Pan-Asian adapted ESMO Clinical Practice Guidelines for the diagnosis, treatment and follow-up of patients with biliary tract cancer

Abstract

The European Society for Medical Oncology (ESMO) Clinical Practice Guidelines for the diagnosis, treatment and follow-up of patients with biliary tract cancer (BTC), published in late 2022 were adapted in December 2023, according to established standard methodology, to produce the Pan-Asian adapted (PAGA) ESMO consensus guidelines for the management of Asian patients with BTC. The adapted guidelines presented in this manuscript represent the consensus opinions reached by a panel of Asian experts in the treatment of patients with BTC representing the oncological societies of China (CSCO), Indonesia (ISHMO), India (ISMPO), Japan (JSMO), Korea (KSMO), Malaysia (MOS), the Philippines (PSMO), Singapore (SSO), Taiwan (TOS) and Thailand (TSCO), co-ordinated by ESMO and the Taiwan Oncology Society (TOS). The voting was based on scientific evidence and was independent of the current treatment practices, drug access restrictions and reimbursement decisions in the different regions of Asia. Drug access and reimbursement in the different regions of Asia are discussed separately in the manuscript. The aim is to provide guidance for the optimisation and harmonisation of the management of patients with BTC across the different countries and regions of Asia, drawing on the evidence provided by both Western and Asian trials, whilst respecting the differences in screening practices and molecular profiling, as well as age and stage at presentation. Attention is drawn to the disparity in the drug approvals and reimbursement strategies, between the different countries.

Keywords: ESMO; Pan-Asian; biliary tract cancer; guidelines; treatment.

Figure 1

Algorithm for the treatment of biliary tract cancer. Purple boxes: general categories or stratification; red boxes: surgery; white boxes: other aspects of management; blue boxes: systemic anticancer therapy; dashed lines: optional recommendation. ChT, chemotherapy; dCCA, distal cholangiocarcinoma; EMA, European Medicines Agency; FDA, US Food and Drug Administration; FGFR2, fibroblast growth factor receptor 2; GBC, gallbladder cancer; HER2, human epidermal growth factor receptor 2; iCCA, intrahepatic cholangiocarcinoma; IDH1, isocitrate dehydrogenase 1; MCBS, ESMO-Magnitude of Clinical Benefit Scale; MDT, multidisciplinary team; NTRK, neurotrophic tyrosine receptor kinase; pCCA, perihilar cholangiocarcinoma; PS, performance status; RFA, radiofrequency ablation; S-1, tegafur, gimeracil and oteracil; SBRT, stereotactic body radiotherapy; RET, rearranged during transfection. ^a Special considerations: (i) consider the need for preoperative drainage; (ii) avoid percutaneous biopsy in resectable d/pCCA; (iii) assess future liver remnant; (iv) neoadjuvant approach (selected cases); (v) completion surgery for incidental GBC stage _T1b. ^b Salvage surgery or local therapies should be considered in responding patients with initially inoperable disease. ^c Clinical trial recommended when available. ^d Molecular profiling should be carried out before/during first-line therapy. Gene panel should include FGFR2, IDH1, HER2/neu and BRAF to test for hotspot mutations, but may also include genes such as NTRK and c-MET. The rapidly evolving landscape of drug targets and predictive biomarkers may necessitate larger panels in the future. ^e Cisplatin-gemcitabine-durvalumab [I, A], cisplatin-gemcitabine-pembrolizumab [I,A] and cisplatin-gemcitabine-S-1 [II, A] are recommended for first-line treatment. Consider gemcitabine monotherapy in patients with a compromised PS or significant debility who are at risk of toxicity from platinum-containing ChT regimens; oxaliplatin or carboplatin can replace cisplatin in the presence of renal insufficiency or ototoxicity; gemcitabine plus S-1 can be considered for patients with or susceptible to peripheral sensory neuropathy. ^f EMA and FDA approved. ^g ESMO-MCBS v1.1³⁶ was used to calculate scores for therapies/indications approved by the EMA or FDA. The scores have been calculated by the ESMO-MCBS Working Group and validated by the ESMO Guidelines Committee (https://www.esmo.org/guidelines/esmo-mcbs/esmo-mcbs-evaluation-forms). ^h Not FDA or EMA approved. (i) Reconsider surgery in the event of adequate response to treatment.

Figure 2

Algorithm for the second- and later-line treatment of biliary tract cancer. Burgundy: general categories or stratification; blue: systemic anticancer therapy. BTC, biliary tract cancer; dMMR, mismatch repair deficiency; EMA, European Medicines Agency; ESCAT, ESMO Scale for Clinical Actionability of Molecular Targets; FDA, Food and Drug Administration; FGFR2, fibroblast growth factor receptor 2; FOLFOX, 5-fluorouracileleucovorineoxaliplatin; HER2, human epidermal growth factor receptor 2; IDH1, isocitrate dehydrogenase 1; MCBS, ESMO-Magnitude of Clinical Benefit Scale; MSI-H, microsatellite instability-high; NTRK, neurotrophic tyrosine receptor kinase; PARP, poly (ADP-ribose) polymerase; PD-1, programmed cell death protein 1; RET, rearranged during transfection; TMB-H, tumour mutational burden-high. ^a ESCAT scores apply to alterations from genomic-driven analyses only. These scores have been defined by the authors of the ESMO biliary tract guidelines and validated by the ESMO Translational Research and Precision Medicine Working Group.^,b ESMO-MCBS v1.1³⁶ was used to calculate scores for therapies/indications approved by the EMA or FDA. The scores have been calculated by the ESMO-MCBS Working Group and validated by the ESMO Guidelines Committee (https://www.esmo.org/guidelines/esmo-mcbs/esmo-mcbs-evaluation-forms). ^c Anti-PD-1 therapy is recommended for patients with MSI-H/dMMR who have not been treated with first-line immunotherapy or for patients with unresectable or metastatic TMB-H (≥10 mutations/megabase) solid tumours that have progressed following prior treatment. ^d EMA approved for MSI-H/dMMR BTC; FDA approved for all MSI-H/dMMR solid tumours. ^e FDA approved; not EMA approved. ^f EMA and FDA approved. ^g Not EMA approved; not FDA approved. ^h Selpercatinib is recommended for adult patients with locally advanced or metastatic tumours with a RET fusion that have progressed following prior treatment. ⁱ Clinical trial recommended when available.