Background & Aims: Immune checkpoint inhibitors (ICIs) are playing a significant role in the treatment of hepatocellular carcinoma (HCC). This study aims to explore the prognostic value of alpha-fetoprotein (AFP) combined with initial tumor shape irregularity in predicting the prognosis of patients treated with ICIs.
Methods: In this retrospective, multi-center cohort study, 296 HCC patients received ICIs. Patients were randomly divided into the training set and the validation set in a 3:2 ratio. The training set was used to evaluate the impact of baseline factors on overall survival (OS) using the Cox model and to develop an easily applicable ATSI (AFP and Tumor Shape Irregularity) score, which was then verified in the validation set.
Results: The ATSI score was developed from two independent prognostic risk actors: baseline AFP ≥ 400 ng/ml (HR 1.71, 95% CI 1.08 ~ 2.73, P =0.023) and initial tumor shape irregularity (HR 1.66, 95% CI 1.01 ~ 2.75, P =0.048). The median OS was not reached in patients who met no criteria (95% CI 28.20 ~ NA), 25.80 months (95% CI 14.17 ~ NA) in patients who met one criterion, and 17.03 months (95% CI 11.73 ~ 23.83) in patients who met two criteria (P =0.001). The median progression-free survival (PFS) was 10.83 months (95% CI 9.27 ~ 14.33) for 0 points, 8.03 months (95% CI 6.77 ~ 10.57) for 1 point, and 5.03 months (95% CI 3.83 ~ 9.67) for 2 points (P <0.001). The validation set effectively verified these results (median OS, 37.43/ 24.27/ 14.03 months for 0/ 1/ 2 points, P =0.028; median PFS, 13.93/ 8.30/ 4.90 months for 0/ 1/ 2 points, P <0.001).
Conclusions: The easily applicable ATSI score based on the baseline AFP levels and initial tumor shape can effectively predict efficacy and survival in HCC patients with ICIs.