Long-term Outcomes of Protocol-based Neoadjuvant Selection and Liver Transplantation Versus Liver Resection for Perihilar Cholangiocarcinoma: A Systematic Review and Meta-analysis
Clinical Snapshot
PICO Framework
| P — Population | Patients with perihilar cholangiocarcinoma (Klatskin tumours) |
| I — Intervention | Protocol-based neoadjuvant selection and liver transplantation |
| C — Comparator | Liver resection |
| O — Outcomes | 5-year overall survival, 5-year disease-free survival, 3-year disease-free survival, R0 resection rate, postoperative mortality |
Bottom Line
This meta-analysis of 768 patients suggests liver transplantation may offer superior long-term survival compared to liver resection for perihilar cholangiocarcinoma. However, the evidence quality is compromised by significant selection bias, retrospective study designs, and potential outcome reporting bias. The apparent survival benefit must be interpreted cautiously, particularly given the inherent selection of healthier patients for transplantation and the risk of post-transplant rather than intention-to-treat outcome reporting. While these findings support the continued evaluation of liver transplantation in highly selected patients, they are insufficient to definitively change practice. Clinicians should continue individualised decision-making within established multidisciplinary frameworks, considering patient factors, tumour characteristics, and institutional expertise. Prospective studies with intention-to-treat analysis are needed to provide more robust evidence for clinical decision-making in this challenging malignancy.
Key Findings
P Value: Significance reported but specific values not provided
Effect Size: Not reported in abstract
Primary Outcome: 5-year overall survival favoured liver transplantation
Nnt Or Sensitivity: Higher R0 resection rate with liver transplantation
Confidence Interval: Not reported
Clinical Application
Limited by organ availability, transplant centre expertise, and stringent selection criteria Relevant to Australian liver transplant centres; would require consideration of organ allocation policies and multidisciplinary team protocols aligned with ANZLTS guidelines Highly selected patients with perihilar cholangiocarcinoma suitable for either liver transplantation or resection
Abstract
BACKGROUND: Perihilar cholangiocarcinoma (CCA) is an aggressive malignancy that presents significant challenges in terms of diagnosis and treatment owing to its anatomical location. Liver resection and transplantation are the primary curative options; however, their long-term outcomes remain controversial. This study aimed to compare the long-term outcomes of protocol-based neoadjuvant selection and liver transplantation with liver resection for the management of perihilar cholangiocarcinoma. METHODS: A systematic review and meta-analysis were conducted according to the PRISMA guidelines. Electronic databases, including the Cochrane Library, PubMed/MEDLINE, and Embase, were searched for relevant clinical trials comparing liver transplantation with liver resection. RESULTS: Six studies comprising 768 patients (265 in the liver transplantation group and 503 in the liver resection group) were included. Liver transplantation was associated with significantly higher 5-year overall survival and 5-year disease-free survival. They also showed a higher 3-year disease-free survival rate. Patients in the liver transplantation group had a higher R0 resection rate. There were no significant differences in the 3- or 30-day postoperative mortality between the two groups. CONCLUSION: Liver transplantation was associated with improved long-term survival in highly selected patients; however, these findings are limited by significant selection bias and the retrospective study design. The apparent long-term benefit observed with LT must be interpreted cautiously because transplant series frequently report post-transplant rather than intention-to-treat outcomes.
References
- 1.Chaouch, M. A., Bigourdan, J. M., Tzedakis, S., da Costa, A. C., Reissfelder, C., Fattal, W., Oweira, H., & Jeddou, H. (2026). Long-term outcomes of protocol-based neoadjuvant selection and liver transplantation versus liver resection for perihilar cholangiocarcinoma: A systematic review and meta-analysis. Journal of Gastrointestinal Cancer. https://doi.org/10.1245/s10434-020-09001-8
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