维奈克拉治疗急性髓系白血病的临床疗效分析——维奈克拉用于急性髓系白血病诱导治疗

Clinical Efficacy Analysis of Venetoclax in the Treatment of Acute Myeloid Leukemia —VEN for Induction Therapy in AML

摘要: 目的：探讨维奈克拉(Venetoclax, VEN)联合去甲基化药物(Hypomethylating agents, HMAs)在不适合强化疗的初诊急性髓系白血病(Newly diagnosed acute myeloid leukemia, ND-AML)患者中的疗效及安全性。方法：回顾性收集2020年10月至2024年1月在安徽医科大学第一附属医院收治的120例AML患者的临床资料，对患者的疗效及不良反应进行分析。结果：在疗效评估中，AML患者的完全缓解(complete response, CR)率为49.2%，CR/完全缓解伴血液学不完全恢复(CR with incomplete hematologic recovery, CRi)率为63.3%，总有效率(overall response rate, ORR)为75%，可检测残留病变(measurable residual disease, MRD)转阴率为62.8%。伴CEBPA突变或低中危染色体核型可能与更好的CR/CRi率相关。在安全性评估中，血液系统不良反应发生率为100%，不同VEN服用疗程之间的血液学毒性和三系脱输注时间无显著差异。结论：VEN + HMAs在初诊AML患者中疗效肯定。几乎所有接受该方案治疗的患者均会出现不良反应，但大多数患者可耐受。

Abstract: Objective: To investigate the efficacy and safety of Venetoclax (VEN) combined with hypomethylating agents (HMAs) in newly diagnosed acute myeloid leukemia (ND-AML) patients ineligible for intensive chemotherapy. Methods: Clinical data of 120 AML patients treated at the First Affiliated Hospital of Anhui Medical University from October 2020 to January 2024 were retrospectively collected, and the efficacy and adverse reactions were analyzed. Results: In efficacy evaluation, the complete response (CR) rate was 49.2%, the CR/complete response with incomplete hematologic recovery (CRi) rate was 63.3%, the overall response rate (ORR) was 75%, and the measurable residual disease (MRD) negativity rate was 62.8% of AML patients. Patients with CEBPA mutations or low-intermediate-risk chromosomal karyotypes were associated with higher CR/CRi rates. In the safety assessment, the incidence of hematologic adverse reactions was 100%, with no significant differences in hematologic toxicity or time to three-lineage transfusion independence between different VEN treatment cycles. Conclusion: The VEN + HMAs regimen demonstrates significant efficacy in newly diagnosed AML patients. Although adverse reactions occur in almost all patients, most are tolerable.