Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Adult Patients With Acute Myeloid Leukemia Harboring KMT2A Rearrangement and Its Prognostic Factors

rearrangement ( -r) in patients with acute myeloid leukemia (AML) is associated with poor outcomes; the prognostic factors after allogeneic hematopoietic stem cell transplantation (allo-HSCT) remain unclear. We investigated 364 adults with AML who underwent allo-HSCT between April 2016 and May 2022, and 45 had -r among them. Propensity score analysis with 1:1 matching and the nearest neighbor matching method identified 42 patients in -r and non- -r cohorts, respectively. The 2-year overall survival (OS), relapse-free survival (RFS), cumulative incidence of relapse (CIR), and non-relapsed mortality rates of patients with -r ( = 45) were 59.1%, 49.6%, 41.5%, and 8.9%, respectively. Using propensity score matching, the 2-year OS rate of patients with -r ( = 42) was lower than that of those without -r ( = 42; 56.1% vs 88.1%, = 0.003). Among patients with -r ( = 45), the prognostic advantage was exhibited from transplantation in first complete remission (CR1) and measurable residual disease (MRD) negative, which was reflected in OS, RFS, and CIR ( < 0.001, < 0.001, and = 0.002, respectively). Furthermore, patients with had poorer outcomes than those with , , and other -r subtypes ( = 0.032, = 0.001, and = 0.001 for OS, RFS, and CIR, respectively). However, no differences were found in the OS, RFS, and CIR between patients with -r with and without mutations (all > 0.05). Univariate and multivariate analyses revealed that achieving CR1 MRD negative before HSCT was a protective factor for OS [hazard ratio (HR) = 0.242, = 0.007], RFS (HR = 0.350, = 0.036), and CIR (HR = 0.271, = 0.021), while was a risk factor for RFS (HR = 2.985, = 0.028) and CIR (HR = 4.675, = 0.004). The prognosis of patients with -r AML was poor, particularly those harboring -related translocation; however, it is not associated with the presence of mutations. These patients can benefit from achieving CR1 MRD negative before HSCT.