Association between complete response and outcomes in transplant-eligible myeloma patients in the era of novel agents

Objectives: Achieving complete response (CR) has been linked to improved progression-free (PFS) and overall (OS) survival in myeloma. A meta-analysis was conducted to investigate whether this holds true in the era of novel agents (bortezomib, lenalidomide, thalidomide).

Methods: A total of 24 studies in newly diagnosed patients undergoing autologous stem cell transplantation (ASCT) that reported associations between responses and long-term outcomes (PFS/OS rates post-ASCT by response, or hazard ratios with 95% confidence intervals from Cox models) were identified and analyzed.

Results: Achievement of CR vs. <CR post-ASCT reduced risk of progression/death by 38% [risk ratio (RR): 0.62, P < 0.0001]; risk of death was 41% lower (RR: 0.59, P < 0.0001). Subgroup meta-analyses showed significant PFS risk reduction with CR post-ASCT with novel (RR: 0.32, P < 0.006) and non-novel (RR: 0.72, P < 0.0001) agents, and corresponding OS risk reduction with novel (RR: 0.33, P = 0.0013) and non-novel (RR: 0.64, P < 0.0001) agents. Risk reduction was greater with novel vs. non-novel agents (PFS: P = 0.047; OS: P = 0.058).

Conclusions: Achieving CR during first-line therapy remains important in the novel-agent era; magnitude of association between achieving CR and outcomes appears higher for CR obtained using novel vs. non-novel agents.