Treatment of Glanzmann's Thrombasthenia: A Prospective Observational Registry
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 218
- 试验地点
- 1
- 主要终点
- For surgery including invasive and dental procedures: Haemoglobin level
研究概览
简要总结
This observational registry is conducted in Europe, Asia, Africa and the United States of America (USA).
The purpose of the registry is to evaluate the efficacy and safety of activated recombinant human factor VII (rFVIIa) during bleeding episodes and for the prevention of bleeding during invasive procedures/surgery in patients with Glanzmann's thrombasthenia (GT) with past or present refractoriness to platelet transfusions. Attention will be directed towards complications related to thrombo-embolic events and concomitant medications especially antifibrinolytics.
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
- •Patients with congenital GT defined as patients with lifelong bleeding tendency characterised by impaired or absent platelet aggregation, impaired clot retraction and prolonged bleeding time or prolonged platelet function analyser closure time. The patient has normal platelet counts and platelet morphology. Optional diagnosis criteria are quantitative or qualitative evaluation of GP (Glycoprotein) IIb/IIIa receptor including flow cytometry and identification of gene defects
- •Signed informed consent by the patient or next of kin or legally acceptable representative to collect data on treatment of a given bleeding episode or surgical event as specified in the protocol. If informed consent is provided by the next of kin or legally acceptable representative, consent must also be obtained from the patient as soon as he/she is able to do so. Informed consent must be obtained before entry of data into the registry
排除标准
- •Patients with acquired thrombasthenic states caused by autoimmune disorders (acute or chronic) or drugs
研究组 & 干预措施
A
干预措施: activated recombinant human factor VII (Drug)
结局指标
主要结局
For surgery including invasive and dental procedures: Haemoglobin level
时间窗: prior to surgery and 24 hours after surgery
For surgery including invasive and dental procedures: Overall haemostatic evaluation by the surgeon
时间窗: 24 hours after surgery
For bleeding episodes: Overall efficacy evaluated by the caregiver/patient
时间窗: within 30 days of end of treatment
次要结局
- Adverse Events (AEs)(during treatment episodes)
- Changes in laboratory parameters (prothrombin time, platelet count, fibrinogen), if available(at the time of administration and two hours after the administration of rFVIIa)
- Serious Adverse Events (SAEs)(during treatment episodes)