Outcomes of Liver Transplantation for Perihilar Cholangiocarcinoma: New Standard of Care?

Julie Heimbach, MD, Mayo Clinic, Rochester, Minnesota, discusses the findings of an international benchmark study that investigated the outcomes of patients who underwent neoadjuvant chemoradiotherapy followed by liver transplantation for perihilar cholangiocarcinoma.

When compared to patients who underwent a liver transplant for indications other than perihilar cholangiocarcinoma and patients who underwent a complete resection for perihilar cholangiocarcinoma, the population which received neoadjuvant chemoradiation followed by liver transplant had similar or improved postoperative outcomes. These outcomes included 90-day complication rate, length of hospital stay, hepatic artery thrombosis rate, and patient and graft survival.

Dr Heimbach concluded that the data from this study suggests that neoadjuvant chemoradiotherapy followed by liver transplant is an effective treatment strategy and can be performed safely worldwide.

Transcript:

Hello, my name is Julie Heimbach. I am a transplant surgeon at Mayo Clinic in Rochester, Minnesota. I'm also a professor of surgery and the director of the transplant center at Mayo Clinic. Today I'm going to speak to you about a recently published study on outcomes of perihilar cholangiocarcinoma patients who underwent neoadjuvant chemo radiotherapy, followed by liver transplant.

This study was performed across multiple centers, in fact, across 17 different programs around the world; it's an international study, performed from 2014 to 2018. The goal of this was to summarize the experience of liver transplant for patients with perihilar cholangiocarcinoma. This is a tumor that is very challenging to treat, and there has been, for a considerable amount of time in the United States, a protocol that was initially designed at 2 centers. The University of Nebraska and Mayo Clinic both led the way in developing a treatment strategy for these patients, which involves neoadjuvant radiotherapy with chemotherapy delivered prior to transplant. Then, for patients that remain eligible, they could proceed to transplant. This is something that we first pioneered way back in 1996, and we've been optimizing this strategy for a long time.

For a long time, it was considered a contraindication, using transplant for the treatment of perihilar cholangiocarcinoma. But combining it with the neoadjuvant chemoradiotherapy followed by transplant has been associated with good outcomes at our center, and at several other centers in the United States. The significance of this study is that it was looked at across the world, and really these recently published data would suggest that this is an effective treatment strategy and can be performed safely worldwide.

More significant is the finding that the outcome of patients that were transplanted for this indication, which is a really challenging tumor that really has no other treatment strategies, so these are patients with unresectable cancer at the bile duct, who are not eligible for resection or any other curative therapies who are then offered neoadjuvant chemo radiotherapy followed by transplant. Outcomes for these patients were compared to outcomes of patients who are undergoing transplant for other indications, like hepatocellular carcinoma, or other standard indications which are not related to cancer, like primary sclerosis and cholangitis, or hepatitis C or other common indications for liver transplant. It was demonstrated that across all of the parameters, the outcomes were similar. Outcomes including 90-day complication rate, length of hospital stay, other technical complications like hepatic artery thrombosis, and especially patient and graft survival, were quite similar to patients undergoing transplant for other indications. This is definitely a strong endorsement of this therapy overall.

Another important thing this study did was compare outcomes for the patients treated with neoadjuvant chemoradiotherapy and liver transplant for perihilar cholangiocarcinoma to those who were undergoing resection. Those would be patients who had lymph node-negative tumors that were resected with a complete resection. And compared to that group who had resection to those that were thought to have unresectable disease who went to transplant, it was found that the patients who went to transplant had still better outcomes. Even though they started with unresectable disease and did have a transplant, which is a therapy that does require immunosuppression and other significant differences, including a prolonged waiting time from enrollment.

Most notably from this study, it was found that this therapy can be applied around the world and that the outcomes are similar to transplant, and certainly encouraging results for this challenging cancer.

Thank you very much for the opportunity to describe our study.

Source:

Breuer E, Mueller M, Doyle MB, et al. Liver transplantation as a new standard of care in patients with perihilar cholangiocarcinoma? Results from an international benchmark study. Ann Surg. 2022;276(5):846-853. doi:10.1097.SLA.0000000000005641