In an analysis reported in The Lancet Oncology, D’Alessio et al found that major pathologic response and complete pathologic response to neoadjuvant immune checkpoint inhibitor therapy was associated with improved relapse-free survival in patients with hepatocellular carcinoma.
Study Details
The study involved a pooled analysis of patient-level data from patients receiving immune checkpoint inhibitor therapy before liver resection as part of a global collaborative consortium (NeoHCC) of five phase I and II trials and standardized observational protocols conducted in 12 tertiary referral centers in the United States, United Kingdom, and Taiwan. Major pathologic response consisted of ≥ 70% tumor regression (100% tumor regression for pathologic complete response).
Key Findings
Among 111 patients included in the analysis, data on pathologic response were available for 104 (94%). Patients received treatment between October 2017 and November 2023, with most (69%) receiving immune checkpoint inhibitor combination treatment. A major pathologic response was observed in 33 patients (32%), and pathologic complete response, in 19 patients (18%).
Median relapse-free survival for the entire cohort was 43.6 months (95% confidence interval [CI] = 28.3 months to not evaluable). Median relapse-free survival was not reached (95% CI = not evaluable to not evaluable) among patients with a major pathologic response vs 28.3 months (95% CI = 12.8–43.8 months) among those without a major pathologic response (hazard ratio [HR] = 0.26; 95% CI = 0.10–0.66; P = .0024). Median relapse-free survival was not reached (95% CI = not evaluable to not evaluable) among patients with a pathologic complete response vs 32.8 months (95% CI = 15.0–50.5 months) among those without a pathologic complete response (HR = 0.19; 95% CI = 0.05–0.78; P= .010). Analysis indicated that a threshold of 90% tumor regression was the optimal level of regression to predict improvement in relapse-free survival.
The investigators concluded: “The extent of tumour regression following neoadjuvant [immune checkpoint inhibitor] therapy could identify patients with improved relapse-free survival following liver resection. The threshold of at least 90% tumour regression should be validated for its surrogate role for relapse-free survival in phase 3 randomised controlled trials.”
David J. Pinato, MD, PhD, of the Division of Cancer, Department of Surgery and Cancer, Imperial College London, is the corresponding author for The Lancet Oncology article.
Disclosure: The investigators reported that there was no funding for the study. For full disclosures of the study authors, visit thelancet.com.
