Predictive value of radiological response, pathological response and relapse-free survival for overall survival in neoadjuvant immunotherapy trials: pooled analysis of 29 clinical trials

• DOI: 10.1016/j.ejca.2023.03.010
• Publication Date: 2023-03-17T20:33:26Z
• Authors: Runcong Nie; Foping Chen; Mariano Provencio; Yun Wang; Tom van den Ende; Hanneke W.M. van ...; Shuqiang Yuan; Miklos Pless; Stefanie Hayoz; Zhiwei Zhou; Yuanfang Li; Sacha I. Rothschild; Muyan Cai
• Keywords: Surrogate endpoint; Clinical endpoint; Neoadjuvant Therapy

An increasing number of clinical trials are being conducted exploring the efficacy neoadjuvant immune checkpoint inhibitors. Surrogate end-points for overall survival (OS) urgently needed.Phase II or III immunotherapy that reported data on OS and surrogate were identified from January 1, 2000, to November 25, 2022. Individual patient data, trial-level requested corresponding authors extracted eligible trials. At individual level, correlations between radiological pathological response measured by Cox model quantified hazard ratio (HR). C-statistic was used quantify predictive performance OS. The coefficient determination (R2) RFS evaluated a bivariate model.A total 29 reporting 2901 patients included. ORR correlated with improved (3-year OS: 87.0% versus 70.4% non-ORR, respectively; HR, 0.34, 95% confidence interval [CI], 0.17-0.68). HRs in achieving MPR pCR 0.24 (95% CI, 0.12-0.46) 0.13 0.05-0.36). benefit maintained after adjusting tumour type. C-statistics ORR, 0.63, 0.63 0.65, respectively. strength association strong (R2 = 0.88, 0.79-0.94).These findings suggest MPR, valid predictors when using Moreover, may be most optimal surrogates