Cohort Study Assessing the Treatment Strategy for High-Risk Myelodysplastic Syndromes in Patients Under 70

NCT05367583 | Unknown

• 1 other identifier: CHUBX 2012/22
• Study Type: observational
• Target: 107
• Locations: 1 country
• Sites: 10

Brief Summary

Myelodysplastic syndromes (MDS) are bone marrow malignant diseases resulting in ineffective haematopoiesis and subsequently, blood cell count decrease. Patients have anaemia responsible of fatigue and high heart frequency, thrombocytopenia responsible of increased risk of bleeding and neutropenia responsible of increased risk of infection. The patients suffering from MDS also are at increased risk of developing acute myeloblastic leukemia (AML). Allogeneic stem cell transplantation (alloSCT) remains the only curative option for patients with aggressive MDS. However, these patients are frequently ineligible for this kind of treatment, because of, for instance, age and co-morbidities. Thus, other treatment options are needed and Azacytidine (AZA), a hypomethylating agent is then proposed. With this COMYRE observatory study, we wanted to analyse which patients undergo alloSCT, why they are not eligible to alloSCT if it is the case, the overall survival of all the patients and if there are some factors which can influence this survival. It could help us to better identify the best candidate for alloSCT and those for other treatments such as AZA.

Conditions

Myelodysplastic Syndromes

Keywords

Myelodysplastic syndromes; Allogeneic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

• Proportion of patient treated with Allogeneic stem cell transplantation (alloSCT) among enrolled patients

  3 months from baseline

Secondary Outcomes (6)

• Evaluate the feasibility of the alloSCT: Proportion of patients with a donor

  3 months from baseline

• Evaluate the effectiveness of iron chelation by measuring plasma ferritin (PF) (PF ≤ 1000 µg/L or PF > 1000 µg/L

  Baseline and before alloSCT or 1 year from baseline for patient not treated with alloSCT

• Evaluate the overall survival of patient treated and not treated with alloSCT

  Until 5 year from baseline

• Evaluate overall survival prognostic factors of patient treated and not treated with alloSCT

  Baseline

• Evaluate response rate to alloSCT or chemotherapy

  Until 5 years from baseline

Interventions

Allogeneic Stem Cell Transplantation PROCEDURE

Allogeneic Stem Cell Transplantation

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with MDS-HR defined by an intermediate or high IPSS score (≥ 1.5), or IPSS revised ≥ to 4.5

You may qualify if:

• Age\> 18 years old and \<70 years old
• MDS-HR defined by an intermediate or high IPSS score (≥ 1.5), or IPSS revised ≥ to 4.5.
• Diagnosis affirmed by 2 myelograms at least 1 month apart or 1 single characteristic myelogram if prior haematological abnormalities on complete blood count present for more than 6 months
• Affiliate or beneficiary of a social security system

You may not qualify if:

• Age ≥ 70 years
• MDS-LR defined by a low IPSS score (\<1.5), or revised IPSS \<4.5
• Women of childbearing potential not using contraception, pregnant or breastfeeding
• Not affiliated to the Social Security system

Sponsors & Collaborators

• University Hospital, Bordeaux: lead

Study Sites (10)

CH AGEN

Agen, France

Location

Ch Bayonne

Bayonne, France

Location

Chu de Bordeaux

Bordeaux, France

Location

Institut Bergonie

Bordeaux, France

Location

CH DAX

Dax, France

Location

Ch Libourne

Libourne, France

Location

Ch Marmande

Marmande, France

Location

Ch Mont de Marsan

Mont-de-Marsan, France

Location

CH PAU

Pau, France

Location

Ch Villeneuve/Lot

Villeneuve-sur-Lot, France

Location

MeSH Terms

Conditions

Myelodysplastic Syndromes

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 2, 2019
• First Posted: May 10, 2022
• Study Start: October 9, 2013
• Primary Completion: October 9, 2021
• Study Completion: October 1, 2024
• Last Updated: July 8, 2022

Record last verified: 2022-07

Data Sharing

• IPD Sharing: Will not share

Locations

FR (10)