A Prospective Observational Registry on the Use of NovoSeven® (Activated Recombinant Human Factor VIIa) for on Demand Treatment of Mild to Moderate Bleeds in Haemophilia A and B Patients With Inhibitors
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 102
- 试验地点
- 1
- 主要终点
- Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 时间视角
- Prospective
入排标准
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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that the patient be somehow lost-to-follow-up over the course of registry participation (not applicable if patient is withdrawn)
排除标准
- •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
研究组 & 干预措施
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
干预措施: eptacog alfa (activated) (Drug)
结局指标
主要结局
Percentage of Bleed Treatments Resulting in Effective Bleed Resolution (Spontaneous Bleed Episodes)
时间窗: 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)
时间窗: 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)
时间窗: 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)
时间窗: 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.
次要结局
- Total Number of Injections (Spontaneous Bleed Episodes)(individual bleed episode)
- 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)
- Total Number of Injections (All Bleed Episodes)(individual bleed episode)
- Percentage of Bleed Treatments Resulting in Patient Satisfaction With Symptom Relief (Spontaneous Bleed Episodes)(duration of bleed episode)
- Overall Time to Cessation of Bleed/Achievement of Haemostasis (Spontaneous Bleed Episodes)(duration of bleed episode)
- Overall Time to Cessation/Achievement of Haemostasis (Spontaneous Bleed Episodes)(duration of bleed episode)
- Childrens' Health Related Quality of Life (Haemo-QoL): Overall Score(Baseline (week 0) and and registry discontinuation (up to 28 months))
- 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)
- 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)
- Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (All Bleed Episodes)(individual bleed episode)
- 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)
- Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (Spontaneous Bleed Episodes)(duration of bleed episode)
- Overall Time to Cessation of Bleed/Achievement of Haemostasis (All Bleed Episodes)(duration of bleed episode)
- Total Exposure (Cumulative Dose) to Activated Recombinant Human Factor VII (Spontaneous Bleed Episodes)(individual bleed episode)
- Percentage of Patients Reporting Satisfaction With Symptom Relief (All Bleed Episodes)(duration of bleed episode)
- Percentage of Bleed Treatments Resulting in Patient Satisfaction With Ease of Use (All Bleed Episodes)(duration of bleed episode)
- Adults' Health Related Quality of Life (Haemo-QoL-A): Overall Score(Baseline (week 0) and and registry discontinuation (up to 28 months))