Moderate Term Musculoskeletal Outcomes With Escalating Dose Prophylaxis: the Canadian Hemophilia Prophylaxis Study Follow-up Study
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 56
- 试验地点
- 2
- 主要终点
- Number of Participants Who Developed Target Joint Bleeding
研究概览
简要总结
Primary prophylaxis given less frequently initially, with the infusion frequency increased if needed (Escalating Dose Prophylaxis), is likely to be less expensive and associated with fewer complications than standard prophylaxis while reducing disability to a greater degree than intermittent therapy.
详细描述
There are 2 specific study objectives. The first is to estimate the incidence of target joint bleeding in patients with severe hemophilia A treated (for primary prophylaxis) with Escalating Dose Prophylactic factor replacement. The second objective is to obtain accurate estimates of the direct and indirect costs associated with this protocol for use in a cost-effectiveness model (comparing Escalating Dose with standard prophylaxis and with intermittent therapy).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 12 Months 至 30 Months（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Severe hemophilia A (factor level less than 2%).
- •Age greater than 1 year and less than or equal to 2.5 years.
- •Normal joints using the World Federation of Hemophilia orthopedic scale.
- •Normal radiographs of joints in which bleeding has occurred using the World Federation of Hemophilia radiographic scale.
- •Platelet count of > 150,
- •Informed consent to participate.
排除标准
- •Three or more clinically determined bleeds into any single elbow, knee or ankle.
- •Presence or past history of a circulating inhibitor (level ≥ 0.5 Bethesda Units).
- •Family judged to be non-compliant by the local hemophilia clinic director.
- •Competing risk (symptomatic HIV infection, juvenile rheumatoid arthritis, metabolic bone disease, or other diseases known to cause or mimic arthritis.)
结局指标
主要结局
Number of Participants Who Developed Target Joint Bleeding
时间窗: 6 months
The number of participants who developed target joint bleeding during the study, which was defined as 3 bleeds into any 1 joint within a period of 3 months.
次要结局
- Number of Patients Who Developed an Inhibitor to FVIII(6 months)
- Complications Arising From Indwelling Venous Catheter(6 months)
- Annualized Factor Use(12 months)
- Annualized Bleeding Rate(6 months)
- Joint Damage as Determined by the Physiotherapy Score(through study completion, a median of 10 years)
- Physical Disability as Measured by the CHAQ(through study completion, a median of 10 years)
研究者
Brian Feldman
Division Head, Rheumatology
The Hospital for Sick Children