Homoharringtonine is a safe and effective substitute for anthracyclines in children younger than 2 years old with acute myeloid leukemia
作者机构：Key Laboratory of Pediatric Hematology and Oncology Ministry of HealthDepartment of Hematology and OncologyPediatric Translational Medicine InstituteShanghai Children's Medical CenterShanghai Jiao Tong University School of MedicineShanghai 200127China
出 版 物：《Frontiers of Medicine》 (医学前沿（英文版）)
年 卷 期：2019年第13卷第3期
页 面：378-387页
核心收录：
基 金：the National Natural Science Foundation of China (No.81270623) the Science and Technology Commission of Shanghai Municipality (No.14411950600) the Public Health 3-Year Project of Shanghai Children's Medical Center(No.GWIV-25).
主 题：homoharringtonine acute myeloid leukemia pediatrics
摘 要：Homoharringtonine (HHT),a plant alkaloid from Cephalotaxus harringtonia,exhibits a unique anticancer mechanism and has been widely used in China to treat patients with acute myeloid leukemia (AML) since the 1970s.Trial SCMC-AML-2009 presented herein was a randomized clinical study designed based on our previous findings that pediatric AML patients younger than two years old may benefit from HHT-containing chemotherapy regimens.Patients randomized to arm A were treated with a standard chemotherapy regimen comprising mainly of anthracyclines and cytarabine (Ara-C),whereas patients in arm B were treated with HHTcontaining regimens in which anthracyclines in all but the initial induction therapy were replaced by HHT.From February 2009 to November 2015,59 patients less than 2 years old with de novo AML (other than acute promyelocytic leukemia) were recruited.A total of 42 patients achieved a morphologic complete remission (CR) after the first course,with similar rates in both arms (70.6% vs.72.0%).At the end of the follow-up period,40 patients remained in CR and 5 patients underwent hematopoietic stem cell transplantation in CR,which could not be considered as events but censors.The 5-year event-free survival (EFS) was 60.2%± 9.6% for arm A and 88.0%± 6.5% for arm B (P=0.024).Patients in arm B experienced shorter durations of leukopenia,neutropenia,and thrombocytopenia and had a lower risk of infection during consolidation chemotherapy with high-dosage Ara-C.Consequently,the homoharringtonine-based regimen achieved excellent EFS and alleviated hematologic toxicity for children aged younger than 2 years with de novo AML compared with the anthracycline-based regimen.
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