Novel Therapy Gilteritinib Effective in Newly Diagnosed AML

By Leah Sherwood - Last Updated: October 12, 2022

A study presented at the 2022 Society of Hematologic Oncology (SOHO) Annual Meeting showed that gilteritinib plus induction and consolidation chemotherapy demonstrated acceptable safety outcomes and efficacy in patients with FLT3-mutated acute myeloid leukemia (AML).

The four-part, open-label, phase I study assessed gilteritinib dose schedules in patients with newly diagnosed AML. Patients received gilteritinib plus 7+3 induction with idarubicin or daunorubicin, plus three or fewer cycles of high-dose cytarabine and gilteritinib consolidation, and two years of single-agent gilteritinib maintenance post-consolidation or transplant.

In this portion of the study, 80 patients were treated, 78 of whom were available for the safety analysis. After a median follow-up of 37.7 months, 24.4% of patients discontinued treatment due to adverse events. About a third of patients (n=27; 33.8%) went on to receive hematopoietic stem cell transplant.

Dose-limiting toxicities occurred in 19.2% of patients (n=15), and the maximum tolerated dose was 120 mg/day.

Among the overall (n=79) and FLT3-mutated (n=44) populations, composite complete remission (CR) occurred in 79.7% and 90.9% of patients, respectively. In these cohorts, the 60-day mortality rates were 1.3% and 0%, respectively, and median disease-free survival was 15.1 months for both cohorts. Estimated three-year overall survival rates were 56.0% and 60.1%, respectively.

“Gilteritinib plus induction and consolidation chemotherapy was well tolerated and resulted in CR, FLT3-ITD clearance, and long-term survival in patients with newly diagnosed FLT3-mutated AML, supporting ongoing randomized trials testing this approach against current standards,” the researchers concluded.


Pratz KW, Cherry M, Podoltsev NA, et al. A phase 1 study of gilteritinib in combination with induction and consolidation chemotherapy in patients with newly diagnosed acute myeloid leukemia: final study results. Abstract #AML-256. Presented at the 2022 Society of Hematologic Oncology (SOHO) Annual Meeting, September 28-October 1, 2022.

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