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目的:探究维奈克拉联合阿扎胞苷（Ven+AZA）在初治不适合强化疗（unfit）及难治（≥1次强化诱导化疗未缓解）/复发（R/R）急性髓系白血病（AML）患者中的近期疗效。方法:回顾性分析北京大学血液病研究所2019年6月1日至2021年5月31日接受Ven+AZA治疗（Ven 100 mg第1天、200 mg第2天、400 mg第3~28天；阿扎胞苷75 mg/mn 2第1~7天）的初治unfit及R/R AML患者60例。分析各组完全缓解/血细胞计数未完全恢复的完全缓解（CR/CRi）率、微小残留病（MRD）转阴率及总体反应率（ORR）。n 结果:60例初治unfit及R/R AML患者，中位年龄54（18~77）岁，男性33例（55.0%），中位随访时间4.8（1.4~26.3）个月。其中初治unfit患者24例（40.0%），R/R患者36例（60.0%）。初治unfit组及R/R组中位治疗疗程均为1（1~5）个。24例初治unfit患者（依据NCCN预后分层：低危8例、中危2例、高危14例）经第1疗程Ven+AZA治疗后，17例（70.8%）达CR/CRi，3例（12.5%）达部分缓解（PR），ORR为83.3%。其中9例患者接受第2疗程治疗，2例接受第3疗程治疗。17例CR/CRi患者中8例（47.1%）经2个疗程治疗后MRD转阴。36例R/R患者经第1疗程诱导治疗后，21例（58.3%）达CR/CRi（其中7例MRD转阴，占33.3%），3例（8.3%）PR，ORR为66.7%。R/R患者中12例治疗≥2个疗程，第2疗程治疗后无新增缓解患者，累计MRD转阴14例（66.7%）。R/R患者中低危组（CR至血液学复发≥18个月）12例，其余24例为高危组。低危R/R组1个疗程后10例（83.3%）达CR/CRi，CR/CRi率显著高于高危R/R组[45.8%（11/24），n P=0.031]。在60例患者中，13例伴IDH1/2突变及4例TP53阳性患者经1个疗程治疗后均达CR/CRi，18例NPM1突变阳性患者1个疗程CR/CRi率为77.8%，5例RUNX1-RUNX1T1伴c-kit D816患者（2例初诊，3例复发）均未获缓解。Ven+AZA作为诱导及再诱导治疗总体耐受性好。n 结论:Ven+AZA在初治unfit及R/R AML患者中均获得较高的治疗反应率，部分缓解患者可快速获得MRD转阴。其在伴NPM1、IDH1/2、TP53等基因突变的AML患者中疗效显著。“,”Objective:To explore the safety and short-term efficacy of venetoclax combined with azacitidine (Ven+AZA) in previously untreated patients unfit for standard chemotherapy and patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) in China.Methods:A retrospective study was conducted in 60 previously untreated patients unfit for standard chemotherapy and patients with R/R AML who received Ven+ AZA (venetoclax, 100 mg D1, 200 mg D2, 400 mg D3-28; azacitidine, 75 mg/mn 2 D1- 7) at the Peking University Institute of Hematology from June 1, 2019 to May 31, 2021. The incidence of adverse events, complete remission (CR) /CR with incomplete hematological recovery (CRi) rate, objective remission rate (ORR) , and minimal residual disease (MRD) status in patients with different risk stratification and gene subtypes were analyzed.n Results:The median age of the patients was 54 (18-77) years, 33 (55.0%) were males, and the median follow-up time was 4.8 (1.4-26.3) months. Among the 60 patients, 24 (40.0%) were previously untreated patients unfit for standard chemotherapy, and 36 (60.0%) were R/R patients. The median mumber cycles of Ven+AZA in the two groups were both 1 (1-5) . According to the prognostic risk stratification of the National Comprehensive Cancer Network, it was divided into 8 cases of favorable-risk, 2 cases of intermediate risk, and 14 cases of poor-risk. In previously untreated patients unfit for standard chemotherapy, after the first cycle of Ven+AZA, 17/24 (70.8%) cases achieved CR/CRi, 3/24 (12.5%) achieved partial remission (PR) , and the ORR was 83.3%. Among them, nine patients received a second cycle chemotherapy and two received a third cycle. Among CR/CRi patients, 8/17 (47.1%) achieved MRD negativity after two cycles of therapy. In the R/R group, after the first cycle of Ven+AZA, 21/36 (58.3%) cases achieved CR/CRi (7/21 achieved MRD negativity) , 3 achieved PR, and the ORR was 66.7%. Among R/R patients, 12 were treated for more than two cycles. There were no new CR/CRi patients after the second treatment cycle, and 14 cases (66.7%) achieved MRD negativity. According to the time from CR to hematological recurrence, the R/R group was divided into 12 cases in the favorable-risk group (CR to hematological recurrence ≥18 months) and 24 in the poor-risk group (CR to hematological recurrence<18 months, no remission after one cycle of therapy, and no remission after two or more cycles of therapy) . Eleven of 24 (45.8%) cases achieved CR/CRi after one cycle of Ven+AZA in the poor-risk R/R group, and 10 of 12 (83.3%) achieved CR/CRi in the favorable-risk R/R group, which was significantly superior to the poor-risk group (n P=0.031) . After one cycle of treatment, 13 patients with IDH1/2 mutations and 4 that were TP53-positive all achieved CR/CRi. The CR/CRi rate of 18 patients with NPM1 mutations was 77.8%. Five patients with RUNX1-RUNX1T1 combined with KIT D816 mutation (two initial diagnoses and three recurrences) had no remission. Ven+ AZA was tolerable for AML patients.n Conclusion:Ven+AZA has acceptable safety in previously untreated patients unfit for standard chemotherapy, patients with R/R AML can achieve a high response rate, and some patients can achieve MRD negativity. It is also effective in NPM1-, IDH1/IDH2-, and TP53-positive patients. The long-term efficacy remains to be observed.