Abstract

Locally advanced cholangiocarcinoma has a poor prognosis, with long-term survival observed only in patients where complete surgical resection is achieved. Pediatric cholangiocarcinoma is exceedingly rare, with an estimated 15-22 cases reported in the last 40 years. As such, no standard therapeutic regimen exists. Novel strategies combining conventional chemotherapy and radiotherapy followed by targeted agents can lead to durable treatment responses and are applicable to pediatric cholangiocarcinoma management. We present a case of a 17-year-old patient with unresectable cholangiocarcinoma whose tumor genetic sequencing revealed a novel, actionable NRG1 translocation, providing an opportunity to utilize combination treatment in the pediatric setting.