Myelodysplasia and Leukemia after Treatment of Aplastic Anemia with G-CSF

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Abstract
Several clinical trials using recombinant human granulocyte colony-stimulating factor (G-CSF) have demonstrated that patients with severe aplastic anemia are responsive to this agent and that neutrophil counts can be increased in the majority of patients without toxicity.1 However, little is known about the long-term effects and toxicity of G-CSF in severe aplastic anemia. We treated three children with severe aplastic anemia in whom myelodysplasia with monosomy 7 developed ( Table 1 ). In two of them, myelodysplasia evolved into acute myelomonocytic leukemia. These three patients represent about 14 percent of all children with aplastic anemia who were treated with G-CSF at our institutions and who survived at least one year without bone marrow transplantation. The incidence of aplastic anemia that evolves into myelodysplasia and leukemia has increased as survival rates have improved and observation times have lengthened.2 , 3 We never observed this phenomenon before the introduction of G-CSF treatment. This small series suggests an increased risk of myelodysplasia and leukemia in patients with severe aplastic anemia who are treated with G-CSF.