Homoharringtonine as a backbone drug for the treatment of newly diagnosed pediatric acute myeloid leukemia: a report from a single institution in China.

Homoharringtonine (HHT) is one of several cephalotaxine alkaloids that has shown clinical efficacy in the treatment of acute myeloid leukemia (AML). The purpose of this study was to evaluate the efficacy and toxicity of HHT for de novo pediatric AML. Patients entered in this study were treated with a regimen including HHT 3.5 mg/m(2) day for 9 days for 6-8 cycles after induction and consolidation with cytarabine plus daunorubicin (DA). One hundred and seventy-one eligible patients, with a median age of 7.58 years, were enrolled. Complete response was obtained in 140/171 (81.9%) cases within 60 days (2 cycles) after DA induction. The 5-year event-free survival was 52.75%. Severe myelosuppression was seen in all patients, with an average minimum WBC count of 686/μl. Following the HHT-including regimen, one patient suffered severe pancreatitis, and a second with a history of congenital hepatitis B suffered liver failure. No significant drug-induced hypotension, fluid retention, hyperglycemia, or cardiac toxicity was detected in this study. Other toxicities, including nausea, vomiting, diarrhea, and mucositis, were mild. HHT-including protocols may emerge as useful therapeutic options in future clinical trials.