- Treatment with gilteritinib improved survival vs standard chemotherapy in patients with FLT3-mutated relapsed/refractory (R/R) acute myeloid leukemia (AML).
Why this matters
- Relapse is common in AML, and up to 40% of patients are refractory to induction chemotherapy.
- R/R AML is associated with short survival and poor response to salvage chemotherapy.
- Poor prognosis is associated with the FLT3 mutation, which is present in about one-third of AML cases diagnosed in the U.S.
- The ADMIRAL trial was a randomized, phase 3 clinical trial of 371 patients with R/R FLT3-mutated AML randomly assigned to gilteritinib (247 patients) or investigators' choice (prior to randomization) of standard chemotherapeutic regimens (124 patients) prior to hematopoietic stem cell transplantation.
- Funding: Astellas Pharma US Inc.
- 84 (34%) patients receiving gilteritinib had complete response (CR) or CR with partial hematologic recovery, compared with 19 (15%) patients who received standard chemotherapy.
- Median OS and 12-month survival were 9.3 months and 37.1% in the gilteritinib arm vs 5.6 months and 16.7% in the standard chemotherapy arm.
- Due to FDA approval of an FLT3-targeted agent during enrollment, a small number of patients in both arms received prior FLT3-targeted therapy, which may have reduced response to gilteritinib.
- Gilteritinib is highly selective in its activity, leaving open the possibility that other mutations might drive growth of the cell, especially in the R/R stage.
- "This is a new, practice-changing strategy that is something that we've been needing in the field of AML therapy for the entire time I've been an oncologist, so to have something like this is really special and important," said Dr. Louis Wiener, co-chair of the AACR Annual Meeting Clinical Trials Committee and director of the Georgetown Lombardi Comprehensive Cancer Center.