Efficacy and Safety of Immune Checkpoint Inhibitors in... : American Journal of Clinical Oncology (original) (raw)

Review Article

Efficacy and Safety of Immune Checkpoint Inhibitors in Hepatocellular Carcinoma

A Systematic Review and Meta-Analysis

Naeem, Muhammad A.B. MBBS*; Paracha, Muhammad R. MBBS*; Noor, Ahmad MBBS*; Khalid, Muhammad H.A. MBBS*; Shahid, Hafiza K. MBBS*; Khan, Maaz MBBS*; Rizvi, Moeaza R. MBBS*; Arshad, Javeeria MBBS*; Abbas, Talha MBBS*; Saeed, Azeem MBBS*; Ashraf, Hamza MBBS*; Ali, Minahil MBBS*; Asif, Mishal MBBS†; Ghouri, Aanusha MD‡

*Department of Medicine, Allama Iqbal Medical College

†Department of Medicine, Fatima Jinnah Medical University, Lahore, Pakistan.

‡Luminis Health Anne Arundel Medical Center Annapolis, MD

M.A.B.N. and M.R.P. contributed equally and are co–first authors.

M.A.B.N.: conceptualization, data curation, writing-original draft, writing-review and editing; M.R.P.: conceptualization, writing-original draft, writing-review and editing; A.N., M.H.A.K., H.K.S., M.Z., M.R.R., J.A., H.A., and M.A.: validation, writing-original draft, and writing-review and editing; T.A.: conceptualization, project administration, supervision, and writing-review and editing; A.S.: validation, writing-review and editing; M.A.: validation and writing-review and editing.

The authors declare no conflicts of interest.

Correspondence: Hamza Ashraf, MBBS, Department of Medicine, Allama Iqbal Medical College, Lahore, Pakistan. E-mail: [email protected].

Supplemental Digital Content is available for this article. Direct URL citations are provided in the HTML and PDF versions of this article on the journal's website, www.amjclinicaloncology.com.

American Journal of Clinical Oncology ():10.1097/COC.0000000000001278, December 22, 2025. | DOI: 10.1097/COC.0000000000001278

Objectives:

Previous meta-analyses have assessed the benefits and safety profile of immune checkpoint inhibitors in hepatocellular carcinoma patients. This meta-analysis provides an updated synthesis by incorporating the newly published studies and previous studies with the revised data.

Methods:

A systematic review and meta-analysis were conducted following PRISMA guidelines. Databases (PubMed, Google Scholar, and Cochrane Library) were searched for randomized controlled trials (RCTs) comparing Immune Checkpoint Inhibitors to Standard Therapy or Placebo in patients with Hepatocellular Carcinoma. Studies were selected based on predefined eligibility criteria, and odds ratios (ORs) and hazard ratios (HRs) for outcomes were calculated using a random effects model.

Results:

Eighteen RCTs involving 9244 patients were included in this study. Compared with the control group, ICIs were associated with a significantly improved objective response rate (ORR) (OR=3.20, 95% CI: 2.44-4.20, _P_=<0.00001), disease control rate (DCR) (OR=1.40, 95% CI: 1.08-1.81, _P_=0.01), stable disease (SD) (OR=2.15, 95% CI: 1.16-3.98, _P_=0.02), overall survival (OS) (HR=0.79, 95% CI: 0.73-0.86, _P_=<0.00001), progression-free survival (PFS) (HR=0.77, 95% CI: 0.69-0.87, _P_=<0.00001) and all-cause grade ≥3 adverse events (OR=1.36, 95% CI: 1.10-1.67, _P_=0.005). No significant differences were observed between the 2 groups in terms of progressive disease (PD) (OR=0.88, 95% CI: 0.67-1.15, _P_=0.34), all-cause any-grade adverse events (OR=1.04, 95% CI: 0.51-2.11, _P_=0.91), treatment-related any-grade adverse events (OR=1.32, 95% CI: 0.67-2.59, _P_=0.42), and treatment-related grade ≥3 adverse events (OR=1.16, 95% CI: 0.65-2.09, _P_=0.61).

Conclusions:

By incorporating the most recent data and the newly published studies, this updated meta-analysis offers a clearer understanding of immune checkpoint inhibitors and reinforces their advantage over other therapeutic options in the treatment of hepatocellular carcinoma. Continued research is encouraged that will further validate these findings.

Plain Language SummaryThis meta-analysis reviewed 18 randomized controlled trials involving 9244 patients to assess the effectiveness of immune checkpoint inhibitors (ICIs) in treating hepatocellular carcinoma. Compared to standard therapy or placebo, ICIs significantly improved objective response rate, disease control rate, stable disease, overall survival, and progression-free survival. However, ICIs were also linked to a higher incidence of severe adverse events. No significant differences were found in progressive disease or any-grade adverse events between ICIs and control groups. This study confirms the benefits of ICIs in hepatocellular carcinoma treatment, but further research is needed to validate these findings.

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