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Gastrointestinal Cancer Liver Transplantation for Cholangiocarcinoma Armin Thelen, 1 Christoph Benckert 1 and Sven Jonas 2 1. Attending Physician; 2. Director, Department of Visceral, Transplantation, Thoracic and Vascular Surgery, Leipzig University, Germany Abstract The treatment of intra- and extrahepatic cholangiocarcinomas remains a medical challenge. Due to the poor efficacy of conventional chemotherapy, surgical treatment modalities represent the only chance of attaining long-term survival and cure. The introduction of new procedures, in particular extended liver resections – which were enabled by increasing surgical expertise and the implementation of multimodal treatment protocols – led to an increasing number of curatively treated patients and significant improvements in long-term results after curative resection. However, numerous patients are not suitable for radical resection because of local tumour growth, intrahepatic metastases, infiltration of main vascular and biliary structures or insufficient remnant liver function. In unresectable tumours, liver transplantation is a curative treatment option for many patients and represents the only chance to achieve long-term survival and cure. Yet, cholangiocarcinomas are not currently a standard indication for liver transplantation, because of the organ shortage and the resulting necessity to allocate available organs to patients with the best prognosis. In recent years, the results of liver transplantation for the different types of cholangiocarcinoma have improved following the application of new treatment protocols. The most promising long-term results were achieved in hilar cholangiocarcinoma by using neoadjuvant radiochemotherapy prior to transplantation. Long-term survival rates were not inferior to those seen in patients receiving a transplantation for benign liver diseases or early-stage hepatocellular carcinoma. The improved long-term outcomes of transplantation for intra- and extrahepatic cholangiocarcinomas have led to a renewed interest for liver transplantation as a treatment for these tumour entities. Keywords Hilar cholangiocarcinoma, intrahepatic cholangiocarcinoma, liver transplantation, results, indications Disclosure: The authors have no conflicts of interest to declare. Received: 2 July 2012 Accepted: 26 July 2012 Citation: European Oncology & Haematology, 2012;8(3):173–8 Correspondence: Armin Thelen, Department of Visceral, Transplantation, Vascular and Thoracic Surgery, Leipzig University, Liebigstrasse 20, 04103 Leipzig, Germany. E: armin.thelen@medizin.uni-leipzig.de Over the past two decades, liver transplantation for cholangiocarcinomas has been controversial. In the early era of transplantation, intrahepatic and hilar cholangiocarcinomas were considered to be ideal indications for liver transplantation. The tumours tend to remain localised within the liver and the liver hilum, respectively, until late in the course of the disease and can often be completely removed by hepatectomy and replacement of a homograft, even if curative resection is not feasible. 1,2 Therefore, liver transplantation represents a curative treatment option in patients with cholangiocarcinomas. However, despite cure obtained in a considerable proportion of patients, the long-term results of the procedure were found to be inferior to those of liver transplantation performed on patients with benign diseases or early-stage hepatocellular carcinoma. 1–4 In the early 1990s, because of the upcoming organ shortage and the resulting necessity to allocate available organs to patients with the best prospects of success in the long term, cholangiocarcinomas started to be refused as an indication for liver transplantation. 5,6 However, numerous patients with cholangiocarcinomas are not suitable for resection due to local tumour extension or insufficient remnant liver function (see Figure 1). In particular, patients with an underlying liver disease, such as primary sclerosing cholangitis (PSC), frequently have unresectable cholangiocarcinomas (see Figure 2). © TOUCH BRIEFINGS 2012 A number of these patients develop cholangiocarcinoma at a younger age, which underlines the need for a curative treatment option in tumours that are not suitable for resection. 7 In this group of patients, liver transplantation remains the only chance of long-term survival and cure. Therefore, liver transplantation remains an important tool in the treatment of patients with cholangiocarcinomas. In recent years, new treatment approaches have markedly improved patient outcomes. However, liver transplantation used in the treatment of cholangiocarcinomas remains complex and the indication needs to take into account the different biological behaviours, the availability of effective alternative treatment modalities, the current transplant allocation criteria and the individual prognosis. Hilar Cholangiocarcinoma The Mayo Clinic Experience In patients with hilar cholangiocarcinoma, the most promising results of liver transplantation were reported by the Mayo Clinic group in the US, with a protocol that included radiochemotherapy prior to transplantation. In the most recent evaluation of its results, the Mayo Clinic group reported a five-year survival rate of 73 % in 120 patients who finished a treatment protocol of liver transplantation after neoadjuvant radiochemotherapy. 8 In the 173