Venetolax plus azacitidine is effective maintenance therapy after allogeneic hematopoietic stem cell transplantation in patients with intermediate/high-risk Acute Myeloid Leukemia

Abstract Background: Relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with intermediate/high-risk acute myeloid leukemia (AML) remains a major cause of treatment failure and associated poor outcomes. Evidence regarding the venetolax plus azacytidine (VEN+AZA) as a prevent relapse therapy for patients with intermediate/high-risk AML after allo-HSCT is scare. Patients and methods： We retrospectively evaluated the outcome of VEN+AZA (n=23) as maintenance therapy to prevent relapse after allo-HSCT for patients with intermediate/high-risk AML enrolled from January 2020 to April 2023, and compared with patients not received any anti-AML therapy as maintenance therapy after allo-HSCT as control group (n=59) (September 2013 - April 2023) in our center. All patients in VEN+AZA group received VEN (200mg/d, on day 1 to 28) and AZA (100mg/d, on day 1 to 5) from 3 months after transplantation. The cycle interval was 1 months, and there were 9 cycles. Patients in the history cohort were not received any anti-AML therapy as maintenance therapy after allo-HSCT. The cumulative incidence of relapse was estimated using competing-risk methods. The probabilities of survival outcomes were calculated according to the Kaplan-Meier method and compared using the long-rank test. Results： The median age of patients in the VEN+AZA group (36 years, [range, 16-63]) compared with control group (35 years, [ranges, 13-57], p=0.706). No significant differences were observed in baseline data, including sex, risk stratification, donor-recipient relationship between two groups. Compared with the control group, the cumulative incidence of relapse was significantly lower in the VEN+AZA group (8.7±6.0% vs. 37.3±6.4%, p=0.029) after transplantation. Overall survival (OS) at three years was better with VEN+AZA group (94.1±5.7%) than control group (61.3±7.0%, p=0.045). Nonrelapse mortality (NRM) at three years in VA group (82.4±12.1%) was also significantly superior to control group (46.5±7.1%, p=0.034). Conclusions： Our data indicate that patients with intermediate/high-risk AML after allo-HSCT received VEN+AZA therapy decreases the relapse rate, and yields better OS and NRM. The role of VEN+AZA as a potential treatment to prevent relapse in intermediate/high-risk AML should be considered.