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Precision Medicine Approaches in the Management of Cholangiocarcinoma
  • CME
  • CE

Christine Chio, PharmD, BCOP
Lipika Goyal, MD
Caroline Kuhlman, NP
Released: May 13, 2022
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  1. National Comprehensive Cancer Network. Clinical practice guidelines in oncology: hepatobiliary cancers. v.1.2022. nccn.org. Accessed April 26, 2022.
  2. Rizzo A, Brandi G. First-line chemotherapy in advanced biliary tract cancer ten years after the ABC-02 trial: “And Yet It Moves!”. Cancer Treat Res Comm. 2021;27:100335.
  3. Valle J, Wasah H, Palmer DH, et al. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med. 2010;362:1273-1281.
  4. Oh D-Y, 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. Presented at: American Society of Clinical Oncology Gastrointestinal Cancers Symposium 2022; January 20-22, 2022. Abstract 328.
  5. Shroff RT, Javle M, 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;5:824-830.
  6. Park JO, Oh DO, Hsu C, et al. Gemcitabine plus cisplatin for advanced biliary tract cancer: a systematic review. Cancer Res Treat. 2015;47:343-361.
  7. Javle M, Bekaii-Saab T, Jain A, et al. Biliary cancer: Utility of next-generation sequencing for clinical management. Cancer. 2016;133:3838-3847.
  8. Infigratinib [prescribing information]. Brisbane, CA: QED Therapeutics; 2021.
  9. Pemigatinib [prescribing information]. Wilmington, DE: Incyte; 2021.
  10. Ivosidenib [prescribing information]. Boston, MA: Servier Pharmaceuticals; 2021.
  11. Larotrectinib [prescribing information]. Whippany, NJ: Bayer HealthCare; 2021.
  12. Entrectinib [prescribing information]. South San Francisco, CA: Genentech; 2021.
  13. Pembrolizumab [prescribing information]. Whitehouse Station, NJ: Merck; 2022.
  14. Dostarlimab [prescribing information]. Philadelphia, PA: GlaxoSmithKline; 2021.
  15. De Dios I, Ma W, Antzoulatos, et al. FGFR expression, fusion and mutation as detected by NGS sequencing of DNA and RNA. Presented at: 2020 Annual Meeting of the American Society of Clinical Oncology; May 29 - June 2, 2020. Abstract e16061.
  16. Marino FZ, Pagliuca F, Ronchi A, et al. NTRK fusions, from the diagnostic algorithm to innovative treatment in the era of precision medicine. Int J Mol Sci. 2020;21:3718.
  17. 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;21:671-684.
  18. Abou-Alfa GK, Sahai V, Hollebecque A, et al. Pemigatinib for previously treated locally advanced/metastatic cholangiocarcinoma (CCA): Update of FIGHT-202. Presented at: 2021 Annual Meeting of the American Society of Clinical Oncology; June 2-8, 2021. Abstract 4086.
  19. 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;6:803-815.
  20. Javle MM, Roychowdhury S, Kelley RK, et al. Final results from a phase II study of infigratinib (BGJ398), an FGFR-selective tyrosine kinase inhibitor, in patients with previously treated advanced cholangiocarcinoma containing FGFR2 fusions/rearrangements. Presented at: American Society of Clinical Oncology Gastrointestinal Cancers Symposium 2021; January 15-17, 2021. Abstract 265.
  21. Lyou Y, Grivas P, Rosenberg JE, et al. Hyperphosphatemia secondary to the selective fibroblast growth factor receptor 1-3 inhibitor infigratinib (BGJ398) is associated with antitumor efficacy in fibroblast growth factor receptor 3-altered advanced/metastatic urothelial carcinoma. Eur Urol. 2020;78:916-924.
  22. Francis JH, Harding JJ, Schram AM, et al. Clinical and morphologic characteristics of fibroblast growth factor receptor inhibitor-associated retinopathy. JAMA Opthalmol. 2021;139:1126-1130.
  23. Lacouture ME, Sibaud V, Anadcat MJ, et al. Dermatologic adverse events associated with selective fibroblast growth factor receptor inhibitors: overview, prevention, and management guidelines. Oncologist. 2021;26:e316-e326.
  24. Sootome H, Fujita H, Ito K, et al. Futibatinib is a novel irreversible FGFR 1–4 inhibitor that shows selective antitumor activity against FGFR-deregulated tumors. Cancer Res. 2020;80:4986-4997.
  25. Bahleda R, Meric-Bernstam F, Goyal L, et al. Phase I, first-in-human study of futibatinib, a highly selective, irreversible FGFR1–4 inhibitor in patients with advanced solid tumors. Ann Oncol. 2020;31:14051-1412.
  26. Meric-Bernstam F, Bahleda R, Hierro C, et al. Futibatinib, an irreversible FGFR1–4 inhibitor, in patients with advanced solid tumors harboring FGF/FGFR aberrations: a phase I dose-expansion study. Cancer Discov. 2022;12:402-415.
  27. Goyal L, Meric-Bernstam F, Hollebecque A, et al. Primary results of phase 2 FOENIX-CCA2: the irreversible FGFR1-4 inhibitor futibatinib in intrahepatic cholangiocarcinoma (iCCA) with FGFR2 fusions/rearrangements. Presented at: American Association for Cancer Research Annual Meeting 2021; April 10-15, 2021. Abstract CT010.
  28. 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;18:85-98.
  29. Goyal L, Shi L, Liu L, et al. TAS-120 overcomes resistance to ATP-competitive FGFR inhibitors in patients with FGFR2 fusion-positive intrahepatic cholangiocarcinoma. Cancer Discov. 2019;9:1064-1079.
  30. Abou-Alfa GK, Macarulla T, Javle MM, et al. Clinical and morphologic characteristics of fibroblast growth factor receptor inhibitor-associated retinopathy. Lancet Oncol. 2020;21:796-807.
  31. Zhu AX, Macarulla T, Javle MM. 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;7:1669-1677.
  32. Lamarca A, Barriuso J, McNamara MG, et al. Molecular targeted therapies: ready for “prime time” in biliary tract cancer. J Hepatol. 2020;73:170-185.
  33. Subbiah V, Lassen U, Elez 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;21:1234-1243.
  34. Javle M, Borad MJ, Azad NSA, 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;22:1290-1300.
  35. Offin M, Liu D, Drilon A. Tumor-agnostic drug development. Am Soc Clin Oncol Educ Book. 2018;38:184-187.
  36. Marabelle A, Le D, Ascierto PA, et al. Efficacy of pembrolizumab in patients with noncolorectal high microsatellite instability/mismatch repair-deficient cancer: results from the phase II KEYNOTE-158 study. J Clin Oncol. 2020;38:1-10.
  37. Marabelle A, Fakih M, Lopez J, et al. Association of tumour mutational burden with outcomes in patients with advanced solid tumours treated with pembrolizumab: prospective biomarker analysis of the multicohort, open-label, phase 2 KEYNOTE-158 study. Lancet Oncol. 2020;21:1353-1365.
  38. Maio M, Ascierto PA, Manzyuk L, et al. Pembrolizumab in microsatellite instability high (MSI-H)/mismatch repair deficient (dMMR) cancers: Updated analysis from phase 2 KEYNOTE-158 study. Presented at: 2021 Annual Meeting of the American Society of Clinical Oncology; June 2-8, 2021. Abstract 2565.
  39. Berton D, Banerjee SN, Curigliano G, et al. Antitumor activity of dostarlimab in patients with mismatch repair-deficient/microsatellite instability–high tumors: A combined analysis of two cohorts in the GARNET study. Presented at: 2021 Annual Meeting of the American Society of Clinical Oncology; June 2-8, 2021. Abstract 2564.
  40. Demetri GD, De Braud F, Drilon A, et al. Updated integrated analysis of the efficacy and safety of entrectinib in patients with NTRK fusion-positive solid tumors. Clin Cancer Res. 2022;28:1302-1312.
  41. Hong DS, DuBois SG, Kummar S, et al. Larotrectinib in patients with TRK fusion-positive solid tumours: a pooled analysis of three phase 1/2 clinical trials. Lancet Oncol. 2020;21:531-540.
  42. Rizzo A, Ricci AD, Brandi G. Detecting and targeting NTRK gene fusions in cholangiocarcinoma: news and perspectives. Expert Rev Precis Med Drug Develop. 2021;6:225-227.
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