Decitabine plus cedazuridine and venetoclax: the promise of an all-oral therapy for patients with myelodysplastic syndromes and chronic myelomonocytic leukaemia
Mené sur 39 patients atteints d'un syndrome myélodysplasique à haut risque d'évolution ou d'une leucémie myélomonocytaire chronique (âge médian : 71 ans), cet essai de phase I/II détermine la dose maximale tolérée de la décitabine dispensée par voie orale en combinaison avec la cédazuridine et le vénétoclax puis évalue l'efficacité de cette combinaison du point de vue du taux de réponse
In The Lancet Haematology, Alex Bataller and colleagues 1 report early results of a phase 1/2 study of decitabine plus cedazuridine and venetoclax for patients with high-risk myelodysplasia and chronic myelomonocytic leukaemia. Bataller and colleagues treated 39 patients, with an impressive overall response rate of 95% (95% CI 83–99; 37/39), a complete remission rate of 44% (28–60), and bone marrow complete remission rate of 51% (35–66); or an overall response rate of 82% (66–92) and 59% (42–74) complete remission, if the International Working Group 2023 response criteria are applied. Most responses occurred during the first cycle of therapy. The delivery of this entirely oral regimen and its associated high and rapid response rate might be paradigm changing for patients with high-risk myelodysplastic syndromes and chronic myelomonocytic leukaemia who currently receive mainly a hypomethylating agent with associated low rates of response and an inconvenient parenteral administration route. There are many potential benefits in high-risk myelodysplastic syndromes and chronic myelomonocytic leukaemia to this regimen, including reduced travel time, chair time, and injection site reactions; improvements in transplantation rates (19 [49%] patients proceeded to transplantation in this study) and overall survival; and the development of molecularly selected triplet therapy combinations of entirely oral drugs with even higher efficacy.