An evaluation of ivosidenib for the treatment of IDH1-mutant cholangiocarcinoma

References

• Banales JM, Marin JJG, Lamarca A, et al. Cholangiocarcinoma 2020: the next horizon in mechanisms and management. Nat Clin Pract Gastroenterol Hepatol. 2020;17(9):557–588.
   Google Scholar
• Mukkamalla SKR, Naseri HM, Kim BM, et al. Trends in incidence and factors affecting survival of patients with cholangiocarcinoma in the United States. J Natl Compr Canc Netw. 2018;16(4):370–376.
   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;21(1):e29–e41.
   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
• Okusaka T, Nakachi K, Fukutomi A, et al. Gemcitabine alone or in combination with cisplatin in patients with biliary tract cancer: a comparative multicentre study in Japan. Br J Cancer. 2010;103(4):469–474.
   PubMed Web of Science ®Google Scholar
• D-Y O, He AR, Qin S, et al. A phase 3 randomized, double-blind, placebo-controlled study of durvalumab in combination with gemcitabine plus cisplatin (GemCis) in patients (pts) with advanced biliary tract cancer (BTC): TOPAZ-1. J Clin Oncol. 2022;40(4_suppl):378.
   Web of Science ®Google Scholar
• Morizane C, Okusaka T, Mizusawa J, et al. Combination gemcitabine plus S-1 versus gemcitabine plus cisplatin for advanced/recurrent biliary tract cancer: the FUGA-BT (JCOG1113) randomized phase III clinical trial. Ann Oncol. 2019;30(12):1950–1958.
   PubMed Web of Science ®Google Scholar
• Shroff RT, Javle MM, Xiao L, et al. Gemcitabine, cisplatin, and nab-paclitaxel for the treatment of advanced biliary tract cancers: a Phase 2 clinical trial. JAMA Oncol. 2019;5(6):824–830.
   PubMed Web of Science ®Google Scholar
• Harsha Tella S, Wessling J, Nathan F, et al. CLO22-088: phase II trial of trifluridine/tipiracil in combination with irinotecan in advanced Biliary Tract Cancers (BTCs). J National Compr Cancer Network. 2022;20(3.5):CLO22–088.
   Google Scholar
• Lamarca A, Palmer DH, Wasan HS, et al. Second-line FOLFOX chemotherapy versus active symptom control for advanced biliary tract cancer (ABC-06): a phase 3, open-label, randomised, controlled trial. Lancet Oncol. 2021;22(5):690–701.
   PubMed Web of Science ®Google Scholar
• Mizrahi J, Gunchick V, Mody K, et al. FOLFIRI in advanced biliary tract cancers. J clin oncol. 2019;37(4_suppl):451.
   Web of Science ®Google Scholar
• Chakrabarti S, Finnes HD, Mahipal A. Fibroblast growth factor receptor (FGFR) inhibitors in cholangiocarcinoma: current status, insight on resistance mechanisms and toxicity management. Expert Opin Drug Metab Toxicol. 2022;18(1):85–98.
   PubMed Web of Science ®Google Scholar
• Abou-Alfa GK, Sahai V, Hollebecque A, et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study. Lancet Oncol. 2020;21(5):671–684.
   PubMed Web of Science ®Google Scholar
• Javle M, Roychowdhury S, Kelley RK, et al. 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;6(10):803–815.
   PubMed Web of Science ®Google Scholar
• Abou-Alfa GK, Sahai V, Hollebecque A, et al. Pemigatinib for previously treated locally advanced/metastatic cholangiocarcinoma (CCA): update of FIGHT-202. J clin oncol. 2021;39(15_suppl):4086.
   Web of Science ®Google Scholar
• Rizzo A, Ricci AD, Brandi G. Pemigatinib: hot topics behind the first approval of a targeted therapy in cholangiocarcinoma. Cancer Treat Res Commun. 2021;27:100337.
   PubMedGoogle Scholar
• Javle M, Bekaii-Saab T, Jain A, et al. Biliary cancer: utility of next-generation sequencing for clinical management. Cancer. 2016;122(24):3838–3847.
   PubMed Web of Science ®Google Scholar
• Subbiah V, Lassen U, Élez E, et al. Dabrafenib plus trametinib in patients with BRAF(V600E)-mutated biliary tract cancer (ROAR): a phase 2, open-label, single-arm, multicentre basket trial. Lancet Oncol. 2020;21(9):1234–1243.
   PubMed Web of Science ®Google Scholar
• Salama AKS, Li S, Macrae ER, et al. Dabrafenib and trametinib in patients with tumors with BRAF(V600E) mutations: results of the NCI-MATCH Trial Subprotocol H. J Clin Oncol. 2020;38(33):3895–3904.
   PubMed Web of Science ®Google Scholar
• Galdy S, Lamarca A, McNamara MG, et al. HER2/HER3 pathway in biliary tract malignancies; systematic review and meta-analysis: a potential therapeutic target? Cancer Metastasis Rev. 2017;36(1):141–157.
   PubMed Web of Science ®Google Scholar
• Javle M, Churi C, Kang HC, et al. HER2/neu-directed therapy for biliary tract cancer. J Hematol Oncol. 2015;8(1):58.
   PubMed Web of Science ®Google Scholar
• Peck J, Wei L, Zalupski M, et al. HER2/neu may not be an interesting target in biliary cancers: results of an early phase II study with lapatinib. Oncology. 2012;82(3):175–179.
   PubMed Web of Science ®Google Scholar
• Javle M, Borad MJ, Azad NS, et al. Pertuzumab and trastuzumab for HER2-positive, metastatic biliary tract cancer (MyPathway): a multicentre, open-label, phase 2a, multiple basket study. Lancet Oncol. 2021;22(9):1290–1300.
   PubMed Web of Science ®Google Scholar
• Meric-Bernstam F, Hanna DL, El-Khoueiry AB, et al. Zanidatamab (ZW25) in HER2-positive biliary tract cancers (BTCs): results from a phase I study. J clin oncol. 2021;39(3_suppl):299.
   Web of Science ®Google Scholar
• Tsurutani J, Iwata H, Krop I, et al. Targeting HER2 with trastuzumab deruxtecan: a dose-expansion, phase i study in multiple advanced solid tumors. Cancer Discov. 2020;10(5):688–701.
   PubMed Web of Science ®Google Scholar
• Harding J, Cleary J, Shapiro G, et al. O-005 - Treating HER2-mutant advanced biliary tract cancer with neratinib: benefits of HER2-directed targeted therapy in the phase 2 SUMMIT ‘basket’ trial. Ann Oncol. 2019;30:iv127.
   Web of Science ®Google Scholar
• Rizzo A, Frega G, Ricci AD, et al. Anti-EGFR monoclonal antibodies in advanced biliary tract cancer: a systematic review and meta-analysis. Vivo. 2020;34(2):479–488.
   PubMed Web of Science ®Google Scholar
• Rizzo A, Ricci AD, Brandi G. Detecting and targeting NTRK gene fusions in cholangiocarcinoma: news and perspectives. Expert Rev Precis Med Drug Dev. 2021;6(4):225–227.
   Web of Science ®Google Scholar
• Casak SJ, Pradhan S, Fashoyin-Aje LA, et al. FDA Approval Summary: ivosidenib for the treatment of patients with advanced unresectable or metastatic, chemotherapy refractory cholangiocarcinoma with an IDH1 mutation. Clin Cancer Res. 2022;28(13):2733–2737.
   PubMed Web of Science ®Google Scholar
• Wu MJ, Shi L, Merritt J, et al. Biology of IDH mutant cholangiocarcinoma. Hepatology. 2022;75(5):1322–1337.
   PubMed Web of Science ®Google Scholar
• Wintheiser G, Zemla T, Shi Q, et al. Isocitrate dehydrogenase-mutated cholangiocarcinoma: natural history and clinical outcomes. JCO Precis Oncol. 2022;6(6):e2100156.
   PubMedGoogle Scholar
• Dhillon S. Ivosidenib: first Global Approval. Drugs. 2018;78(14):1509–1516.
   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;9(4):300–305.
   PubMed Web of Science ®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;38(2):433–444.
   PubMed 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;4(9):711–720.
   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;21(6):796–807.
   PubMed Web of Science ®Google Scholar
• Zhu AX, Macarulla T, Javle MM, et al. 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;7(11):1669–1677.
   PubMed Web of Science ®Google Scholar
• Lapin M, Huang HJ, Chagani S, et al. Monitoring of dynamic changes and clonal evolution in circulating tumor DNA from patients With IDH -mutated cholangiocarcinoma treated with isocitrate dehydrogenase inhibitors. JCO Precis Oncol. 2022;6(6):e2100197.
   PubMedGoogle Scholar
• Bridgewater J, Palmer D, Cunningham D, et al. Outcome of second-line chemotherapy for biliary tract cancer. Eur J Cancer. 2013;49(6):1511.
   PubMed Web of Science ®Google Scholar
• Saha SK, Parachoniak CA, Ghanta KS, et al. Mutant IDH inhibits HNF-4α to block hepatocyte differentiation and promote biliary cancer. Nature. 2014;513(7516):110–114.
   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 Advances. 2020;4(9):1894–1905.
   PubMed Web of Science ®Google Scholar