Observational study reports changes in molecular aberrations during decitabine therapy, suggesting implications for treatment response in AML patients.
Key Points
To investigate the changes in molecular aberrations in AML patients undergoing treatment with oral decitabine/cedazuridine.
Monitored molecular markers during treatment in a subset of five AML patients.
Assessed changes in the mutational landscape and blast cell counts throughout the therapy.
Evaluated overall survival based on treatment response.
No significant reduction in mutated AML clones for four out of five patients.
One patient achieved complete remission with a decrease in variant allele frequency (VAF) of mutated clones.
Two patients experienced significant decreases in blast cells without changes in their molecular profiles.
Patients with complete or partial response had significantly longer overall survival compared to non-responders.
80% of patients had potentially targetable mutations at diagnosis, including IDH2, NPM1, and FLT3.