Trial Outcomes & Findings for Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors (NCT NCT00703911)
NCT ID: NCT00703911
Last Updated: 2017-01-11
Results Overview
The percentage of bleed treatments successfully resulting in bleed resolution. Analysis only considers the patient's opinion of effectiveness at 9 hours, with a rating of "Effective" considered as successful treatment.
Recruitment status
COMPLETED
Target enrollment
102 participants
Primary outcome timeframe
within 9 hours of first injection
Results posted on
2017-01-11
Participant Flow
Participant milestones
| Measure |
Activated Recombinant Human Factor VII
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Overall Study
STARTED
|
102
|
|
Overall Study
COMPLETED
|
92
|
|
Overall Study
NOT COMPLETED
|
10
|
Reasons for withdrawal
| Measure |
Activated Recombinant Human Factor VII
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Overall Study
Death
|
1
|
|
Overall Study
Physician Decision
|
6
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors
Baseline characteristics by cohort
| Measure |
Activated Recombinant Human Factor VII
n=102 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Age, Continuous
|
23.3 years
STANDARD_DEVIATION 16.37 • n=5 Participants
|
|
Age, Customized
2 - 12 years
|
35 participants
n=5 Participants
|
|
Age, Customized
above 12 - 17 years
|
14 participants
n=5 Participants
|
|
Age, Customized
18 years or above
|
53 participants
n=5 Participants
|
|
Gender
Female
|
0 Participants
n=5 Participants
|
|
Gender
Male
|
102 Participants
n=5 Participants
|
|
Region of Enrollment
Algeria
|
17 participants
n=5 Participants
|
|
Region of Enrollment
Austria
|
3 participants
n=5 Participants
|
|
Region of Enrollment
Belgium
|
4 participants
n=5 Participants
|
|
Region of Enrollment
Czech Republic
|
1 participants
n=5 Participants
|
|
Region of Enrollment
France
|
16 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
4 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
16 participants
n=5 Participants
|
|
Region of Enrollment
Netherlands
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
9 participants
n=5 Participants
|
|
Region of Enrollment
Portugal
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Saudi Arabia
|
2 participants
n=5 Participants
|
|
Region of Enrollment
South Africa
|
8 participants
n=5 Participants
|
|
Region of Enrollment
Sweden
|
1 participants
n=5 Participants
|
|
Region of Enrollment
Turkey
|
6 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
7 participants
n=5 Participants
|
|
Region of Enrollment
Venezuela
|
6 participants
n=5 Participants
|
|
Peak inhibitor titre
At least 5 Bethesda Units
|
91 participants
n=5 Participants
|
|
Peak inhibitor titre
Less than 5 Bethesda Units
|
11 participants
n=5 Participants
|
|
Outcome of immune tolerance induction (ITI) treatment
None
|
66 participants
n=5 Participants
|
|
Outcome of immune tolerance induction (ITI) treatment
Unsuccessful
|
20 participants
n=5 Participants
|
|
Outcome of immune tolerance induction (ITI) treatment
Successful, with inhibitor recurrence
|
5 participants
n=5 Participants
|
|
Outcome of immune tolerance induction (ITI) treatment
Ongoing ITI
|
11 participants
n=5 Participants
|
|
Estimated number of bleeds for the 6 months preceding enrolment in registry
None
|
9 participants
n=5 Participants
|
|
Estimated number of bleeds for the 6 months preceding enrolment in registry
1 - 6 (1 or less per month)
|
65 participants
n=5 Participants
|
|
Estimated number of bleeds for the 6 months preceding enrolment in registry
6 - 12 (1 - 2 per month)
|
13 participants
n=5 Participants
|
|
Estimated number of bleeds for the 6 months preceding enrolment in registry
12 - 18 (2 - 3 per month)
|
12 participants
n=5 Participants
|
|
Estimated number of bleeds for the 6 months preceding enrolment in registry
18 (more than 3 per month)
|
2 participants
n=5 Participants
|
|
Estimated number of bleeds for the 6 months preceding enrolment in registry
Data missing
|
1 participants
n=5 Participants
|
|
Primary treatment pattern for the 6 months preceding enrolment in registry
On-demand
|
88 participants
n=5 Participants
|
|
Primary treatment pattern for the 6 months preceding enrolment in registry
Prophylaxis
|
13 participants
n=5 Participants
|
|
Primary treatment pattern for the 6 months preceding enrolment in registry
Data missing
|
1 participants
n=5 Participants
|
|
Primary treatment type and dose for the 6 months preceding enrolment in registry
Multiple dose 90 mcg/kg NovoSeven®
|
44 participants
n=5 Participants
|
|
Primary treatment type and dose for the 6 months preceding enrolment in registry
Single dose 270 mcg/kg NovoSeven®
|
28 participants
n=5 Participants
|
|
Primary treatment type and dose for the 6 months preceding enrolment in registry
Other dose regimen NovoSeven®
|
17 participants
n=5 Participants
|
|
Primary treatment type and dose for the 6 months preceding enrolment in registry
Other bypassing agent (e.g., FEIBA)
|
13 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: within 9 hours of first injectionPopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level
The percentage of bleed treatments successfully resulting in bleed resolution. Analysis only considers the patient's opinion of effectiveness at 9 hours, with a rating of "Effective" considered as successful treatment.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
High initial dose, n=211
|
56.9 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
Any initial dose, n=494
|
61.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
Low initial dose, n=156
|
67.9 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)
Intermediate initial dose, n=127
|
62.2 percentage of bleed episodes
|
PRIMARY outcome
Timeframe: within 9 hours of first injectionPopulation: Analysis set is spontaneous bleed episodes treated with any initial dose
The percentage of bleed treatments successfully resulting in bleed resolution. Analysis only considers the patient's opinion of effectiveness at 9 hours, with a rating of "Effective" considered as successful treatment.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)
Any initial dose, n=324
|
59.0 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)
Low initial dose, n=92
|
62.6 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)
Intermediate initial dose, n=91
|
60.4 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)
High initial dose, n=141
|
55.7 percentage of bleed episodes
|
PRIMARY outcome
Timeframe: within 9 hours of first injectionPopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level
The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)
Any initial dose, n=494
|
71.3 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)
Low initial dose, n=156
|
82.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)
Intermediate initial dose, n=127
|
60.6 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)
High initial dose, n=211
|
69.7 percentage of bleed episodes
|
PRIMARY outcome
Timeframe: within 9 hours of first injectionPopulation: Analysis set is spontaneous bleed episodes treated with any initial dose
The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)
Intermediate initial dose, n=91
|
58.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)
High initial dose, n=141
|
67.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)
Any initial dose, n=324
|
68.0 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)
Low initial dose, n=92
|
79.1 percentage of bleed episodes
|
SECONDARY outcome
Timeframe: 1 hour, 3 hours and 6 hours, respectively, after first injectionPopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level
Effective haemostasis at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Any initial dose (1 hour), n=494
|
21.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Low initial dose (1 hour), n=156
|
16.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Intermediate initial dose (1 hr), n=127
|
25.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
High initial dose (1 hour), n=211
|
21.5 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Any initial dose (3 hours), n=494
|
36.9 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Low initial dose (3 hours), n=156
|
32.5 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Intermediate initial dose (3 hrs), n=127
|
41.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
High initial dose (3 hours), n=211
|
37.6 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Any initial dose (6 hours), n=494
|
50.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Low initial dose (6 hours), n=156
|
51.3 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
Intermediate initial dose (6 hrs), n=127
|
51.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)
High initial dose (6 hours), n=211
|
50.2 percentage of bleed episodes
|
SECONDARY outcome
Timeframe: 1 hour, 3 hours and 6 hours, respectively, after first injectionPopulation: Analysis set is spontaneous bleed episodes treated with any initial dose
Effective haemostasis at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Any initial dose (1 hour), n=324
|
19.3 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Low initial dose (1 hour), n=92
|
7.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Intermediate initial dose (1 hr), n=91
|
24.4 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
High initial dose (1 hour), n=141
|
23.6 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Any initial dose (3 hours), n=324
|
36.9 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Low initial dose (3 hours), n=92
|
27.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Intermediate initial dose (3 hrs), n=91
|
40.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
High initial dose (3 hours), n=141
|
40.3 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Any initial dose (6 hours), n=324
|
48.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Low initial dose (6 hours), n=92
|
43.3 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
Intermediate initial dose (6 hrs), n=91
|
50.5 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)
High initial dose (6 hours), n=141
|
49.6 percentage of bleed episodes
|
SECONDARY outcome
Timeframe: 1 hour, 3 hours and 6 hours, respectively, after first injectionPopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level
Effective pain relief at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Any initial dose (1 hour), n=494
|
27.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Low initial dose (1 hour), n=156
|
29.9 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Intermediate initial dose (1 hr), n=127
|
19.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
High initial dose (1 hour), n=211
|
30.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Any initial dose (3 hours), n=494
|
53.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Low initial dose (3 hours), n=156
|
59.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Intermediate initial dose (3 hrs), n=127
|
47.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
High initial dose (3 hours), n=211
|
51.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Any initial dose (6 hours), n=494
|
66.0 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Low initial dose (6 hours), n=156
|
77.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
Intermediate initial dose (6 hrs), n=127
|
57.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)
High initial dose (6 hours), n=211
|
62.3 percentage of bleed episodes
|
SECONDARY outcome
Timeframe: 1 hour, 3 hours and 6 hours, respectively, after first injectionPopulation: Analysis set is spontaneous bleed episodes treated with any initial dose
Effective pain relief at 3 different time points for spontaneous bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Any initial dose (1 hour), n=324
|
24.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Low initial dose (1 hour), n=92
|
24.4 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Intermediate initial dose (1 hr), n=91
|
18.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
High initial dose (1 hour), n=141
|
28.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Any initial dose (3 hours), n=324
|
49.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Low initial dose (3 hours), n=92
|
55.6 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Intermediate initial dose (3 hrs), n=91
|
44.0 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
High initial dose (3 hours), n=141
|
48.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Any initial dose (6 hours), n=324
|
62.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Low initial dose (6 hours), n=92
|
77.5 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
Intermediate initial dose (6 hrs), n=91
|
55.6 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)
High initial dose (6 hours), n=141
|
58.0 percentage of bleed episodes
|
SECONDARY outcome
Timeframe: individual bleed episodePopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level
The median number of injections required to treat individual bleed episodes.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Total Number of Injections (All Bleed Episodes)
Any initial dose, n=494
|
2 injections
Interval 1.0 to 3.0
|
|
Total Number of Injections (All Bleed Episodes)
Low dose, n=156
|
2 injections
Interval 2.0 to 3.0
|
|
Total Number of Injections (All Bleed Episodes)
Intermediate dose, n=127
|
2 injections
Interval 1.0 to 3.0
|
|
Total Number of Injections (All Bleed Episodes)
High dose, n=211
|
1 injections
Interval 1.0 to 2.0
|
SECONDARY outcome
Timeframe: individual bleed episodePopulation: Analysis set is spontaneous bleed episodes treated with any initial dose
The median number of injections required to treat individual bleed episodes.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Total Number of Injections (Spontaneous Bleed Episodes)
Any initial dose, n=324
|
2 injections
Interval 1.0 to 3.0
|
|
Total Number of Injections (Spontaneous Bleed Episodes)
Low dose, n=92
|
2 injections
Interval 2.0 to 3.0
|
|
Total Number of Injections (Spontaneous Bleed Episodes)
Intermediate dose, n=91
|
2 injections
Interval 1.0 to 3.0
|
|
Total Number of Injections (Spontaneous Bleed Episodes)
High dose, n=141
|
1 injections
Interval 1.0 to 2.0
|
SECONDARY outcome
Timeframe: individual bleed episodePopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level. Excludes bleeds episodes with greater than 24 hours between 1st and 2nd rFVII dose where total cumulative dose recorded likely reflects treatment of re-bleeding or prophylactic/maintenance dosing
The median total cumulative dose required to treat individual bleed episodes.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)
Any initial dose, n=494
|
270 mcg/kg
Interval 43.0 to 2160.0
|
|
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)
Low dose, n=156
|
267 mcg/kg
Interval 43.0 to 1632.0
|
|
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)
Intermediate dose, n=127
|
256 mcg/kg
Interval 123.0 to 2160.0
|
|
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)
High dose, n=211
|
270 mcg/kg
Interval 250.0 to 1822.0
|
SECONDARY outcome
Timeframe: individual bleed episodePopulation: Analysis set is spontaneous bleed episodes. Excludes bleeds episodes with \> 24 hours between 1st and 2nd rFVII dose where total cumulative dose recorded likely reflects treatment of re-bleeding or prophylactic/maintenance dosing, and 2 bleed episodes categorised as CNS that would not meet criteria of mild to moderate spontaneous bleed episodes
The median total cumulative dose required to treat individual bleed episodes.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)
Any initial dose, n=324
|
184 mcg/kg
Interval 90.0 to 1632.0
|
|
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)
Low dose, n=92
|
284 mcg/kg
Interval 123.0 to 2160.0
|
|
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)
Intermediate dose, n=91
|
270 mcg/kg
Interval 250.0 to 1822.0
|
|
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)
High dose, n=141
|
270 mcg/kg
Interval 90.0 to 2160.0
|
SECONDARY outcome
Timeframe: duration of bleed episodePopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level
Patient rate of satisfaction with symptom relief for the bleed episode overall on a 7-point Likert scale. Completion of questionnaire was voluntary. A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured. Scale answers ranged from extremely satisfied to extremely dissatisfied. The percentage of bleed episodes for which patients reported "extremely satisfied", "very satisfied" or "satisfied" is presented.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)
Any dose, n=494
|
72.7 percentage of bleed episodes
|
|
Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)
Low dose, n=156
|
82.1 percentage of bleed episodes
|
|
Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)
Intermediate dose, n=127
|
69.3 percentage of bleed episodes
|
|
Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)
High dose, n=211
|
67.8 percentage of bleed episodes
|
SECONDARY outcome
Timeframe: duration of bleed episodePopulation: Analysis set is spontaneous bleed episodes treated with any initial dose
Patient rate of satisfaction with symptom relief for the bleed episode overall on a 7-point Likert scale. Completion of questionnaire was voluntary. A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured. Scale answers ranged from extremely satisfied to extremely dissatisfied. The percentage of bleed episodes for which patients reported "extremely satisfied", "very satisfied" or "satisfied" is presented.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)
Any dose, n=324
|
68.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)
Low dose, n=92
|
75.0 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)
Intermediate dose, n=91
|
69.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)
High dose, n=141
|
63.1 percentage of bleed episodes
|
SECONDARY outcome
Timeframe: duration of bleed episodePopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level
Rate of ease of use related to activated recombinant human factor VII on a 7-point Likert scale. Completion of questionnaire was voluntary. A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured. Scale answers ranged from extremely difficult to extremely easy. The percentage of bleed episodes for which patients reported "extremely easy", "very easy" or "easy" is presented.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)
Any dose, n=494
|
74.9 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)
Low dose, n=156
|
84.6 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)
Intermediate dose, n=127
|
74.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)
High dose, n=211
|
67.8 percentage of bleed episodes
|
SECONDARY outcome
Timeframe: duration of bleed episodePopulation: Analysis set is spontaneous bleed episodes treated with any initial dose
Rate of ease of use related to activated recombinant human factor VII on a 7-point Likert scale. Completion of questionnaire was voluntary. A Likert scale is an ordered, multiple choice questionnaire from which respondents choose one option that best aligns with their view of the particular outcome being measured. Scale answers ranged from extremely difficult to extremely easy. The percentage of bleed episodes for which patients reported "extremely easy", "very easy" or "easy" is presented.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)
Any dose, n=324
|
69.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)
Low dose, n=92
|
81.5 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)
Intermediate dose, n=91
|
69.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)
High dose, n=141
|
61.0 percentage of bleed episodes
|
SECONDARY outcome
Timeframe: duration of bleed episodePopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level
Median time to achievement of haemostasis/bleed cessation calculated using Kaplan Meier life table methods. If approximately 50% or less of bleeds achieved the endpoint in a given initial dose subgroup, the median cannot be calculated and it is reported as not applicable
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)
Low dose, n=156
|
720 minutes
Interval 540.0 to 1430.0
|
|
Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)
Any dose, n=494
|
1200 minutes
Interval 720.0 to
Using Kaplan-Meier methods to calculate the median time to event takes into account the censoring of data at the upper part of the distribution. If the upper limit falls to the right of the censoring time, then it will be missing
|
|
Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)
Intermediate dose, n=127
|
NA minutes
Since 50% or less of bleeds achieved the endpoint in the intermediate dose subgroup, the median cannot be calculated and it is reported as "not applicable" (NA)
|
|
Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)
High dose, n=211
|
1200 minutes
Interval 660.0 to
Using Kaplan-Meier methods to calculate the median time to event takes into account the censoring of data at the upper part of the distribution. If the upper limit falls to the right of the censoring time, then it will be missing
|
SECONDARY outcome
Timeframe: duration of bleed episodePopulation: Analysis set is spontaneous bleed episodes treated with any initial dose
Median time to achievement of haemostasis/bleed cessation calculated using Kaplan Meier life table methods. If approximately 50% or less of bleeds achieved the endpoint in a given initial dose subgroup, the median cannot be calculated and it is reported as not applicable
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)
Low dose, n=92
|
600 minutes
Interval 540.0 to
Using Kaplan-Meier methods to calculate the median time to event takes into account the censoring of data at the upper part of the distribution. If the upper limit falls to the right of the censoring time, then it will be missing
|
|
Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)
Any dose, n=324
|
1200 minutes
Interval 600.0 to
Using Kaplan-Meier methods to calculate the median time to event takes into account the censoring of data at the upper part of the distribution. If the upper limit falls to the right of the censoring time, then it will be missing
|
|
Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)
Intermediate dose, n=91
|
NA minutes
Since 50% or less of bleeds achieved the endpoint in the intermediate dose subgroup, the median cannot be calculated and it is reported as "not applicable" (NA)
|
|
Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)
High dose, n=141
|
1200 minutes
Interval 540.0 to
Using Kaplan-Meier methods to calculate the median time to event takes into account the censoring of data at the upper part of the distribution. If the upper limit falls to the right of the censoring time, then it will be missing
|
SECONDARY outcome
Timeframe: duration of bleed episodePopulation: Analysis set is spontaneous bleed episodes treated with any initial dose, excluding bleed episodes treated with more than one dose for which \> 24 hours between the 1st and 2nd dose was reported (bleed episodes with \> 24 hours between 1st and 2nd rFVIIa dose likely reflects treatment of re-bleeding or prophylactic/maintenance dosing)
Median time to achievement of haemostasis/bleed cessation calculated using Kaplan Meier life table methods. If approximately 50% or less of bleeds achieved the endpoint in a given initial dose subgroup, the median cannot be calculated and it is reported as not applicable.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)
Low dose, n=87
|
600 minutes
Interval 540.0 to
Using Kaplan-Meier methods to calculate the median time to event takes into account the censoring of data at the upper part of the distribution. If the upper limit falls to the right of the censoring time, then it will be missing
|
|
Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)
Any dose, n=270
|
600 minutes
Interval 480.0 to 780.0
|
|
Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)
Intermediate dose, n=80
|
NA minutes
Since 50% or less of bleeds achieved the endpoint in the intermediate dose subgroup, the median cannot be calculated and it is reported as "not applicable" (NA)
|
|
Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)
High dose, n=103
|
480 minutes
Interval 330.0 to 720.0
|
SECONDARY outcome
Timeframe: Baseline (week 0) and and registry discontinuation (up to 28 months)Population: A subset of paediatric subjects age 4 to 16 (inclusive) included in the study that completed the questionnaire at baseline and/or at discontinuation
The Haemo-QoL is a specific multidimensional validated and reliable questionnaire used to assess quality of life in patients with haemophilia. Scores are reported on a 0 to 100 scale-higher scores indicate more impairment.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=7 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score
Baseline, n=7
|
47.49 scores on a scale
Standard Deviation 11.08
|
|
Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score
Discontinuation, n=7
|
54.12 scores on a scale
Standard Deviation 10.19
|
SECONDARY outcome
Timeframe: Baseline (week 0) and and registry discontinuation (up to 28 months)Population: A subset of adult subjects above 16 years included in the study that completed the questionnaire at baseline and/or at discontinuation
The adult Haemo-QoL-A is a specific multidimensional validated and reliable questionnaire used to assess quality of life in patients with haemophilia. Scores are reported on a 0 to 100 scale-higher scores indicate more impairment.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=11 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score
Baseline, n=11
|
35.31 scores on a scale
Standard Deviation 9.69
|
|
Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score
Discontinuation, n=10
|
34.46 scores on a scale
Standard Deviation 12.14
|
OTHER_PRE_SPECIFIED outcome
Timeframe: within 9 hours after first injectionPopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level
Percentage of bleeds with effective haemostasis within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "effective" = bleed resolved or substantially improved; "partially effective" = bleed with some improvement; "ineffective" = bleed with no change or with worsening.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Any, effective
|
61.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Any, partially effective
|
26.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Any, ineffective
|
11.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Low, effective
|
67.5 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Low, partially effective
|
21.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Low, ineffective
|
11.3 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Intermediate, effective
|
62.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Intermediate, partially effective
|
31.5 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
Intermediate, ineffective
|
6.5 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
High, effective
|
57.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
High, partially effective
|
27.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (All Bleed Episodes)
High, ineffective
|
15.1 percentage of bleed episodes
|
OTHER_PRE_SPECIFIED outcome
Timeframe: within 9 hours after first injectionPopulation: Analysis set is spontaneous bleed episodes treated with any initial dose
Percentage of bleeds with effective haemostasis within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "effective" = bleed resolved or substantially improved; "partially effective" = bleed with some improvement; "ineffective" = bleed with no change or with worsening.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Any, effective
|
59.0 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Any, partially effective
|
29.3 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Any, ineffective
|
11.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Low, effective
|
62.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Low, partially effective
|
21.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Low, ineffective
|
16.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Intermediate, effective
|
60.4 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Intermediate, partially effective
|
36.3 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
Intermediate, ineffective
|
3.3 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
High, effective
|
56.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
High, partially effective
|
29.5 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleed) by Dose Level (Spontaneous Bleed Episodes)
High, ineffective
|
14.4 percentage of bleed episodes
|
OTHER_PRE_SPECIFIED outcome
Timeframe: within 9 hours after first injectionPopulation: Analysis set is all bleed episodes, regardless of bleed type or initial dose level
Percentage of bleeds with effective pain relief within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "Better" = pain resolved or decreased substantially; "Same" = no change; "Worsened" = pain worsening.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Any, better
|
71.3 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Any, same
|
25.6 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Any, worsened
|
3.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Low, better
|
60.0 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Low, same
|
39.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Low, worsened
|
0.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Intermediate, better
|
82.4 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Intermediate, same
|
15.0 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
Intermediate, worsened
|
2.6 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
High, better
|
69.9 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
High, same
|
25.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (All Bleed Episodes)
High, worsened
|
4.9 percentage of bleed episodes
|
OTHER_PRE_SPECIFIED outcome
Timeframe: within 9 hours after first injectionPopulation: Analysis set is spontaneous bleed episodes treated with any initial dose
Percentage of bleeds with effective pain relief within 9 hours of first injection of activated recombinant human factor VII. Patient reported assessments were sorted into 3 sub-categories: "Better" = pain resolved or decreased substantially; "Same" = no change; "Worsened" = pain worsening.
Outcome measures
| Measure |
Activated Recombinant Human Factor VII
n=85 Participants
Male patients above 2 years of age with haemophilia A or B who have developed inhibitors and have been prescribed on-demand treatment of activated recombinant human factor VII at any dose for treatment of mild to moderate spontaneous bleeds
|
|---|---|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Any, better
|
68.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Any, same
|
28.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Any, worsened
|
3.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Low, better
|
79.8 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Low, same
|
16.9 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Low, worsened
|
3.4 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Intermediate, better
|
58.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Intermediate, same
|
40.7 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
Intermediate, worsened
|
1.1 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
High, better
|
67.2 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
High, same
|
28.5 percentage of bleed episodes
|
|
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Initial Dose (Spontaneous Bleed Episodes)
High, worsened
|
4.4 percentage of bleed episodes
|
Adverse Events
Activated Recombinant Human Factor VII
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If an investigator wishes to publish information from the study, a copy of the manuscript must be provided to Novo Nordisk for review at least 60 days before submission for publication or presentation. The investigator will recognize the integrity of a multi-centre study by not publishing data derived from the individual site until the combined results from the completed registry have been published in full, or the sponsor confirms there will be no multi-centre registry publication.
- Publication restrictions are in place
Restriction type: OTHER