Evaluating Minimal Residual Disease Negativity as a Surrogate Endpoint for Treatment Efficacy in Multiple Myeloma: A Meta-Analysis of Randomized Controlled Trials

Abstract

This meta-analysis examined the association between minimal residual disease (MRD) negativity and survival outcomes in 15 304 patients with multiple myeloma (MM) enrolled in randomized controlled trials published until June 2, 2024. Overall, there was a significant, negative and strong association between MRD negativity odds ratios and survival hazard ratios (β_PFS = -0.20, p < 0.001, β_OS = -0.12, p = 0.023). These associations remained significant for newly diagnosed patients (β_PFS = -0.35, p < 0.001), and they were consistent but not significant for relapsed/refractory patients (β_PFS = -0.06, p = 0.635). Sustained MRD negativity at 1 year was strongly correlated with prolonged PFS (β_PFS = -0.30, p < 0.001). In conclusion, this comprehensive meta-analysis supports MRD as a surrogate for survival in MM.