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MDS 2021 | Long-term follow-up: Phase II study of APR-246 + aza in TP53-mutated MDS & AML

Thomas Cluzeau, MD, PhD, Central University Hospital of Nice, Nice, France, discusses the long-term follow-up results from a Phase II study (NCT03588078) of APR-246 (eprenetapopt) plus azacitidine (aza) in TP53-mutated myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Prof. Cluzeau reports a median overall survival (OS) of 12 months for patients with MDS and 15 months for patients with AML and explains that patients with a measurable residual disease of less than 5% experience a significant increase in OS. Four patients that were allotransplanted were still alive after 22-30 months. Prof. Cluzeau concludes that patients with a TP53 mutation could benefit from the combination of APR-246 and other drugs such as magrolimab or sabatolimab with azacitidine, and highlights the possibility of allotransplants to potentially cure these patients. This interview took place at the 16th International Congress on Myelodysplastic Syndromes, held virtually in 2021.

Disclosures

Thomas Cluzeau, MD, PhD, has participate in clinical research with Novartis, Alexion, Celgene/BMS, Amgen, Syros, Kartos, Arog and Takeda, has participated in advisory boards with Celgene, Abbvie, Jazz Pharma, Roche, Novartis and Agios; has participated in educational roles with Novartis, Amgen, Sanofi, Astellas and Celgene/BMS; and has participated in international congresses with Sanofi, Pfizer, Celgene/BMS and Novartis.