Real-world experience with gilteritinib maintenance following allogeneic transplantation in relapsed/refractory AML...

Gilteritinib is an established FLT3 inhibitor used to treat patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) harboring FLT3 mutations. Prior studies have predominantly evaluated gilteritinib as a posttransplant maintenance therapy in patients who undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT) in complete remission. Nonetheless, evidence remains limited for patients who receive gilteritinib as salvage therapy in a relapsed or refractory setting, proceed to allo-HSCT, and subsequently continue gilteritinib maintenance. This real-world study aimed to address this knowledge gap in an Asian cohort.

Patients with FLT3-mutated R/R AML received gilteritinib before transplantation, and those achieving engraftment without grade ≥ 2 acute graft-versus-host disease were offered posttransplant maintenance between days 30 and 90. The cohort (median age, 48 years; 92.0% FLT3-ITD) achieved a complete response (CR) rate of 76.2%. At a median follow-up of 46.2 months, gilteritinib maintenance therapy was associated with longer relapse-free and overall survival and a significantly lower 1-year cumulative incidence of relapse. Exploratory measurable residual disease (MRD) subgroup analyses suggested a numerically greater survival difference in patients without flow cytometry-detectable MRD before or after transplantation; however, the findings are limited by the small sample size and lack of FLT3-targeted next-generation sequencing-based MRD assessment.

Gilteritinib was generally well-tolerated, with no observed increase in cytomegalovirus (CMV)-related or graft-versus-host complications. This study provides real-world evidence in a clinically relevant scenario and supports the use of gilteritinib maintenance in patients with FLT3-mutated AML who undergo transplantation during R/R disease.