Evaluation of Efficacy, Safety and Costs of Recombinant FVIII Products Applied to Severe Hemophilia A Patients: Observational Data Collection Study Evaluating On-demand Treatment and Secondary Prophylaxis
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- Bayer
- 入组人数
- 73
- 主要终点
- Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group
研究概览
简要总结
The project is an observational, multi-central, prospective, non-interventional and open-label data collection study on secondary prophylaxis with recombinant FVIII products in adolescents and adults with severe hemophilia A (FVIII < 1%).
It will be a controlled observation of patients on secondary prophylaxis versus on-demand treatment regimen. Patients will be enrolled preferably on a 1:1 basis with regards to prophylaxis and on-demand treatment.
The patient enrollment period will be 2 years with a follow-up (observation period) of 2 years for each patient. Based on the primary effectiveness parameters (joint bleeds and overall bleeds per year) an observation period of 2 years is considered sufficient although it has to be admitted that it is rather short to assess the progression of orthopedic status. Previously treated prophylaxis patients with at least 50 exposure days and patients with continuing prophylaxis treatment will be included.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 18 years
- •Severe hemophilia A (FVIII<1%) diagnosis
- •Prior treatment or ongoing treatment with on-demand or prophylaxis regimens according to Turkish guidelines and requirements
- •Previously treated patients with at least >50 exposure days
- •Written informed consent signed by patient/legal representative
排除标准
- •Currently on immune tolerance treatment
- •Platelet count < 75,000/mm3
- •Participation in another study
- •Existence of inhibitors in the past and in the last currently available blood sample prior to study start (Bethesda titer < 0.6 BU/ml)
- •Existence of inhibitor history in family members who also are diagnosed with hemophilia A
- •Having been on primary prophylaxis as defined in the introduction
- •Participation in another study
结局指标
主要结局
Mean ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group
时间窗: After 4 years
Median ±SD, range of number of joint bleeds per year of prophylaxis versus on-demand group
时间窗: After 4 years
次要结局
- Musculoskeletal evaluation recommended by World Federation of Hemophilia: Orthopedic Joint Score (Gilbert Score)(After 4 years)
- Number of overall bleeding episodes(After 4 years)
- Cost-utility(After 4 years)
- Quality of life as measured with the SF-36(Baseline and after 4 years)
- Cost-effectiveness (cost of additional joint bleed)(After 4 years)
- Number of spontaneous bleeds(After 4 years)
- Quality of life as measured with Hemo-QoL(Baseline and after 4 years)
- Musculoskeletal evaluation recommended by World Federation of Hemophilia: Radiological evaluation (Pettersson Score)(After 4 years)
- Comparison of patient compliance between prophylaxis and on-demand therapy groups(After 4 years)