Abstract

Background Paediatric acute myeloid leukaemia (AML) may have a different biology from adult AML. Furthermore, different paediatric groups from across the world use different protocols to treat the disease. There are no data to show whether a standard 3 + 7 approach used in adult patients would be an adequate therapy for paediatric AML. Methods We retrospectively analysed the outcomes of all consecutive paediatric AML patients treated at our centre from January 2008 to May 2013. Overall survival (OS) and progression-free survival (PFS) were estimated using Kaplan–Meier methods, and outcomes were compared across the patient parameters and treatment regimens used. The treatment regimens were DA (daunorubicin [DNR] 60 mg/m 2 for 3 days and cytarabine [Ara-C] 100 mg/m 2 for 7 days) in 39/65 (60%) patients and ADE (Ara-C 100 mg/m 2 intravenously twice daily for 10 days plus DNR 50 mg/m 2 for 3 days plus etoposide 100 mg/m 2 for 5 days) in 26/65 (40%) patients. Findings Sixty-five patients with a median age of 9 years (1–17 years) were included in the study, 45 (69%) of whom were male. The commonest subtype was M2 (26%). According to conventional cytogenetics, patients were classified as good risk ( n = 18, 27%), intermediate risk ( n = 13, 20%) and high risk ( n = 12, 18%). A total of 72% of patients (47/65) attained complete remission (24/39 [62%] in the DA group versus 23/26 (88%) in the ADE group; p = 0.52). Median OS was 14.7 months (95% confidence interval 4.4–24.8) and was not significantly different between the two treatment regimens (37 months in the DA group versus 40 months in the ADE group; p = 0.4). There were more grade 4–5 haematological toxicities in the ADE group than in the DA group, but the differences were not significant. The only factor that predicted the outcome was cytogenetic risk stratification (OS in good-risk patients according to cytogenetics was 65%; p = 0.004). Interpretation Even though a high complete response rate was achieved with both regimens, OS was still poor because of disease relapses among the intermediate-risk and high-risk patients. Increasing coverage with allogeneic stem-cell transplantation in both intermediate and high-risk cytogenetic patients might help to improve outcomes.

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