Condition: Bone Marrow Failure Syndrome · Sponsor: Assistance Publique - Hôpitaux de Paris
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-p…
This description comes directly from the study's public registry record.
Régis Peffault De Latour · +33142385073 · regis.peffaultdelatour@aphp.fr
Matthieu Resche-Rigon · +3341499742 · matthieu.resche-rigon@u-paris.fr
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| Hématologie Greffe | Paris, France | Recruiting |
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Source record: clinicaltrials.gov/study/NCT04781790