Real-world outcomes of patients with advanced intrahepatic cholangiocarcinoma treated with programmed cell death protein-1-targeted immunotherapy

References

• Sung H, Ferlay J, Siegel RL, et al. Global cancer statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2021;71(3):209–249.
   PubMed Web of Science ®Google Scholar
• Valle JW, Kelley RK, Nervi B, et al. Biliary tract cancer. Lancet. 2021;397(10272):428–444.
   PubMed Web of Science ®Google Scholar
• Sirica AE, Gores GJ, Groopman JD, et al. Intrahepatic cholangiocarcinoma: continuing challenges and translational advances. Hepatology. 2019;69(4):1803–1815.
   PubMed Web of Science ®Google Scholar
• Patel T. Cholangiocarcinoma-controversies and challenges. Nat Rev Gastroenterol Hepatol. 2011;8(4):189–200.
   PubMed Web of Science ®Google Scholar
• Mavros MN, Economopoulos KP, Alexiou VG, et al. Treatment and prognosis for patients with intrahepatic cholangiocarcinoma: systematic review and meta-analysis. JAMA Surg. 2014;149(6):565–574.
   PubMed Web of Science ®Google Scholar
• Le DT, Uram JN, Wang H, et al. PD-1 blockade in tumors with mismatch-repair deficiency. N Engl J Med. 2015;372(26):2509–2520.
   PubMed Web of Science ®Google Scholar
• Kansy BA, Concha-Benavente F, Srivastava RM, et al. PD-1 status in CD8+ T cells associates with survival and anti-PD-1 therapeutic outcomes in head and neck cancer. Cancer Res. 2017;77(22):6353–6364.
   PubMed Web of Science ®Google Scholar
• Migden MR, Rischin D, Schmults CD, et al. PD-1 blockade with cemiplimab in advanced cutaneous squamous-cell carcinoma. N Engl J Med. 2018;379(4):341–351.
   PubMed Web of Science ®Google Scholar
• Sato Y, Kinoshita M, Takemura S, et al. The PD-1/PD-L1 axis may be aberrantly activated in occupational cholangiocarcinoma. Pathol Int. 2017;67(3):163–170.
   PubMed Web of Science ®Google Scholar
• Ye Y, Zhou L, Xie X, et al. Interaction of B7-H1 on intrahepatic cholangiocarcinoma cells with PD-1 on tumor-infiltrating T cells as a mechanism of immune evasion. J Surg Oncol. 2009;100(6):500–504.
   PubMed Web of Science ®Google Scholar
• Gani F, Nagarajan N, Kim Y, et al. Program death 1 immune checkpoint and tumor microenvironment: Implications for patients with intrahepatic cholangiocarcinoma. Ann Surg Oncol. 2016;23(8):2610–2617.
   PubMed Web of Science ®Google Scholar
• Ricci AD, Rizzo A, Brandi G. Immunotherapy in biliary tract cancer: worthy of a second look. Cancer Control. 2020;27(3):1073274820948047.
   Web of Science ®Google Scholar
• Boilève A, Hilmi M, Smolenschi C, et al. Immunotherapy in advanced biliary tract cancers. Cancers (Basel). 2021;13(7):1569.
   PubMed Web of Science ®Google Scholar
• Rizzo A, Ricci AD, Brandi G. Recent advances of immunotherapy for biliary tract cancer. Expert Rev Gastroenterol Hepatol. 2021;15(5):527–536.
   PubMed Web of Science ®Google Scholar
• Guo X, Shen W. Latest evidence on immunotherapy for cholangiocarcinoma. Oncol Lett. 2020;20(6):381.
   PubMed Web of Science ®Google Scholar
• Rizzo A, Ricci AD, Brandi G. Durvalumab: an investigational anti-PD-L1 antibody for the treatment of biliary tract cancer. Expert Opin Investig Drugs. 2021;30(4):343–350.
   PubMed Web of Science ®Google Scholar
• Nagtegaal ID, WHO Classification of Tumours Editorial Board, Odze RD, Klimstra D, et al. The 2019 WHO classification of tumours of the digestive system. Histopathology. 2020;76(2):182–188.
   PubMed Web of Science ®Google Scholar
• Eisenhauer EA, Therasse P, Bogaerts J, et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer. 2009;45(2):228–247.
   PubMed Web of Science ®Google Scholar
• Lencioni R, Llovet JM. Modified RECIST (mRECIST) assessment for hepatocellular carcinoma. Semin Liver Dis. 2010;30(1):52–60.
   PubMed Web of Science ®Google Scholar
• Johnson PJ, Berhane S, Kagebayashi C, et al. Assessment of liver function in patients with hepatocellular carcinoma: a new evidence-based approach-the ALBI grade. J Clin Oncol. 2015;33(6):550–558.
   PubMed Web of Science ®Google Scholar
• Chan A, Leung H, Chong C, et al. Validating the ALBI grade: Its current and future use in HCC prognostication. J Hepatol. 2017;66(3):661–663.
   PubMed Web of Science ®Google Scholar
• Pinato DJ, Sharma R, Allara E, et al. The ALBI grade provides objective hepatic reserve estimation across each BCLC stage of hepatocellular carcinoma. J Hepatol. 2017;66(2):338–346.
   PubMed Web of Science ®Google Scholar
• Deng M, Ng S, Cheung ST, et al. Clinical application of Albumin-Bilirubin (ALBI) score: the current status. Surgeon. 2020;18(3):178–186.
   PubMed Web of Science ®Google Scholar
• Brindley PJ, Bachini M, Ilyas SI, et al. Cholangiocarcinoma. Nat Rev Dis Primers. 2021;7(1):65.
   PubMed Web of Science ®Google Scholar
• Lee SH, Lee HS, Lee SH, et al. Efficacy and safety of pembrolizumab for gemcitabine/Cisplatin-Refractory biliary tract cancer: a multicenter retrospective study. J Clin Med. 2020;9(6):1769.
   Web of Science ®Google Scholar
• Kang J, Jeong JH, Hwang HS, et al. Efficacy and safety of pembrolizumab in patients with refractory advanced biliary tract cancer: tumor proportion score as a potential biomarker for response. Cancer Res Treat. 2020;52(2):594–603.
   PubMed Web of Science ®Google Scholar
• Pinato DJ, Kaneko T, Saeed A, et al. Immunotherapy in hepatocellular cancer patients with mild to severe liver dysfunction: adjunctive role of the ALBI grade. Cancers (Basel). 2020;12(7):1862.
   PubMed Web of Science ®Google Scholar
• Wong J, Kwok G, Tang V, et al. Ipilimumab and nivolumab/pembrolizumab in advanced hepatocellular carcinoma refractory to prior immune checkpoint inhibitors. J Immunother Cancer. 2021;9(2):e001945.
   PubMed Web of Science ®Google Scholar
• Hilmi M, Neuzillet C, Calderaro J, et al. Angiogenesis and immune checkpoint inhibitors as therapies for hepatocellular carcinoma: current knowledge and future research directions. J Immunother Cancer. 2019;7(1):333.
   PubMed Web of Science ®Google Scholar
• D'Andrea MA, Reddy GK. Systemic immunostimulatory effects of radiation therapy improves the outcomes of patients with advanced NSCLC receiving immunotherapy. Am J Clin Oncol. 2020;43(3):218–228.
   PubMed Web of Science ®Google Scholar
• Bang YJ, Golan T, Dahan L, et al. Ramucirumab and durvalumab for previously treated, advanced non-small-cell lung cancer, gastric/gastro-oesophageal junction adenocarcinoma, or hepatocellular carcinoma: an open-label, phase Ia/b study (JVDJ). Eur J Cancer. 2020;137:272–284.
   PubMed Web of Science ®Google Scholar
• Zhu AX, Finn RS, Edeline J, et al. Pembrolizumab in patients with advanced hepatocellular carcinoma previously treated with sorafenib (KEYNOTE-224): a non-randomised, open-label phase 2 trial. Lancet Oncol. 2018;19(7):940–952.
   PubMed Web of Science ®Google Scholar
• Finn RS, Qin S, Ikeda M, et al. Atezolizumab plus bevacizumab in unresectable hepatocellular carcinoma. N Engl J Med. 2020;382(20):1894–1905.
   PubMed Web of Science ®Google Scholar
• Lai E, Astara G, Ziranu P, et al. Introducing immunotherapy for advanced hepatocellular carcinoma patients: too early or too fast. Crit Rev Oncol Hematol. 2021;157:103167.
   PubMed Web of Science ®Google Scholar
• Razumilava N, Gores GJ. Cholangiocarcinoma. Lancet. 2014;383(9935):2168–2179.
   PubMed Web of Science ®Google Scholar
• Banales JM, Marin J, Lamarca A, et al. Cholangiocarcinoma 2020: the next horizon in mechanisms and management. Nat Rev Gastroenterol Hepatol. 2020;17(9):557–588.
   PubMed Web of Science ®Google Scholar
• Massironi S, Pilla L, Elvevi A, et al. New and emerging systemic therapeutic options for advanced cholangiocarcinoma. Cells. 2020;9(3):688.
   PubMed Web of Science ®Google Scholar
• Valle J, Wasan H, Palmer DH, et al. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med. 2010;362(14):1273–1281.
   PubMed Web of Science ®Google Scholar
• Ebata T, Bile Duct Cancer Adjuvant Trial (BCAT) Study Group, Hirano S, Konishi M, et al. Randomized clinical trial of adjuvant gemcitabine chemotherapy versus observation in resected bile duct cancer. Br J Surg. 2018;105(3):192–202.
   PubMed Web of Science ®Google Scholar
• Edeline J, Benabdelghani M, Bertaut A, et al. Gemcitabine and oxaliplatin chemotherapy or surveillance in resected biliary tract cancer (PRODIGE 12-ACCORD 18-UNICANCER GI): a randomized phase III study. J Clin Oncol. 2019;37(8):658–667.
   PubMed Web of Science ®Google Scholar
• Scheuermann U, Kaths JM, Heise M, et al. Comparison of resection and transarterial chemoembolisation in the treatment of advanced intrahepatic cholangiocarcinoma–a single-center experience. Eur J Surg Oncol. 2013;39(6):593–600.
   PubMed Web of Science ®Google Scholar
• Li J, Wang Q, Lei Z, et al. Adjuvant transarterial chemoembolization following liver resection for intrahepatic cholangiocarcinoma based on survival risk stratification. Oncologist. 2015;20(6):640–647.
   PubMed Web of Science ®Google Scholar
• Hammad AY, Berger NG, Eastwood D, et al. Is radiotherapy warranted following intrahepatic cholangiocarcinoma resection? The impact of surgical margins and lymph node status on survival. Ann Surg Oncol. 2016;23(S5):912–920.
   PubMedGoogle Scholar
• Sha M, Jeong S, Xia Q. Antiviral therapy improves survival in patients with HBV infection and intrahepatic cholangiocarcinoma undergoing liver resection: novel concerns. J Hepatol. 2018;68(6):1315–1316.
   PubMed Web of Science ®Google Scholar
• Ueno M, Ikeda M, Morizane C, et al. Nivolumab alone or in combination with cisplatin plus gemcitabine in japanese patients with unresectable or recurrent biliary tract cancer: a non-randomised, multicentre, open-label, phase 1 study. Lancet Gastroenterol Hepatol. 2019;4(8):611–621.
   PubMed Web of Science ®Google Scholar
• Piha-Paul SA, Oh DY, Ueno M, et al. Efficacy and safety of pembrolizumab for the treatment of advanced biliary cancer: results from the KEYNOTE-158 and KEYNOTE-028 studies. Int J Cancer. 2020;147(8):2190–2198.
   PubMed Web of Science ®Google Scholar
• Kim RD, Chung V, Alese OB, et al. A phase 2 multi-institutional study of nivolumab for patients with advanced refractory biliary tract cancer. JAMA Oncol. 2020;6(6):888–894.
   PubMed Web of Science ®Google Scholar
• Yoo C, Oh DY, Choi HJ, et al. Phase I study of bintrafusp alfa, a bifunctional fusion protein targeting TGF-β and PD-L1, in patients with pretreated biliary tract cancer. J Immunother Cancer. 2020;8(1):e000564.
   PubMed Web of Science ®Google Scholar