Mayo Clinic Study Assessed Impact of Neoadjuvant Chemoradiation in Patients with Unresectable Perihilar CCA

2022 Year in Review: Cholangiocarcinoma — January 4, 2023

The Mayo Clinic protocol is the standard to determine eligibility for liver transplantation and is based on strict selection criteria that include neoadjuvant chemoradiation before patients are eligible for liver transplantation. A multicenter retrospective study assessed whether survival outcomes associated with the Mayo Clinic protocol in patients with unresectable perihilar cholangiocarcinoma (CCA) were “truly due to the strict selection or due to the neoadjuvant chemoradiation,” per Flavio Rocha, MD, FACS, FSSP, Physician-in-Chief of the Knight Cancer Institute. Results from this study were shared at the 2022 International Hepato-Pancreato-Biliary Association World Congress.

In this international study, 2 centers in the Netherlands and France reviewed charts of 49 patients with unresectable perihilar CCA between 2011 and 2021 who were assessed with the Mayo Clinic selection criteria and stratified by neoadjuvant chemoradiation. The primary end point was overall survival; secondary end points included post-transplant morbidity and vascular complications.

Of the 49 patients who underwent liver transplantation, 27 received chemoradiation and 22 did not; median age was 55 years. In the cohort that received neoadjuvant chemoradiation (median follow-up of 25 months), 1-year survival rate was 65%, 3-year survival rate was 51%, and 5-year survival rate was 41%; the 1-year, 3-year, and 5-year survival rates of the cohort who had not received neoadjuvant chemoradiation were 91%, 68%, and 53%, respectively. The tumor 5-year recurrence rate was similar between the groups, 32% in the neoadjuvant chemoradiation cohort versus 53% in the cohort that did not receive chemoradiation. However, major vascular complications (defined as Clavien-Dindo >IIIa) were significantly higher in the chemoradiation group (41% vs 14%).

In summary, Dr Rocha stated, “This was an interesting analysis that looked at survival after liver transplantation in a very highly selected patient cohort. They found that there was no difference in survival between patients who had received neoadjuvant chemoradiation and those who had not; however, there was an increased rate of vascular complications in those that did have chemoradiation.”

He also noted, “The Mayo Clinic protocol has taken the test of time, and the chemoradiation piece does have significant treatment effect, with some folks even suggesting that the transplant is actually adjuvant therapy for chemoradiation. Although it is a hypothesis-generating approach, it is certainly food for thought, as we continue to evolve these transplant protocols on omitting chemoradiation.”

Sources: Hoogwater FJH, Kuipers H, de Meijer VE, et al. Liver transplantation for unresectable perihilar cholangiocarcinoma: is neo-adjuvant chemo-radiation therapy really necessary? An international, multicenter comparison. International Hepato-Pancreato-Biliary Association World Congress 2022. Abstract FP16-4.

Van Keulen A-M, Olthof PB, Cescon M, et al. Actual 10-year survival after resection of perihilar cholangiocarcinoma: what factors preclude a chance for cure? Cancers (Basel). 2021;13:6260.

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