Register für Schwere Immunzytopenien - Severe Immune Cytopenia Registry (SIC-Reg.Org)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 80
- 试验地点
- 1
- 主要终点
- underlying disease that causes or is associated with severe immune cytopenia
研究概览
简要总结
Prospective registry study for children and young adults with severe immune cytopenias (persisting/chronic immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome) to improve the management, facilitate the differential diagnostic work-up, and document the clinical course under various treatments.
Time points: at inclusion, after 6 months, after 12 months, then yearly up to 4 years after inclusion.
No intervention, mere observation and documentation. Guided pre-inclusion (differential) diagnostic work-up.
详细描述
The study aims to improve the management and care of patients with severe immune cytopenias, to identify underlying causes of severe immune cytopenias and to develop a strategy for early treatment stratification based on a standardized diagnostic algorithm, potentially supported by biomarker analyses and (off study) genetic analyses, where clinically indicated.
Primary Goal:
Rapid detection of underlying causes of severe immune cytopenias with the aid of a structured diagnostic approach and access to a clinical care network of the participating centers, allowing early treatment stratification
Secondary Goals:
- Collection of data about epidemiology of rare diseases
- Systemic documentation of response rates to various treatments
- Identification of biomarkers and modifiers of immune tolerance
- Collection of data about the usage of novel/experimental therapeutic agents
- Documentation of physician-reported outcome measures/performance scores
- Consultation of the caring physicians through a regular SIC-Reg board
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
- 6 Months 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Autoimmune hemolytic anemia (AIHA)
- •Evans syndrome (ES)
- •Persistent or chronic immune thrombocytopenia (ITP; >6 months after first manifestation)
排除标准
- •(history of) malignancies
- •(history of) hematopoietic stem cell transplantation
结局指标
主要结局
underlying disease that causes or is associated with severe immune cytopenia
时间窗: 0-4 years
identify the underlying condition or other disease, e.g., primary immunodeficiency or bone marrow failure syndrome by diagnostic procedures according to a standardized algorithm
次要结局
- Clinical course(0-4 years)
- Biomarkers - Blood(0-4 years)
- Routine laboratory parameters(0-4 years)
- Biomarkers - Stool(0-4 years)
- Number of participants with the diagnosis of severe immune cytopenia per participating centre and per year(0-4 years)