title: Erythroblastic and/or megakaryoblastic leukemia in Down syndrome: treatment with low-dose arabinosyl cytosine.
abstract: OBJECTIVE
  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.
  
  
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Hematology, Centre Hospitalier de Bicêtre, Le Kremlin Bicêtre, France.
authors.forename: G
authors.initials: G
authors.lastname: Tchernia
authors.forename: F
authors.initials: F
authors.lastname: Lejeune
authors.forename: J F
authors.initials: JF
authors.lastname: Boccara
authors.forename: M F
authors.initials: MF
authors.lastname: Denavit
authors.forename: J P
authors.initials: JP
authors.lastname: Dommergues
authors.forename: F
authors.initials: F
authors.lastname: Bernaudin
mesh_terms: D000964:Antimetabolites, Antineoplastic / Q000008:administration & dosage / Q000627:therapeutic use*; D002675:Child, Preschool; D003561:Cytarabine / Q000008:administration & dosage / Q000627:therapeutic use*; D018572:Disease-Free Survival; D004314:Down Syndrome / Q000150:complications*; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007223:Infant; D004915:Leukemia, Erythroblastic, Acute / Q000150:complications* / Q000188:drug therapy*; D007947:Leukemia, Megakaryoblastic, Acute / Q000150:complications* / Q000188:drug therapy*; D008297:Male; D012074:Remission Induction
chemical_list: D000964:Antimetabolites, Antineoplastic; D003561:Cytarabine