Tomizawa Daisuke, Tanaka Shiro, Hasegawa Daisuke, Iwamoto Shotaro, Hiramatsu Hidefumi, Kiyokawa Nobutaka, Miyachi Hayato, Horibe Keizo, Saito Akiko Moriya, Taga Takashi, Adachi Souichi
Division of Leukemia and Lymphoma, Children's Cancer Center, National Center for Child Health and Development, Tokyo.
Clinical Research Center, National Hospital Organization, Nagoya Medical Center, Nagoya.
Jpn J Clin Oncol. 2018 Jun 1;48(6):587-593. doi: 10.1093/jjco/hyy061.
The purpose of this study is to compare the efficacy and safety of combination therapy (HD-ECM) including high-dose cytarabine in initial induction therapy with that of combination therapy (ECM) involving the continuous administration of cytarabine for previously untreated, newly diagnosed patients with AML at <18 years of age. This is a seamless Phase II-III clinical trial, consisting of Phase II and III parts. In the Phase II part, the safety of the experimental treatment (HD-ECM) will be examined. Subsequently, the Phase III study will compare the efficacy and safety of HD-ECM with that of standard ECM. The primary endpoint of the Phase II study is the early mortality rate. The primary endpoints of the Phase III study are the 3-year event-free survival rate and the positive minimal residual disease rate by flow cytometry after initial induction therapy. This trial has been registered at the UMIN Clinical Trials Registry (UMIN000013288).
本研究的目的是比较含大剂量阿糖胞苷的联合治疗（HD-ECM）与持续给予阿糖胞苷的联合治疗（ECM）在初始诱导治疗中对年龄小于18岁、既往未治疗的新诊断急性髓系白血病（AML）患者的疗效和安全性。这是一项无缝衔接的II-III期临床试验，由II期和III期部分组成。在II期部分，将检验实验性治疗（HD-ECM）的安全性。随后，III期研究将比较HD-ECM与标准ECM的疗效和安全性。II期研究的主要终点是早期死亡率。III期研究的主要终点是3年无事件生存率和初始诱导治疗后流式细胞术检测的微小残留病阳性率。本试验已在日本大学医学情报网络临床试验注册中心注册（UMIN000013288）。
