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

This is an interventional treatment trial for MDS focused on measuring Low risk MDS, Transplantation Read More

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