Transplant outcomes after intensive chemotherapy or venetoclax plus azacitidine in acute myeloid leukemia: a multicenter retrospective study in Japan

The present retrospective multicenter study in Japan evaluated the role of venetoclax plus azacitidine (V/A) before first allogeneic hematopoietic stem cell transplantation (SCT) in 144 transplant-eligible acute myeloid leukemia (AML). Thirty-seven patients received V/A prior to SCT, mainly as salvage therapy for refractory and adverse-risk AML. Patients were classified into four groups according to remission status at SCT and V/A use. The 1-year overall survival (OS), cumulative incidence of relapse (CIR) and non-relapse mortality (NRM) of those who achieved complete remission (CR) with intensive chemotherapy (IC) were 93%, 5% and 4%, respectively. The 1-year OS, CIR and NRM of those who were refractory/intolerant to IC and achieved CR with V/A were 93%, 7% and 7%, respectively. The 1-year OS, CIR and NRM of those who were refractory to IC and proceeded directly to SCT were 52%, 54% and 13%, respectively. The 1-year OS, CIR and NRM of those who were refractory/intolerant to IC and refractory to V/A were 41%, 39% and 29%, respectively. Overall, use of V/A prior to SCT salvaged some AML patients who were refractory/intolerant to IC, and the transplant outcomes of these patients were favorable. Further studies are needed to optimize treatment for AML prior to SCT.