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Updated: Jan 26, 2026

Y-90 Radioembolization and PD-1 Inhibitor as Neoadjuvant Treatment in Hepatocellular Carcinoma
Published on: May 24, 2024
Programmed cell death protein-1 (PD-1)-targeted immunotherapy in advanced hepatocellular carcinoma: efficacy and
Bernhard Scheiner1, Martha M Kirstein2, Florian Hucke3
1Vienna, Austria.
Background:
Programmed cell death protein-1-targeted immunotherapy has shown promising results in phase II studies of hepatocellular carcinoma.
Aim:
To evaluate safety and efficacy of nivolumab and pembrolizumab in an international, multicentre, real-world cohort of patients with advanced hepatocellular carcinoma.
Methods:
Sixty-five patients treated with nivolumab (n = 34) or pembrolizumab (n = 31) between July 10, 2015 and December 31, 2018 (data cut-off) across six centres in Austria and Germany were retrospectively analysed.
Results:
Child-Pugh class A/B/C was 32 (49%)/28 (43%)/5 (8%). Immunotherapy was used as systemic first-/second-/third-/fourth-line treatment in 9 (14%)/27 (42%)/26 (40%)/3 (5%) patients. Fifty-four patients had at least one follow-up imaging and were, therefore, available for radiological response assessment. The overall response and disease control rates were 12% and 49% respectively. Of 52 evaluable patients, four (8%) had hyperprogressive disease. Median time to progression was 5.5 (95% CI, 3.5-7.4) months, median progression-free survival was 4.6 (95% CI, 3.0-6.2) months, and median overall survival was 11.0 (95% CI, 8.2-13.8) months. Most common adverse events were infections (n = 7), rash (n = 6), pruritus (n = 3), fatigue (n = 3), diarrhoea (n = 3) and hepatitis (n = 3). Efficacy and safety results were comparable between Child-Pugh A and B patients; however, median overall survival (OS) was shorter in Child-Pugh B patients (16.7 vs 8.6 months; P = 0.065). There was no difference in terms of efficacy and adverse events between patients who received immunotherapy as first-/second-line and third-/fourth-line respectively.
Conclusions:
Programmed cell death protein-1-targeted immunotherapy with nivolumab or pembrolizumab showed promising efficacy and safety in patients with advanced hepatocellular carcinoma, including subjects with Child-Pugh stage B and patients with intensive pretreatment.
Insights
Programmed cell death protein-1 (PD-1) targeted immunotherapy with nivolumab or pembrolizumab demonstrated favorable safety and efficacy in advanced hepatocellular carcinoma patients. These findings support PD-1 inhibitors for treating this challenging cancer, even in patients with Child-Pugh stage B.
Area of Science:
- Hepatocellular Carcinoma (HCC) treatment
- Immunotherapy
- Oncology
Background:
- Programmed cell death protein-1 (PD-1) targeted immunotherapy shows promise in phase II studies for hepatocellular carcinoma (HCC).
- Real-world data is crucial for understanding the safety and efficacy of these treatments in advanced HCC.
Purpose of the Study:
- To evaluate the safety and efficacy of nivolumab and pembrolizumab in a real-world, international cohort of advanced HCC patients.
- To assess treatment outcomes across different lines of therapy and in patients with varying liver function (Child-Pugh scores).
Main Methods:
- Retrospective analysis of 65 patients with advanced HCC treated with nivolumab or pembrolizumab between July 2015 and December 2018.
- Patients were categorized by Child-Pugh class (A/B/C) and line of immunotherapy treatment (1st-4th line).
- Radiological response, time to progression, progression-free survival (PFS), and overall survival (OS) were assessed.
Main Results:
- The overall response rate was 12%, and disease control rate was 49%. Median PFS was 4.6 months, and median OS was 11.0 months.
- Hyperprogressive disease was observed in 8% of evaluable patients. Common adverse events included infections, rash, and fatigue.
- Efficacy and safety were comparable between Child-Pugh A and B patients, though OS was shorter in Child-Pugh B patients (8.6 months vs. 16.7 months).
Conclusions:
- PD-1 targeted immunotherapy with nivolumab or pembrolizumab demonstrates promising efficacy and safety in advanced HCC.
- Treatment outcomes were consistent across different lines of therapy and in patients with Child-Pugh stage B.
- These findings support the use of PD-1 inhibitors in advanced HCC, including heavily pretreated patients and those with impaired liver function.
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