ASH 2020 | Venetoclax plus azacitidine for higher-risk MDS

Jacqueline Garcia, MD, Dana-Farber Cancer Institute, Boston, MA, shares the results of a Phase Ib (NCT02942290) study investigating whether adding the BCL-2 inhibitor venetoclax (ven) to azacitidine (aza) improves outcomes in treatment-naïve higher-risk myelodysplastic syndrome (MDS) patients. These patients have extremely poor overall survival and no standard of care is currently established. Favorable outcomes with this combination treatment in older acute myeloid leukemia patients provided the rationale for this safety and preliminary efficacy trial in higher-risk MDS. Grade ≥3 adverse events were experienced by 97% of patients, with febrile neutropenia being the most common serious adverse effect. Patients treated with ven + aza achieved an overall response rate (ORR) of 79%, over half of whom achieved complete remission (CR). A promising median response duration of 14.8 months was also shown, with high rates of transfusion independence attained. The recommended Phase II dose of venetoclax was established as 400mg and patients receiving this dose maintained physical functioning for the duration of the treatment. This interview took place during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, 2020.