Gilteritinib significantly improved survival among patients with relapsed/refractory FLT3-mutated acute myeloid leukemia (AML), according to a study published in The New England Journal of Medicine.
This phase III trial randomized 371 adults (median age = 62 years) with relapsed/refractory FLT3-mutated AML 2:1 to receive gilteritinib 120 mg daily (n=247) or salvage chemotherapy (n=124).
Improved survival with gilteritinib
After a median follow-up of 17.8 months, median overall survival (primary endpoint) was 9.3 months in the gilteritinib group compared with 5.6 months in the chemotherapy cohort (hazard ratio [HR] for death = 0.64; 95% CI, 0.49-0.83; P<0.001). One-year survival rates were 37.1% with gilteritinib and 16.7% with salvage chemotherapy. Median event-free survival was 2.8 months and 0.7 months, respectively (HR for treatment failure or death = 0.79; 95% CI, 0.58-1.09).
The percentage of patients who had complete remission (CR) with full or partial hematologic recovery was 34.0% in the gilteritinib group and 15.3% in the chemotherapy group (risk difference = 18.6 percentage points; 95% CI, 9.8-27.4). The percentage of patients in CR were 21.1% and 10.5%, respectively (risk difference = 10.6 percentage points; 95% CI, 2.8-18.4).
Grade ≥3 adverse events (AEs) and serious AEs occurred less frequently in the gilteritinib group (19.34 vs. 42.44); the most common AEs that occurred in the gilteritinib group were febrile neutropenia (45.9%), anemia (40.7%), and thrombocytopenia (22.8%).