French National Registry of Bone Marrow Failures

Condition(s) studied

Investigational drug(s) / intervention(s)

Bone Marrow Failure: without interventional intervention . biological collection in the routine care Standard of care of patients with bone marrow failure.

Study summary

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.

Eligibility

Primary outcome measure(s)

• Prevalence of bone marrow failure — at 10 years
• Distribution of different bone marrow failure syndromes — at 10 years

Trial sites (1)

More Assistance Publique - Hôpitaux de Paris trials in France