Long‐term results of an ultra low‐dose cytarabine‐based regimen for the treatment of acute megakaryoblastic leukaemia in children with Down syndrome

Abstract

Children with acute megakaryoblastic leukaemia (AMKL) and Down syndrome (DS) show a favourable response to chemotherapy, probably due to increased sensitivity of the leukaemic blasts to cytarabine. In contrast, dose-intensive approaches have resulted in disproportionate treatment-related mortality in this group. The survival of children with AMKL and DS was retrospectively compared following treatment with a low-dose chemotherapy protocol, consisting of cytarabine (10 mg/m2/dose), retinylpalmitate and vincristine or standard chemotherapy. Event-free (67 +/- 11%) and overall survival (77 +/- 10%) at 5 years were not significantly different in both groups. Further reduction of treatment intensity in AMKL of children with DS, therefore, appears feasible.