AbstractObjective: To investigate the efficacy and safety of venetoclax-based induction chemotherapy in newly diagnosed (ND) patients ineligible for intensive therapy and patients with relapsed/refractory (R/R) acute myeloid leukemia (AML).
Methods: The clinical data of 51 newly diagnosed patients ineligible for intensive therapy and patients with R/R AML treated in the Department of Hematology of Xijing Hospital from February 1, 2021 to April 30, 2022 were retrospectively analyzed. The incidence of complete remission (CR)/CR with incomplete hematological recovery (CRi), objective remission rate (ORR), minimal residual disease (MRD) status, advense events (AE), overall survival (OS) and progression-free survival (PFS) were analyzed.
Results: Among 51 patients, 32 patients were newly diagnosed patients unfit for intensive therapy, with a median age of 60 (29-88) years, and 19 patients were R/R patients, with a median age of 49 (22-92) years. The median cycles of VEN-based treatment in the two groups were both 2. The CR/CRi rates in the ND-AML and R/R-AML group after one course of induction treatment were 65.6% and 36.9%, respectively, and the ORR were 81.3% and 42.1%, respectively. The cumulative CR/CRi rates after 1-3 courses of VEN-based treatment were 71.9% and 47.4%, respectively. The MRD negativity rates of patients achieving CR/CRi were 69.6% and 33.3%, respectively. In the ND-AML and R/R-AML group, the median PFS were 8(5-11) and 3(1-5) months, and the median OS were 13 (6-20) and 5 (3-7) months, respectively. The median OS of patients achieving CR/CRi in both groups was significantly better than that of patients not achieving CR/CRi (13 months vs 4 months; OS not reached vs 4 months). During the first induction cycle, the incidence of grade 3 or higher granulocytopenia, anemia and thrombocytopenia was 96%, 90.2% and 84.3%, respectively. 30 patients (58.8%) had granulocytopenia with fever. The most common non-hematological AE was infection (12/51, 23.5%), followed by gastrointestinal symptoms (6/51, 11.8%).
Conclusion: The VEN-based strategy has good treatment response and tolerance in newly diagnosed patients unfit for intensive therapy and R/R AML. The most common AEs are hematological toxicities and infection.
题目: 维奈克拉单药或联合治疗急性髓系白血病疗效及安全性分析.
目的: 探讨基于维奈克拉（VEN）的诱导治疗在初治不宜强化化疗及复发难治（R/R）急性髓系白血病（AML）患者中的疗效及安全性.
方法: 回顾性分析西京医院血液内科于2021年2月1日至2022年4月30日收治的使用含维奈克拉诱导治疗的初治不适合强化化疗（初治虚弱组）及复发/难治性（R/R组） AML患者共51例的临床资料，分析患者的诱导治疗缓解率（CR/CRi）、总体反应率（ORR）、微小残留病（MRD）、治疗相关不良事件、总生存期（OS）及无进展生存期（PFS）.
结果: 初治虚弱组患者32例，中位年龄60（29-88）岁；R/R组患者19例，中位年龄49（22-92）岁。初治虚弱组和R/R组接受VEN治疗的中位疗程数均为2个。初治虚弱组及R/R组经1个疗程诱导治疗后CR/CRi率分别为65.6%和36.9%，ORR分别为81.3%和42.1%，经1-3个疗程治疗后累计CR/CRi率分别为71.9%和47.4%，达到CR/CRi患者的MRD转阴率分别为69.6%和33.3%。初治虚弱组及R/R组患者的中位PFS分别为8（5-11）和3（1-5）个月，中位OS分别为13（6-20）和5（3-7）个月。两组达到CR/CRi的患者中位OS均显著优于未达到CR/CRi的患者（13 vs 4个月、OS未达到 vs 4个月）。首次诱导治疗期间有3级以上粒细胞、血红蛋白及血小板减少的患者分别占96%、90.2%、84.3%，粒细胞缺乏伴发热患者有30例（58.8%）。最常见的非血液学AE为感染（12/51，23.5%），其次为胃肠道反应（6/51，11.8%）.
结论: 以VEN为基础的治疗方案在初治不宜强化化疗及R/R AML患者中诱导治疗反应率高，总体耐受性良好，最常见的不良反应为血液学毒性及感染.
Keywords: acute myeloid leukemia; hypomethylating agent; newly diagnosed; relapsed/ refractory; venetoclax.