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Excellent single-center outcomes of neoadjuvant chemoradiation and liver transplantation for unresectable perihilar cholangiocarcinoma caused the United Network of Organ Sharing to offer a standardized model of end-stage liver disease (MELD) exception for this disease. Dr Sarwa Darwish Murad and colleagues from Minnesota, USA analyzed data from multiple centers to determine the effectiveness of this treatment and the appropriateness of the MELD exception. The researchers collected and analyzed data from 12 large-volume transplant centers in the United States. These centers met the inclusion criteria of treating 3 or more patients with perihilar cholangiocarcinoma using neoadjuvant therapy, followed by liver transplantation, from 1993 to 2010. Center-specific protocols and medical charts were reviewed on-site.  | | Recurrence-free survival rate was 78% at 2 years after therapy | | Gastroenterology |
The patients completed external radiation, brachytherapy, radiosensitizing therapy, and/or maintenance chemotherapy. The team found that 71 patients dropped out before liver transplantation. Intent-to-treat survival rates were 68% and 53%, 2 and 5 years after therapy, respectively. Post-transplant, recurrence-free survival rates were 78% and 65% at 2 and 5 years after therapy, respectively. Patients outside the United Network of Organ Sharing criteria or with a prior malignancy had significantly shorter survival times. The research team found no differences in outcomes among patients based on differences in surgical staging or brachytherapy. The team noted that although most patients came from 1 center, the other 11 centers had similar survival times after therapy. Dr Murad's team commented, "Patients with perihilar cholangiocarcinoma who were treated with neoadjuvant therapy followed up by liver transplantation at 12 US centers had a 65% rate of recurrence-free survival after 5 years, showing this therapy to be highly effective." "A 12% drop-out rate after 4 months of therapy indicates the appropriateness of the MELD exception." "Rigorous selection is important for the continued success of this treatment."
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