Combination therapy of BCL-2 antagonist venetoclax and demethylase inhibitor azacitidine for the treatment of multiple myeloma: a clinical study

This clinical trial evaluated assess the efficacy and safety of combination therapy with the BCL-2 antagonist venetoclax and the demethylase inhibitor azacitidine in 440 patients diagnosed with multiple myeloma. The primary endpoints included overall response rate (ORR), complete response (CR) rate, progression-free survival (PFS), and safety profile. The venetoclax-azacytidine combination demonstrated comparable efficacy, with enhanced depth of response in patients with high-risk cytogenetic abnormalities. Adverse events were generally manageable, with neutropenia observed in 25% and fatigue in 30% of patients. Minimal residual disease (MRD) negativity and specific cytogenetic markers, such as t(11;14), were identified as significant independent predictors of favorable treatment response. The therapy demonstrated clinical benefit with a manageable toxicity profile, suggesting potential utility as a treatment option in selected patient subgroups. However, the need for longer follow-up and expanded cohorts remains critical to validate these outcomes and optimize patient selection. These findings support the further exploration of venetoclax and azacitidine as a viable treatment option for patients with refractory or high-risk multiple myeloma, warranting additional prospective trials before consideration for standard-of-care implementation.