Patients with hepatocellular carcinoma whose tumors initially exceeded the limits for liver transplantation lived longer and had a lower risk of cancer recurrence after surgery when they received a pretransplant treatment strategy built around immune checkpoint inhibitors, according to a Chinese study published in JAMA Oncology. The benefit came with a trade-off, however: a nearly doubled rate of acute rejection within 90 days of transplantation.
The Milan criteria, the standard benchmark for transplant eligibility in liver cancer, allow a single tumor no larger than 5 cm or two to three tumors no larger than 3 cm each. For patients whose tumors initially surpassed these thresholds, the study examined whether downstaging with an immune checkpoint inhibitor–based multimodal regimen — typically combined with locoregional and/or targeted therapies — could improve posttransplant outcomes.
Researchers at eight high-volume Chinese centers, each performing more than 55 liver transplantations annually, retrospectively evaluated patients who underwent transplantation between January 2019 and December 2024. Using propensity score matching to balance measured characteristics between groups, the investigators compared 208 recipients who received immune checkpoint inhibitors before transplantation with 208 recipients treated without them.
The results favored the immune checkpoint inhibitor approach. Median overall survival after transplantation was 4.6 years versus 2.5 years (P < .001), and median recurrence-free survival was 1.5 years versus 0.9 years (P = .02). Multivariable Cox analyses showed the strategy was associated with improved overall survival (hazard ratio [HR] = 0.63; 95% CI, 0.43–0.92) and recurrence-free survival (HR = 0.59; 95% CI, 0.44–0.80), while a Fine-Gray model indicated a lower risk of posttransplant recurrence (subdistribution HR = 0.54; 95% CI, 0.40–0.72). Yet acute rejection within 90 days was more frequent with the strategy, occurring in 16.3% of patients versus 8.2% of those treated without immune checkpoint inhibitors (P = .01). “The results of this cohort study suggest that an immune checkpoint inhibitor–based multimodal pretransplant strategy was associated with better posttransplant oncologic outcomes and survival in selected patients with hepatocellular carcinoma that was initially beyond the Milan criteria, despite more frequent acute rejection,” the investigators concluded. They added that by characterizing this post–liver transplantation benefit-risk profile, the multicenter matched cohort study “may inform future prospective studies of immune checkpoint inhibitor–based pretransplant strategies in liver transplantation candidates and help clinical decision-making.” Looking ahead, the authors wrote that “prospective multicenter studies and randomized clinical trials are needed to validate these findings and determine how this strategy should be integrated into transplant candidate selection and peritransplant rejection risk management.” Qi Ling, MD, PhD, of The First Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China, and Guangming Li, MD, of YouAn Hospital, Capital Medical University, Beijing, are the corresponding authors. The study was supported by grants from the National Science and Technology Major Projects and the Leading Goose Research and Development Program of Zhejiang Province, and the authors reported no conflicts of interest. Original Source: https://ascopost.com/news/october-2026/immunotherapy-based-downstaging-linked-to-improved-outcomes-after-liver-transplantation-in-hepatocellular-carcinoma/ Category: Tags: Publish Date: 2026-10-19 22:17:00
