Trial reportGenome medicine2022
Hepatocellular carcinoma patients with high circulating cytotoxic T cells and intra-tumoral immune signature benefit from pembrolizumab: results from a single-arm phase 2 trial.
Trial report in Genome medicine, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 64 papers, 4 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
64 citing papers in PubMed, 4 syntheses or guidelines pooled it, 132 citations in OpenAlex.
- Efficacy and safety of pembrolizumab in the treatment of advanced hepatocellular carcinoma: a systematic review and meta-analysis.European journal of clinical pharmacology · 2025Pooled it
- Monocyte-related markers as predictors of immune checkpoint inhibitor efficacy and immune-related adverse events: a systematic review and meta-analysis.Cancer metastasis reviews · 2025Pooled it
- Pretreatment neutrophil-to-lymphocyte ratio is associated with immunotherapy efficacy in patients with advanced cancer: a systematic review and meta-analysis.Scientific reports · 2025Pooled it
- Phase I-IV Drug Trials on Hepatocellular Carcinoma in Asian Populations: A Systematic Review of Ten Years of Studies.International journal of molecular sciences · 2024Pooled it
- Durvalumab plus HAIC-FOLFOX followed by maintenance durvalumab for hepatocellular carcinoma with major portal invasion: phase 2 DurHope study.Nature communications · 2026Trial
- Dynamic transcriptional immune landscape in response to NK-cell therapy combined with gemcitabine plus S-1 in advanced pancreatic cancer: a phase 1b/2 trial.Signal transduction and targeted therapy · 2025Trial
- Hepatocellular carcinoma patients with high circulating cytotoxic T cells and intra-tumoral immune signature benefit from pembrolizumab: results from a single-arm phase 2 trial.Genome medicine · 2022Trial
- From spectroscopic analysis to clinical imaging: emerging applications and persistent challenges of raman spectroscopy in CTCs-based liquid biopsies.Annals of medicine · 2026Review
- High Serum IgA Identifies Poor Responders to Immune Checkpoint Inhibitors in Patients with Unresectable Esophageal Squamous Cell Carcinoma.Annals of surgical oncology · 2026Article
- KLF16 promotes immune escape in hepatocellular carcinoma cells by upregulating PD-L1 expression via PRKDC.Translational oncology · 2026Article
- Clustering of quantitative CT features identifies HCC subtypes with distinct prognosis and immune signatures.European radiology experimental · 2026Article
- Pan-cancer analysis of single-cell profiles with polygenic signals reveals genetic influences on tumor immunity across cancers.Communications biology · 2026Article
- WNT-β-catenin signalling in hepatocellular carcinoma: from bench to clinical trials.Nature reviews. Gastroenterology & hepatology · 2026Review
- Novel models for predicting individualized outcomes in patients with advanced hepatocellular carcinoma receiving immunotherapy.Frontiers in oncology · 2026Article
- Tumor-based biomarkers and circulating tumor DNA for precision medicine in advanced hepatocellular carcinoma.Clinical and molecular hepatology · 2026Review
- Metabolic Reprogramming ofCancer communications (London, England) · 2026Article
- Current Perspectives on the Use of Adjuvant Pembrolizumab for Hepatocellular Carcinoma.Journal of hepatocellular carcinoma · 2026Review
- Structural and temporal dynamics analysis of PD-1/PD-L1 immunotherapy in hepatocellular carcinoma: History, research hotspots, and emerging trends.Human vaccines & immunotherapeutics · 2025Article
- Multidimensional tumor-blood profiling uncovers systemic lymphocyte-monocyte imbalance in pituitary neuroendocrine tumors.Signal transduction and targeted therapy · 2025Article
- Reversal of tumour immune evasion via enhanced MHC-Class-I antigen presentation by a dual-functional RNA regulated system.Molecular cancer · 2025Article
4 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
22 authors at 5 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundA limited number of studies have characterized genomic properties of hepatocellular carcinoma (HCC) patients in response to anti-PD-1 immunotherapy.
methodsHerein, we performed comprehensive molecular characterization of immediate (D-42 to D-1) pre-treatment tumor biopsy specimens from 60 patients with sorafenib-failed HCC in a single-arm prospective phase II trial of pembrolizumab. Objective response rate was the primary efficacy endpoint. We used whole-exome sequencing, RNA sequencing, and correlative analysis. In addition, we performed single-cell RNA sequencing using peripheral blood mononuclear cells.
resultsThe overall response rate of pembrolizumab in sorafenib-failed HCC patients was 10% ([6/60] 95% CI, 2.4-17.6). In a univariate analysis using clinicopathological features, female gender, PD-L1 positivity, and low neutrophil-to-lymphocyte ratio (NLR) were identified as contributing factors to pembrolizumab response. Somatic mutations in CTNNB1 and genomic amplifications in MET were found only in non-responders. Transcriptional profiles through RNA sequencing identified that pembrolizumab responders demonstrated T cell receptor (TCR) signaling activation with expressions of MHC genes, indicating increased levels of T cell cytotoxicity. In single-cell sequencing from 10 pre- and post-treatment peripheral blood mononuclear cells (PBMCs), patients who achieved a partial response or stable disease exhibited immunological shifts toward cytotoxic CD8+ T cells. Conversely, patients with progressive disease showed an increased number of both CD14+ and CD16+ monocytes and activation of neutrophil-associated pathways.
conclusionsTaken together, HCC patients with infiltration of cytotoxic T cells, along with increased active circulating CD8+ T cells during pembrolizumab treatment and down-regulation of neutrophil-associated markers, significantly benefited from pembrolizumab treatment.
trial registrationNCT#03163992 (first posted: May 23, 2017).
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.