PT - JOURNAL ARTICLE AU - Leila Khoja AU - Marcus O. Butler AU - S. Peter Kang AU - Scot Ebbinghaus AU - Anthony M. Joshua TI - Pembrolizumab AID - 10.1186/s40425-015-0078-9 DP - 2015 Dec 01 TA - Journal for ImmunoTherapy of Cancer PG - 36 VI - 3 IP - 1 4099 - http://jitc.bmj.com/content/3/1/36.short 4100 - http://jitc.bmj.com/content/3/1/36.full SO - J Immunother Cancer2015 Dec 01; 3 AB - The development of the cytotoxic T-lymphocyte-associated protein 4 inhibitor ipilimumab and its approval in 2011 for the treatment of metastatic melanoma has heralded a new era in immuno-oncology. Subsequently, novel agents against the programmed death receptor 1 (PD-1)/programmed death receptor ligand 1 (PD-L1) axis have shown significant activity in melanoma and a variety of other tumor types. Pembrolizumab was the first anti-PD-1 antibody to be approved by the US Food and Drug Administration for the treatment of patients with unresectable or metastatic melanoma with disease progression following ipilimumab, and if BRAF V600 mutation positive, a BRAF inhibitor. Pembrolizumab has also received breakthrough status for the treatment of EGFR mutation-negative, ALK rearrangement-negative non-small cell lung cancer (NSCLC) that has progressed on or following platinum-based chemotherapy. There remain a number of pivotal trials in progress to further evaluate the optimal use of pembrolizumab alone and in combination for melanoma, NSCLC, and other tumor types. In this article, we review the efficacy and toxicity profile of pembrolizumab and evaluate its future development.Abbreviations:AEAdverse eventALKAnaplastic lymphoma kinaseALTAlanine aminotransferaseAPCAntigen-presenting cellCIConfidence intervalCRComplete responseDLBCLDiffuse large B-cell lymphomaDRDrug-relatedDRAEDrug-related adverse eventEGFREpidermal growth factor receptorFDAUS Food and Drug AdministrationGBMGlioblastoma multiformeHNSCCHead and neck squamous cell carcinomaHPVHuman papilloma virusHRHazard ratioIFN-γInterferon gammaIHCImmunohistochemicalirRCImmune-related response criteriaLDHLactate dehydrogenaseMRIMagnetic resonance imagingNRNot reached/not reportedNSCLCNon-small cell lung cancerORROverall response rateOSOverall survivalPD-1Programmed death receptor 1PD-L1Programmed death receptor ligand 1PD-L2Programmed death receptor ligand 2PFSProgression-free survivalPRPartial responseQ2WOnce every 2 weeksQ3WOnce every 3 weeksRECIST v1.1Response Evaluation Criteria in Solid Tumors version 1.1ROCReceiver operating curveSDStable diseaseTNFTumor necrosis factor