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Erythroblastic and/or Megakaryoblastic Leukemia in Down Syndrome: Treatment with Low‐Dose Arabinosyl Cytosine

Purpose We report here the clinical response to low-dose arabinosyl cytosine (Ara-C) in seven children with Down syndrome (DS) and acute leukemia in which blast cells express markers of erythroid and/or megakaryoblastic lineages. Following an initial course of treatment with Ara-C, complete remission was obtained in all seven patients. Maintenance therapy with Ara-C was continued during complete remission. Four patients subsequently relapsed; the three others are disease-free. Based on these data, we suggest that when conventional therapy is contraindicated by associated malformations, low-dose Ara-C can be used for treating DS patients with erythroblastic or megakaryoblastic leukemia. The aim of this study was to assess the efficacy of low-dose Ara-C in treating megakaryoblastic and/or erythroblastic leukemia associated with DS. Patients and Methods Seven patients with DS presented with leukemia in which blast cells displayed early markers of the erythroblastic and/or megakaryoblastic lineage. Low-dose subcutaneous Ara-C (10 mg/m2 two times per day) was given for 21 days as induction therapy, followed by a 5–10-day course each month for 2 years as a maintenance treatment. Results Low-dose Ara-C treatment resulted in complete remission in all seven patients and in long-term disease-free survival in three patients. Conclusion In cases in which conventional chemotherapy is contraindicated, low-dose Ara-C should be considered as a therapeutic alternative for treatment of DS-associated erythroblastic or megakaryocytic leukemia.