PDF(Pubmed)
Abstract：
Over the past two decades, the prognosis in adolescents and young adults (AYAs) diagnosed with acute myeloid leukemia (AML) has significantly improved. The standard intensive cytotoxic treatment approach for AYAs with AML, consisting of induction chemotherapy with anthracycline/cytarabine combination followed by consolidation chemotherapy or stem cell transplantation, has lately been shifting toward novel targeted therapies, mostly in the fields of clinical trials. One of the most recent advances in treating AML is the combination of the B-cell lymphoma 2 (Bcl-2) inhibitor venetoclax with hypomethylating agents, which has been studied in elderly populations and was approved by the Food and Drug Administration (FDA) for patients over 75 years of age or patients excluded from intensive chemotherapy induction schemas due to comorbidities. Regarding the AYA population, venetoclax combination therapy could be a therapeutic option for patients with refractory/relapsed (R/R) AML, although data from real-world studies are currently limited. Venetoclax is frequently used by AYAs diagnosed with advanced hematologic malignancies, mainly acute lymphoblastic leukemia and myelodysplastic syndromes, as a salvage therapeutic option with considerable efficacy and safety. Herein, we aim to summarize the evidence obtained from clinical trials and observational studies on venetoclax use in AYAs with AML. Based on the available evidence, venetoclax is a safe and effective therapeutic option for R/R AML AYA patients. However, further research in larger cohorts is needed to confirm these data, establishing the benefits of a venetoclax-based regimen for this special population.
摘要：
在过去的二十年里，诊断为急性髓系白血病(AML)的青少年和年轻成人(AYAs)的预后显著改善.AYAs合并AML的标准强化细胞毒性治疗方法，包括蒽环类/阿糖胞苷联合诱导化疗，然后进行巩固化疗或干细胞移植，最近一直在转向新的靶向疗法，主要是在临床试验领域。治疗AML的最新进展之一是B细胞淋巴瘤2（Bcl-2）抑制剂venetoclax与低甲基化药物的组合，该研究已在老年人群中进行，并已获得美国食品药品监督管理局（FDA）的批准，适用于75岁以上的患者或由于合并症而被排除在强化化疗诱导方案之外的患者。关于AYA人口，维奈托克联合治疗可能是难治性/复发性（R/R）AML患者的治疗选择，尽管来自真实世界研究的数据目前有限。被诊断为晚期血液系统恶性肿瘤的AYAs经常使用维奈托克。主要是急性淋巴细胞白血病和骨髓增生异常综合征，作为一种挽救性治疗选择，具有相当的疗效和安全性。在这里，我们的目的是总结从临床试验和观察性研究中获得的关于venetoclax用于AYAs合并AML的证据.根据现有证据,维奈托克是R/RAMLAYA患者安全有效的治疗选择。然而,需要在更大的队列中进行进一步的研究来证实这些数据，确定基于venetoclax的方案对这一特殊人群的益处。
