Azacitidine Plus Venetoclax Outcomes Comparable to Azacitidine Monotherapy for MDS

In a retrospective, multicenter study of patients with high-risk myelodysplastic syndrome (HR-MDS), investigators compared outcomes from azacitidine plus venetoclax therapy with those of azacitidine monotherapy. The study was presented at the Society of Hematologic Oncology 2024 Annual Meeting in Houston, Texas.

The investigators found that the two regimens had closely comparable overall survival (OS), overall response rate (ORR), and adverse event (AE) results, although they noted that “the azacitidine-venetoclax group had a shorter time to response, consistent with previous studies.”

The study cohort included 51 patients with HR-MDS, was 68.6% male, and had a median age of 67 years. Of the total cohort, 65% was treated with azacitidine plus venetoclax and 35% with azacitidine monotherapy. The investigators noted no significant baseline differences between the two groups.

In the total cohort, ORR was 74.5%, 23.5% of the cohort achieved complete response (CR), and 10% achieved CR with incomplete cell count recovery. Of the patients who responded, 34.2% relapsed.

Median OS for the total cohort was 24.2 months. Median OS for patients who received azacitidine plus venetoclax was 20.4 months, and for patients on azacitidine monotherapy it was 29.5 months (P=0.618).

Grade 3 or 4 AEs occurred in 70.6% of the total cohort. Regarding the prevalence of specific hematologic toxicities, anemia affected 82% of the azacitidine plus venetoclax group and 67% of the azacitidine group (P=0.738), thrombocytopenia affected 85% of the azacitidine plus venetoclax group and 73% of the azacitidine group (P=0.375), neutropenia affected 93% of the azacitidine plus venetoclax group and 80% of the azacitidine group (P=0.407), and febrile neutropenia affected 52% of the azacitidine plus venetoclax group and 60% of the azacitidine group (P=0.357).

“Further research, including molecular genotype subgroup analysis, is needed to validate the potential benefit of this treatment strategy as first-line therapy in MDS patients,” the investigators concluded.

Reference

Chacon-Rangel LC, Rodriguez-Rodriguez S, Ontiveros-Austria JL, et al. Impact of venetoclax on high-risk myelodysplastic syndromes: A retrospective multicenter analysis of azacitidine-venetoclax vs azacitidine monotherapy in Mexico. Abstract #MDS-460. Presented at the Society of Hematologic Oncology 2024 Annual Meeting; September 4-7, 2024; Houston, Texas.