Background:Cladribine-based combination chemotherapy has demonstrated promising efficacy in patients with relapsed/refractory adult acute myeloid leukemia (AML), prompting its increased utilization in the frontline; in pediatrics, it has been typically reserved for relapsed or refractory cases. While fludarabine has been used more commonly as a purine analog in intensive regimens, cladribine may be an important alternative.Methods:We performed a retrospective study at MD Anderson Cancer Center from January 2015 to July 2023, which included patients aged 1–21 years with refractory or relapsed AML who received cladribine outside of a transplant conditioning.Results:A total of 30 patients were included, with a median age of 20 years (range, 2–21), and 55% being male. Similar to adults, cladribine exhibited good tolerability in pediatric and adolescent patients, with the most common adverse events being febrile neutropenia and myelosuppression. The most common grade 3 or 4 adverse events included febrile neutropenia (55%) and sepsis (26%), and there were no treatment discontinuations due to adverse events. Among patients with a median number of 2 (0–7) prior treatments, the overall response rate (CR/CRi) was 45%, and median event-free and overall survival were 6 and 12 months, respectively. Disease progression resulted in 4 deaths within 30 days of treatment.Conclusions:Cladribine was tolerated in pediatrics. No new safety signals were seen with cladribine regimens in this cohort. Response assessment is limited due to the heavily pretreated cohort. Further prospective studies are warranted on the safety and efficacy of cladribine and establish its role in pediatric, adolescent, and early young adult patients with AML.
背景：基于克拉屈滨的联合化疗在复发/难治性成人急性髓系白血病（AML）患者中显示出良好的疗效，这促使其在临床一线治疗中的应用日益增多；而在儿科领域，该方案通常仅用于复发或难治性病例。尽管氟达拉滨作为嘌呤类似物在强化化疗方案中应用更为普遍，但克拉屈滨可能是一种重要的替代选择。 方法：我们在MD安德森癌症中心开展了一项回顾性研究，时间跨度为2015年1月至2023年7月，纳入了年龄在1-21岁之间、在非移植预处理方案中接受克拉屈滨治疗的难治性或复发性AML患者。 结果：研究共纳入30例患者，中位年龄20岁（范围2-21岁），其中55%为男性。与成人患者相似，克拉屈滨在儿童和青少年患者中表现出良好的耐受性，最常见的不良事件为发热性中性粒细胞减少和骨髓抑制。最常见的3级或4级不良事件包括发热性中性粒细胞减少（55%）和败血症（26%），且未出现因不良事件而终止治疗的情况。在既往接受过中位数为2次（范围0-7次）治疗的患者中，总体缓解率（完全缓解/完全缓解伴血细胞不完全恢复）为45%，中位无事件生存期和总生存期分别为6个月和12个月。疾病进展导致4例患者在治疗30天内死亡。 结论：克拉屈滨在儿科患者中具有可耐受性。本队列研究中未发现克拉屈滨方案的新安全性信号。由于该队列患者既往接受过大量治疗，疗效评估存在局限性。有必要对克拉屈滨的安全性和有效性开展进一步前瞻性研究，以明确其在儿童、青少年及年轻成人AML患者治疗中的作用。
肿瘤(癌症)患者之家