Hematology Clinic, Osmaniye State Hospital, 80000 Osmaniye, Türkiye.
Department of Hematology, Faculty of Medicine, Zonguldak Bulent Ecevit University, 67100 Zonguldak, Türkiye.
Medicina (Kaunas). 2024 Oct 4;60(10):1623. doi: 10.3390/medicina60101623.
Acute myeloid leukemia and myelodysplastic syndrome are both clonal hematologic malignancies that primarily affect older adults. Current treatments for AML/MDS are both limited in number and efficacy. This study aims to evaluate venetoclax-based therapies in AML/MDS, focusing on overall survival and recurrence-free survival rates, and to expand real-world data on its use. Clinical and laboratory data on patients with AML/MDS aged 18≥ treated with venetoclax between January 2019 and July 2022 were included. Survival analysis was calculated based on the period from 2019 to December 2023. A total of 161 AML and 40 patients with MDS were included. The median age was 63.53 ± 15.30 years for AML and 70.12 ± 10.21 years for MDS. In both groups, over 55% are male. A total of 77.6% of patients with AML and 75% of patients with MDS received treatment prior to venetoclax. Venetoclax was administered in combination with azacitidine to 84.5% of AML and 67.5% of MDS. The relapse rate in AML is approximately 15%. Overall, the 2-year survival rate is 46% and 18.73 months. The overall CR/CRi rate for patients with AML is 49.1%, while for patients with MDS, it is 50%. The 2-year survival rate for patients with MDS is 52.7%. The 2-year RFS rate was 75.5% for AML and 90.9% for MDS. The relapse rate in AML is approximately 15%. The percentage of adverse events leading to treatment discontinuation among those with grade 3-4 toxicity is low; 26.7% for AML ( = 43) and 15% for MDS ( = 6). Our real-world data demonstrate that venetoclax has the potential to improve overall survival rates when used in combination with HMAs and supports its use in patients with AML/MDS.
急性髓系白血病和骨髓增生异常综合征均为主要影响老年人的克隆性血液系统恶性肿瘤。目前 AML/MDS 的治疗方法数量有限且疗效有限。本研究旨在评估 Venetoclax 在 AML/MDS 中的治疗效果，重点关注总生存率和无复发生存率，并扩大其应用的真实世界数据。
纳入了 2019 年 1 月至 2022 年 7 月期间接受 Venetoclax 治疗的年龄≥18 岁的 AML/MDS 患者的临床和实验室数据。生存分析基于 2019 年至 2023 年 12 月的时间段进行计算。
共纳入 161 例 AML 和 40 例 MDS 患者。AML 的中位年龄为 63.53±15.30 岁，MDS 的中位年龄为 70.12±10.21 岁。两组中超过 55%为男性。AML 患者中有 77.6%和 MDS 患者中有 75%在接受 Venetoclax 治疗前接受了治疗。Venetoclax 联合阿扎胞苷用于 84.5%的 AML 和 67.5%的 MDS。AML 的复发率约为 15%。总的来说，2 年生存率为 46%，无进展生存期为 18.73 个月。AML 患者的总体完全缓解/完全缓解伴血细胞计数不完全恢复率为 49.1%，而 MDS 患者的这一比例为 50%。MDS 患者的 2 年生存率为 52.7%。AML 的 2 年无复发生存率为 75.5%，MDS 为 90.9%。AML 的复发率约为 15%。3-4 级毒性导致治疗中断的不良事件比例较低；AML 为 26.7%（=43），MDS 为 15%（=6）。
我们的真实世界数据表明，Venetoclax 与 HMAs 联合使用时有可能提高总生存率，支持其在 AML/MDS 患者中的应用。
