A single-center retrospective cohort analysis of venetoclax in relapsed/refractory multiple myeloma

Abstract

Venetoclax, a small molecule inhibitor of BCL-2, has promising pre-clinical and early clinical activity in relapsed refractory multiple myeloma (RRMM). However, the clinical use of venetoclax remains under evaluation. We performed a single-center, retrospective analysis of patients with RRMM treated with off-label venetoclax. Forty-seven patients with a median of seven lines of prior therapy were identified. Most patients (87%) received venetoclax plus proteasome inhibitor, though there was heterogeneity in the venetoclax-containing regimen patients received, and 38% had known t(11;14). The overall response rate (ORR) was 39%, with 17% achieving ≥ very good partial response (VGPR). In the t(11;14) cohort, the ORR was 71%, with 24% achieving ≥ VGPR. The median progression-free survival was 2.1 months and overall survival was 15.6 months. One treatment-related death (related to infection) occurred. Venetoclax appears safe and efficacious in patients with RRMM, especially in those with t(11;14). Prospective trials are ongoing to further evaluate this treatment.