Observational Registry of NovoSeven® Used as On-demand Treatment of Bleeds in Patients With Haemophilia A and B With Inhibitors

Completed

Conditions: Congenital Bleeding Disorder
Haemophilia A With Inhibitors
Haemophilia B With Inhibitors

Interventions: Drug: eptacog alfa (activated)

Registration Number: NCT00703911

Lead Sponsor: Novo Nordisk A/S

Brief Summary: This study was conducted in Africa, Europe, the Middle-East and South America. The primary objective of this registry was to observe the use of single dose and multi-dose use of activated recombinant human factor VII and to compare short-term outcomes, including effectiveness, safety, quality of life and treatment satisfaction with the approved treatments.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: Male

Target Recruitment: 102

Inclusion Criteria

Diagnosed with haemophilia A or B with inhibitors
Experience mild to moderate spontaneous bleeds which require on-demand treatment and who are currently prescribed activated recombinant human factor VII
Be able and willing to provide informed consent (or proxy consent by caregiver, if applicable), as required by local research ethics committee, governmental or regulatory authorities
Be willing to provide information on at least one alternate contact person in the event that the patient be somehow lost-to-follow-up over the course of registry participation (not applicable if patient is withdrawn)

Exclusion Criteria

Known hypersensitivity to the active substance or the excipients in the formulation of activated recombinant human factor VII, or to mouse, hamster or bovine protein

Study & Design

Study Type: OBSERVATIONAL

Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
activated recombinant human factor VII	eptacog alfa (activated)	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

Primary Outcome Measures

Name	Time	Method
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)	within 9 hours of first injection	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.
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)	within 9 hours of first injection	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.
Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)	within 9 hours of first injection	The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.
Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)	within 9 hours of first injection	The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.

Secondary Outcome Measures

Name	Time	Method
Total Number of Injections (Spontaneous Bleed Episodes)	individual bleed episode	The median number of injections required to treat individual bleed episodes.
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)	1 hour, 3 hours and 6 hours, respectively, after first injection	Effective pain relief at 3 different time points for spontaneous bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
Total Number of Injections (All Bleed Episodes)	individual bleed episode	The median number of injections required to treat individual bleed episodes.
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)	duration of bleed episode	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.
Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)	duration of bleed episode	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
Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)	duration of bleed episode	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.
Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score	Baseline (week 0) and and registry discontinuation (up to 28 months)	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.
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)	1 hour, 3 hours and 6 hours, respectively, after first injection	Effective haemostasis at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)	1 hour, 3 hours and 6 hours, respectively, after first injection	Effective pain relief at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)	individual bleed episode	The median total cumulative dose required to treat individual bleed episodes.
Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)	1 hour, 3 hours and 6 hours, respectively, after first injection	Effective haemostasis at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)	duration of bleed episode	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.
Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)	duration of bleed episode	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
Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)	individual bleed episode	The median total cumulative dose required to treat individual bleed episodes.
Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)	duration of bleed episode	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.
Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)	duration of bleed episode	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.
Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score	Baseline (week 0) and and registry discontinuation (up to 28 months)	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.