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

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.

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