Biomarker-driven trials double response rates in refractory biliary tract cancers, systematic review finds

Despite recent research advances, Biliary tract cancers (BTCs) remain challenging tumors with persistent difficulties in clinical trial recruitment and achieving positive outcomes. Current standard-of-care, often cytotoxic chemotherapy, has yielded only modest incremental survival benefits over decades. This systematic review aimed to comprehensively analyze the landscape of BTC clinical trials over the past 45 years, specifically evaluating the impact and potential of precision medicine and immunotherapy to address these therapeutic gaps and improve patient outcomes.

Researchers conducted a systematic review and meta-analysis, searching Embase, PubMed, Scopus, and Web of Science for prospective trials on biliary tract cancers (BTCs) over 45 years. Trials were included if they reported specific patient data for BTCs. Data on epidemiological aspects and outcomes were extracted from 427 trials involving 23,024 patients. A meta-analysis of randomized trials specifically compared the efficacy of precision medicine approaches against cytotoxic chemotherapy as a standard comparator, focusing on response rates and overall survival.