The following is a summary of “Progression-free survival as a surrogate endpoint for overall survival in patients with relapsed or refractory multiple myeloma,” published in the April 2024 issue of Oncology by Dimopoulos et al.
This investigation aimed to establish a quantitative correlation between median PFS and median OS in patients specifically diagnosed with relapsed/refractory multiple myeloma (RRMM), drawing from data from published randomized controlled trials (RCTs).
Methodologically, a comprehensive search of two major bibliographic databases, PubMed and Embase, from 1970 to 2017, was conducted to identify RCTs focusing on RRMM that provided data on both OS and PFS. Additionally, an updated search was performed for studies published between 2010 and 2022 across three databases: Embase, MEDLINE, and EBM Reviews. The association between median PFS and median OS was evaluated using nonparametric Spearman rank and parametric Pearson correlation coefficients. Furthermore, a quantitative assessment of the relationship between PFS and OS was conducted using weighted least-squares regression, accounting for covariates such as age, sex, and publication year, with the study arms weighted by the number of patients in each arm.
The analysis encompassed 31 RCTs, totaling 56 treatment arms and involving 10,450 patients diagnosed with RRMM. The mean median PFS and median OS were 7.1 months (SD 5.5) and 28.1 months (SD 11.8). Strong correlation coefficients were observed between median PFS and median OS, both through Spearman (0.80, P < 0.0001) and Pearson (0.79, P < 0.0001) analyses. On an individual treatment arm level, an increment of 1 month in median PFS was associated with a 1.72-month increase in median OS (95% CI 1.26–2.17).
In conclusion, the comprehensive analysis incorporating recent data underscores the utility of PFS as a predictive measure for OS in RRMM, consolidating its role as a surrogate endpoint in clinical trials assessing treatments for this disease entity.
Source: bmccancer.biomedcentral.com/articles/10.1186/s12885-024-12263-0