■小儿急性髓系白血病(AML)预后差,复发率和死亡率高,和探索新的治疗方案仍然是至关重要的。
■为了总结我们对小儿AML的新治疗策略的结果,其特征是双重诱导和急性淋巴细胞白血病(ALL)元件巩固。
■回顾性,单臂研究。
■从2012年7月至2019年12月,对新诊断的AML患儿采用强化化疗方案,其中包含双重感应,基于高剂量阿糖胞苷的三个疗程的巩固,和两个由大剂量甲氨蝶呤组成的巩固疗程,长春新碱,天冬酰胺酶,和巯基嘌呤(ALL样元素)。间隔进行骨髓涂片监测,化疗期间进行了两次腰椎穿刺。我们回顾性分析了这项研究的有效性和安全性。最后一次随访是2023年5月26日。
■共包括70个儿科AMLs。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,11人100×109/L经过双重诱导,完全缓解(CR)62例,5例部分缓解,3例未缓解。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WBC<100×109/L(n=59)和100×109/L(n=11)的患者的5年EFS分别为76.4%±5.7%和45.5%±15%(p=0.013),分别。共发生650例医院感染。主要感染原因为呼吸道感染(26.92%),败血症(18.46%),口腔炎(11.85%),皮肤和软组织感染(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,并且没有在ClinicalTrials.gov上注册。
UNASSIGNED: 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.
UNASSIGNED: 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.
UNASSIGNED: Retrospective, single-arm study.
UNASSIGNED: 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.
UNASSIGNED: 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 × 109/L, 11 of whom ⩾100 × 109/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 × 109/L (n = 59) and ⩾100 × 109/L (n = 11) were 76.4% ± 5.7% and 45.5% ± 15% (p = 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%).
UNASSIGNED: This intensive treatment protocol with dual induction and ALL-like elements is effective and safe for childhood AML. Initial WBC ⩾ 100 × 109/L was the only independent risk factor in this cohort.
UNASSIGNED: It is a retrospective study, and no registration on ClinicalTrials.gov.