Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A." POTTER-10
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Bayer
- 入组人数
- 43
- 主要终点
- Number of joint bleeding episodes per year
研究概览
简要总结
Following the performance of the POTTER observational study, whose primary objective was to collect data on the benefits of secondary prophylaxis versus on demand treatment in terms of prevention of bleeding episodes, the present study aims to extend the observation time up to 10 years, of the same population involved in the previous study.
The present study has been designed to allow a focus on long term disease-related damage at joint level in subjects with severe haemophilia A, as well as on the pharmacoeconomics impact of the two different treatment regimens.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 17 Years 至 60 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Completion of follow-up in the original Potter study without having being excluded from the Intention To Treat efficacy analysis of the primary endpoint;
- •Written informed consent specifically issued for the 5-year extension.
排除标准
- •Switching of treatment to a Factor VIII concentrate different from Kogenate Bayer/Helixate NexGen after the end of the previous follow-up period.
研究组 & 干预措施
Prophylaxis
Prophylaxis group: treatment is based on regularly repeated infusions of clotting factor, 20-30 IU/kg -3 times a week
干预措施: Recombinant Factor VIII (Kogenate FS, BAY14-2222) (Drug)
On-demand
On-demand group: treatment administered when bleeding episode occur
干预措施: Recombinant Factor VIII (Kogenate FS, BAY14-2222) (Drug)
结局指标
主要结局
Number of joint bleeding episodes per year
时间窗: 5 years period
次要结局
- Total amount of Recombinant Factor VIII consumption(5 years period)
- Total number of bleeding episodes per year(5 years period)
- Changes of prophylaxis dose(5 years period)
- Changes of frequency of infusions(5 years period)
- Musculoskeletal evaluation by calculating the World Federation of Haemophilia Orthopedic Joint Score (Gilbert score)(5 years period)
- Proportions of patients who will change the therapeutic regimen during the study(5 years period)
- Musculoskeletal evaluation by calculating the Radiological score (Pettersson Score)(5 years period)
- Evaluating the Questionnaires of Health-Related Quality of Life _Haemo Quality of Life (HaemoQol)(5 years period)
- Evaluating the Questionnaires of Health-Related Quality of Life _Short Form 36 questions (SF36)(5 years period)
- Evaluating the Questionnaires of Health-Related Quality of Life _ Euro Quality 5 Dimension (EQ5D)(5 years period)
- Number of days missed from work or school of patients and caregivers because of all the events haemophilia-related(5 years period)
- Number of patients with poor compliance(5 years period)
- Number of patients with adverse events(5 years period)