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

Novo Nordisk A/S102 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

102

Conditions

Congenital Bleeding Disorder Haemophilia A With Inhibitors Haemophilia B With Inhibitors

Interventions

eptacog alfa (activated)DRUG

Treatment of patients experiencing bleeds at the discretion of the physician/caregiver

Eligibility

Sex: MALE

Medical Language ↔ Plain English

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that the patient be somehow lost-to-follow-up over the course of registry participation (not applicable if patient is withdrawn) Exclusion Criteria: * Known hypersensitivity to the active substance or the excipients in the formulation of activated recombinant human factor VII, or to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

Locations (16)

Novo Nordisk Investigational Site

Algiers, Algeria

Novo Nordisk Investigational Site

Vienna, Austria

Novo Nordisk Investigational Site

Outcomes

Primary Outcomes

Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (All Bleed Episodes)

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.

Time frame: within 9 hours of first injection

Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)

Time frame: within 9 hours of first injection

Percentage of Bleed Treatments Resulting in Effective Pain Relief (All Bleed Episodes)

The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.

Time frame: within 9 hours of first injection

Percentage of Bleed Treatments Resulting in Effective Pain Relief (Spontaneous Bleed Episodes)

The percentage of participants with effective pain relief. Pain relief was a subjective assessment made by the patient during treatment of a bleed episode.

Time frame: within 9 hours of first injection

Secondary Outcomes

Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (All Bleed Episodes)

Effective haemostasis at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.

Time frame: 1 hour, 3 hours and 6 hours, respectively, after first injection

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Novo Nordisk Investigational Site

Prague, Czechia

Novo Nordisk Investigational Site

Paris La Défense Cedex, France

Novo Nordisk Investigational Site

Mainz, Germany

Novo Nordisk Investigational Site

Rome, Italy

Novo Nordisk Investigational Site

Alphen aan den Rijn, Netherlands

Novo Nordisk Investigational Site

Warsaw, Poland

Novo Nordisk Investigational Site

Paço de Arcos, Portugal

...and 6 more locations

Percentage of Bleed Treatments Resulting in Effective Haemostasis (Cessation of Bleeds) by Time Point (Spontaneous Bleed Episodes)

Effective haemostasis at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.

Time frame: 1 hour, 3 hours and 6 hours, respectively, after first injection

Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (All Bleed Episodes)

Effective pain relief at 3 different time points for all bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.

Time frame: 1 hour, 3 hours and 6 hours, respectively, after first injection

Percentage of Bleed Treatments Resulting in Effective Pain Relief by Time Point (Spontaneous Bleed Episodes)

Effective pain relief at 3 different time points for spontaneous bleeds. Patient reported outcomes are reported over 1 hour, 3 hours and 6 hours.

Time frame: 1 hour, 3 hours and 6 hours, respectively, after first injection

Total Number of Injections (All Bleed Episodes)

The median number of injections required to treat individual bleed episodes.

Time frame: individual bleed episode

Total Number of Injections (Spontaneous Bleed Episodes)

The median number of injections required to treat individual bleed episodes.

Time frame: individual bleed episode

Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)

The median total cumulative dose required to treat individual bleed episodes.

Time frame: individual bleed episode

Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)

The median total cumulative dose required to treat individual bleed episodes.

Time frame: individual bleed episode

Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)

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.

Time frame: duration of bleed episode

Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)

Time frame: duration of bleed episode

Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)

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.

Time frame: duration of bleed episode

Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)

Time frame: duration of bleed episode

Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)

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

Time frame: duration of bleed episode

Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)

Time frame: duration of bleed episode

Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)

Time frame: duration of bleed episode

Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score

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.

Time frame: Baseline (week 0) and and registry discontinuation (up to 28 months)

Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score

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.

Time frame: Baseline (week 0) and and registry discontinuation (up to 28 months)