Ivosidenib for the treatment of isocitrate dehydrogenase-1 mutant cholangiocarcinoma

References

• Rizvi S, Gores GJ. Pathogenesis, diagnosis, and management of cholangiocarcinoma. Gastroenterology. 2013 Dec;145(6):1215–1229.
   PubMed Web of Science ®Google Scholar
• Chapman RW. Risk factors for biliary tract carcinogenesis. Ann Oncol. 1999;10(Suppl 4):308–311.
   PubMed Web of Science ®Google Scholar
• Bergquist A, Von Seth E. Epidemiology of cholangiocarcinoma. Best Pract Res Clin Gastroenterol. 2015 Apr;29(2):221–232.
   PubMed Web of Science ®Google Scholar
• Shin HR, Oh JK, Masuyer E, et al. Comparison of incidence of intrahepatic and extrahepatic cholangiocarcinoma–focus on East and South-Eastern Asia. Asian Pac J Cancer Prev. 2010;11(5):1159–1166.
   PubMedGoogle Scholar
• Gad MM, Saad AM, Faisaluddin M, et al. Epidemiology of cholangiocarcinoma; United States incidence and mortality trends. Clin Res Hepatol Gastroenterol. 2020 Nov;44(6):885–893.
   PubMed Web of Science ®Google Scholar
• Khan SA, Emadossadaty S, Ladep NG, et al. Rising trends in cholangiocarcinoma: is the ICD classification system misleading us? J Hepatol. 2012 Apr;56(4):848–854.
   PubMed Web of Science ®Google Scholar
• DeOliveira ML, Cunningham SC, Cameron JL, et al. Cholangiocarcinoma: thirty-one-year experience with 564 patients at a single institution. Ann Surg. 2007 May;245(5):755–762.
   PubMed Web of Science ®Google Scholar
• Jarnagin WR. Cholangiocarcinoma of the extrahepatic bile ducts. Semin Surg Oncol. 2000 Sep-Oct;19(2):156–176.
   PubMedGoogle Scholar
• Shaib YH, El-Serag HB, Davila JA, et al. Risk factors of intrahepatic cholangiocarcinoma in the United States: a case-control study. Gastroenterology. 2005 Mar;128(3):620–626.
   PubMed Web of Science ®Google Scholar
• Khan SA, Davidson BR, Goldin RD, et al. Guidelines for the diagnosis and treatment of cholangiocarcinoma: an update. Gut. 2012 Dec;61(12):1657–1669.
   PubMed Web of Science ®Google Scholar
• Jarnagin WR, Fong Y, DeMatteo RP, et al. Staging, resectability, and outcome in 225 patients with hilar cholangiocarcinoma. Ann Surg. 2001 Oct;234(4):507–517. discussion 517–9
   PubMed Web of Science ®Google Scholar
• Sapisochin G, Javle M, Lerut J, et al. Liver transplantation for cholangiocarcinoma and mixed hepatocellular cholangiocarcinoma: working group report from the ILTS transplant oncology consensus conference. Transplantation. 2020 Jun;104(6):1125–1130.
   PubMed Web of Science ®Google Scholar
• Primrose JN, Fox RP, Palmer DH, et al. Capecitabine compared with observation in resected biliary tract cancer (BILCAP): a randomised, controlled, multicentre, phase 3 study. Lancet Oncol. 2019 May;20(5):663–673.
   PubMed Web of Science ®Google Scholar
• Nagino M, Ebata T, Yokoyama Y, et al. Evolution of surgical treatment for perihilar cholangiocarcinoma: a single-center 34-year review of 574 consecutive resections. Ann Surg. 2013 Jul;258(1):129–140.
   PubMed Web of Science ®Google Scholar
• Ribero D, Pinna AD, Guglielmi A, et al. Surgical approach for long-term survival of patients with intrahepatic cholangiocarcinoma: a multi-institutional analysis of 434 patients. Arch Surg. 2012 Dec;147(12):1107–1113.
   PubMedGoogle Scholar
• Valle J, Wasan H, Palmer DH, et al. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med. 2010 Apr 8;362(14):1273–1281.
   PubMed Web of Science ®Google Scholar
• Valle JW, Borbath I, Khan SA, et al. Biliary cancer: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2016 Sep;27(suppl 5):v28–v37.
   PubMed Web of Science ®Google Scholar
• National Comprehensive Cancer Network. Hepatobiliary cancers (version 5.2020) 2020 [cited 2021 Jan 31]. Available from: https://www.nccn.org/professionals/physician_gls/pdf/hepatobiliary_blocks.pdf
   Google Scholar
• Lamarca A, Hubner RA, David Ryder W, et al. Second-line chemotherapy in advanced biliary cancer: a systematic review. Ann Oncol. 2014 Dec;25(12):2328–2338.
   PubMed Web of Science ®Google Scholar
• Lowery MA, Goff LW, Keenan BP, et al. Second-line chemotherapy in advanced biliary cancers: a retrospective, multicenter analysis of outcomes. Cancer. 2019 Dec 15;125(24):4426–4434.
   PubMed Web of Science ®Google Scholar
• Lamarca A, Palmer DH, Wasan H, et al. ABC-06 | a randomised phase III, multi-centre, open-label study of active symptom control (ASC) alone or ASC with oxaliplatin/5-FU chemotherapy (ASC+mFOLFOX) for patients (pts) with locally advanced/metastatic biliary tract cancers (ABC) previously-treated with cisplatin/gemcitabine (CisGem) chemotherapy. J clin oncol. 2019;37(15_suppl):4003. .
   Web of Science ®Google Scholar
• Demols A, Borbath I, Van Den Eynde M, et al. Regorafenib after failure of gemcitabine and platinum-based chemotherapy for locally advanced/metastatic biliary tumors: REACHIN, a randomized, double-blind, phase II trial. Ann Oncol. 2020 Sep;31(9):1169–1177. .
   PubMed Web of Science ®Google Scholar
• Tella SH, Kommalapati A, Borad MJ, et al. Second-line therapies in advanced biliary tract cancers. Lancet Oncol. 2020 Jan;21(1):e29–e41.
   PubMed Web of Science ®Google Scholar
• DiPeri TP, Javle MM, Meric-Bernstam F. Next generation sequencing for biliary tract cancers. Expert Rev Gastroenterol Hepatol. 2021 Mar;10:1–4.
   Web of Science ®Google Scholar
• Jusakul A, Cutcutache I, Yong CH, et al. Whole-genome and epigenomic landscapes of etiologically distinct subtypes of cholangiocarcinoma. Cancer Discov. 2017 Oct;7(10):1116–1135.
   PubMed Web of Science ®Google Scholar
• Lowery MA, Ptashkin R, Jordan E, et al. Comprehensive molecular profiling of intrahepatic and extrahepatic cholangiocarcinomas: potential targets for intervention. Clin Cancer Res off J Am Assoc Cancer Res. 2018 Sep 1;24(17):4154–4161.
   PubMed Web of Science ®Google Scholar
• Javle M, Bekaii-Saab T, Jain A, et al. Biliary cancer: utility of next-generation sequencing for clinical management. Cancer. 2016 Dec 15;122(24):3838–3847.
   PubMed Web of Science ®Google Scholar
• Nakamura H, Arai Y, Totoki Y, et al. Genomic spectra of biliary tract cancer. Nat Genet. 2015 Sep;47(9):1003–1010.
   PubMed Web of Science ®Google Scholar
• Boscoe AN, Rolland C, Kelley RK. Frequency and prognostic significance of isocitrate dehydrogenase 1 mutations in cholangiocarcinoma: a systematic literature review. J Gastrointest Oncol. 2019 Aug;10(4):751–765.
   PubMed Web of Science ®Google Scholar
• Wang P, Dong Q, Zhang C, et al. Mutations in isocitrate dehydrogenase 1 and 2 occur frequently in intrahepatic cholangiocarcinomas and share hypermethylation targets with glioblastomas. Oncogene. 2013 Jun 20;32(25):3091–3100.
   PubMed Web of Science ®Google Scholar
• Abou-Alfa GK, Macarulla T, Javle MM, et al. Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study. Lancet Oncol. 2020 Jun;21(6):796–807. .
   PubMed Web of Science ®Google Scholar
• Popovici-Muller J, Lemieux RM, Artin E, et al. Discovery of AG-120 (Ivosidenib): a first-in-class mutant IDH1 inhibitor for the treatment of IDH1 mutant cancers. ACS Med Chem Lett. 2018 Apr 12;9(4):300–305.
   PubMed Web of Science ®Google Scholar
• Agios Pharmaceuticals TIBSOVO (ivosidenib). Highlights of prescribing information 2019 [cited 2021 Feb 3rd]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/211192s001lbl.pdf
   Google Scholar
• Fan B, Mellinghoff IK, Wen PY, et al. Clinical pharmacokinetics and pharmacodynamics of ivosidenib, an oral, targeted inhibitor of mutant IDH1, in patients with advanced solid tumors. Invest New Drugs. 2020 Apr;38(2):433–444.
   PubMed Web of Science ®Google Scholar
• Valle JW, Lamarca A, Goyal L, et al. New Horizons for Precision Medicine in Biliary Tract Cancers. Cancer Discov. 2017 Sep;7(9):943–962.
   PubMed Web of Science ®Google Scholar
• Ward PS, Patel J, Wise DR, et al. The common feature of leukemia-associated IDH1 and IDH2 mutations is a neomorphic enzyme activity converting alpha-ketoglutarate to 2-hydroxyglutarate. Cancer Cell. 2010 Mar 16;17(3):225–234.
   PubMed Web of Science ®Google Scholar
• Dang L, White DW, Gross S, et al. Cancer-associated IDH1 mutations produce 2-hydroxyglutarate. Nature. 2009 Dec 10;462(7274):739–744.
   PubMed Web of Science ®Google Scholar
• Lu C, Ward PS, Kapoor GS, et al. IDH mutation impairs histone demethylation and results in a block to cell differentiation. Nature. 2012 Feb 15;483(7390):474–478.
   PubMed Web of Science ®Google Scholar
• Xu W, Yang H, Liu Y, et al. Oncometabolite 2-hydroxyglutarate is a competitive inhibitor of alpha-ketoglutarate-dependent dioxygenases. Cancer Cell. 2011 Jan 18;19(1):17–30.
   PubMed Web of Science ®Google Scholar
• Saha SK, Parachoniak CA, Ghanta KS, et al. Mutant IDH inhibits HNF-4alpha to block hepatocyte differentiation and promote biliary cancer. Nature. 2014 Sep 4;513(7516):110–114.
   PubMed Web of Science ®Google Scholar
• Hansen E, Quivoron C, Straley K, et al. AG-120, an oral, selective, first-in-class, potent inhibitor of mutant IDH1, reduces intracellular 2HG and induces cellular differentiation in TF-1 R132H cells and primary human IDH1 mutant AML patient samples treated ex vivo. Blood. 2014;124(21):3734.
   Web of Science ®Google Scholar
• Lowery MA, Burris HA 3rd, Janku F, et al. Safety and activity of ivosidenib in patients with IDH1-mutant advanced cholangiocarcinoma: a phase 1 study. Lancet Gastroenterol Hepatol. 2019 Sep;4(9):711–720. .
   PubMed Web of Science ®Google Scholar
• Bunse L, Pusch S, Bunse T, et al. Suppression of antitumor T cell immunity by the oncometabolite (R)-2-hydroxyglutarate. Nat Med. 2018 Aug;24(8):1192–1203.
   PubMed Web of Science ®Google Scholar
• Aguado-Fraile E, Tassinari A, Ishii Y, et al. Molecular and morphological changes induced by ivosidenib correlate with efficacy in mutant-IDH1 cholangiocarcinoma. Future Oncol. 2021 Mar 12;
   PubMed Web of Science ®Google Scholar
• Stein EM, DiNardo CD, Fathi AT, et al. Ivosidenib or enasidenib combined with intensive chemotherapy in patients with newly diagnosed AML: a phase 1 study. Blood. 2020 Oct 5;135:463–471.
   Google Scholar
• Goyal L, Govindan A, Sheth RA, et al. Prognosis and clinicopathologic features of patients with advanced stage isocitrate dehydrogenase (IDH) mutant and IDH wild-type intrahepatic cholangiocarcinoma. Oncologist. 2015 Sep;20(9):1019–1027.
   PubMed Web of Science ®Google Scholar
• Choe S, Wang H, DiNardo CD, et al. Molecular mechanisms mediating relapse following ivosidenib monotherapy in IDH1-mutant relapsed or refractory AML. Blood Adv. 2020 May 12;4(9):1894–1905.
   PubMed Web of Science ®Google Scholar