Experts’ position on durvalumab treatment for cholangiocarcinoma patients
Abstract
In the Polish population, biliary tract cancers account for about 1% of the incidence and 2.5% of deaths due to all cancers, with 85% of all cases diagnosed in patients over the age of 60. Biliary tract cancer is a neoplasm that originates from intrahepatic or extrahepatic biliary epithelial cells and shows features of cholangiocyte differentiation. It can also develop from perihilar glands and hepatocytes. It is characterized by a poor prognosis, while the use of exclusive chemotherapy in the treatment leads to a slight improvement in the treatment effect. The paper presents current guidelines for imaging and endoscopic diagnosis in patients with biliary tract cancer and summarizes data on the efficacy and safety of durvalumab from clinical trials as well as everyday clinical practice.
Keywords: cholangiocrcinomaimmunotherapydurvalumab
References
- Razumilava N, Gores GJ. Cholangiocarcinoma. Lancet. 2014; 383(9935): 2168–2179.
- Zhu Y, Kwong LN. Insights Into the Origin of Intrahepatic Cholangiocarcinoma From Mouse Models. Hepatology. 2020; 72(1): 305–314.
- Kendall T, Verheij J, Gaudio E, et al. Anatomical, histomorphological and molecular classification of cholangiocarcinoma. Liver Int. 2019; 39 Suppl 1: 7–18.
- Fabris L, Perugorria MJ, Mertens J, et al. The tumour microenvironment and immune milieu of cholangiocarcinoma. Liver Int. 2019; 39 Suppl 1(Suppl 1): 63–78.
- Blechacz B, Komuta M, Roskams T, et al. Clinical diagnosis and staging of cholangiocarcinoma. Nat Rev Gastroenterol Hepatol. 2011; 8(9): 512–522.
- Ilyas SI, Khan SA, Hallemeier CL, et al. Cholangiocarcinoma - evolving concepts and therapeutic strategies. Nat Rev Clin Oncol. 2018; 15(2): 95–111.
- Khan SA, Tavolari S, Brandi G. Cholangiocarcinoma: Epidemiology and risk factors. Liver Int. 2019; 39 Suppl 1: 19–31.
- Paul A, Kaiser GM, Molmenti EP, et al. Klatskin tumors and the accuracy of the Bismuth-Corlette classification. Am Surg. 2011; 77(12): 1695–1699.
- Nakeeb A, Pitt HA, Sohn TA, et al. Cholangiocarcinoma. A spectrum of intrahepatic, perihilar, and distal tumors. Ann Surg. 1996; 224(4): 463–73; discussion 473.
- Izquierdo-Sanchez L, Lamarca A, La Casta A, et al. Cholangiocarcinoma landscape in Europe: Diagnostic, prognostic and therapeutic insights from the ENSCCA Registry. J Hepatol. 2022; 76(5): 1109–1121.
- Brindley PJ, Bachini M, Ilyas SI, et al. Cholangiocarcinoma. Nat Rev Dis Primers. 2021; 7(1): 65.
- Didkowska J, Wojciechowska U, Michalek IM, et al. Cancer incidence and mortality in Poland in 2019. Sci Rep. 2022; 12(1): 10875.
- Didkowska J, Barańska K, Miklewska M, et al. Rak dróg żółciowych w Polsce – obraz epidemiologiczny wraz z analizą postępowania terapeutycznego w latach 2016-2020. 2023.
- Banales JM, Marin JJG, Lamarca A, et al. Cholangiocarcinoma 2020: the next horizon in mechanisms and management. Nat Rev Gastroenterol Hepatol. 2020; 17(9): 557–588.
- Pericleous M, Khan SA. Epidemiology of HPB malignancy in the elderly. Eur J Surg Oncol. 2021; 47(3 Pt A): 503–513.
- Khan S, Thomas H, Davidson B, et al. Cholangiocarcinoma. The Lancet. 2005; 366(9493): 1303–1314.
- Rushbrook SM, Kendall TJ, Zen Y, et al. British Society of Gastroenterology guidelines for the diagnosis and management of cholangiocarcinoma. Gut. 2023; 73(1): 16–46.
- Saluja SS, Sharma R, Pal S, et al. Differentiation between benign and malignant hilar obstructions using laboratory and radiological investigations: a prospective study. HPB (Oxford). 2007; 9(5): 373–382.
- Trikudanathan G, Navaneethan U, Njei B, et al. Diagnostic yield of bile duct brushings for cholangiocarcinoma in primary sclerosing cholangitis: a systematic review and meta-analysis. Gastrointest Endosc. 2014; 79(5): 783–789.
- Ni Q, Wang H, Zhang Y, et al. MDCT assessment of resectability in hilar cholangiocarcinoma. Abdom Radiol (NY). 2017; 42(3): 851–860.
- Joo I, Lee JM. Imaging bile duct tumors: pathologic concepts, classification, and early tumor detection. Abdom Imaging. 2013; 38(6): 1334–1350.
- Lamarca A, Barriuso J, Chander A, et al. 18F-fluorodeoxyglucose positron emission tomography (18FDG-PET) for patients with biliary tract cancer: Systematic review and meta-analysis. J Hepatol. 2019; 71(1): 115–129.
- Ruys AT, van Beem BE, Engelbrecht MRW, et al. Radiological staging in patients with hilar cholangiocarcinoma: a systematic review and meta-analysis. Br J Radiol. 2012; 85(1017): 1255–1262.
- Huang X, Yang J, Li J, et al. Comparison of magnetic resonance imaging and 18-fludeoxyglucose positron emission tomography/computed tomography in the diagnostic accuracy of staging in patients with cholangiocarcinoma: A meta-analysis. Medicine (Baltimore). 2020; 99(35): e20932.
- Shimada K, Isoda H, Hirokawa Y, et al. Comparison of gadolinium-EOB-DTPA-enhanced and diffusion-weighted liver MRI for detection of small hepatic metastases. Eur Radiol. 2010; 20(11): 2690–2698.
- Koc Z, Erbay G. Optimal b value in diffusion-weighted imaging for differentiation of abdominal lesions. J Magn Reson Imaging. 2014; 40(3): 559–566.
- Franken LC, Coelen RJS, Erdmann JI, et al. Multidetector computed tomography assessment of vascular involvement in perihilar cholangiocarcinoma. Quant Imaging Med Surg. 2021; 11(11): 4514–4521.
- Francica G, Meloni MF, de Sio I, et al. Biopsy of Liver Target Lesions under Contrast-Enhanced Ultrasound Guidance - A Multi-Center Study. Ultraschall Med. 2018; 39(4): 448–453.
- Novikov A, Kowalski TE, Loren DE. Practical Management of Indeterminate Biliary Strictures. Gastrointest Endosc Clin N Am. 2019; 29(2): 205–214.
- Yoon SB, Moon SH, Ko SW, et al. Brush Cytology, Forceps Biopsy, or Endoscopic Ultrasound-Guided Sampling for Diagnosis of Bile Duct Cancer: A Meta-Analysis. Dig Dis Sci. 2022; 67(7): 3284–3297.
- Zhai J. UroVysion Multi-Target Fluorescence in situ Hybridization Assay for the Detection of Malignant Bile Duct Brushing Specimens: A Comparison with Routine Cytology. Acta Cytol. 2018; 62(4): 295–301.
- Naitoh I, Nakazawa T, Kato A, et al. Predictive factors for positive diagnosis of malignant biliary strictures by transpapillary brush cytology and forceps biopsy. J Dig Dis. 2016; 17(1): 44–51.
- Sun Xi, Zhou Z, Tian J, et al. Is single-operator peroral cholangioscopy a useful tool for the diagnosis of indeterminate biliary lesion? A systematic review and meta-analysis. Gastrointest Endosc. 2015; 82(1): 79–87.
- Navaneethan U, Hasan MK, Lourdusamy V, et al. Single-operator cholangioscopy and targeted biopsies in the diagnosis of indeterminate biliary strictures: a systematic review. Gastrointest Endosc. 2015; 82(4): 608–14.e2.
- Arechederra M, Rullán M, Amat I, et al. Next-generation sequencing of bile cell-free DNA for the early detection of patients with malignant biliary strictures. Gut. 2022; 71(6): 1141–1151.
- Patel AH, Harnois DM, Klee GG, et al. The utility of CA 19-9 in the diagnoses of cholangiocarcinoma in patients without primary sclerosing cholangitis. Am J Gastroenterol. 2000; 95(1): 204–207.
- Valle JW, Borbath I, Khan SA, et al. ESMO Guidelines Committee. Biliary cancer: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2016; 27(suppl 5): v28–v37.
- Lamarca A, Palmer DH, Wasan HS, et al. Advanced Biliary Cancer Working Group, ABC-02 Trial Investigators. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med. 2010; 362(14): 1273–1281.
- Sharma A, Kalyan Mohanti B, Pal Chaudhary S, et al. Modified gemcitabine and oxaliplatin or gemcitabine + cisplatin in unresectable gallbladder cancer: Results of a phase III randomised controlled trial. Eur J Cancer. 2019; 123: 162–170.
- Oh DY, Ruth He A, Qin S, et al. Durvalumab plus Gemcitabine and Cisplatin in Advanced Biliary Tract Cancer. NEJM Evid. 2022; 1(8): EVIDoa2200015.
- AstraZeneca AB. IMFINZI (durvalumab): EU summary of prod- uct characteristics. 2024. https://www.ema.europa.eu/.
- Oh DY, He A, Bouattour M, et al. Durvalumab or placebo plus gemcitabine and cisplatin in participants with advanced biliary tract cancer (TOPAZ-1): updated overall survival from a randomised phase 3 study. Lancet Gastroenterol Hepatol. 2024; 9(8): 694–704.
- Burris HA, Okusaka T, Vogel A, et al. Durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer (TOPAZ-1): patient-reported outcomes from a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Oncol. 2024; 25(5): 626–635.
- Di Maio M, Perrone F, Conte P. Real-World Evidence in Oncology: Opportunities and Limitations. Oncologist. 2020; 25(5): e746–e752.
- Seung SJ, Saherawala H, Syed I, et al. Real-world treatment patterns and survival outcomes for treated biliary tract cancer patients using administrative databases in Ontario. J Gastrointest Oncol. 2023; 14(4): 1806–1816.
- Rimini M, Fornaro L, Lonardi S, et al. Durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer: An early exploratory analysis of real‐world data. Liver International. 2023; 43(8): 1803–1812.
- Rimini M, Masi G, Lonardi S, et al. Durvalumab Plus Gemcitabine and Cisplatin Versus Gemcitabine and Cisplatin in Biliary Tract Cancer: a Real-World Retrospective, Multicenter Study. Target Oncol. 2024; 19(3): 359–370.
- Rimini M, Fornaro L, Rizzato MD, et al. Durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer: A large real-life worldwide population. Eur J Cancer. 2024; 208: 114199.
- Obwieszczenie Ministra Zdrowia z dnia 18 września 2024 r. w sprawie wykazu refundowanych leków, środków spożywczych specjalnego przeznaczenia żywieniowego oraz wyrobów medycznych na 1 października 2024 r. Program Lekowy B.5. https://www.gov.pl/web/zdrowie/obwieszczenia-ministra-zdrowia-lista-lekow-refundowanych.