Blood MALT1 serves as a potential biomarker reflecting the response and survival of immune‑checkpoint‑inhibitor therapy in advanced hepatocellular carcinoma.

Treatment modalities involving an immune-checkpoint-inhibitor (ICI) have emerged as therapeutic options in advanced hepatocellular carcinoma (HCC). Nonetheless, auxiliary biomarkers are required to evaluate their efficacy. The present study aimed to assess the potential of blood mucosa-associated lymphoid tissue 1 (MALT1) in reflecting clinical response and prognosis in patients with advanced HCC who received ICI therapy. Peripheral blood was collected from 51 patients with advanced HCC who were about to receive ICI or ICI-based treatment. Blood MALT1 levels were determined using reverse transcription-quantitative PCR, and the blood MALT1 levels in 50 healthy controls (HCs) were also assessed. Besides, the treatment response and survival data were collected. The Wilcoxon rank-sum test was used for comparison analysis and the Spearman's rank correlation coefficient test was used for correlation analysis. The prognostic value of MALT1 was determined by Kaplan-Meier curve analysis with the log-rank test. Univariate and multivariate Cox regression models were used to identify factors associated with progression-free survival (PFS) and overall survival (OS). The results demonstrated that blood MALT1 levels were significantly increased in patients with advanced HCC compared with that in HCs (P<0.001). Blood MALT1 levels were increased in patients with portal vein invasion (vs. without portal vein invasion; P=0.010), extrahepatic disease (vs. without extrahepatic disease; P=0.026) and α-fetoprotein (AFP) ≥200 ng/ml (vs. AFP <200 ng/ml; P=0.040). After 4 cycles of ICI therapy, the objective response rate (ORR) and disease control rate (DCR) was 29.4 and 68.6%, respectively. Blood MALT1 levels were also significantly and negatively associated with the ORR (P=0.043) and DCR (P=0.004). Furthermore, PFS and OS were shortened in patients with high blood MALT1 levels (cut-off by the median) compared to those with low blood MALT1 levels. After adjusting using multivariate Cox regression models, high blood MALT1 levels were demonstrated to be a significant independent risk factor for shortened PFS [hazard ratio (HR)=2.419; P=0.009] and OS (HR=2.706, P=0.018) in patients with advanced HCC who received ICI therapy. In summary, blood MALT1 levels serve as a potential biomarker to reflect treatment response and survival in patients with advanced HCC who receive ICI therapy.