Beijing Children's Hospital, Capital Medical University
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 55
- 试验地点
- 1
- 主要终点
- Success rate
研究概览
简要总结
The study start on January 18, 2017. The Severe(FⅧ<1%) and moderate hemophilia A (FⅧ1%～5%)children with high titer inhibitor(historical peak inhibitor titer≥5BU ) combining with poor ITI risk(s) were enrolled. The low-dose ITI was alone or combined with immunosuppression.
详细描述
Poor risk(s) includes:①peak historical inhibitor titer≥200BU ②inhibitor titer≥10BU before ITI initiation ③peak inhibitor titer during ITI≥200BU ④time to titer decline to<10BU before ITI≥24 months ⑤age≥8 years at start of ITI ⑥ITI initiated ≥5 years after inhibitor diagnosis ⑦interruptions in ITI≥2 weeks in duration. The low-dose ITI strategy consist of FⅧ（25-50IU/kg）alone or combining with immunosuppression: prednisone and Rituximab when the inhibitor titer ≥40BU ml/ml before or during ITI.
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
- 1 Year 至 14 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •from 1 to 14 years old
- •severe or moderate hemophilia A;
- •inhibitors positive before ITI started.
排除标准
- •<1 or >14 years old
- •hemophilia B or mild haemophilia A;
- •inhibitor negative before ITI started.
研究组 & 干预措施
ITI strategy
The low-dose ITI was Coagulation Factor VIII (50IU/kg, every other day) alone or combined with prednisone (2mg Kg-1/day, one month, then taper in three months) depending on the tendency of inhibitor, and Rituximab (375mg/square meter every week for 4 weeks) when the inhibitor titer ≥40BU/ml before or during ITI.Inhibitor and hemorrhage should be retested and recorded periodically.
干预措施: Coagulation Factor VIII (Drug)
ITI strategy
The low-dose ITI was Coagulation Factor VIII (50IU/kg, every other day) alone or combined with prednisone (2mg Kg-1/day, one month, then taper in three months) depending on the tendency of inhibitor, and Rituximab (375mg/square meter every week for 4 weeks) when the inhibitor titer ≥40BU/ml before or during ITI.Inhibitor and hemorrhage should be retested and recorded periodically.
干预措施: Prednisone (Drug)
ITI strategy
The low-dose ITI was Coagulation Factor VIII (50IU/kg, every other day) alone or combined with prednisone (2mg Kg-1/day, one month, then taper in three months) depending on the tendency of inhibitor, and Rituximab (375mg/square meter every week for 4 weeks) when the inhibitor titer ≥40BU/ml before or during ITI.Inhibitor and hemorrhage should be retested and recorded periodically.
干预措施: Rituximab (Drug)
结局指标
主要结局
Success rate
时间窗: 2 years
Success rate
次要结局
- Annualized Bleeding Rate(2 year)
- Success time(2 years)
- Annualized Joint Bleeding Rate(2 year)
研究者
Runhui WU
Capital Medical Univercity
Beijing Children's Hospital