Multinational real-world study of effectiviness of gilteritinib beyond second-line therapy in relapsed/refractory FLT3-mutated acute myeloid leukemia

Gilteritinib is a selective FLT3 inhibitor approved for the treatment of relapsed or refractory (R/R) FLT3-mutated AML following ≥ 1 prior line of therapy. Information on its effectiviness in later-line settings is limited. A retrospective study across centers in Italy, Spain, Turkey, and the United Kingdom evaluated gilteritinib as third-line or beyond treatment for R/R FLT3-mutated AML. Among 171 patients, 84% carried FLT3-ITD mutations. The composite complete remission rate was 28%, and the objective response rate was 47%. Patients who were younger and presented with relapsed (versus refractory) disease had better outcomes. Prior exposure to venetoclax or allogeneic hematopoietic stem cell transplantation was associated with inferior response. Gilteritinib enabled HSCT in 12% of patients. Median overall survival was 7.1 months (95% CI 5.9-10.1) and was significantly improved among responders and those who underwent HSCT (21.5 months; 12.8-NR). The authors conclude that, despite heavy pretreatment, gilteritinib retained clinically relevant activity in later-line R/R FLT3-mutated AML (graphical abstract).