Weng Guang-Yang, You Wei-Wen, Liu Huan-Xun, Cai Yun, DU Xin
Department of Hematology, Shenzhen Second People's Hospital, Shenzhen 518000, Guangdong Province, China.
Department of Hematology, Shenzhen Second People's Hospital, Shenzhen 518000, Guangdong Province, China.E-mail:
Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2023 Oct;31(5):1333-1339. doi: 10.19746/j.cnki.issn.1009-2137.2023.05.013.
To explore the efficacy of venetoclax (VEN) plus azacitidine (AZA) in patients with mutated relapsed/refractory acute myeloid leukemia ( R/R AML) and analyze the molecular genetic characteristics of the patients.
Clinical baseline characteristics and follow-up data of 16 R/R AML patients treatd with VEN plus AZA in the hematology department of Shenzhen Second People's Hospital from November 2018 to April 2021 were collected. Leukemia related genes were detected by next-generation sequencing(NGS) or PCR. The relationship between the efficacy of VEN plus AZA and molecular genetics characteristics of patients with R/R AML were analyzed.
14.3% (1/7) of the patients in group and 22.2% (2/9) of the patients in group achieved complete remission (CR)/CR with incomplete blood count recovery (CRi), respectively, with no significant difference (=0.69). There was no significant difference in overall response rate (ORR) (CR/CRi+PR) between group and group [42.9%(3/7) 44.4%(4/9), =0.95], too. The median overall survival (OS) time of patients was significantly shorter than that of patients (130 300 days, respectively) ( =0.02). Co-existing mutations of and were detected in one patient who achieved CR. Co-existing mutations of and were found in one patient who achieved PR. Three R/R AML patients accompanied with mutation had no response to VEN plus AZA.
VEN plus AZA showed a certain effect on patients with R/R AML. To improve OS of the patients, bridging transplantation is need. and mutations might predict that patients with R/R AML have treatment response to VEN plus AZA, while the combination of mutation may indicate poor response.
探讨维奈克拉(VEN)联合阿扎胞苷(AZA)治疗突变型复发/难治性急性髓系白血病(R/R AML)患者的疗效,并分析患者的分子遗传学特征。
收集2018年11月至2021年4月在深圳市第二人民医院血液科接受VEN联合AZA治疗的16例R/R AML患者的临床基线特征和随访数据。采用二代测序(NGS)或聚合酶链反应(PCR)检测白血病相关基因。分析VEN联合AZA的疗效与R/R AML患者分子遗传学特征之间的关系。
A组14.3%(1/7)的患者和B组22.2%(2/9)的患者分别达到完全缓解(CR)/血细胞计数未完全恢复的CR(CRi),差异无统计学意义(P=0.69)。A组和B组的总缓解率(ORR,CR/CRi+PR)也无显著差异[分别为42.9%(3/7)和44.4%(4/9),P=0.95]。A组患者的中位总生存期(OS)明显短于B组患者(分别为130天和300天)(P=0.02)。1例达到CR的患者检测到IDH2和FLT3共突变。1例达到PR的患者发现IDH1和NRAS共突变。3例伴有TP53突变的R/R AML患者对VEN联合AZA无反应。
VEN联合AZA对R/R AML患者有一定疗效。为提高患者的OS,需要进行桥接移植。IDH1和IDH2突变可能预测R/R AML患者对VEN联合AZA有治疗反应,而TP53突变可能提示反应不佳。