French National Registry of Bone Marrow Failures
ConditionBone Marrow Failure Syndrome
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorAssistance Publique - Hôpitaux de Paris
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
None
Trial population
Patients with bone marrow failure syndromes (BMF)
Trial design
Design model
Other
Time perspective
Prospective
Treatments tested in this trial
Bone Marrow Failure
Other interventionwithout interventional intervention . biological collection in the routine care Standard of care of patients with bone marrow failure.
Treatment groups
5,000 Participants
are divided into 1 treatment groupGroup A: Patients with Bone marrow failure1 intervention
Trial outcomes
Primary outcomes
1
Prevalence of bone marrow failure
Time frame
at 10 years2
Distribution of different bone marrow failure syndromes
Time frame
at 10 yearsSecondary outcomes
1
Quality of life assessed by EORTC QLQ-C30- v3 questionnaire
Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems. EORTC QLQ-C30 Scoring Manual. Fayers PM et al. on behalf of the EORTC Quality of Life Group. EORTC, 2001. ISBN: 2-9300.
Time frame
at inclusion2
Quality of life assessed by EORTC QLQ-C30- v3 questionnaire
Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems. EORTC QLQ-C30 Scoring Manual. Fayers PM et al. on behalf of the EORTC Quality of Life Group. EORTC, 2001. ISBN: 2-9300.
Time frame
at 10 years3
mechanisms underlying a clonal evolution or AML/MDS syndroms
Time frame
at 10 years4
Proportion of patients with new forms of constitutional aplasias
Time frame
at 10 yearsSponsors and contacts
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Source ClinicalTrials.gov