In this prospective donor vs. no-donor analysis of adults (age 18 to 60 years) having AML with the NPM1 mutation, allogeneic HCT in first remission using HLA-identical sibling donors led to a significantly prolonged relapse-free survival (RFS) compared to consolidation or autologous HCT. This multi-center trial of 1,179 patients included 304 eligible patients with NPM1 mutation and intermediate-risk karyotype; 77 patients had a sibling donor and 227 had no available matched family donor. Three-year RFS in the donor and no-donor groups were 71% and 47%, respectively, (p=0.005). The study also found no statistically significant difference in overall survival in donor and no-donor groups, 70% vs. 60%, respectively, (p=0.114), which the authors noted was most likely because AML patients with the NPM1 mutation who relapse respond well to salvage treatment.
Donor vs. No Donor Study: Better Outcome with Allo-HCT in NPM1-Mutated AML