Refractory patients with favorable/intermediate‐risk acute myeloid leukemia benefit from azacytidine maintenance therapy following allogeneic hematopoietic stem cell transplantation

Abstract

AbstractRecurrence following allogeneic hematopoietic stem cell transplantation (allo‐HSCT) is the major cause of treatment failure in patients with myeloid malignancy. Azacytidine (AZA) maintenance is a promising therapy to prevent relapse and improve survival. We conducted a prospective, one‐arm study involving 78 patients with myeloid malignancy at a high risk of recurrence who were enrolled between September 2019 and April 2022. Furthermore, 102 matched historical controls were selected using propensity score matching. With a median follow‐up time of 19.6 (3.5–91.7) months, AZA maintenance therapy significantly improved relapse‐free survival (RFS; log‐rank test, p = 0.01). The AZA and control groups had a 1‐year RFS of 87.7% (95% confidence interval [CI], 0.80–0.96) and 72.2% (95% CI, 0.64–0.82), respectively, with a hazard ratio (HR) of 0.21 (95% CI, 0.09–0. 47; p < 0.01). There were no grade 4 adverse effects or deaths related to AZA. Refractory patients with favorable/intermediate‐risk acute myeloid leukemia (AML) benefited more from AZA maintenance therapy than those with adverse‐risk AML according to the European Leukemia Net guidelines (RFS in favorable/intermediate‐risk AML, HR = 0.29, 95% CI, 0.11–0.79; RFS in adverse‐risk AML, HR = 0.57, 95% CI, 0.21–1.6; p for interaction = 0.03). Our findings suggest that AZA maintenance therapy following allo‐HSCT was safe and could reduce the incidence of relapse, particularly for refractory patients with favorable/intermediate‐risk AML.