Dr. Cluzeau on the Efficacy of APR-246/Azacitidine in TP53-Mutated MDS and AML

Thomas Cluzeau, MD, PhD, head of hematology department, Central University Hospital of Nice, in Nice, France, discusses the response rates of a phase 2 study with APR-246 and azacitidine in TP53-mutated myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML).

Patients enrolled in the study were treated with APR-246 at 4500 mg intravenously from days 1 to 4 followed by azacitidine at 75mg/m2 from days 4 to 10 in 28-day cycles. A total of 52 patients were enrolled on the trial. Results pertaining to response to the treatment were promising, says Cluzeau; the overall response rate (ORR) was 58%, with 37% of patients achieving a complete response (CR).

Specifically, in 39 evaluable patients who received ≥3 cycles of treatment, the ORR was 77%, with 49% of patients achieving a CR, adds Cluzeau. When analyzing by subgroups, in 34 evaluable patients with MDS, the ORR was 75%, with 57% of patients experiencing a CR. For patients with AML with 20% to 30% of blasts, the ORR was 78%, with 33% of patients experiencing a CR.

Finally, in terms of survival, after a median follow-up of 9.7 months, the median overall survival (OS) was 12.1 months. In patients who received ≥3 cycles of treatment, the median OS was 13.7 months. Thus, the results from this study demonstrated great responseswith this treatment in this high-risk patient population, concludes Cluzeau.