Trial Outcomes & Findings for PROPACT: Retrospective Prophylaxis Patient Case Collection (NCT NCT00882778)
NCT ID: NCT00882778
Last Updated: 2017-01-09
Results Overview
Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period
COMPLETED
86 participants
Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
2017-01-09
Participant Flow
Participating sites: Argentina (1), Canada (1), Croatia (1), Czech Republic (1), Germany (1), France (1), Ireland (1), Italy (4), Slovakia (1), Spain (3), Sweden (2), Switzerland (2), United Kingdom (1), and the United States of America (11)
Participant milestones
| Measure |
Activated Recombinant Human Factor VII
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
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|---|---|
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Overall Study
STARTED
|
86
|
|
Overall Study
COMPLETED
|
86
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
PROPACT: Retrospective Prophylaxis Patient Case Collection
Baseline characteristics by cohort
| Measure |
Activated Recombinant Human Factor VII
n=86 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
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|---|---|
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Age, Continuous
All, n=86
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11.6 years
STANDARD_DEVIATION 13.03 • n=5 Participants
|
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Age, Continuous
Bleeding population, n=74
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10.9 years
STANDARD_DEVIATION 12.45 • n=5 Participants
|
|
Age, Continuous
Frequent bleeding population, n=36
|
12.1 years
STANDARD_DEVIATION 12.67 • n=5 Participants
|
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Age, Customized
Below 12 years
|
61 participants
n=5 Participants
|
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Age, Customized
Between 12 and 17 years
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7 participants
n=5 Participants
|
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Age, Customized
18 years or above
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18 participants
n=5 Participants
|
|
Gender
Female
|
0 Participants
n=5 Participants
|
|
Gender
Male
|
86 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
12 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
33 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
41 Participants
n=5 Participants
|
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Race (NIH/OMB)
American Indian or Alaska Native
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1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
7 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
43 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
33 Participants
n=5 Participants
|
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Region of Enrollment
Argentina
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4 participants
n=5 Participants
|
|
Region of Enrollment
Canada
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1 participants
n=5 Participants
|
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Region of Enrollment
Croatia
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2 participants
n=5 Participants
|
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Region of Enrollment
Czech Republic
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1 participants
n=5 Participants
|
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Region of Enrollment
France
|
17 participants
n=5 Participants
|
|
Region of Enrollment
Germany
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3 participants
n=5 Participants
|
|
Region of Enrollment
Ireland
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5 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
7 participants
n=5 Participants
|
|
Region of Enrollment
Slovakia
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Spain
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10 participants
n=5 Participants
|
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Region of Enrollment
Sweden
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4 participants
n=5 Participants
|
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Region of Enrollment
Switzerland
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2 participants
n=5 Participants
|
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Region of Enrollment
United Kingdom
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1 participants
n=5 Participants
|
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Region of Enrollment
United States
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28 participants
n=5 Participants
|
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Peak historical inhibitor titre recorded
All, n=81
|
517.7 Bethesda Units (BU)/mL
STANDARD_DEVIATION 1164.41 • n=5 Participants
|
|
Peak historical inhibitor titre recorded
Bleeding population, n=72
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571 Bethesda Units (BU)/mL
STANDARD_DEVIATION 1224.88 • n=5 Participants
|
|
Peak historical inhibitor titre recorded
Frequent bleeding population, n=35
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737.9 Bethesda Units (BU)/mL
STANDARD_DEVIATION 1457 • n=5 Participants
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|
Peak historical inhibitor titre recorded
Missing, n=5
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NA Bethesda Units (BU)/mL
STANDARD_DEVIATION NA • n=5 Participants
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Reason for prophylaxis
Prevention of recurrent bleeding
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79 participants
n=5 Participants
|
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Reason for prophylaxis
In preparation for surgery
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0 participants
n=5 Participants
|
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Reason for prophylaxis
Prep. for rehab./planned phys. activity
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2 participants
n=5 Participants
|
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Reason for prophylaxis
Other
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2 participants
n=5 Participants
|
|
Reason for prophylaxis
Missing
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3 participants
n=5 Participants
|
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Relationship to timing of Immune Tolerance Induction (ITI)
Pre-ITI
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19 participants
n=5 Participants
|
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Relationship to timing of Immune Tolerance Induction (ITI)
During ITI
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8 participants
n=5 Participants
|
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Relationship to timing of Immune Tolerance Induction (ITI)
Post-ITI
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25 participants
n=5 Participants
|
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Relationship to timing of Immune Tolerance Induction (ITI)
ITI dates unknown
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0 participants
n=5 Participants
|
|
Relationship to timing of Immune Tolerance Induction (ITI)
Not candidate for ITI
|
27 participants
n=5 Participants
|
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Relationship to timing of Immune Tolerance Induction (ITI)
Not reported
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7 participants
n=5 Participants
|
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Duration of prophylaxis
All, n=86
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288 days
n=5 Participants
|
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Duration of prophylaxis
Bleeding population, n=74
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293 days
n=5 Participants
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Duration of prophylaxis
Frequent bleeding population, n=36
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293 days
n=5 Participants
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Weekly dose
All, n=82
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710 mcg/kg
n=5 Participants
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Weekly dose
Bleeding population, n=73
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742.5 mcg/kg
n=5 Participants
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Weekly dose
Frequent bleeding population, n=36
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656.2 mcg/kg
n=5 Participants
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|
Weekly dose
Missing, n=4
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NA mcg/kg
n=5 Participants
|
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Dose frequency
All, n=82
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5.2 doses per week
n=5 Participants
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Dose frequency
Bleeding population, n=73
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5.8 doses per week
n=5 Participants
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Dose frequency
Frequent bleeding population, n=36
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5.2 doses per week
n=5 Participants
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Dose frequency
Missing, n=4
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NA doses per week
n=5 Participants
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PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=74 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
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|---|---|
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Percent Change in Total Bleed Episodes Per Month - Bleeding Population
|
-45 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=36 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
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Percent Change in Total Bleed Episodes Per Month - Frequent Bleeding Population
|
-51 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=74 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
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|---|---|
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Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population
Paediatric, n=54
|
-48 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population
Adolescent, n=7
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0 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population
Adult, n=13
|
-56 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=36 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
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|---|---|
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Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population
Adult, n=6
|
-49 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population
Paediatric, n=27
|
-56 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population
Adolescent, n=3
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5 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=1 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
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|---|---|
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Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing
All, n=1
|
-6 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing
Paediatric, n=1
|
-6 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing
Adolescent, n=0
|
0 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing
Adult, n=0
|
0 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=32 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
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|---|---|
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Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week
All, n=32
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-52 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week
Paediatric, n=18
|
-50 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week
Adolescent, n=3
|
12 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week
Adult, n=11
|
-72 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=29 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
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|---|---|
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Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing
All, n=29
|
-48 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing
Paediatric, n=26
|
-48 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing
Adolescent, n=2
|
-43 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing
Adult, n=1
|
-51 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=11 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing
All, n=11
|
-18 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing
Paediatric, n=8
|
-47 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing
Adolescent, n=2
|
40 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing
Adult, n=1
|
35 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=1 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing
All, n=1
|
-6 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing
Paediatric, n=1
|
-6 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing
Adolescent, n=0
|
0 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing
Adult, n=0
|
0 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=17 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week
All, n=17
|
-56 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week
Padiatric, n=11
|
-57 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week
Adolescent, n=2
|
-10 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week
Adult, n=4
|
-76 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=15 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing
Adolescent, n=1
|
47 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing
All, n=15
|
-59 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing
Paediatric, n=13
|
-62 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing
Adult, n=1
|
-51 percent change (%) in bleeds per month
|
PRIMARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=3 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing
All, n=3
|
33 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing
Paediatric, n=2
|
-13 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing
Adolescent, n=0
|
0 percent change (%) in bleeds per month
|
|
Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing
Adult, n=1
|
35 percent change (%) in bleeds per month
|
SECONDARY outcome
Timeframe: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=53 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric
Infrequent dosing, n=1
|
133.3 mcg/kg
Interval 133.3 to 133.3
|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric
Dosing three times per week, n=18
|
185.4 mcg/kg
Interval 26.9 to 301.8
|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric
Daily dosing, n=26
|
141.9 mcg/kg
Interval 54.5 to 395.4
|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric
Frequent dosing, n=8
|
135 mcg/kg
Interval 103.3 to 155.5
|
SECONDARY outcome
Timeframe: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=7 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent
Infrequent dosing, n=0
|
0 mcg/kg
Interval 0.0 to 0.0
|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent
Dosing three times per week, n=3
|
198 mcg/kg
Interval 77.9 to 301.8
|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent
Daily dosing, n=2
|
138.9 mcg/kg
Interval 54.5 to 395.4
|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent
Frequent dosing, n=2
|
138 mcg/kg
Interval 120.0 to 155.5
|
SECONDARY outcome
Timeframe: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=13 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult
Infrequent dosing, n=0
|
0 mcg/kg
Interval 0.0 to 0.0
|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult
Dosing three times per week, n=11
|
159.7 mcg/kg
Interval 26.9 to 290.9
|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult
Daily dosing, n=1
|
117.6 mcg/kg
Interval 117.6 to 117.6
|
|
Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult
Frequent dosing, n=1
|
103.3 mcg/kg
Interval 103.3 to 103.3
|
SECONDARY outcome
Timeframe: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=27 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric
Infrequent dosing, n=1
|
133.3 mcg/kg
Interval 133.3 to 133.3
|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric
Dosing three times per week, n=11
|
197.2 mcg/kg
Interval 77.9 to 270.0
|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric
Daily dosing, n=13
|
135.9 mcg/kg
Interval 57.3 to 395.4
|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric
Frequent dosing, n=2
|
131 mcg/kg
Interval 127.0 to 135.0
|
SECONDARY outcome
Timeframe: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=3 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent
Infrequent dosing, n=0
|
0 mcg/kg
Interval 0.0 to 0.0
|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent
Dosing three times per week, n=2
|
138 mcg/kg
Interval 96.0 to 180.0
|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent
Daily dosing, n=1
|
280 mcg/kg
Interval 280.0 to 280.0
|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent
Frequent dosing, n=0
|
0 mcg/kg
Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=6 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult
Infrequent dosing, n=0
|
0 mcg/kg
Interval 0.0 to 0.0
|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult
Dosing three times per week, n=4
|
162 mcg/kg
Interval 26.9 to 240.0
|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult
Daily dosing, n=1
|
117.6 mcg/kg
Interval 117.6 to 117.6
|
|
Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult
Frequent dosing, n=1
|
103.3 mcg/kg
Interval 103.3 to 103.3
|
SECONDARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approx. 6 months. Patients from the French sites were not included because bleed location was not collected. Bleed episodes with no recorded locations were not included in the analysis
Percent change in bleed episodes per month between pre-prophylaxis period and prophylaxis period by location of joint, target joint (defined as 3 or more documented bleeds in the same joint over the course of 6 months) or non-joint. All joints = target joints and non-target joints.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=56 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population
All joints
|
-39 percent change (%) in bleeds per month
|
|
Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population
Target joints
|
-50 percent change (%) in bleeds per month
|
|
Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population
Non-joints
|
-33 percent change (%) in bleeds per month
|
SECONDARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approx. 6 months. Patients from the French sites were not included because bleed location was not collected in these patients. Bleed episodes with no recorded locations were not included in the analysis
Percent change in bleed episodes per month between pre-prophylaxis period and prophylaxis period by location of joint, target joint (= 3 or more documented bleeds in the same joint over the course of 6 months) or non-joint. All joints = target joints and non-target joints.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=27 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population
All joints
|
-46 percent change (%) in bleeds per month
|
|
Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population
Target joints
|
-56 percent change (%) in bleeds per month
|
|
Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population
Non-joints
|
-42 percent change (%) in bleeds per month
|
SECONDARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Whole population of all male patients diagnosed with haemophilia A or B with inhibitor, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days
Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis to the prophylaxis period.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=83 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients
Out patient clinical visits
|
-0.011 change in events per month
Standard Deviation 1.07
|
|
Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients
Physician consultations
|
0.043 change in events per month
Standard Deviation 1.66
|
|
Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients
Hospital admissions
|
-0.097 change in events per month
Standard Deviation 0.27
|
SECONDARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=74 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population
Physician consultations
|
0.053 change in events per month
Standard Deviation 1.76
|
|
Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population
Out patient clinical visits
|
0.0090 change in events per month
Standard Deviation 1.117
|
|
Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population
Hospital admissions
|
-0.11 change in events per month
Standard Deviation 0.28
|
SECONDARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=36 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population
Out patient clinical visits
|
0.0576 change in events per month
Standard Deviation 1.39
|
|
Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population
Physician consultations
|
0.213 change in events per month
Standard Deviation 2.49
|
|
Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population
Hospital admissions
|
-0.11 change in events per month
Standard Deviation 0.33
|
SECONDARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Whole population of all male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (RFVIIa) for at least 30 days.
Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=83 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients
Total hospital length of stay
|
-0.654 change in days per month
Standard Deviation 3.43
|
|
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients
School/work absences
|
-1.365 change in days per month
Standard Deviation 1.99
|
SECONDARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Bleeding population with at least one bleed in the pre-prophylaxis of approximately 6 months.
Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=74 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population
School/work absences
|
-1.365 change in days per month
Standard Deviation 1.99
|
|
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population
Total hospital length of stay
|
-0.794 change in days per month
Standard Deviation 3.55
|
SECONDARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months.
Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=36 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population
Total hospital length of stay
|
-1.064 change in days per month
Standard Deviation 2.38
|
|
Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population
School/work absences
|
-1.927 change in days per month
Standard Deviation 2.29
|
SECONDARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Whole population of all male patients diagnosed with haemophilia A or B with inhibitor, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days
Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=86 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Physician Reported Outcome Assessment in Prophylaxis in Number of Patients
Successful
|
35 patients
|
|
Physician Reported Outcome Assessment in Prophylaxis in Number of Patients
Partially successful
|
20 patients
|
|
Physician Reported Outcome Assessment in Prophylaxis in Number of Patients
Unsuccessful
|
7 patients
|
|
Physician Reported Outcome Assessment in Prophylaxis in Number of Patients
Unable to determine
|
9 patients
|
|
Physician Reported Outcome Assessment in Prophylaxis in Number of Patients
Data missing or not reported
|
15 patients
|
SECONDARY outcome
Timeframe: Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.Population: Whole population of all male patients diagnosed with haemophilia A or B with inhibitor, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days
Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=86 Participants
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients
Successful
|
40 percentage of patients
|
|
Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients
Partially successful
|
23 percentage of patients
|
|
Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients
Unsuccessful
|
8 percentage of patients
|
|
Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients
Unable to determine
|
10 percentage of patients
|
|
Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients
Data missing or not reported
|
17 percentage of patients
|
Adverse Events
Activated Recombinant Human Factor VII
Serious adverse events
| Measure |
Activated Recombinant Human Factor VII
n=86 participants at risk
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
|
|---|---|
|
Blood and lymphatic system disorders
Septicaemia of Candida albicans
|
1.2%
1/86 • Number of events 1 • Onset date 15-Dec-2007, outcome date 22-Dec-2007
Safety population is all patients in the study. This is a non-interventional study, and thus only adverse events (AEs) and serious adverse events (SAEs) reported by the investigator is possible or probably related to drug and/or medical events of special interest (MESI) are included in the table
|
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place