Multimodal treatment and tumour biology-driven long-term survival in PSC-associated hilar cholangiocarcinoma: A case report.

Abstract

Primary sclerosing cholangitis (PSC) is a recognised risk factor for hilar cholangiocarcinoma (hCCA). In selected patients, neoadjuvant chemoradiotherapy followed by liver transplantation provides the optimal chance of long-term survival. However, for the patient described in the present case report, at the time of the patient s treatment, the UK did not have an approved transplant programme for cholangiocarcinoma, and access to liver transplantation was limited, often necessitating upfront surgical resection despite its complexity and limited curative potential. The present study describes the case of a 52-year-old male patient with PSC who was diagnosed with hCCA and underwent an extended right hepatectomy. After 26 months, progressive liver dysfunction due to PSC-related cirrhosis prompted liver transplantation, which was approved following a lengthy appeals process. Over the following years, the patient developed metastases in the bowel, lungs and abdominal wall, all of which were successfully managed with surgical resections. He remained disease-free for 8 years following his initial diagnosis before developing intrahepatic recurrence. The tumour was HER2-positive, and the compassionate use of zanidatamab was initiated following progression on standard therapies. At the time of the writing of the present case report, the patient remained alive 101 months following this initial diagnosis. On the whole, the present case report highlights the potential impact of tumour biology and multimodal treatment in PSC-associated hCCA. The prolonged survival of the patient despite delayed transplant and metastatic recurrence suggests that PSC-related hCCA may follow a more indolent course compared to de novo cases. Future efforts are required to focus on tumour profiling and stratified therapeutic approaches to better guide treatment in this complex disease.