[Efficacy of Venetoclax Plus Azacitidine in Relapsed/Refractory Acute Myeloid Leukemia Patients with Mutation].

Objective: 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.

Methods: 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.

Results: 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.

Conclusion: 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.