An observational study in Bone Marrow Failure Syndrome, sponsored by Assistance Publique - Hôpitaux de Paris. Recruiting at 1 site in France. Per ClinicalTrials.gov, last updated 2021-03-04.
Sponsored by Assistance Publique - Hôpitaux de Paris · Observational
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:
Research:
85 studies on the registry are indexed under Bone Marrow Failure Disorders; 34 are open to participants now.
This study's planned enrollment of 5,000 is above the median of 350 across 19 observational studies indexed under Bone Marrow Failure Disorders.
Browse Bone Marrow Failure Disorders studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,576 studies on the registry; 1,028 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients with bone marrow failure syndromes (BMF)
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
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
Standard of care of patients with bone marrow failure
Other: Bone Marrow Failure
without interventional intervention . biological collection in the routine care Standard of care of patients with bone marrow failure.
Prevalence of bone marrow failure
Time frame: at 10 years
Distribution of different bone marrow failure syndromes
Time frame: at 10 years
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 inclusion
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 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
Started Feb 2017 (actual). Primary completion is estimated for Feb 2027, 120 months after the start.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Bone Marrow Failure Disorders →
Assistance Publique - Hôpitaux de Paris →