Cited by 6,198Cancer Immunotherapy and BiomarkersLung Cancer Treatments and MutationsLung Cancer Research Studies

Pembrolizumab for the Treatment of Non–Small-Cell Lung Cancer

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Garon, Edward B., Rizvi, Naiyer A., Hui, Rina, Leighl, Natasha, Balmanoukian, Ani S., Eder, Joseph Paul, Patnaik, Amita, Aggarwal, Charu +19 more·2015·New England Journal of Medicine

Abstract

BACKGROUND: We assessed the efficacy and safety of programmed cell death 1 (PD-1) inhibition with pembrolizumab in patients with advanced non-small-cell lung cancer enrolled in a phase 1 study. We also sought to define and validate an expression level of the PD-1 ligand 1 (PD-L1) that is associated with the likelihood of clinical benefit. METHODS: We assigned 495 patients receiving pembrolizumab (at a dose of either 2 mg or 10 mg per kilogram of body weight every 3 weeks or 10 mg per kilogram every 2 weeks) to either a training group (182 patients) or a validation group (313 patients). We assessed PD-L1 expression in tumor samples using immunohistochemical analysis, with results reported as the percentage of neoplastic cells with staining for membranous PD-L1 (proportion score). Response was assessed every 9 weeks by central review. RESULTS: Common side effects that were attributed to pembrolizumab were fatigue, pruritus, and decreased appetite, with no clear difference according to dose or schedule. Among all the patients, the objective response rate was 19.4%, and the median duration of response was 12.5 months. The median duration of progression-free survival was 3.7 months, and the median duration of overall survival was 12.0 months. PD-L1 expression in at least 50% of tumor cells was selected as the cutoff from the training group. Among patients with a proportion score of at least 50% in the validation group, the response rate was 45.2%. Among all the patients with a proportion score of at least 50%, median progression-free survival was 6.3 months; median overall survival was not reached. CONCLUSIONS: Pembrolizumab had an acceptable side-effect profile and showed antitumor activity in patients with advanced non-small-cell lung cancer. PD-L1 expression in at least 50% of tumor cells correlated with improved efficacy of pembrolizumab. (Funded by Merck; KEYNOTE-001 ClinicalTrials.gov number, NCT01295827.).

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Crossref · 10.1056/nejmoa1501824

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References 80 · Cited by 6,198

Authors
Garon, E. B., Rizvi, N. A., Hui, R., Leighl, N., Balmanoukian, A. S., Eder, J. P., Patnaik, A., Aggarwal, C., Gubens, M., Horn, L., Carcereny, E., Ahn, M.-J., Felip, E., Lee, J.-S., Hellmann, M. D., Hamid, O., Goldman, J. W., Soria, J.-C., Dolled-Filhart, M., Rutledge, R. Z., Zhang, J., Lunceford, J. K., Rangwala, R., Lubiniecki, G. M., Roach, C., Emancipator, K., & Gandhi, L.
Affiliations
  • University of California, Los Angeles (US)
  • Columbia University (US)
  • The University of Sydney (AU)
  • Princess Margaret Cancer Centre (CA)
  • Angeles Clinic and Research Institute (US)
  • Yale University (US)
  • South Texas Accelerated Research Therapeutics (US)
  • Abramson Cancer Center (US)
  • University of California, San Francisco (US)
  • Vanderbilt University (US)
  • Institut Català d'Oncologia (ES)
  • Samsung Medical Center (KR)
  • Vall d'Hebron Hospital Universitari (ES)
  • Seoul National University (KR)
  • Memorial Sloan Kettering Cancer Center (US)
  • Université Paris-Sud (FR)
  • Merck & Co., Inc., Rahway, NJ, USA (United States) (US)
  • Dana-Farber Cancer Institute (US)
Venue
New England Journal of Medicine
Published
2015-05-21
Volume
372
Issue
21
Pages
2018-2028
ISSN
  • 0028-4793 (ISSN-L)
  • 1533-4406
OpenAlex
W1982488143
Cited by
6,198
References
80
Citations by year
2026: 206 · 2025: 342 · 2024: 406 · 2023: 511 · 2022: 552 · 2021: 673 · 2020: 712 · 2019: 662 · 2018: 662 · 2017: 685 · 2016: 606 · 2015: 170 · 2014: 3 · 2013: 1
FWCI
306.88
Type
journal article
Language
en
Open access
Locations
  • New England Journal of Medicine (publishedVersion)
  • PubMed (publishedVersion)
  • IRIS Research product catalog (Sapienza University of Rome) (submittedVersion)
Topics
  • Health Sciences › Medicine › Oncology › Cancer Immunotherapy and Biomarkers
  • Health Sciences › Medicine › Pulmonary and Respiratory Medicine › Lung Cancer Treatments and Mutations
  • Health Sciences › Medicine › Oncology › Lung Cancer Research Studies
Keywords
pembrolizumab, non-small cell lung cancer, anti-PD-1 therapy, PD-L1 expression, predictive biomarkers, immune checkpoint inhibitors, objective response rate, progression-free survival, overall survival, adverse events
MeSH terms
Adult, Aged, Aged, 80 and over, Antineoplastic Agents, Carcinoma, Non-Small-Cell Lung, Female, Humans, Infusions, Intravenous, Lung Neoplasms, Male, Middle Aged, ROC Curve, Biomarkers, Survival Analysis, Disease-Free Survival, B7-H1 Antigen, Antibodies, Monoclonal, Humanized

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