Alternate Therapy Is Safe, Effective for Treating AML, MDS

By Melissa Badamo - Last Updated: February 8, 2024

Oral decitabine plus cedazuridine is a safe and effective alternative to intravenous decitabine for treating myelodysplastic syndromes (MDS), chronic myelomonocytic leukemia (CMML), and acute myeloid leukemia (AML), according to results from the phase III ASCERTAIN study.

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The multicenter, open-label, crossover study was led by Guillermo Garcia-Manero, MD, of the University of Texas MD Anderson Cancer Center, and published in The Lancet Haematology.

A total of 138 patients were randomized to receive either decitabine 35 mg plus cedazuridine 100 mg orally for five days or intravenous decitabine 20 mg/m2 per day in a 28-day cycle followed by five days of oral decitabine plus cedazuridine in the next cycle.

The primary endpoint was total decitabine exposure over five days. At a median follow-up of 966 days, total exposure of oral decitabine plus cedazuridine versus intravenous decitabine was 98.93% (90% CI, 92.66-105.60). This finding indicates “equivalent pharmacokinetic exposure on the basis of the area under the curve,” according to Dr. Garcia-Manero and colleagues.

The researchers observed similar safety patterns in the two treatment groups. A total of 31% of patients receiving oral decitabine plus cedazuridine experienced serious adverse events (AEs) compared with 18% of those receiving intravenous decitabine. The most frequent grade 3 AEs included thrombocytopenia, neutropenia, and anemia. Five treatment-related deaths occurred, two from oral treatment and three from intravenous treatment.

Funding for this study was provided by Astex Pharmaceuticals.

Reference

Garcia-Manero, G, McCloskey J, Griffiths EA, et al. Oral decitabine–cedazuridine versus intravenous decitabine for myelodysplastic syndromes and chronic myelomonocytic leukaemia (ASCERTAIN): a registrational, randomised, crossover, pharmacokinetics, phase 3 study. Lancet Haematol. 2024. doi.org/10.1016/S2352-3026(23)00338-1

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