French National Registry of Bone Marrow Failures: Prospective and Retrospective Database Associated to a Collection of Biological Samples: RIME Project
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 5,000
- 试验地点
- 1
- 主要终点
- Prevalence of bone marrow failure
研究概览
简要总结
This is a unique clinical and biological database that collects standardized clinical information during the management of all patients with bone marrow failure syndromes (BMF) in France (multicenter registry), from diagnosis and throughout follow-up during the natural history of the disease, treated or not. In parallel, biological samples (blood and/or bone marrow and/or skin) are collected during clinical care and are biobanked in Saint-Louis Hospital (Hematology laboratory) in order to be used in translational research related to bone marrow failure diseases.
This registry has two main objectives:
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Public health care evaluation and improvement: to assess the medical and social needs inherent to the management of these rare diseases; to precisely assess the level of diagnosis and management of bone marrow failure syndromes in France; to evaluate the impact and guidance of the French reference center guidelines for diagnosis and treatment; to evaluate the real-life efficacy and tolerance of any given specific treatments; to analyze treatment's cost-effectiveness according to each situation.
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Research:
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Epidemiology: to determine the incidence, prevalence, and distribution of different bone marrow failure syndromes at the national level;
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Biology: to better understand the pathophysiology of BMF; to identify and to study complications within each entity, such as mechanisms underlying clonal evolution, new forms of inherited BMF and acute myeloid leukemia (AML)/MDS-predisposition syndromes, and to better and deeper characterize known entities;
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Treatment: to identify prognostic factors and predictors of response; to identify side effects and impact of treatment on others organs and natural functions; to assess patients' quality of life as early as possible since diagnosis and throughout follow-up.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 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 diagnostic of BMF
- •Having given his non-opposition to registry after understand overall aims
- •Having signed a written informed consent (2 parents for patients aged less than 18) for collection of biological samples
- •With health insurance coverage
排除标准
- •With myelodysplastic syndrome occurring in a patient over the age of 50 in absence of genetical predispositions, familial forms and history of medullary hypoplasia
结局指标
主要结局
Prevalence of bone marrow failure
时间窗: at 10 years
Distribution of different bone marrow failure syndromes
时间窗: at 10 years
次要结局
- Prognostic factor ans treatment response(at 10 years)
- Proportion of patients with new forms of constitutional aplasias(at 10 years)
- Pathophysiology of bone marrow failures(at 10 years)
- Incidence and outcome of extra hematological complications including solid tumors, fertility(within 10 years)
- Quality of life assessed by EORTC QLQ-C30- v3 questionnaire(at 10 years)
- mechanisms underlying a clonal evolution or AML/MDS syndroms(at 10 years)
- Proportion of patients with complications within each entity(at 10 years)
- Global response to treatment(at 10 years)