French National Registry of Bone Marrow Failures
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Bone Marrow Failure.
- Who it may be relevant to
- Registry conditions: Bone Marrow Failure Syndrome. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
French National Registry of Bone Marrow Failures: Prospective and Retrospective Database Associated to a Collection of Biological Samples: RIME Project
Overview
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: * 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. * Research: * Epidemiology: to determine the incidence, prevalence, and distribution of different bone marrow failure syndromes at the national level; * 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; * 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.
Interventions
- Other Bone Marrow Failure
without interventional intervention . biological collection in the routine care Standard of care of patients with bone marrow failure.
Primary outcome measures
- Prevalence of bone marrow failure [Time frame: at 10 years]
- Distribution of different bone marrow failure syndromes [Time frame: at 10 years]
Secondary outcome measures (9)
- Quality of life assessed by EORTC QLQ-C30- v3 questionnaire [Time frame: at inclusion]
- Quality of life assessed by EORTC QLQ-C30- v3 questionnaire [Time frame: at 10 years]
- mechanisms underlying a clonal evolution or AML/MDS syndroms [Time frame: at 10 years]
- Proportion of patients with new forms of constitutional aplasias [Time frame: at 10 years]
- Proportion of patients with complications within each entity [Time frame: at 10 years]
- Prognostic factor ans treatment response [Time frame: at 10 years]
- Pathophysiology of bone marrow failures [Time frame: at 10 years]
- Global response to treatment [Time frame: at 10 years]
- Incidence and outcome of extra hematological complications including solid tumors, fertility [Time frame: within 10 years]
Eligibility criteria
Inclusion criteria
- All age
- 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
Exclusion criteria
With myelodysplastic syndrome occurring in a patient over the age of 50 in absence of genetical predispositions, familial forms and history of medullary hypoplasia
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Other
Study locations
France · 1 center
- Hématologie Greffe — Paris
Identifiers
NCT: NCT04781790 · NI17045J