Anthony Boutros El-Khoueiry, MD

Associate Professor of Clinical Medicine

Norris Cancer Center CISO Chair

USC NCCC Clinical Trials Network

Section Chief for Developmental Therapeutics in the Division of Medical Oncology

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Anthony El-Khoueiry, MD, is an assistant professor of clinical medicine at the Keck School of Medicine of USC and director of clinical translation at the Southern California Clinical and Translational Science Institute (SC CTSI). He has extensive experience in clinical and translational research through his roles as medical director of the Clinical Investigations Support Office and director of the phase I drug development program at the USC Norris Comprehensive Cancer Center. He is the recipient of the National Cancer Institute Cancer Investigator Team Leadership Award in 2011.

Dr. El-Khoueiry has established a national reputation in clinical research-related hepatobiliary (liver, gall bladder and bile duct) and pancreatic cancers. He is a member of the National Cancer Institute Hepatobiliary Cancers Task Force and chair of the Southwest Oncology Group hepatobiliary cancers subcommittee. In addition, he is the principal investigator of several phase I, including first-in-human, clinical trials aimed at developing novel therapies for solid tumors. Dr. El-Khoueiry works with several basic scientists at USC on joint translational projects designed to identify molecular predictors of outcome and survival for patients with advanced gastrointestinal cancers.

Dr. El-Khoueiry completed his medical degree, residency in internal medicine and fellowship in hematology and oncology at USC. He unravels scientific and non-scientific mysteries in four languages: English, Arabic, French and Spanish.


  • National Cancer Institute : Clinical Investigator Team Leadership Award, 2011-2012
  • Southwest Oncology Group: Young Investigator Training Award, 2004
  • Division of Hematology: Researcher of the Year, 2003
  • USC: Dean’s Scholar, 1998


  • CCALGB 80802 (Alliance): Impact of Sorafenib with and without Doxorubicin on Hepatitis C infection in Patients with advanced hepatocellular carcinoma Cancer Res Commun. 2024 Feb 16. . View in PubMed
  • Nivolumab in sorafenib-naive and sorafenib-experienced patients with advanced hepatocellular carcinoma: 5-year follow-up from CheckMate 040 Ann Oncol. 2023 Dec 25. . View in PubMed
  • Adjuvant Chemoradiation in Patients with Lymph Node-Positive Biliary Tract Cancers: Secondary Analysis of a Single-Arm Clinical Trial (SWOG 0809) Ann Surg Oncol. 2023 Mar; 30(3):1354-1363. . View in PubMed
  • Exposure-response analysis for nivolumab plus ipilimumab combination therapy in patients with advanced hepatocellular carcinoma (CheckMate 040) Clin Transl Sci. 2023 08; 16(8):1445-1457. . View in PubMed
  • Phase 1 study of GSK3368715, a type I PRMT inhibitor, in patients with advanced solid tumors Br J Cancer. 2023 08; 129(2):309-317. . View in PubMed
  • Profiling of Circulating Tumor Cells for Screening of Selective Inhibitors of Tumor-Initiating Stem-Like Cells Adv Sci (Weinh). 2023 05; 10(14):e2206812. . View in PubMed
  • Nivolumab Plus Cabozantinib With or Without Ipilimumab for Advanced Hepatocellular Carcinoma: Results From Cohort 6 of the CheckMate 040 Trial J Clin Oncol. 2023 03 20; 41(9):1747-1757. . View in PubMed
  • Association of Body Mass Index With the Safety Profile of Nivolumab With or Without Ipilimumab JAMA Oncol. 2023 01 01; 9(1):102-111. . View in PubMed
  • Zanidatamab, a novel bispecific antibody, for the treatment of locally advanced or metastatic HER2-expressing or HER2-amplified cancers: a phase 1, dose-escalation and expansion study Lancet Oncol. 2022 Dec; 23(12):1558-1570. . View in PubMed
  • Safety and efficacy of cabozantinib for patients with advanced hepatocellular carcinoma who advanced to Child-Pugh B liver function at study week 8: a retrospective analysis of the CELESTIAL randomised controlled trial BMC Cancer. 2022 Apr 09; 22(1):377. . View in PubMed
  • First-in-human study of an OX40 (ivuxolimab) and 4-1BB (utomilumab) agonistic antibody combination in patients with advanced solid tumors J Immunother Cancer. 2022 10; 10(10). . View in PubMed
  • Quality of life assessment of cabozantinib in patients with advanced hepatocellular carcinoma in the CELESTIAL trial Eur J Cancer. 2022 06; 168:91-98. . View in PubMed
  • Molecular pathogenesis and systemic therapies for hepatocellular carcinoma Nat Cancer. 2022 04; 3(4):386-401. . View in PubMed
  • Efficacy and safety of cabozantinib for patients with advanced hepatocellular carcinoma based on albumin-bilirubin grade Br J Cancer. 2022 03; 126(4):569-575. . View in PubMed
  • Nivolumab versus sorafenib in advanced hepatocellular carcinoma (CheckMate 459): a randomised, multicentre, open-label, phase 3 trial Lancet Oncol. 2022 01; 23(1):77-90. . View in PubMed
  • Outcomes Based on Plasma Biomarkers for the Phase 3 CELESTIAL Trial of Cabozantinib versus Placebo in Advanced Hepatocellular Carcinoma Liver Cancer. 2022 Jan; 11(1):38-47. . View in PubMed
  • A Phase I, Open-Label, Dose-Escalation Study of the OX40 Agonist Ivuxolimab in Patients with Locally Advanced or Metastatic Cancers Clin Cancer Res. 2022 01 01; 28(1):71-83. . View in PubMed
  • DNMT and EZH2 inhibitors synergize to activate therapeutic targets in hepatocellular carcinoma Cancer Lett. 2022 11 01; 548:215899. . View in PubMed
  • Final Overall Survival Efficacy Results of Ivosidenib for Patients With Advanced Cholangiocarcinoma With IDH1 Mutation: The Phase 3 Randomized Clinical ClarIDHy Trial JAMA Oncol. 2021 Nov 01; 7(11):1669-1677. . View in PubMed
  • Etiology and Outcomes of Hepatocellular Carcinoma in an Ethnically Diverse Population: The Multiethnic Cohort Cancers (Basel). 2021 Jul 12; 13(14). . View in PubMed
  • Cabozantinib: An evolving therapy for hepatocellular carcinoma Cancer Treat Rev. 2021 Jul; 98:102221. . View in PubMed
  • Comparative Efficacy of Atezolizumab plus Bevacizumab and Other Treatment Options for Patients with Unresectable Hepatocellular Carcinoma: A Network Meta-Analysis Liver Cancer. 2021 Jun; 10(3):240-248. . View in PubMed
  • Pharmacokinetics and safety of niraparib in patients with moderate hepatic impairment Cancer Chemother Pharmacol. 2021 11; 88(5):825-836. . View in PubMed
  • Infigratinib (BGJ398) in previously treated patients with advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements: mature results from a multicentre, open-label, single-arm, phase 2 study Lancet Gastroenterol Hepatol. 2021 10; 6(10):803-815. . View in PubMed
  • Society for Immunotherapy of Cancer (SITC) clinical practice guideline on immunotherapy for the treatment of hepatocellular carcinoma J Immunother Cancer. 2021 09; 9(9). . View in PubMed
  • CheckMate 040 cohort 5: A phase I/II study of nivolumab in patients with advanced hepatocellular carcinoma and Child-Pugh B cirrhosis J Hepatol. 2021 09; 75(3):600-609. . View in PubMed
  • A phase I study of intravenous fenretinide (4-HPR) for patients with malignant solid tumors Cancer Chemother Pharmacol. 2021 04; 87(4):525-532. . View in PubMed
  • Presentation, Management, and Outcomes Across the Rural-Urban Continuum for Hepatocellular Carcinoma JNCI Cancer Spectr. 2021 02; 5(1). . View in PubMed
  • Liver Cancer Immunity Hepatology. 2021 01; 73 Suppl 1:86-103. . View in PubMed
  • IMbrave 151: a randomized phase II trial of atezolizumab combined with bevacizumab and chemotherapy in patients with advanced biliary tract cancer Ther Adv Med Oncol. 2021; 13:17588359211036544. . View in PubMed
  • Phase I, First-in-Human Study of the Probody Therapeutic CX-2029 in Adults with Advanced Solid Tumor Malignancies Clin Cancer Res. 2021 08 15; 27(16):4521-4530. . View in PubMed
  • Efficacy and Safety of Nivolumab Plus Ipilimumab in Patients With Advanced Hepatocellular Carcinoma Previously Treated With Sorafenib: The CheckMate 040 Randomized Clinical Trial JAMA Oncol. 2020 Nov 01; 6(11):e204564. . View in PubMed
  • Atezolizumab and Bevacizumab Combination Therapy for Hepatocellular Carcinoma Gastroenterol Hepatol (N Y). 2020 Mar; 16(3):145-148. . View in PubMed
  • Association of inflammatory biomarkers with clinical outcomes in nivolumab-treated patients with advanced hepatocellular carcinoma J Hepatol. 2020 12; 73(6):1460-1469. . View in PubMed
  • Phase II Trial of Neoadjuvant Bevacizumab with Modified FOLFOX7 in Patients with Stage II and III Rectal Cancer Oncologist. 2020 12; 25(12):e1879-e1885. . View in PubMed
  • Second-line cabozantinib after sorafenib treatment for advanced hepatocellular carcinoma: a subgroup analysis of the phase 3 CELESTIAL trial ESMO Open. 2020 08; 5(4). . View in PubMed
  • Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study Lancet Oncol. 2020 06; 21(6):796-807. . View in PubMed
  • Randomised phase II trial (SWOG S1310) of single agent MEK inhibitor trametinib Versus 5-fluorouracil or capecitabine in refractory advanced biliary cancer Eur J Cancer. 2020 05; 130:219-227. . View in PubMed
  • Diagnosis and management of toxicities of immune checkpoint inhibitors in hepatocellular carcinoma J Hepatol. 2020 02; 72(2):320-341. . View in PubMed
  • Phase I and Biomarker Study of the Wnt Pathway Modulator DKN-01 in Combination with Gemcitabine/Cisplatin in Advanced Biliary Tract Cancer Clin Cancer Res. 2020 12 01; 26(23):6158-6167. . View in PubMed
  • Serum Alpha-fetoprotein Levels and Clinical Outcomes in the Phase III CELESTIAL Study of Cabozantinib versus Placebo in Patients with Advanced Hepatocellular Carcinoma Clin Cancer Res. 2020 09 15; 26(18):4795-4804. . View in PubMed
  • Assessment of Treatment With Sorafenib Plus Doxorubicin vs Sorafenib Alone in Patients With Advanced Hepatocellular Carcinoma: Phase 3 CALGB 80802 Randomized Clinical Trial JAMA Oncol. 2019 Nov 01; 5(11):1582-1588. . View in PubMed
  • Adjuvant Therapy for Resected Biliary Tract Cancer: ASCO Clinical Practice Guideline J Clin Oncol. 2019 04 20; 37(12):1015-1027. . View in PubMed
  • Mandatory Research Biopsy Requirements Delay Initiation of Clinical Trials Front Oncol. 2019; 9:968. . View in PubMed
  • Monitoring Twitter Conversations for Targeted Recruitment in Cancer Trials in Los Angeles County: Protocol for a Mixed-Methods Pilot Study JMIR Res Protoc. 2018 Sep 25; 7(9):e177. . View in PubMed
  • Cabozantinib in Patients with Advanced and Progressing Hepatocellular Carcinoma N Engl J Med. 2018 Jul 05; 379(1):54-63. . View in PubMed
  • A multi-arm phase I dose escalating study of an oral NOTCH inhibitor BMS-986115 in patients with advanced solid tumours Invest New Drugs. 2018 12; 36(6):1026-1036. . View in PubMed
  • Integrative Epigenetic Analysis Reveals Therapeutic Targets to the DNA Methyltransferase Inhibitor Guadecitabine (SGI-110) in Hepatocellular Carcinoma Hepatology. 2018 10; 68(4):1412-1428. . View in PubMed
  • A phase 1, dose-escalation study of PF-06664178, an anti-Trop-2/Aur0101 antibody-drug conjugate in patients with advanced or metastatic solid tumors Invest New Drugs. 2018 10; 36(5):836-847. . View in PubMed
  • A phase I trial of escalating doses of cixutumumab (IMC-A12) and sorafenib in the treatment of advanced hepatocellular carcinoma Cancer Chemother Pharmacol. 2018 05; 81(5):957-963. . View in PubMed
  • Evaluation of hepatic impairment on pharmacokinetics and safety of crizotinib in patients with advanced cancer Cancer Chemother Pharmacol. 2018 04; 81(4):659-670. . View in PubMed
  • Clinical relevance of EMT and stem-like gene expression in circulating tumor cells of metastatic colorectal cancer patients Pharmacogenomics J. 2018 01; 18(1):29-34. . View in PubMed
  • Phase II Study of BGJ398 in Patients With FGFR-Altered Advanced Cholangiocarcinoma J Clin Oncol. 2018 01 20; 36(3):276-282. . View in PubMed
  • A Randomized Phase II Open-Label Multi-Institution Study of the Combination of Bevacizumab and Erlotinib Compared to Sorafenib in the First-Line Treatment of Patients with Advanced Hepatocellular Carcinoma Oncology. 2018; 94(6):329-339. . View in PubMed
  • AKT Inhibition in Solid Tumors With AKT1 Mutations J Clin Oncol. 2017 Jul 10; 35(20):2251-2259. . View in PubMed
  • Nivolumab in patients with advanced hepatocellular carcinoma (CheckMate 040): an open-label, non-comparative, phase 1/2 dose escalation and expansion trial Lancet. 2017 Jun 24; 389(10088):2492-2502. . View in PubMed
  • Combination epigenetic therapy in metastatic colorectal cancer (mCRC) with subcutaneous 5-azacitidine and entinostat: a phase 2 consortium/stand up 2 cancer study Oncotarget. 2017 May 23; 8(21):35326-35338. . View in PubMed
  • Effect of First-Line Chemotherapy Combined With Cetuximab or Bevacizumab on Overall Survival in Patients With KRAS Wild-Type Advanced or Metastatic Colorectal Cancer: A Randomized Clinical Trial JAMA. 2017 06 20; 317(23):2392-2401. . View in PubMed
  • The Promise of Immunotherapy in the Treatment of Hepatocellular Carcinoma Am Soc Clin Oncol Educ Book. 2017; 37:311-317. . View in PubMed
  • A Phase I Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Combination Therapy with Refametinib plus Sorafenib in Patients with Advanced Cancer Clin Cancer Res. 2016 05 15; 22(10):2368-76. . View in PubMed
  • Guadecitabine (SGI-110) priming sensitizes hepatocellular carcinoma cells to oxaliplatin Mol Oncol. 2015 Nov; 9(9):1799-814. . View in PubMed
  • SWOG S0809: A Phase II Intergroup Trial of Adjuvant Capecitabine and Gemcitabine Followed by Radiotherapy and Concurrent Capecitabine in Extrahepatic Cholangiocarcinoma and Gallbladder Carcinoma J Clin Oncol. 2015 Aug 20; 33(24):2617-22. . View in PubMed
  • Integrin genetic variants and stage-specific tumor recurrence in patients with stage II and III colon cancer Pharmacogenomics J. 2015 Jun; 15(3):226-34. . View in PubMed
  • Phase I trial of sorafenib following liver transplantation in patients with high-risk hepatocellular carcinoma Liver Cancer. 2015 Mar; 4(2):115-25. . View in PubMed
  • A phase I, pharmacokinetic, and pharmacodynamic evaluation of the DNA methyltransferase inhibitor 5-fluoro-2′-deoxycytidine, administered with tetrahydrouridine Cancer Chemother Pharmacol. 2015 Mar; 75(3):537-46. . View in PubMed
  • A phase II trial of bevacizumab plus temsirolimus in patients with advanced hepatocellular carcinoma Invest New Drugs. 2015 Feb; 33(1):241-6. . View in PubMed
  • A phase I study of PF-04449913, an oral hedgehog inhibitor, in patients with advanced solid tumors Clin Cancer Res. 2015 03 01; 21(5):1044-51. . View in PubMed
  • Phase I study of the halichondrin B analogue eribulin mesylate in combination with cisplatin in advanced solid tumors Br J Cancer. 2014 Dec 09; 111(12):2268-74. . View in PubMed
  • Phase I clinical trial of temsirolimus and vinorelbine in advanced solid tumors Cancer Chemother Pharmacol. 2014 Dec; 74(6):1227-34. . View in PubMed
  • Impact of sex on the survival of patients with hepatocellular carcinoma: a Surveillance, Epidemiology, and End Results analysis Cancer. 2014 Dec 01; 120(23):3707-16. . View in PubMed
  • S0941: a phase 2 SWOG study of sorafenib and erlotinib in patients with advanced gallbladder carcinoma or cholangiocarcinoma Br J Cancer. 2014 Feb 18; 110(4):882-7. . View in PubMed
  • Sustained inhibition of deacetylases is required for the antitumor activity of the histone deactylase inhibitors panobinostat and vorinostat in models of colorectal cancer Invest New Drugs. 2013 Aug; 31(4):845-57. . View in PubMed
  • The effects of liver impairment on the pharmacokinetics of brivanib, a dual inhibitor of fibroblast growth factor receptor and vascular endothelial growth factor receptor tyrosine kinases Cancer Chemother Pharmacol. 2013 Jul; 72(1):53-64. . View in PubMed
  • Pharmacogenetic profiling of CD133 is associated with response rate (RR) and progression-free survival (PFS) in patients with metastatic colorectal cancer (mCRC), treated with bevacizumab-based chemotherapy Pharmacogenomics J. 2013 Apr; 13(2):173-80. . View in PubMed
  • First-in-man phase I study of GC33, a novel recombinant humanized antibody against glypican-3, in patients with advanced hepatocellular carcinoma Clin Cancer Res. 2013 Feb 15; 19(4):920-8. . View in PubMed
  • SWOG 0514: a phase II study of sorafenib in patients with unresectable or metastatic gallbladder carcinoma and cholangiocarcinoma Invest New Drugs. 2012 Aug; 30(4):1646-51. . View in PubMed
  • A randomized phase II of gemcitabine and sorafenib versus sorafenib alone in patients with metastatic pancreatic cancer Invest New Drugs. 2012 Jun; 30(3):1175-83. . View in PubMed
  • The Cyclin D1 (CCND1) A870G polymorphism predicts clinical outcome to lapatinib and capecitabine in HER2-positive metastatic breast cancer Ann Oncol. 2012 Jun; 23(6):1455-64. . View in PubMed
  • A phase II trial of gemcitabine and capecitabine in patients with unresectable or metastatic gallbladder cancer or cholangiocarcinoma: Southwest Oncology Group study S0202 Cancer Chemother Pharmacol. 2011 Dec; 68(6):1595-602. . View in PubMed
  • Common cancer stem cell gene variants predict colon cancer recurrence Clin Cancer Res. 2011 Nov 01; 17(21):6934-43. . View in PubMed
  • Pharmacogenetic angiogenesis profiling for first-line Bevacizumab plus oxaliplatin-based chemotherapy in patients with metastatic colorectal cancer Clin Cancer Res. 2011 Sep 01; 17(17):5783-92. . View in PubMed
  • Phase I dose-escalation study to determine the safety, pharmacokinetics and pharmacodynamics of brivanib alaninate in combination with full-dose cetuximab in patients with advanced gastrointestinal malignancies who have failed prior therapy Br J Cancer. 2011 Jun 28; 105(1):44-52. . View in PubMed
  • The dual EGFR/HER2 inhibitor lapatinib synergistically enhances the antitumor activity of the histone deacetylase inhibitor panobinostat in colorectal cancer models Cancer Res. 2011 May 15; 71(10):3635-48. . View in PubMed
  • Phase I clinical trial of pegylated liposomal Doxorubicin and docetaxel in patients with advanced solid tumors Am J Clin Oncol. 2011 Feb; 34(1):27-31. . View in PubMed
  • A phase I/II trial of vorinostat in combination with 5-fluorouracil in patients with metastatic colorectal cancer who previously failed 5-FU-based chemotherapy Cancer Chemother Pharmacol. 2010 Apr; 65(5):979-88. . View in PubMed
  • Genetic variations in angiogenesis pathway genes associated with clinical outcome in localized gastric adenocarcinoma Ann Oncol. 2010 Jan; 21(1):78-86. . View in PubMed
  • Accomplishments in 2008 in the management of hepatobiliary cancers Gastrointest Cancer Res. 2009 Sep; 3(5 Supplement 2):S28-36. . View in PubMed
  • Rates and predictors of chemotherapy use for stage III colon cancer: a systematic review Cancer. 2008 Dec 15; 113(12):3279-89. . View in PubMed
  • Polymorphisms in cyclooxygenase-2 and epidermal growth factor receptor are associated with progression-free survival independent of K-ras in metastatic colorectal cancer patients treated with single-agent cetuximab Clin Cancer Res. 2008 Dec 01; 14(23):7884-95. . View in PubMed
  • Gender-related survival differences associated with EGFR polymorphisms in metastatic colon cancer Cancer Res. 2008 Apr 15; 68(8):3037-42. . View in PubMed
  • Thymidylate synthase haplotype is associated with tumor recurrence in stage II and stage III colon cancer Pharmacogenet Genomics. 2008 Feb; 18(2):161-8. . View in PubMed
  • Serum lactate dehydrogenase levels and glycolysis significantly correlate with tumor VEGFA and VEGFR expression in metastatic CRC patients Pharmacogenomics. 2007 Dec; 8(12):1705-13. . View in PubMed
  • Association of methylenetetrahydrofolate reductase gene polymorphisms and sex-specific survival in patients with metastatic colon cancer J Clin Oncol. 2007 Aug 20; 25(24):3726-31. . View in PubMed
  • Gene expression levels of epidermal growth factor receptor, survivin, and vascular endothelial growth factor as molecular markers of lymph node involvement in patients with locally advanced rectal cancer Clin Colorectal Cancer. 2006 Nov; 6(4):305-11. . View in PubMed
  • Reversible posterior leukoencephalopathy syndrome induced by RAF kinase inhibitor BAY 43-9006 J Clin Oncol. 2006 Oct 01; 24(28):e48. . View in PubMed
  • Epidermal growth factor receptor and epidermal growth factor receptor variant III gene expression in metastatic colorectal cancer Clin Colorectal Cancer. 2006 Sep; 6(3):214-8. . View in PubMed
  • Gene expression in tumor-adjacent normal tissue is associated with recurrence in patients with rectal cancer treated with adjuvant chemoradiation Pharmacogenet Genomics. 2006 Aug; 16(8):555-63. . View in PubMed
  • Should continuous infusion 5-fluorouracil become the standard of care in the USA as it is in Europe? Cancer Invest. 2006 Feb; 24(1):50-5.. View in PubMed
  • Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells J Virol. 1995 Feb; 69(2):748-55. . View in PubMed