Liver transplantation for perihilar cholangiocarcinoma: patient selection and outcomes

ABSTRACT

Introduction: Peri-hilar cholangiocarcinoma is an aggressive bile duct cancer. Long-term survival is possible with margin-negative surgery. Historically, unresectable disease was approached with non-curative treatment options. In recent decades, an innovative approach of neoadjuvant chemoradiation and liver transplantation has demonstrated long-term survival for highly selected patients.

Areas covered: This is a critical analysis of studies published to date on neoadjuvant chemoradiation and liver transplantation for selected patients with peri-hilar cholangiocarcinoma. A PubMed literature search was conducted for years 1970–2020 with the following search criteria: [‘hilar’ OR ‘peri-hilar’ AND ‘cholangiocarcinoma’] AND [‘treatment’ OR ‘transplantation’ OR ‘survival’ OR ‘outcome’]; ‘neoadjuvant chemoradiation’ AND ‘unresectable cholangiocarcinoma’. All peer-reviewed original research studies were selected for review.

Expert opinion: Neoadjuvant chemoradiation and liver transplantation for patients with early stage unresectable peri-hilar cholangiocarcinoma can achieve long-term survival in highly selected patients who survive to transplantation without disease progression. There are observed differences in survival for patients with PSC-associated versus de novo cholangiocarcinoma and transplanted versus resected patients; however, these differences are not contextualized by established tumor and patient factors that influence recurrence and survival. Therefore, these results must be interpreted within the limitations of the study designs upon which they are based.

KEYWORDS:

• Cholangiocarcinoma
• hilar cholangiocarcinoma
• liver transplantation
• Mayo protocol
• peri-hilar cholangiocarcinoma

Article highlights

• Peri-hilar cholangiocarcinoma is a rare but aggressive bile duct cancer

• In resectable patients, long-term survival is achievable with margin-negative surgery

• Locally unresectable disease has historically been treated with non-curative approaches including radiation and/or chemotherapy

• Neoadjuvant chemoradiation followed by orthotopic liver transplantation is a novel treatment approach that can achieve long-term survival in patients with early stage unresectable peri-hilar cholangiocarcinoma. However, long-term survival and appropriate use of donor organs is contingent upon strict adherence to the patient selection criteria inherent in this approach.

• Although there are observed survival differences between PSC-associated and de novo peri-hilar cholangiocarcinoma, studies demonstrating these results are heavily influenced by selection bias.

• Neoadjuvant chemoradiation and liver transplantation have the potential to achieve long-term survival in highly selected patients with early stage locally unresectable disease who survive to transplantation without disease progression or medical decline.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.