Hematopoietic stem cell transplantation for adult patients with isolated NPM1 mutated acute myeloid leukemia in first remission

Abstract

Acute myeloid leukemia (AML) in first remission (CR1) with isolated NPM1 mutation (iNPM1m) is considered a good prognosis genotype, although up to one third relapse. To evaluate the best transplant strategy, we retrospectively compared autologous stem cell transplantation (auto‐SCT), related (MSD) and fully matched unrelated (MUD) allogeneic stem cell transplantation (allo‐SCT). We identified 256 adult patients including 125 auto‐SCT, 72 MSD and 59 MUD. The 2‐year leukemia‐free survival (LFS) was 62% in auto‐SCT, 69% in MUD and 81% in MSD (p=0.02 for MSD versus others). The 2‐year overall survival (OS) was not different among auto‐SCT, MUD and MSD, reaching 83% (p=0.88). The 2‐year non‐relapse mortality (NRM) was 2.5% in auto‐SCT and 7.5% in allo‐SCT (p=0.04). The 2‐year cumulative incidence of relapse (RI) was higher after auto‐SCT (30%) than after MUD (22%) and MSD (12%, p=0.01). In multivariate analysis, MSD versus auto‐SCT but not MUD versus auto‐SCT was associated with lower RI (pNPM1m AML in CR1. Auto‐SCT was followed by worse RI and LFS, but similar OS to both allo‐SCT modalities.