Transplant outcomes in WT1 mutant vs wild-type WT1 AML

A retrospective cohort study from the Acute Leukemia Working Party (ALWP) of the European Society for Blood and Marrow Transplantation (EBMT) compared transplant outcomes in 703 patients with de novo or secondary acute myeloid leukemia (AML) with Wilms tumor mutation (mWT1) (n = 50) vs those with wild-type WT1 (wtWT1) (n = 653). The study endpoints included 2-year overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), non-relapse mortality (NRM), and chronic GvHD (cGvHD). Nagler et al. published the results in Bone Marrow Transplantation.

Key data: Patients with mWT1 were younger (p < 0.001), with a higher proportion of females (p = 0.01), and had a higher frequency of FLT3-ITD (p = 0.01) and CEBPA (p = 0.03) mutations vs those with wtWT1. In the matched-pair analysis (mWT1 [n = 49] vs wtWT1 [n = 127]), transplant outcomes at 2 years were similar: RI (28.8% vs 30.4%; p = 0.64), NRM (15.5% vs 9.9%; p = 0.49), LFS (55.7% vs 59.6%; p = 0.39), OS (65.4% vs 73.3%; p = 0.66), and cGvHD (24.3% vs 25.4%; p = 0.95).

Key learning: Harboring mWT1 did not influence transplantation outcomes in patients with AML in first remission, suggesting it may not predict adverse prognosis in the transplant setting.