Impact of Implementation of Prophylaxis With Emicizumab in Children With Severe Hemophilia A in Ivory Coast
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 33
- 试验地点
- 1
- 主要终点
- Change in annual bleeding rate
研究概览
简要总结
Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast
详细描述
Assessment of the outcomes of prophylaxis with Emicizumab in children with severe hemophilia A in Ivory Coast.
Duration of the study: 1 year after initiation of prophylaxis with Emicizumab
Inclusion criteria: boys aged > 2 years, affected with severe hemophilia A with or without inhibitors
Evaluated outcomes: annual bleeding rate, days of absence from school, the need for additional facteur VIII consumption, satisfaction about the treatment and quality of life.
Tools used to assess quality of life are the Ivoirian version of the Canadian Haemophilia Outcomes-Kids' Life Assessment Tool version 2 (CHO-KLAT) and EQ-5D
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
- 2 Years 至 18 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •severe hemophilia A with or without inhibitors -
排除标准
- •parents refusal
研究组 & 干预措施
Ivoirian Boys with severe Hemophilia A treated with Emicizumab
All Ivoirian boys with severe Hemophilia A (with and without inhibitors) on prophylaxis with Emicizumab
干预措施: Prophylaxis with Emicizumab (Drug)
结局指标
主要结局
Change in annual bleeding rate
时间窗: change in annual bleeding rate from baseline at 6 months and 12 months
Using the patients' logbook
Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D in children treated with Emicizumab
时间窗: Change in the scores of HR-QoL tools CHO-KLAT and EQ-5D at 6 months.
Using the CHO-KLAT and EQ-5D. The scales range from 0 to 100 with 0 representing the worst quality of life and 100 the best quality of life score
Satisfaction of the parents about the treatment with Emicizumab after 6 month
时间窗: Assessment at 6 months after initiation of Emicizumab
Usign the PGIC (scale ranging from extremely satisfied to extremely unsatisfied)
次要结局
未报告次要终点
研究者
Lambert Catherine
Clinical Professor. Hematology department
Cliniques universitaires Saint-Luc- Université Catholique de Louvain