Clinical trial NCT06956755: Registry of Myelodysplastic Syndromes and Therapy-related Acute Myeloid Leukemia
Official title: Registry of Myelodysplastic Syndromes and Therapy-related Acute Myeloid Leukemia
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Myelodysplastic Syndromes
Leukemia, Myeloid, Acute
Keywords: MDS
t-AML
Lead sponsor: Groupe Francophone des Myelodysplasies
Sponsor class: OTHER
First posted: 2025-05-04
Enrollment: 6990
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Included in the "Registry" are all patients over 18 years of age with myelodysplastic syndrome and therapy-related acute myeloid leukemia

Brief summary: The Registry MDS is an ongoing, observational study that has collected longitudinal data on diagnostics, demographics, clinical parameters, and health Care Interventions (HCI) from patients with MDS and therapy-related acute myeloid leukemia

Detailed description: The Registry MDS is an ongoing, observational study that has collected longitudinal data on diagnostics, demographics, clinical parameters, and health Care Interventions (HCI) from patients with MDS and therapy-related acute myeloid leukemia.

This registry was created in July 2003 by GFM Group. It is conducted in accordance with the French law on data processing and freedom relating to the processing of personal data in the field of health (law n°78-17 of 6 January 1978 as amended). It has received the favorable opinion of the ethics committee for research and the authorization of the National Commission on Informatics and Freedoms. (CNIL) Included in the registry are all patients over 18 years of age with MDS or secondary chemo- and/or radio-induced leukemia diagnosed after July 2003.

The registry allows, after obtaining the patient's consent, the anonymous recording of his or her demographic (sex, age), epidemiological (medical and family history, exposure to toxicants), clinical, biological, cytological and cytogenetic data. Included in the registry are all patients over 18 years of age with MDS or secondary chemo- and/or radio-induced leukemia diagnosed after July 2003.

The registry allows, after obtaining the patient's consent, the anonymous recording of his or her demographic (sex, age), epidemiological (medical and family history, exposure to toxicants), clinical, biological, cytological and cytogenetic data.

To date, 70 GFM centers participate in this registry, 5300 patients have been included. Approximately 400 to 500 new patients are currently included per year.

Data are recorded and entered through the web-based e-CRF by haematology centers in 70 centers of GFM at inclusion and Follow-up of the patients every six months.

Data analyses are conducted by the SBIM (Medical and Biostatistical Computing at service of Pr Sylvie CHEVRET, Hospital Saint Louis). Data quality control including monitoring of both clinical execution and data collection implemented from the initiation of the Registry has resulted in high quality data.

Registry MDS has included the most patients and has proven itself as a well-established operational, and constantly evolving project in an elderly population.

The registration of new patients is still ongoing, and the follow-up of the project is unlimited.

Eligibility criteria: Inclusion Criteria:

* Male or female
* Age \> 18 years
* Patients with myelodysplastic syndrome and therapy-related acute myeloid leukemia
* Able and willing to provide written informed consent

Exclusion Criteria:

* Age \<18 years
* Patient is unwilling or unable to give consent

Primary outcomes:
measure: Hemoglobin level Unit of Measure g/dL
timeFrame: At enrollment
measure: Absolute neutrophil count / Unit of Measure G/L
timeFrame: At enrollment
measure: Platelet count Unit of Measure / Unit of Measure: %G/L
timeFrame: At enrollment
measure: Percentage of bone marrow blasts at enrollment / Unit of Measure: percent
timeFrame: At enrollment
measure: Presence of multilineage dysplasia at enrollment / Unit of Measure percent of patients
timeFrame: At enrollment
measure: Cytogenetic abnormalities at enrollment / Unit of Measure percent of patients
timeFrame: At enrollment
measure: IPSS-R risk classification at enrollment / Unit of Measure percent of patients by category
timeFrame: At enrollment
measure: Presence of somatic mutations at enrollment / Unit of Measure: percent of patients
timeFrame: At enrollment
measure: Demographic characteristics at enrollment / Unit of Measure: Descriptive (e.g., mean ± SD for age, % for sex distribution)
timeFrame: At enrollment
Secondary outcomes:
measure: Overall survival
timeFrame: From diagnosis until death or last follow-up, up to 120 months
measure: Progression-free survival
timeFrame: From diagnosis until progression, AML transformation, or death, assessed up to 120 months
measure: Incidence of bone marrow failure events (anemia, neutropenia, thrombocytopenia)
timeFrame: From diagnosis until last follow-up, up to 120 months
measure: Duration of first-line treatment for MDS
timeFrame: From treatment initiation until discontinuation or last follow-up, assessed up to 60 months
measure: Time to first documented treatment response
timeFrame: From treatment start to first response, assessed up to 60 months
measure: Time to transformation to acute myeloid leukemia (AML)
timeFrame: From diagnosis until AML confirmation or last follow-up, up to 120 months
measure: Time to transfusion dependency
timeFrame: From diagnosis to transfusion dependency or last follow-up, up to 120 months
measure: Cytogenetic and molecular response rates
timeFrame: Assessed during follow-up, up to 120 months
measure: Time to progression on IPSS score
timeFrame: From initial classification to IPSS progression or last follow-up, up to 120 months
Other outcomes:
Locations:
city: Rouen
country: France
facility: Aspasia Stamatoullas
Study officials:
References: