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Unmet needs in CCA — 1 of 3
Diagnosis of CCA presents significant clinical challenges
CCA is usually asymptomatic in its early stages, often resulting in late diagnosis when patients are already at an advanced stage7,10
20–25%
of iCCA cases are diagnosed because of an incidental finding, owing to non-specific symptoms7,11
90%
of patients with eCCA present with painless jaundice; 10% present with acute cholangitis5,10
Lack of absolute diagnostic criteria and low specificity of diagnostic modalities make diagnosis of CCA difficult7,8,10
What are the symptoms of advanced CCA?
Symptoms of advanced CCA7,10,11
- Asthaenia
- Abdominal pain
- Malaise
- Nausea
- Anorexia/weight loss
- Jaundice
Date of preparation: February 2022
References
- PEMAZYRE® (pemigatinib) Swissmedic Professional Information, June 2021; https://www.swissmedicinfo.ch/ (accessed 02 December 2021).
- Rizvi S and Borad MJ. The rise of the FGFR inhibitor in advanced biliary cancer: the next cover of Time magazine? J Gastrointest Oncol. 2016;7:789–96.
- Ghouri YA, et al. Cancer review: Cholangiocarcinoma. J Carcinog. 2015;14:1.
- Sharma P and Yadav S. Demographics, tumor characteristics, treatment, and survival of patients with Klatskin tumors. Ann Gastroenterol. 2018;31:231–6.
- Blechacz B. Cholangiocarcinoma: Current knowledge and new developments. Gut Liver. 2017;11:13–26.
- Valle JW, et al. New horizons for precision medicine in biliary tract cancers. Cancer Discov. 2017;7:943–62.
- Bañales JM, et al. Cholangiocarcinoma 2020: The next horizon in mechanisms and management. Nat Rev Gastroenterol Hepatol. 2020;17:557–88.
- Bañales JM, et al. Expert consensus document: Cholangiocarcinoma: Current knowledge and future perspectives consensus statement from the European Network for the Study of Cholangiocarcinoma (ENS-CCA). Nat Rev Gastroenterol Hepatol. 2016;13:261–80.
- Bertuccio P, et al. A comparison of trends in mortality from primary liver cancer and intrahepatic cholangiocarcinoma in Europe. Ann Oncol. 2013;24:1667–74.
- Blechacz B, et al. Clinical diagnosis and staging of cholangiocarcinoma. Nat Rev Gastroenterol Hepatol. 2011;8:512–22.
- Forner A, et al. Clinical presentation, diagnosis and staging of cholangiocarcinoma. Liver Int. 2019;39(suppl 1):98–107.
- Valle JW, et al. Biliary cancer: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2016;27(suppl 5):v28–v37.
- Lamarca A, et al. Advanced intrahepatic cholangiocarcinoma: Post hoc analysis of the ABC-01, -02, and -03 clinical trials. J Natl Cancer Inst. 2020;112:200–10.
- Valle JW, et al. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med. 2010;362:1273–81.
- Lamarca A, 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;22:690–701.
- Lowery MA, et al. Comprehensive molecular profiling of intrahepatic and extrahepatic cholangiocarcinomas: potential targets for intervention. Clin Cancer Res. 2018;24:4154–61.
- Jain A, et al. Cholangiocarcinoma with FGFR genetic aberrations: A unique clinical phenotype. JCO Precis Oncol. 2018;2:1–12.
- Ross JS, et al. New routes to targeted therapy of intrahepatic cholangiocarcinomas revealed by next-generation sequencing. Oncologist. 2014;19:235–42.
- Farshidfar F, et al. Integrative genomic analysis of cholangiocarcinoma identifies distinct IDH-mutant molecular profiles. Cell Rep. 2017;18:2780–94.
- Graham RP, et al. Fibroblast growth factor receptor 2 translocations in intrahepatic cholangiocarcinoma. Hum Pathol. 2014;45:1630–8.
- Churi CR, et al. Mutation profiling in cholangiocarcinoma: Prognostic and therapeutic implications. PLoS One. 2014;9:e115383.
- Arai Y, et al. Fibroblast growth factor receptor 2 tyrosine kinase fusions define a unique molecular subtype of cholangiocarcinoma. Hepatology. 2014;59:1427–34.
- Borad MJ, et al. Fibroblast growth factor receptor 2 fusions as a target for treating cholangiocarcinoma. Curr Opin Gastroenterol. 2015;31:264–68.
- Silverman IM, et al. Clinicogenomic analysis of FGFR2-rearranged cholangiocarcinoma identifies correlates of response and mechanisms of resistance to pemigatinib. Cancer Discov. 2021;11:326–39.
- Barr FG. Fusion genes in solid tumors: The possibilities and the pitfalls. Expert Rev Mol Diagn. 2016;16:921–3.
- Mosele F, et al. Recommendations for the use of next-generation sequencing (NGS) for patients with metastatic cancers: A report from the ESMO Precision Medicine Working Group. Ann Oncol. 2020;31:1491–505.
- Liu PCC, et al. INCB054828 (pemigatinib), a potent and selective inhibitor of fibroblast growth factor receptors 1, 2, and 3, displays activity against genetically defined tumor models. PLoS One. 2020;15:e0231877.
- Abou-Alfa GK, et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: A multicentre, open-label, phase 2 study. Lancet Oncol. 2020;21:671–84.
All references are available upon request
Abbreviations
Abbreviated Prescribing Information
▼ This medicinal product is subject to additional monitorings. For further information, see product information Pemazyre on www.swissmedicinfo.ch.
PEMAZYRE (pemigatinib), tablets of 13.5 mg, 9 mg, 4.5 mg. I: : As monotherapy for the treatment of adults with locally advanced, unresectable or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one line of systemic therapy. P: Treatment should be initiated by a physician experienced in the diagnosis and treatment of patients with biliary tract cancer. FGFR2 fusion positivity status must be confirmed prior to initiation of therapy. Pemazyre 13.5 mg is taken orally once per day for 14 days, followed by 7 days off therapy. For dose adjustments due to adverse reactions, see www.swissmedicinfo.ch.For patients with severe hepatic or renal impairment, the dose should be reduced to the next lower strength. CI: : Hypersensitivity to the active substance or to any of the excipients. Concomitant use with St John’s wort. W/P: Pemazyre is associated with hyper- and hypophosphatemia, serous retinal detachment, dry eye and blood creatinine increase. Pemazyre can cause fetal harm. Women of childbearing potential should be advised not to become pregnant and men not to father a child during treatment. IA: Concomitant administration with proton pump inhibitors, strong CYP3A4 inhibitors or inducers should be avoided. Administration should be separated by at least 6 hours before or after administration of P-gp substrates with a narrow therapeutic index. Close clinical surveillance is recommended when co-administered with CYP2B6 substrates. UE: The most common adverse reactions (≥ 30%) were hyperphosphatemia, alopecia, diarrhea, nail toxicity, fatigue, nausea, dysgeusia, stomatitis, constipation and dry mouth. The most common serious adverse reactions were hyponatremia and increased blood creatinine. For further information on UE, see www.swissmedicinfo.ch. Dispensing cat.: A. Revision date: June 2021. Marketing authorisation holder: Incyte Biosciences International Sàrl, CH-1110 Morges. Refer to www.swissmedicinfo.ch for detailed information.Resources and support
For further information, please refer to the PEMAZYRE Swissmedic Professional Information.1
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