Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A. POTTER
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Bayer
- 入组人数
- 58
- 主要终点
- Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per year
研究概览
简要总结
The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII < 1%).
The observational period will cover at least 5 years per patient.
The long-term secondary prophylaxis group will be compared versus on-demand treatment group by the assessment of orthopedic status progression and pharmacoeconomics evaluation.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 12 Years 至 55 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •age ≥ 12 years and ≤ 55 years
- •severe haemophilia A (FVIII < 1%)
- •absence of inhibitors (Bethesda titre < 0.6 BU/ml)
- •Previous Treated Patients (prior exposure days > 200)
- •Kogenate Bayer administered 20-30 IU/kg -3 times a week, for the prophylaxis group
- •≥ 6 joint bleeds requiring treatment with FVIII concentrates in the previous 6 months before enrollment, for on-demand group
- •written informed consent
排除标准
- •concomitant severe and chronic diseases or congenital skeletal malformation
- •unreliability of patient or likelihood of follow-up failure
- •presence of inhibitors or history of inhibitors (in the previous 2 years)
- •currently on immune tolerance treatment
- •hepatic cirrhosis or liver disease in rapid progression
- •platelet count < 75,000/mm3
- •presence of conditions that influence negatively patient´s compliance
- •participation in another study
研究组 & 干预措施
Group 1
干预措施: Recombinant Factor VIII (Kogenate FS, BAY14-2222) (Drug)
Group 2
干预措施: Recombinant Factor VIII (Kogenate FS, BAY14-2222) (Drug)
结局指标
主要结局
Evaluate the efficacy, safety and pharmacoeconomic of long-term secondary prophylaxis with Kogenate Bayer in comparison to on-demand treatment with Kogenate Bayer in terms of joint bleeding episodes per year
时间窗: Every 6 months
次要结局
- Efficacy, safety, pharmacoeconomic impact of secondary prophylaxis versus on demand group in terms of overall bleeding episodes(Every 6 months)
- Muscolo skeletal evaluation by Orthopedic Joint Score(Every 12 months)
- Radiological Evaluation by Pettersson Score(Baseline and after 3-5 years)
- Health related quality of life(Every 12 months)
- cost-effectiveness and utility, patient compliance, adverse events(Every 6 months)