Erythroblastic and/or megakaryoblastic leukemia in Down syndrome: treatment with low-dose arabinosyl cytosine

J Pediatr Hematol Oncol. 1996 Feb;18(1):59-62. doi: 10.1097/00043426-199602000-00011.


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.

Publication types

  • Case Reports

MeSH terms

  • Antimetabolites, Antineoplastic / administration & dosage
  • Antimetabolites, Antineoplastic / therapeutic use*
  • Child, Preschool
  • Cytarabine / administration & dosage
  • Cytarabine / therapeutic use*
  • Disease-Free Survival
  • Down Syndrome / complications*
  • Drug Administration Schedule
  • Female
  • Humans
  • Infant
  • Leukemia, Erythroblastic, Acute / complications*
  • Leukemia, Erythroblastic, Acute / drug therapy*
  • Leukemia, Megakaryoblastic, Acute / complications*
  • Leukemia, Megakaryoblastic, Acute / drug therapy*
  • Male
  • Remission Induction


  • Antimetabolites, Antineoplastic
  • Cytarabine