Relationship Between Salvage Strategy and Outcomes in Patients With CBF or NPM1-mutated AML and First Molecular...

Given the uncertainty regarding the optimal management of molecular relapse in patients with CBF or NPM1-mutated AML, we retrospectively analyzed the outcome of 121 adults from 12 centers with CBF (n=28) or NPM1-mutated (n=93) AML and first molecular relapse according to the salvage strategy used (upfront allogeneic HCT [n=19], intensive chemotherapy [IC; n=21], venetoclax and azacitidine [VEN-AZA]; n=70), and other strategies (n=11; including AZA, gemtuzumab ozogamicin, selective inhibitors). At three years, OS was not statistically different between the four groups (84% for upfront allo vs. 81% for IC vs. 79% for VEN-AZA vs. 64% for other, P=0.31). Allogeneic HCT was received by 98 patients (81%) with a cumulative incidence of allogeneic HCT at 12 weeks of 100% for upfront allo, 71% for IC, 73% for VEN-AZA, and 82% for other (P<0.001) with better outcomes in transplanted patients. In patients who received allogeneic HCT, type of salvage therapy was not statistically associated with post-HCT relapse, relapse-free survival, or OS. Our data suggests that upfront allogeneic is a valuable option, if feasible, while other salvage strategies are associated with favorable outcomes and relatively low non-relapse mortality after allogeneic HCT.