Ficlatuzumab/Cytarabine Combination Yields Promising Results in Chemo-Refractory High-Risk Acute Myeloid Leukemia

Patients with chemotherapy-refractory, high-risk acute myeloid leukemia achieved promising benefit from treatment with ficlatuzumab and cytarabine.

Treatment with ficlatuzumab (previously AV-299) and cytarabine resulted in promising clinical activity in a population of patients with chemotherapy-refractory, high-risk acute myeloid leukemia (AML) and may have utility in conjunction with multiplexed single cell analyses to detect on-target activity, according to findings from a phase 1b trial (NCT02109627).

Investigators reported an overall response rate (ORR) of 53% (n= 9/17) based on bone marrow biopsy, all responses of which were complete responses (CR). A total of 16 patients were assessed for minimal residual disease (MRD) status, with 4 of 9 responders testing negative for MRD.

Trial eligibility required patients to be aged 18 years or older and have been diagnosed with histologically confirmed refractory AML or have relapsed within 12 months of their first CR/CR with incomplete hematologic recovery. Patients could not have received more than 2 cycles of AML-directed therapy, 1 of which needed to be an anthracycline/cytarabine combination.

The investigator-initiated trial utilized a 3+3 design, with patients being treated with 4 intravenous doses of ficlatuzumab every 14 days plus cytarabine. Dosage for ficlatuzumab started at 10 mg/kg in cohort 1, 15 mg/kg in cohort 2, and 20 mg/kg in cohort 3. Cytarabine was administered at a fixed dose of 2000 mg/m2 daily.

The primary outcome of the study was safety and determining the maximum tolerated dose of ficlatuzumab and cytarabine. Key secondary end points included ORR, CR rate, and progression-free survival (PFS), with exploratory end points including overall survival (OS) and biomarker changes over the course of treatment.

Investigators included 18 patients who consented from January 2014 to October 2018, 17 of whom enrolled on the study. One patient was excluded due to active fungal pneumonia.

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