维奈克拉联合阿扎胞苷治疗复发难治性急性髓系白血病疗效分析

Efficacy of Venetoclax Combined with Azacitidine in the Treatment of Refractory and Relapsed Acute Myeloid Leukemia

目的:探讨维奈克拉联合阿扎胞苷治疗复发难治性(refractory relaspse,R/R)急性髓系白血病(acute my⁃eloid leukemia,AML)的疗效及安全性。方法:回顾性分析2020年10月至2024年1月本院血液内科收治的19例R/R AML患者,评估该方案的疗效及安全性。结果:19例患者经过1个疗程维奈克拉联合阿扎胞苷方案治疗后,完全缓解(complete response,CR)/完全缓解伴不完全血液学恢复(CR with incomplete blood count recovery,CRi)率为68.42%,客观缓解率(objective remission rate,ORR)为73.68%。与Non-CR/CRi患者相比,获得CR/CRi患者中复发患者占比高,且这些患者中NPM1、CEBPA、TET2突变比率高(均P<0.05)。19例R/R AML患者中位随访时间为321 d,中位OS为287 d;与Non-CR/CRi患者相比,CR/CRi患者的生存时间显著改善(P<0.05)。血液学不良反应以贫血多见,其中≥3级贫血的发生率达52.63%;非血液学不良反应中,革兰阳性细菌性肺炎最为常见,发生率为52.63%。结论:维奈克拉联合阿扎胞苷对R/R AML患者有良好的治疗效果,缓解率高且安全性好。NPM1、CEB⁃PA、TET2突变可能是预测患者治疗效果的生物学标志。

Objective To explore the efficacy and safety of venetoclax combined with azacitidine in the treatment of patients with refractory and relapsed acute myeloid leukemia(R/R AML).Methods A retrospective analysis was conducted on 19 R/R AML patients admitted to the Department of Hematology of Taihe Hospital,Hubei University of Medicine from October 2020 to January 2024,to evaluate the therapeutic efficacy and safety of this regimen.Results After one course of treat⁃ment,the complete remission(CR)with with incomplete count recovery(CRi)rate was 68.42%,and the objective remis⁃sion rate(ORR)was 73.68%.Patients with CR/CRi had higher recurrence and mutations of NPM1,CEBPA,and TET2 than those with non-CR/CRi(all P<0.05).The median follow-up time was 321 d among the 19 R/R AML patients,with a median overall survival(OS)of 287 d.Patients with CR/CRi had improved survival compared with those with non-CR/CRi(P<0.05).Hematological adverse reactions,particularly anemia,were common,with the prevalence of anemia≥grade 3 being 52.63%.Among non-hematological adverse reactions,gram-positive bacterial pneumonia was the most com⁃mon(52.63%).Conclusion Venetoclax combined with azacitidine is an effective and safe treatment option for R/R AML patients,with high remission.Mutations of NPM1,CEBPA,and TET2 serve as potential markers for predicting therapeutic outcomes in patients.