Preliminary Results of a Placebo-Controlled, Double-Blind Trial Evaluating Sequential HD-AraC/Mitoxantrone Induction Chemotherapy with or Without Granulocyte-Macrophage Colony-Stimulating Factor in Patients with High-Risk Myelodysplastic Syndromes

Patients with high-risk myelodysplastic syndromes (MDS) face a poor prognosis with a median survival time of less than 12 months. The only curvative treatment known so far is allogeneic bone marrow transplantation which is available for younger patients with an HLA-matched sibling and hence for a minority of cases only. Acute myeloid leukemia (AML)-type combination chemotherapy can achieve complete remissions (CR) in the range of 15%–51%, however, remission duration is short (1, 2). Treatment failures are due to primary drug resistance and a high early death rate. The latter may be related to a longer duration of treatment-induced aplasia as compared to AML.