Surrogate end points for survival in patients with advanced hepatocellular carcinoma treated with immune checkpoint inhibitors

Aim: The authors investigated surrogacy of radiology-based end points for clinical trials investigating immune checkpoint inhibitors in advanced hepatocellular carcinoma. Methods: Data were collected from electronic databases reporting median overall survival (OS), median progression-free survival (PFS) and objective response rate (ORR). Weighted Pearson correlation coefficients and 95% confidence intervals (CIs) were calculated. Results: 26 clinical trials (41 treatment arms, 5144 patients) were included. ORR (coefficient: 0.71; 95% CI: 0.52-0.84) and PFS (coefficient: 0.63; 95% CI: 0.21-0.92) were positively correlated with OS. Sensitivity analyses suggested liver function, line of therapy and study phase did not greatly impact results. The COSMIC-312 study negatively impacted the overall weighted correlation. Conclusion: ORR and PFS are positively correlated with OS in patients with advanced hepatocellular carcinoma.This study was conducted to determine how effective measurements of tumor size are at predicting how long patients with liver cancer survive when they receive a drug that blocks proteins involved in immune system regulation, which are made by some types of immune system cells in the body. The authors calculated how closely measurements of a patient's tumor were related to survival by examining data from 26 separate clinical trials that included more than 5000 patients. This study showed measurements of tumor size and the response (shrinkage, growth or no change in size) to checkpoint inhibitor drugs were likely to be related to the length of survival. This information is important for designing future clinical trials and may help speed the delivery of effective medicines to patients with liver cancer.