High-dose methylprednisolone (HDMP) treatment has been shown to induce differentiation of myeloid leukemic cells in children with acute promyelocytic leukemia and other subtypes (FAB AML M1-M2-M4) of acute myeloblastic leukemia. In the present study, a child with acute megakaryoblastic leukemia (AMKL) was given HDMP (30 mg/kg/day) orally in a single dose for the first 4 days of induction therapy. A marked decrease in peripheral blood blast cells and an increase in platelet count associated with a striking change in bone marrow (BM) morphology was observed following a short-course of HDMP treatment alone. BM cells developed distinct morphology characterized by cytoplasmic blebbing and some appeared as platelet producing micromegakaryocytes. Flow cytometric analysis of the BM cells 4 days after HDMP treatment demonstrated a decrease in the percentage of cells co-expressing CD34 and CD117 antigens and a marked increase in CD42a antigen. These changes in BM morphology and immunophenotype may suggest maturation effect of HDMP on megakaryocytic leukemic cells. In addition ultrastructural analysis of BM cells cultured with methylprednisolone (10(-3) and 10(-6) M) for 24 and 48 h showed numerous apoptotic cells. This was coincident with a significant increase in the percentage of annexin positive cells. These results suggest that HDMP treatment may induce differentiation and apoptosis of leukemic cells in a child with AMKL and it could be a promising agent for remission induction of patients with AMKL.