Objective: To investigate the efficacy and safety of venetoclax (VEN) combined with demethylating agents (HMA) in the treatment of relapsed/refractory acute myeloid leukemia (R/R AML).
Methods: The clinical data of 26 adult R/R AML patients who received the combination of VEN with azacitidine (AZA) or decitabine (DAC) in Huai'an Second People's Hospital from February 2019 to November 2021 were retrospectively analyzed. The treatment response, adverse events as well as survival were observed, and the factors of influencing the efficacy and survival were explored.
Results: The overall response rate (ORR) of 26 patients was 57.7% (15 cases), including 13 cases of complete response (CR) and CR with incomplete count recovery (CRi) and 2 cases of partial response (PR). Among the 13 patients who got CR/CRi, 7 cases achieved CRm (minimal residual disease negative CR) and 6 cases did not, with statistically significant differences in overall survival (OS) and event-free survival (EFS) between the two groups (P=0.044, 0.036). The median OS of all the patients was 6.6 (0.5-15.6) months, and median EFS was 3.4 (0.5-9.9) months. There were 13 patients in the relapse group and refractory group, respectively, with response rate of 84.6% and 30.8% (P=0.015). The survival analysis showed that the relapse group had a better OS than the refractory group (P=0.026), but there was no significant difference in EFS (P=0.069). Sixteen patients who treated for 1-2 cycles and 10 patients who treated for more than 3 cycles achieved response rates of 37.5% and 90.0%, respectively (P=0.014), and patients treated for more cycles had superior OS and EFS (both P<0.01). Adverse effects were mainly bone marrow suppression, complicated by various degrees of infection, bleeding, and gastrointestinal discomfort was common, but these could be all tolerated by patients.
Conclusion: VEN combined with HMA is an effective salvage therapy for patients with R/R AML and is well tolerated by patients. Achieving minimal residual disease negativity is able to improve long-term survival of patients.
题目: 维奈克拉联合去甲基化药物治疗复发/难治急性髓系白血病的临床观察.
目的: 探讨维奈克拉联合去甲基化药物治疗复发/难治急性髓系白血病（R/R AML）的有效性和安全性。.
方法: 回顾性分析2019年2月至2021年11月在淮安市第二人民医院接受维奈克拉与阿扎胞苷或地西他滨联合治疗的26例成人R/R AML患者的临床资料，观察患者治疗反应、不良反应及生存情况，并探讨影响疗效和生存的因素。.
结果: 26例患者的总反应率为57.7%（15例），其中完全缓解（CR/CRi）13例，部分缓解（PR）2例；13例获得CR/CRi的患者中达CRm（微小病变残存阴性的CR）7例，未达CRm 6例，两组总生存期（OS）和无事件生存期（EFS）比较差异均有统计学意义（P=0.044，0.036）。患者总体中位OS为6.6（0.5-15.6）个月，中位EFS为3.4（0.5-9.9）个月，复发组、难治组患者的治疗反应率分别为84.6%（11/13）和30.8%（4/13）（P=0.015），生存分析结果显示，复发组较难治组具有更佳的OS（P=0.026），但EFS差异无统计学意义（P=0.069）。16例治疗1-2个疗程，10例治疗≥3个疗 程，反应率分别为37.5%和90.0%（P=0.014），疗程数多的患者具有更优的OS和EFS（两者均P<0.01）。患者不良反应主要是骨髓抑制，并发不同程度的感染、出血，胃肠道不适反应较为常见，但患者均可耐受。.
结论: 维奈克拉联合去甲基化药物是R/R AML患者有效的挽救性治疗方案，患者耐受性较好。治疗效果达微小病变残存阴性即更深程度的缓解能够改善患者的远期生存。.
Keywords: azacitidine; decitabine; demethylating agen; relapsed/refractory acute myeloid leukemia; venetoclax.