Neoadjuvant pemigatinib as a bridge to living donor liver transplantation for intrahepatic cholangiocarcinoma with FGFR2 gene rearrangement.

We report a case of a 55-year-old male with intrahepatic cholangiocarcinoma (iCCA) who underwent living donor liver transplantation (LDLT) after complete radiographic response on second-line pemigatinib. LDLT for iCCA is controversial, but recent reports have cited the potential benefit for patients with unresectable disease, especially those with disease stability after 6 months of systemic therapy. Concomitantly, genomic profiling has identified potentially treatable oncologic targets in iCCA. This patient's tumor genomic profile revealed an FGFR2 rearrangement and was treated with pemigatinib, a competitive inhibitor for fibroblast growth factor receptors 1, 2, and 3. This resulted in a complete radiographic and metabolic response after 2 months of treatment. He was considered eligible for LDLT after 6 months of observation on treatment with a sustained response. He underwent an uncomplicated LDLT (including an uncomplicated donor surgery) and at a 1-year follow-up is without evidence of disease recurrence. We believe this is the first report of LDLT for this indication.