علاج سرطان القناة الصفراوية خارج الكبد غير القابل للاستئصال أو المنتشر مع وجود تغيير جزيئي قابل للاستهداف

References

DOI: 10.1016/j.jhep.2025.03.007

• Gemcitabine and cisplatin in combination with either durvalumab or pembrolizumab should be considered standard of care for the first-line systemic treatment of patients with unresectable or metastatic eCCA (LoE 2, strong recommendation, consensus).
• Patients with unresectable or metastatic eCCA should receive molecular profiling to identify and therapeutically address actionable alterations and to support inclusion into clinical trials (LoE 2, strong recommendation, strong consensus).
• Currently, FDA and EMA approvals exist for futibatinib and pemigatinib in patients with FGFR2 fusion/rearrangements, and, based on phase III data, for ivosidenib in IDH1 R132 mutant biliary tract cancer.
• A tumour-agnostic FDA approval exists for the combination of dabrafenib and trametinib in patients with solid tumours harbouring a BRAF^V600E mutation, and who have progressed following prior treatment.
• For HER2 positive (IHC 3+) biliary cancers, the bispecific antibody zanidatamab has received FDA approval for patients with previously treated, unresectable or metastatic disease.
• Further, the antibody-drug conjugate trastuzumab-deruxtecan was granted tumour-agnostic FDA approval in adult patients with unresectable or metastatic HER2-positive (IHC 3+) solid tumours who have received prior systemic treatment.
• Entity-independent EMA and FDA approvals exist for NTRK-fusion positive tumours (entrectinib and larotrectinib).
• Repotrectinib has been approved by the FDA and EMA for tyrosine kinase inhibitor-naïve and -pretreated solid tumours harbouring NTRK gene fusions, and a positive CHMP opinion (EMA) was issued in November 2024 for this indication.
• In patients with advanced RET fusion-positive solid tumours, selpercatinib was approved by the FDA and EMA.