Efficacy of allogeneic haematopoietic stem cell transplantation in acute myeloid leukemia with myelodysplasia-related genetics

Acute myeloid leukemia with myelodysplasia-related (AML-MR) gene mutations is associated with high rates of relapse and mortality and has limited prognostic benefits. To assess the efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in AML-MR patients, 196 AML-MR patients who underwent allo-HSCT at our center were retrospectively analyzed. Patients who received hypomethylating agents (HMAs) +/- venetoclax (VEN) as bridging therapy had improved overall survival (OS) and reduced relapse-free survival (RFS) compared with those who did not. Patients who failed to achieve cCR prior to transplantation benefit from HMA/VA bridging therapy compared with those who proceeded directly to allo-HSCT. Among patients in CR, those with AML-MR associated cytogenetic abnormalities at diagnosis also benefit from bridging therapy. Multivariate analysis revealed that older age, positive MFC-MRD, positive NGS-MRD and bridging therapy were independent risk factors. Thus, HMA/VA bridging therapy improves survival in AML-MR patients, especially those with positive AML-MR genetics before transplantation.