Li Jia-Nan, Chen Yi-Jun, Fan Zhong, Li Qiao-Ru, Liao Liu-Hua, Ke Zhi-Yong, Li Yu, Wang Li-Na, Yang Cui-Yun, Luo Xue-Qun, Tang Yan-Lai, Zhang Xiao-Li, Huang Li-Bin
Department of Pediatrics, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, China.
Department of Pediatrics, Zhongshan People's Hospital, Zhongshan, China.
Ther Adv Hematol. 2024 May 31;15:20406207241256894. doi: 10.1177/20406207241256894. eCollection 2024.
Pediatric acute myeloid leukemia (AML) has poor prognosis and high rate of relapse and mortality, and exploration of new treatment options is still critically needed.
To summarize the outcome of our new treatment strategies for pediatric AML, which is characterized by dual induction and acute lymphoblastic leukemia (ALL) elements consolidation.
Retrospective, single-arm study.
From July 2012 to December 2019, an intensive chemotherapy protocol was used for newly diagnosed children with AML, which contains dual induction, three courses of consolidations based on high-dose cytarabine, and two courses of consolidations composed of high-dose methotrexate, vincristine, asparaginase, and mercaptopurine (ALL-like elements). Blasts were monitored by bone marrow smears at intervals, and two lumbar punctures were performed during chemotherapy. We retrospectively analyzed the efficacy and safety of this study. The last follow-up was on 26 May 2023.
A total of 70 pediatric AMLs were included. The median age at diagnosis was 6.7 (0.5-16.0) years. The median initial WBC count was 23.74 × 10/L, 11 of whom ⩾100 × 10/L. After dual induction, there were 62 cases of complete remission (CR), 5 cases of partial remission, and 3 cases of nonremission. The CR rate was 88.57%. The median follow-up time was 5.8 (0.2-9.4) years, the 5-year overall survival was 78.2% ± 5%, the event-free survival (EFS) was 71.2% ± 5.6%, and the cumulative recurrence rate was 27.75%. The 5-year EFS of patients with initial WBC < 100 × 10/L ( = 59) and ⩾100 × 10/L ( = 11) were 76.4% ± 5.7% and 45.5% ± 15% ( = 0.013), respectively. A total of 650 hospital infections occurred. The main causes of infection were respiratory tract infection (26.92%), septicemia (18.46%), stomatitis (11.85%), and skin and soft-tissue infection (10.46%).
This intensive treatment protocol with dual induction and ALL-like elements is effective and safe for childhood AML. Initial WBC ⩾ 100 × 10/L was the only independent risk factor in this cohort.
It is a retrospective study, and no registration on ClinicalTrials.gov.
儿童急性髓系白血病（AML）预后较差，复发率和死亡率较高，因此仍迫切需要探索新的治疗方案。
总结我们以双重诱导和急性淋巴细胞白血病（ALL）样元素巩固为特征的儿童AML新治疗策略的结果。
回顾性单臂研究。
2012年7月至2019年12月，对新诊断的儿童AML采用强化化疗方案，包括双重诱导、基于大剂量阿糖胞苷的三个巩固疗程，以及由大剂量甲氨蝶呤、长春新碱、天冬酰胺酶和巯嘌呤（ALL样元素）组成的两个巩固疗程。定期通过骨髓涂片监测原始细胞，并在化疗期间进行两次腰椎穿刺。我们回顾性分析了本研究的疗效和安全性。最后一次随访时间为2023年5月26日。
共纳入70例儿童AML。诊断时的中位年龄为6.7（0.5 - 16.0）岁。初始白细胞计数中位数为23.74×10⁹/L，其中11例≥100×10⁹/L。双重诱导后，完全缓解（CR）62例，部分缓解5例，未缓解3例。CR率为88.57%。中位随访时间为5.8（0.2 - 9.4）年，5年总生存率为78.2%±5%，无事件生存率（EFS）为71.2%±5.6%，累积复发率为27.75%。初始白细胞<100×10⁹/L（n = 59）和≥100×10⁹/L（n = 11）患者的5年EFS分别为76.4%±5.7%和45.5%±15%（P = 0.013）。共发生650次医院感染。感染的主要原因是呼吸道感染（26.92%）、败血症（18.46%）、口腔炎（11.85%）和皮肤及软组织感染（10.46%）。
这种具有双重诱导和ALL样元素的强化治疗方案对儿童AML有效且安全。初始白细胞≥100×10⁹/L是该队列中唯一的独立危险因素。
这是一项回顾性研究，未在ClinicalTrials.gov上注册。
