Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Myelodysplastic Syndrome Low Risk

Not Applicable

Completed

• Conditions: MDS
• Interventions: Other: transplantation

• Registration Number: NCT02757989
• Lead Sponsor: Groupe Francophone des Myelodysplasies

Brief Summary

Comparison of survival in patients with or without a matched donor at 36 months

Detailed Description

Patients with a matched donor (8/8 at molecular level unrelated donor or matched sibling) received an allogeneic hematopoietic stem cell transplantation.

Patients without a matched donor received the best available treatment. All patients will be followed at least 36 months or until the end of the study.

Study Timeline

• Study First Submitted: 2016-03-31
• Study First Submitted QC: 2016-04-27
• Study First Posted: 2016-05-02
• Study Start: 2016-05-31
• Status Verified: 2024-02
• Primary Completion: 2024-06-07
• Study Completion: 2024-06-07
• Last Update Submitted: 2024-08-01
• Last Update Posted: 2024-08-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 79

Inclusion Criteria

1. Signed Informed consent

2. Classical IPSS intermediate 1 or low myelodysplastic syndrome associated with at least one poor prognosis feature:

   1. Intermediate or higher risk revised IPSS
   2. RBC transfusion dependent anemia and failure to 2 or more lines or therapy (including EPO, Lenalidomide or demethylating agent...)
   3. thrombocytopenia < 20 G/L requiring transfusion
   4. neutropenia < 0.5 G/L associated with severe infection (defined as requiring hospitalization)

3. Patient aged ≥ 18 and < 70 years For young patients, 18-45 years, Fanconi disease and dyskeratosis should be ruled out

4. Patient for whom a transplantation from a matched donor, (8/8 (HLA A, B, C, DRB1) identical at molecular level)unrelated donor or matched sibling), is considered irrespective of donor availability

5. Performance status 0-2 on the Eastern Cooperative Oncology Group (ECOG) Scale (At time of screening)

6. Negative pregnancy and adequate contraception (including in male patients wishing to father), if relevant.

7. Wash-out of at least 30 days since a previous treatment with Vidaza, Lenalidomide, EPO or any other treatment inducing cytopenias.

Exclusion Criteria

1. MDS classified according to classical IPSS as intermediate 2 or High risk

2. Transformation in Acute myeloid Leukemia (AML)

3. Severe active infection or any other uncontrolled severe condition.

4. Organ dysfunctions including the following

   • Hepatic : total bilirubin > 2 times upper limit of normal (ULN) (except moderate unconjugated hyperbilirubinemia due to intra medullary hemolysis or Gilbert syndrome) , alanine transaminase (ALT) and aspartate transaminase (AST) > 3xULN
   • Symptomatic respiratory chronic failure
   • Symptomatic cardiac failure
   • Renal clearance < 60ml/min

5. Prior malignancy (except in situ cervix carcinoma, limited basal cell carcinoma, or other tumors if not active during the last 3 years)

6. MDS with the following causal germline disease : Fanconi anemia, GATA2 related syndromes and telomere disorders

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Patients with donor	transplantation	Patients with a matched donor (8/8 at molecular level unrelated donor or matched sibling)

Primary Outcome Measures

Name	Time	Method
overall survival	36 months	comparison of overall survival in patients with or without a matched donor (8/8 unrelated donor or matched sibling) at 36 months

Secondary Outcome Measures

Name	Time	Method
quality of life	12, 24 and 36 months	comparison of quality of life in patients with or without a matched donor, quality of life assessed by questionnaire (EORTC version 3) at inclusion, 12, 24 and 36 months
proportion of patients with iron overload	16 months	proportion of patients with iron overload (Serum Ferritin (SF)\>1000 ng/mL or Red Blood Cells transfusion\>20) at time of inclusion and at 16 month after inclusion for non-transplanted patients and 12 months post-transplant for transplanted patients
number of patients with adverse events grade III and IV as assessed by CTCAE v4.0	36 months	comparison between patients with or without a donor for number of Grade III and IV toxicities (hematological and non-hematological) recorded according to NCI CTCAE criteria versions 4.0 during the 36 months
number of patients with complete response at 36 month	36 months	comparison between patients with or without a donor for cumulative incidence of complete response at 36 month
efficiency of chelation	3 and 16 months	the effect of chelation will be assessed at 3 month after inclusion for all patient and post transplant by measuring Serum ferritin level
number of patients with transformation in AML at 36 month	36 months	comparison between patients with or without a donor for cumulative incidence of transformation in AML at 36 month
evolution of innovative iron markers including Non-transferrin binding iron (NTBI), labile plasmatic Iron (LPI) and Hepcidine	3 and 16 months	evolution of innovative iron markers including Non-transferrin binding iron (NTBI), labile plasmatic Iron (LPI) and Hepcidine measured at time of inclusion, at 3 month and 16 month post-inclusion for all patients; In transplanted patients these markers will be measured just before conditioning regimen (J-5), Just before the transplantation (J0), at D7, 30, 100 and 12 month after transplant.

Trial Locations

Locations (37)

Centre hospitalier Victor Dupouy; 🇫🇷 Argenteuil, France

CHSF Gilles de Corbeil; 🇫🇷 Corbeil-Essonnes, France

CHU d'Angers; 🇫🇷 Angers, France

CHRU Côte de Nacre; 🇫🇷 Caen, France

Hôpital Huriez; 🇫🇷 Lille, France

Hôpital Dupuytren; 🇫🇷 Limoges, France

Centre Henri Becquerel; 🇫🇷 Rouen, France

Hôpital Necker; 🇫🇷 Paris, France

Hôpital Avicenne; 🇫🇷 Bobigny, France

CHU Estaing; 🇫🇷 Clermont Ferrand, France

CHU d'Amiens; 🇫🇷 Amiens, France

CHU de Nîmes; 🇫🇷 Nîmes, France

CH René Dubos; 🇫🇷 Pontoise, France

Hôpital Pontchaillou; 🇫🇷 Rennes, France

Institut Curie; 🇫🇷 Saint-Cloud, France

Hôpital Pitié Salpétrière; 🇫🇷 Paris, France

Hôpital Henri Mondor; 🇫🇷 Créteil, France

Hôpital Saint Vincent de Paul; 🇫🇷 Lille, France

CHRU de Montpellier; 🇫🇷 Montpellier, France

CHU de Poitiers; 🇫🇷 Poitiers, France

Institut de cancérologie Lucien Neuwirth; 🇫🇷 Saint-Priest-en-Jarez, France

Hôpital civil; 🇫🇷 Strasbourg, France

CHU de Grenoble; 🇫🇷 Grenoble, France

CH Le Mans; 🇫🇷 Le Mans, France

Hôpital de Brabois; 🇫🇷 Vandoeuvre les nancy, France

IUCT-Oncopole; 🇫🇷 Toulouse, France

CHU de Nantes; 🇫🇷 Nantes, France

Centre hospitalier Lyon Sud; 🇫🇷 Lyon, France

Hôpital Cochin; 🇫🇷 Paris, France

CHU Jean Minjoz; 🇫🇷 Besançon, France

CHU de Haut Lévèque; 🇫🇷 Bordeaux, France

GHEF, site de Meaux; 🇫🇷 Meaux, France

CHU de Nice; 🇫🇷 Nice, France

Hôpital Saint Louis; 🇫🇷 Paris, France

CH Joffre; 🇫🇷 Perpignan, France

CHU de Reims; 🇫🇷 Reims, France

Hôpital Bretonneau; 🇫🇷 Tours, France