ABSTRACT This study was designed to provide prospective evidence for the combination of hepatic arterial infusion chemotherapy (HAIC) and toripalimab, which had suggested encouraging antitumor activity and safety in advanced hepatocellular carcinoma (HCC) previously. This single‐center, non‐comparative, randomized phase II study (NCT04135690) recruited locally advanced HCC participants (1:1) to receive HAIC plus either toripalimab (TorHAIC) or sorafenib (SoraHAIC) per 3 weeks. The primary endpoint was the progression‐free survival (PFS) rate at 6 months. Seventy‐two participants were randomly assigned to received TorHAIC ( n = 36) or SoraHAIC ( n = 36). The 6‐month PFS rate was 63.9% in the TorHAIC group and 61.1% in the SoraHAIC group. The median OS was 20.9 months in the TorHAIC group and 16.4 months in the SoraHAIC group, while the median PFS was 9.1 and 7.2 months, respectively. There were 12 participants (33.3%) developed grade 3–4 adverse events (AEs) in the TorHAIC group and 16 participants (44.4%) in the SoraHAIC group. Serious AEs were reported in two participants in the TorHAIC group and five participants in the SoraHAIC group. Our study suggested that the TorHAIC regimen had a favorable safety and efficacy profile in locally advanced HCC. However, these findings warrant validation in a phase III trial.
Lai et al. (Mon,) studied this question.