Abstract: Objective To investigate the clinical efficacy and safety of Venetoclax combined with Azacitidine and Chidamide in treating the patients with refractory and relapsed acute myeloid leukemia (R/R AML). Methods The efficacy and safety of Venetoclax combined with Azacitidine and Chidamide for 6 R/R AML patients treated in the Department of Hematology, Peking University People's Hospital, from March 15, 2021 to November 30, 2021, were retrospectively analyzed. The steady-state trough concentrations (Cmin) of Venetoclax at different times during the induction period (day 7, 14, 21 and 28) were monitored and counted. Results Among the 6 R/R AML patients, 3 patients showed clinical response, including 2 complete remission (CR) and 1 CR with incomplete hematological recovery (CRi). The median follow-up time was 178 days. There were 5 patients currently alive, with an overall survival (OS) rate of 83.3%(5/6). The steady-state Cmin of Venetoclax on day 7, 14, 21 and 28 were (3 181.67±1 631.90) ng/mL, (2 688.67±1 926.36) ng/mL, (2 142.17±1 380.35) ng/mL, (2 766.67±1 138.55) ng/mL, respectively. Conclusions Venetoclax combined with Azacitidine and Chidamide can improve the clinical efficacy in R/R AML patients, and such a combination is expected to be one of the feasible options for the salvage therapy of R/R AML patients.

Key words: Acute myeloid leukemia, Refractory/relapsed, Venetoclax, Azacitidine, Chidamide