Acute Myeloid Leukemia Coverage From Every Angle

ASH 2020: Phase III Trial of Gilteritinib Plus Azacitidine for FLT3-Mutated AML

By: Julia Fiederlein
Posted: Wednesday, December 23, 2020

Researchers are currently recruiting patients for a multicenter phase III trial studying the safety and efficacy of treatment with azacitidine alone and in combination with the FMS-like tyrosine kinase 3 (FLT3) inhibitor gilteritinib in patients with newly diagnosed FLT3-mutated acute myeloid leukemia (AML) who are ineligible for intensive induction chemotherapy. Eunice S. Wang, MD, of Roswell Park Comprehensive Cancer Center, Buffalo, and colleagues shared the study design and preliminary data during the virtual edition of the 2020 American Society of Hematology (ASH) Annual Meeting and Exposition (Abstract 27).

The trial was initiated with a safety cohort; a total of 15 patients were administered 80 mg of gilteritinib as the initial dose (or 120 mg as the next dose level) plus 75 mg/m2 of azacitidine. The target enrollment for the randomization cohort is approximately 250 patients. In a 2:1 allocation ratio, they will be randomly assigned to receive either 75 mg/m2 of azacitidine alone or in combination with gilteritinib; as of June 29, 2020, 114 patients were administered one of these treatments. The gilteritinib-alone arm was removed from the current protocol; a total of 22 patients were randomly assigned to this treatment.  

The investigators identified overall survival as the primary endpoint. Event-free survival, best response, remission rates and duration, transfusion conversion and maintenance rates, leukemia-free survival, patient-reported fatigue, and safety and tolerability will be evaluated as secondary endpoints.

In the safety cohort, a total of 14 patients died. Mature remission data revealed a composite complete remission rate of 67%. Based on this result, the investigators chose to administer 120 mg of gilteritinib plus azacitidine to the randomization cohort. A death rate of 61% was observed in these patients. Thus far, the safety profile appears to be congruent with previous reports.

Disclosure: For full disclosures of the study authors, visit

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