The European Paediatric Network for Haemophilia Management and the PedNet Haemophilia Registry
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 4,000
- 试验地点
- 39
- 主要终点
- Number of patients with antibody development to exogenous clotting factors
研究概览
简要总结
Rationale:
Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias.
Objective:
To collect data on bleeding during neonatal period, endogenous (genetic) and exogenous (treatment-related) determinants of inhibitor development and long term outcome.
详细描述
Design: Multicenter Prospective Observational Birth Cohort Study
Population:
Patients with haemophilia A and B with FVIII/IX levels of <1 to 25% born between 1-1-2000 and 1-1-2040.
Intervention:
No intervention; only documentation of patient characteristics and parameters of routine patient care and outcome
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosed with Haemophilia A or B
- •Factor VIII/ IX activity of <1 to 25%
- •Complete records of Factor treatment and bleeds
- •Treated in one of the participating centres
排除标准
- •Patients referred because of an inhibitor*
- •Informed consent not obtained
结局指标
主要结局
Number of patients with antibody development to exogenous clotting factors
时间窗: Until patient reaches age of 18
Allo-antibodies against Factor VIII and IX; Blood test: measurement in Bethesda units (BU), positive according to local standards, for most labs \>0.5 BU
次要结局
- Long term outcome of haemophilia on joint status using the Hemophilia Joint Health Score (HJHS) and MRI techniques.(From diagnose every 5 years until patient reaches age of 18)
- Long term outcome different Immune Tolerance Induction (ITI) therapies in patients with inhibitor.(From date first positive inhibitor titer preferably every 3 years until patient reaches age of 18)
研究者
Gili Kenet
Director
PedNet Haemophilia Research Foundation