Pomalidomide, Bortezomib, and Dexamethasone Versus Bortezomib and Dexamethasone in Relapsed or Refractory Multiple Myeloma: Final Survival and Subgroup Analyses From the OPTIMISMM Trial

ABSTRACT Introduction In the OPTIMISMM trial, pomalidomide/bortezomib/dexamethasone (PVd) significantly prolonged median progression‐free survival (PFS) versus bortezomib/dexamethasone (Vd) in lenalidomide‐exposed relapsed and refractory multiple myeloma (RRMM). We report final overall survival (OS) and updated efficacy analyses. Methods Adults with RRMM who had 1–3 prior regimens, including lenalidomide (≥ 2 cycles), were assigned (1:1) to PVd or Vd. Primary endpoint: PFS. Prespecified secondary endpoint: OS. Prespecified exploratory endpoints: PFS2 and subgroup efficacy analyses. Results With an overall event rate of 70.0%, OS data were mature in the intent‐to‐treat population ( N = 559). After median follow‐up of 64.5 months (data cutoff: May 13, 2022), median OS was 35.6 months with PVd versus 31.6 months with Vd (HR 0.94, 95% CI 0.77–1.15, p = 0.571); adjusting for subsequent therapies, OS improved with PVd versus Vd (HR 0.76, 95% CI 0.619–0.931, p = 0.008). Median PFS2 was 22.1 versus 16.9 months, respectively (HR 0.77, 95% CI 0.64–0.94, nominal p = 0.008). Treatment‐emergent adverse events led to study drug discontinuation in 92 (33.1%) and 53 (19.6%) patients in PVd and Vd arm, respectively. Conclusions Findings showed a nonsignificant trend towards improved OS with PVd versus Vd. PFS2 favored PVd, supporting its use in RRMM.