Multicenter Observational Ambispective Study for Congenital Platelet Disorders
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 200
- 试验地点
- 2
- 主要终点
- congenital platelet diseases
研究概览
简要总结
Inherited platelet disorders (IPD) are a heterogeneous group of rare bleeding diseases associated with a reduction of platelet number and/or function and with a bleeding tendency ranging from mild to severe. The frequency of inherited thrombocytopenias has been estimated to be 2.7/100,000 while the prevalence of inherited platelet function disorders is unknown, partly because they are frequently overlooked due to their difficult diagnosis.
详细描述
This a no-profit national multicenter ambispectic (retrospective and prospective) observational study.
After collection of informed consent form each patient, each center will enroll the patient and will collect general, laboratory and clinical data on an electronic CRF on a REDCAP platform. Each patient will receive a unique identification number. All clinical events will be reported in the data base.
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
- •All patients with established diagnosis of inherited number or function platelet disorder (IPN and IPD, respectively), on the basis of internationally established clinical, laboratory and genetic criteria will be enrolled.
- •For patients <18 ys, consensus will be signed by parents or legal responsible
排除标准
- •No written consent
- •Undocumented or uncertain diagnosis
结局指标
主要结局
congenital platelet diseases
时间窗: 48 months
The main objectives of the study are to collect information on the diagnosis and management of these rare disorders and to create clinical and scientific collaborations between participating centres, and the aim of this project is to create for the first time an ambiseptic multicentre database on clinical and laboratory data on patients with congenital platelet disorders and to observe the prevalence of different congenital platelet disorders
次要结局
- QoL(48 months)
研究者
De Candia Erica
Associate Professor of Internal Medicine
Fondazione Policlinico Universitario Agostino Gemelli IRCCS