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Triplet Therapy Achieves ‘Encouraging Survival’ in FLT3-Mutated AML

By Melissa Badamo - Last Updated: February 6, 2024

Azacitidine, venetoclax, and gilteritinib combination therapy achieved high complete remission (CR)/CR with incomplete hematologic recovery (CRi) rates in patients with newly diagnosed FLT3-mutated acute myeloid leukemia (AML), according to a recent study.

The phase I/II trial was led by Nicholas Short, MD, of the University of Texas MD Anderson Cancer Center and published in the Journal of Clinical Oncology.

Dr. Short and colleagues evaluated the triplet therapy in 52 patients with either frontline (n=30) or relapsed or refractory (n=22) AML. Patients received the recommended phase II dose of gilteritinib at 80 mg once a day in combination with azacitidine and venetoclax. The primary endpoints were the maximum tolerated dose of gilteritinib and the combined CR/CRi rate.

The CR/CRi rate was 96% for the frontline cohort, compared with 27% for the relapsed or refractory cohort. Nine additional patients with relapsed or refractory AML (41%) achieved a morphologic leukemia-free state.

Within four cycles, 65% of patients achieved FLT3-internal tandem duplication measurable residual disease <5 × 10–5. The median relapse-free survival (RFS) and overall survival (OS) have not been reached after a median follow-up of 19.3 months. The 18-month RFS and OS rates are 71% and 72%, respectively.

The most common grade ≥3 nonhematologic adverse events included infection (62%) and febrile neutropenia (38%), which occurred more frequently in the relapsed or refractory cohort.



Short NJ, Daver N, Dinardo CD, et al. Azacitidine, venetoclax, and gilteritinib in newly diagnosed and relapsed or refractory FLT3-mutated AML. J Clin Oncol. 2024. doi:10.1200/JCO.23.01911

Original Source: Triplet Therapy Achieves ‘Encouraging Survival’ in FLT3-Mutated AML | Blood Cancers Today


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