.84
|
15.6 Scores on a scale
Standard Deviation 4.42
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Treatment- Month 6 (N=4, 5)
|
30.6 Scores on a scale
Standard Deviation 16.50
|
56.8 Scores on a scale
Standard Deviation 15.89
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Future- Change (Month 6 - Termination)(N=2, 2)
|
-3.1 Scores on a scale
Standard Deviation 4.42
|
15.6 Scores on a scale
Standard Deviation 4.42
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Others- Screening (N=4, 5)
|
42.7 Scores on a scale
Standard Deviation 33.74
|
26.7 Scores on a scale
Standard Deviation 20.11
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Others- Month 6 (N=4, 5)
|
33.3 Scores on a scale
Standard Deviation 40.11
|
16.7 Scores on a scale
Standard Deviation 15.02
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
PH- Screening (N=4, 5)
|
67.0 Scores on a scale
Standard Deviation 24.98
|
42.1 Scores on a scale
Standard Deviation 32.28
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
PH- Month 6 (N=4, 5)
|
49.1 Scores on a scale
Standard Deviation 14.69
|
33.9 Scores on a scale
Standard Deviation 16.66
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
View-Month 6 (N=4, 5)
|
39.3 Scores on a scale
Standard Deviation 33.02
|
32.4 Scores on a scale
Standard Deviation 19.41
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
PH- Termination (N=4, 5)
|
41.1 Scores on a scale
Standard Deviation 23.05
|
25.7 Scores on a scale
Standard Deviation 10.83
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
PH- Change (Screening - Month 6) (N=4, 5)
|
17.9 Scores on a scale
Standard Deviation 19.56
|
8.2 Scores on a scale
Standard Deviation 34.43
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
PH- Change(Screening - Termination)(N=4, 5)
|
25.9 Scores on a scale
Standard Deviation 14.40
|
16.4 Scores on a scale
Standard Deviation 29.73
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
View- Change (Screening -Termination)(N=4, 5)
|
13.5 Scores on a scale
Standard Deviation 16.56
|
-0.3 Scores on a scale
Standard Deviation 10.77
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Total-Month 6 (N=4, 5)
|
37.4 Scores on a scale
Standard Deviation 17.91
|
37.7 Scores on a scale
Standard Deviation 9.87
|
|
Hemophilia-specific Quality of Life Questionnaire for Children and Adolescents < 16 Years Old (Haemo-QoL) - Child's Evaluation
Total- Termination (N=4, 5)
|
32.2 Scores on a scale
Standard Deviation 17.96
|
35.3 Scores on a scale
Standard Deviation 9.06
|
SECONDARY outcome
Timeframe: Baseline, 6 months and 12 months ± 14 daysPopulation: Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - comprised of all participants ≥12 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
General pain was assessed using a VAS pain scale at screening, 6 months, and at termination. Unlike the VAS pain assessment for pain of bleeding episodes (Outcome above), this general pain assessment did not take use of analgesics into account. For the pain scale, a higher number indicates worse pain. The visual analog scale ranges from 0 to 100 where the endpoints are labeled 'Worst imaginable health state' (=0) and 'Best imaginable health state' (=100). A positive change from baseline indicates improvement. Change in VAS scores at 6 months and study termination were also compared relative to Baseline/Screening scores (ie, (Baseline/Screening VAS score) - (VAS score at 6 months and study termination).
Outcome measures
| Measure |
On-demand Arm
n=17 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=15 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old
Month 6 (N= 16, 14)
|
36.6 Scores on a scale
Standard Deviation 25.81
|
32.7 Scores on a scale
Standard Deviation 26.24
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old
Change (Month 6 - Termination) (N= 16, 14)
|
4.2 Scores on a scale
Standard Deviation 21.82
|
2.9 Scores on a scale
Standard Deviation 19.62
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old
Screening (N= 17, 15)
|
35.2 Scores on a scale
Standard Deviation 30.15
|
55.5 Scores on a scale
Standard Deviation 23.68
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old
Termination (N= 16, 14)
|
32.4 Scores on a scale
Standard Deviation 21.97
|
29.8 Scores on a scale
Standard Deviation 30.48
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old
Change (Screening - Month 6) (N= 16, 14)
|
0.8 Scores on a scale
Standard Deviation 31.77
|
20.3 Scores on a scale
Standard Deviation 38.91
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Adults and Adolescents ≥12 Years Old
Change (Screening - Termination) (N= 16, 14)
|
5.0 Scores on a scale
Standard Deviation 28.70
|
23.2 Scores on a scale
Standard Deviation 46.61
|
SECONDARY outcome
Timeframe: Baseline, 6 months and 12 months ± 14 daysPopulation: Health-Related Quality of Life (HRQoL) Intent-to-Treat Analysis Dataset - comprised of all participants \<12 years old who were randomized, had any available assessments at any available study visits (baseline, 6-month, and 12-month) as defined in the protocol
General pain was assessed using the children's VAS pain scale (a facial expression scale with one end marked as no pain and the opposite end marked as the worst possible pain). Assessments were done at the screening, 6 months, and termination visits. Scores on the children's VAS scale are presented as: -No Pain -Mild Pain -Moderate pain -Severe pain -Very severe pain Unlike the VAS pain assessment for pain of bleeding episodes (Outcome above), this general pain assessment did not take use of analgesics into account. Change in VAS scores at 6 months and study termination were also compared relative to Baseline/Screening scores (ie, (Baseline/Screening VAS score) - (VAS score at 6 months and study termination).
Outcome measures
| Measure |
On-demand Arm
n=2 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated): FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=2 Participants
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month prophylactic period
|
|---|---|---|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Month 6 - No Pain
|
0 participants
|
0 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Termination visit - No Pain
|
0 participants
|
1 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Termination visit - Moderate Pain
|
1 participants
|
1 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Screening - No Pain
|
1 participants
|
0 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Screening - Mild Pain
|
0 participants
|
1 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Screening - Moderate Pain
|
1 participants
|
0 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Screening - Severe Pain
|
0 participants
|
1 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Screening - Very Severe Pain
|
0 participants
|
0 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Month 6 - Mild Pain
|
1 participants
|
2 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Month 6 - Moderate Pain
|
1 participants
|
0 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Month 6 - Severe Pain
|
0 participants
|
0 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Month 6 - Very Severe Pain
|
0 participants
|
0 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Termination visit - Mild Pain
|
0 participants
|
0 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Termination visit - Severe Pain
|
0 participants
|
0 participants
|
|
Health-Related Quality of Life (HRQoL) - General Pain Assessment Using a Visual Analogue Scale (VAS) in Pediatrics <12 Years Old
Termination visit - Very Severe Pain
|
1 participants
|
0 participants
|
Adverse Events
On-demand Arm
Serious events: 7 serious events
Other events: 4 other events
Deaths: 0 deaths
Prophylaxis Arm
Serious events: 6 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
On-demand Arm
n=19 participants at risk
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : Standard FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 participants at risk
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month ± 14 days prophylactic period
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal Wall Haematoma
|
0.00%
0/19 • Throughout the study period of 3 years and 7 months
|
5.9%
1/17 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
|
Hepatobiliary disorders
Cholecystitis Acute
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Infections and infestations
Haematoma Infection
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Injury, poisoning and procedural complications
Femoral Neck Fracture
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Investigations
Hepatitis B Surface Antibody Positive
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
11.8%
2/17 • Number of events 2 • Throughout the study period of 3 years and 7 months
|
|
Musculoskeletal and connective tissue disorders
Arthropathy
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Musculoskeletal and connective tissue disorders
Haemarthrosis
|
5.3%
1/19 • Number of events 3 • Throughout the study period of 3 years and 7 months
|
11.8%
2/17 • Number of events 8 • Throughout the study period of 3 years and 7 months
|
|
Musculoskeletal and connective tissue disorders
Muscle Haemorrhage
|
0.00%
0/19 • Throughout the study period of 3 years and 7 months
|
5.9%
1/17 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
|
Renal and urinary disorders
Haematuria
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Surgical and medical procedures
Catheter Removal
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Vascular disorders
Arteriosclerosis
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Vascular disorders
Haematoma
|
0.00%
0/19 • Throughout the study period of 3 years and 7 months
|
5.9%
1/17 • Number of events 2 • Throughout the study period of 3 years and 7 months
|
|
Vascular disorders
Haemorrhage
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Vascular disorders
Hypertensive Crisis
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
Other adverse events
| Measure |
On-demand Arm
n=19 participants at risk
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : Standard FEIBA NF dose and dosing interval as prescribed by the treating physician
|
Prophylaxis Arm
n=17 participants at risk
Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated) : 85 ± 15 U/kg of FEIBA NF every other day during the 12-month ± 14 days prophylactic period
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
10.5%
2/19 • Number of events 2 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Gastrointestinal disorders
Diarrhoea
|
5.3%
1/19 • Number of events 2 • Throughout the study period of 3 years and 7 months
|
5.9%
1/17 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
|
Gastrointestinal disorders
Nausea
|
10.5%
2/19 • Number of events 2 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Gastrointestinal disorders
Vomiting
|
10.5%
2/19 • Number of events 2 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
General disorders
Pyrexia
|
10.5%
2/19 • Number of events 4 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
10.5%
2/19 • Number of events 3 • Throughout the study period of 3 years and 7 months
|
5.9%
1/17 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
|
Musculoskeletal and connective tissue disorders
Arthropathy
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
0.00%
0/17 • Throughout the study period of 3 years and 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
5.9%
1/17 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
5.9%
1/17 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
|
Skin and subcutaneous tissue disorders
Acne
|
5.3%
1/19 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
5.9%
1/17 • Number of events 1 • Throughout the study period of 3 years and 7 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Baxter's agreements with PIs may vary per requirements of individual PI, but contain common elements. For this study: PIs may require prior written approval, or be restricted from independently publishing results until the earlier of the primary multicenter publication or ≤36 months after study completion. Baxter requires a review of results communications (e.g., for confidential information) prior to submission for publication, and shall review and if necessary amend the manuscript in ≤60 days
- Publication restrictions are in place
Restriction type: OTHER