Prognostic impact of WT1 dynamics in peripheral blood before and after allogeneic HSCT in patients with AML and MDS

Wilms tumor gene 1 (WT1) is frequently identified in hematologic malignancies; however, its utility for measurable residual disease (MRD) assessment remains controversial. We investigated the prognostic impact of WT1 dynamics in peripheral blood (PB) before and after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

Of 85 patients with AML or MDS who underwent allo-HSCT at our institution between January 2016 and March 2025, 51 who had WT1 in PB assessed before and approximately 100 days after transplantation were analyzed retrospectively. Patients were stratified by WT1 status before and/or after allo-HSCT. WT1 expression < 50 copies/µg RNA was defined as MRD-negative.

In univariate analysis, 2-year progression-free survival (PFS), overall survival (OS), and cumulative incidence of relapse (CIR) differed significantly between Group A (MRD−/MRD−), Group B (MRD+/MRD−), and Group C (MRD+/MRD+) (PFS: 93.8% vs 67.0% vs 31.2%; OS: 93.8% vs 85.4% vs 48.6%; CIR: 0% vs 28.2% vs 68.7%). In multivariate analysis, conversion from WT1 positivity to negativity after allo-HSCT was associated with a favorable prognosis. No patients who were WT1-negative before allo-HSCT relapsed in this cohort.

WT1 status before and after allo-HSCT is a relevant prognostic marker.