Informations générales (source: ClinicalTrials.gov)
French National Registry of Bone Marrow Failures: Prospective and Retrospective Database Associated to a Collection of Biological Samples: RIME Project (RIME)
Observational
Assistance Publique - Hôpitaux de Paris (Voir sur ClinicalTrials)
février 2017
février 2027
29 juin 2024
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.
Etablissements
| Les établissements d'Île-de-France ayant mis à jour leurs données Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| HOPITAL FOCH | YOLAND SCHOINDRE | 21/10/2024 07:07:20 | Contacter | ||
| HOPITAL NOVO | SARGHI | 04/07/2024 11:05:05 | Contacter | ||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| Hématologie Greffe - Paris - France | Regis PEFFAULT DE LATOUR | Contact (sur clinicalTrials) | |||
Critères
Tous
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
- 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
With myelodysplastic syndrome occurring in a patient over the age of 50 in absence of
genetical predispositions, familial forms and history of medullary hypoplasia