What question did this study set out to answer?

This research seeks to identify prognostic factors affecting outcomes in patients with NPM1-mutated AML after transplantation.

February 14, 2026Open Access

Prognostic impact of clinico-genetic characteristics and donor types in NPM1 -mutated AML undergoing allogeneic hematopoietic stem cell transplantation

Puntos clave

This research seeks to identify prognostic factors affecting outcomes in patients with NPM1-mutated AML after transplantation.
Enrolled 139 patients with NPM1-mutated AML who underwent allogeneic HSCT.
Analyzed clinical and genetic characteristics associated with prognosis.
Conducted multivariable analysis to identify significant predictors of outcomes.
3-year cumulative incidence of relapse was 15.1%; leukemia-free survival was 78.4%; overall survival was 78.0%.
FLT3-ITD mutations linked to higher relapse rates and lower survival outcomes.
Matched sibling donor HSCT had worse outcomes compared to unrelated donors.
Pre-HSCT minimal residual disease (MRD) status was a critical predictor for outcomes.

Resumen

NPM1 -mutated acute myeloid leukemia (AML) is a distinct entity with specific biological and clinicopathological features. Prognosis varies depending on the associated mutations and clinical characteristics. This study evaluated the prognostic factors of NPM1 -mutated AML in patients who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT). We enrolled 139 patients with NPM1 -mutated AML who underwent allo-HSCT. The median age at allo-HSCT was 47 years with a median follow-up of 1,134 days. The 3-year cumulative incidence of relapse (CIR), leukemia-free survival (LFS), and overall survival (OS) rates were 15.1%, 78.4%, and 78.0%, respectively. FLT3-ITD mutations showed higher CIR (22.6% vs 5.3%, P = 0.006), lower LFS (70.9% vs 88.1%, P = 0.019), and reduced OS (70.8% vs 87.5%, P = 0.036). FLT3-ITD and DTA mutations indicated adverse prognosis, while IDH1/2 mutations showed improved outcomes. Matched sibling donor (MSD) allo-HSCT patients had higher CIR and lower LFS and OS than unrelated donor (URD) or haploidentical-related donor (HRD) recipients. Pre-HSCT MRD status affected the outcomes, with MRD+ and nCR patients showing worse outcomes. Multivariable analysis identified FLT3-ITD , MSD, and pre-MRD status as significant predictors. In conclusion, FLT3-ITD and DTA mutations are associated with an adverse prognosis, whereas HRD or URD transplants may offer better outcomes than MSD allo-HSCT.

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