Haema 2012; 3(2): 151-157
by Athanasios Galanopoulos
Hematologist, General Hospital «G. Gennimatas», Athens
Abstract
Treatment of MDS should be patient- and risk-adapted, based on transfusion requirements, bone marrow blast-cell percentage and on their cytogenetic profile. The goals of treatment are different in lower risk patients than in higher risk. In lower risk patients, with a median survival of 6-8 years and lower probability of transformation to AML, goals are the achievement of a better quality of life and the decrease of transfusion requirements and of AML transformation. In higher risk, the goal is to prolong survival. Current available therapies, in low risk MDS, include growth factor support, lenalidomide, hypomethylating agents, immunosuppressive therapy, thalidomide as well as other agents like danazol, and arsenic trioxide, which have been used separately or in various combinations. Lenalidomide has significant clinical activity in patients with lower risk disease, anemia and Del-5q and has been approved as first line treatment, in USA, but not in Europe where it is proposed as second line treatment (after failure of an erythropoiesis stimulating agent). Lenalidomide has also been used in lower risk, non del(5q)-MDS, but with lower activity. Two hypomethylating agents, 5-azacytidine and decitabine have been approved for MDS: 5-azacytidine is approved for all subsets of MDS, whereas decitabine for those with Int-1 disease and above. None of them have been shown to modify the natural history of patients with lower risk disease. Combined immunosuppressive therapy with antithymocyte globulin, cyclosporine and corticosteroids is indicated especially in younger patients (<60 years), HLA-DR15 positive and with a shorter duration of transfusion dependency. Thalidomide, even at daily doses of 50 mg, for at least one year, is equivalent with higher doses, without the risk of deep vein thrombosis. Allogeneic transplantation is not usually recommended for patients with lower risk disease even if they are young. Younger patients with hypoplastic MDS should be considered for this approach upfront.