High-dose methylprednisolone (HDMP) has been shown to induce differentiatio
n of myeloid leukemic cells with a remarkable antileukemic effect in childr
en with various subtypes of acute myeloblastic leukemia (AML). Here the ben
eficial effect of short-course HDMP therapy in a child with myelodysplastic
syndrome (AIDS) is reported. Oral methylprednisolone sodium succinate (Pre
dnol-L) was administered at a single daily dose of 30 mg/kg for 5 days to a
4-year-old girl with refractory, anemia with excess of blasts and hypocell
ular bone marrow before the initiation of chemotherapy. In addition to dram
atic clinical improvement, the patient's while blood cell count increased f
rom 2.3 x 10(9)/L to 5.0 x 10(9)/L, and peripheral blood blast cells disapp
eared 4 days after HDMP treatment. Repeated bone marrow aspirate 1 week aft
er the initiation of HDMP disclosed increased cellularity with no blasts. F
urthermore, short-course HDMP treatment stimulated the increase in the numb
er of peripheral blood lymphocytes and CD3(+), CD4(+), CD8(+), CD19(+), CD3
4(+), and NK cells. Results obtained with HDMP from the previous studies an
d the present case suggest that high-dose methylprednisolone is a promising
agent in the treatment of MDS and it is recommended as an initial treatmen
t especially for MDS children with hypocellular bone marrow at presentation
.