Table 2 Research priorities in cholangiocarcinoma
From: Cholangiocarcinoma 2026: status quo, unmet needs and priorities
Number | Research priority | Grade (consensus agreement) |
|---|---|---|
I | Although the current results suggest a protective effect of aspirin and/or statins on CCA development in the general population, none of these should be currently recommended due to the lack of strong evidence, and more research should be conducted | A (93.0%) |
II | The surveillance recommendations of patients with PSC for early CCA detection are suboptimal and may benefit from further research | U (100%) |
III | Strategies aiming to modulate the CCA tumour immune microenvironment deserve further investigations to expand therapeutic targets | U (100%) |
IV | CCA classes identified by molecular and immune characterization have been proposed to be clinically relevant in terms of patient outcomes. These molecular classifications should be clinically investigated in prospective trials | A (95.7%) |
V | Further research is needed to assess if patient-derived advanced 3D culture models (that is, organoids, organ-on-a-chip, precision-cut liver slices, among others) can be used to tailor therapeutic approaches related to specific signalling pathways or altered processes | A (98.6%) |
VI | Highly proliferative CCA cells rely on increased lipid and lipoprotein uptake and metabolization to support their growth. More research is now needed to determine the clinical implications of these findings to evaluate the potential implications of dieting advice and the targeting of lipid metabolism in CCA | A (98.5%) |
VII | International validation studies of biomarkers for CCA are mandatory to translate the preliminary results available from multi-omic studies into clinical practice through evidence-based recommendations. These should include large cohorts of patients with histologically proven diagnoses, appropriate control groups (for example, cirrhotic, non-cirrhotic, HBV, HCV, MASLD), and further prospective validation in the setting of clinical trials | A (98.6%) |
VIII | Further research on biomarkers for systemic therapy response is needed | U (100%) |
IX | The role of liquid biopsy to monitor tumour evolution during treatment should be investigated in prospective studies | A (98.6%) |
X | Predictive biomarkers of treatment response to immunotherapy are highly needed | A (99.3%) |
XI | Consensus on radiological criteria for resectability of CCA is needed | A (97.8%) |
XII | Further clinical investigation is needed to identify the role of local therapy in CCA and the appropriate combination with systemic treatments | U (100%) |
XIII | Understanding the effect of chemotherapy and immunotherapy in CCAs with specific actionable genetic alterations is important and may impact clinical decisions | A (97.9%) |
XIV | The impact of systemic treatment according to the location of the CCA should be assessed in the future | A (98.6%) |
XV | Clinical investigation of the role of neoadjuvant systemic treatment in non-metastatic CCA is needed | U (100%) |
XVI | Serum CA19-9 and CEA levels should be used as comparators for the development of novel biomarkers for the early diagnosis of CCA | B (83.3%) |
XVII | Collection of biological samples should be pursued within clinical trials and clinical practice to improve understanding of the disease and the impact of therapies, and ultimately improve patient outcomes | U (100%) |
XVIII | Investigation of the role of targeted therapies in first-line should be assessed in patients with advanced (unresectable or metastatic) disease | A (99.3%) |
