"French National Cohort of Patients With Pediatric-onset of Autoimmune Cytopenia (OBS'CEREVANCE Cohort)"
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 3,500
- 试验地点
- 9
- 主要终点
- AIC context
研究概览
简要总结
From 2004, OBS'CEREVANCE is a national real-world prospective clinical cohort of patients with auto-immune cytopenia of pediatric-onset : Immune thrombocytopenia (ITP), Autoimmune Hemolytic anemia (AIHA), or Evans syndrome (all bi or tri cytopenias). Thanks to the collaboration of the 30 French pediatric hematologic centers, this cohort supports all of the Rare Disease Centre CEREVANCE (Centre de Référence National des Cytopénies Auto-Immunes de l'Enfant) missions for care, education and research. Specifically, this original unbiased database allows to describe the long-term health of adult patients, to identify the heterogenous genetic underlying pathophysiologic contexts, and to study the benefit-risk balance of treatments, including the growing development of targeted therapies.
详细描述
Immune thrombocytopenic purpura (ITP) and autoimmune hemolytic anemia (AHAI) are rare childhood diseases that involve autoimmune destruction of platelets and erythrocytes respectively.
They may be associated with an even rarer entity, Evans syndrome (ES). These three conditions are referred to as autoimmune cytopenias (AIC).
In association with CAI, patients may present with various immunopathological (IM) manifestations such as lymphoproliferation, autoimmune autoimmune/autoinflammatory organ diseases that may be absent at the time of at the time of diagnosis of CAI and develop during follow-up.
Since 2004, the CEREVANCE reference center for childhood autoimmune CEREVANCE has been coordinating a national prospective cohort of patients with pediatric-onset CAI including over 1900 patients (data 05/2023).
Thanks to the collaboration of the 30 French pediatric hematologic centers, this cohort supports all of the Rare Disease Centre CEREVANCE (Centre de Référence National des Cytopénies Auto-Immunes de l'Enfant) missions for care, education and research. Specifically, this original unbiased database allows to describe the long-term health of adult patients, to identify the heterogenous genetic underlying pathophysiologic contexts, and to study the benefit-risk balance of treatments, including the growing development of targeted therapies.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- — 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of ITP, AIHA, Evans Syndrome
- •Onset before the age of 18
排除标准
- •Opposition of legal representative or to data collection
结局指标
主要结局
AIC context
时间窗: Baseline
Number of patients with immunopathological manifestations (IM), systemic erythematosus lupus (SLE), primary immunodeficiency (PID).
次要结局
- Events(every 6 months after baseline up to 19 years)
- Treatment lines(every 6 months after baseline up to 19 years)
- Adverse drug reactions(every 6 months after baseline up to 19 years)
- AIC context(every 6 months after baseline up to 19 years)