Matthew M Byrne, Richard F Dunne, Jennifer I Melaragno, Mariana Chávez-Villa, Aram Hezel, Xiaoyan Liao, Marco Ertreo, Bandar Al-Judaibi, Mark Orloff, Roberto Hernandez-Alejandro, Koji Tomiyama
{"title":"Neoadjuvant Pemigatinib as a Bridge to Living Donor Liver Transplantation for Intrahepatic Cholangiocarcinoma with FGFR2 Rearrangement.","authors":"Matthew M Byrne, Richard F Dunne, Jennifer I Melaragno, Mariana Chávez-Villa, Aram Hezel, Xiaoyan Liao, Marco Ertreo, Bandar Al-Judaibi, Mark Orloff, Roberto Hernandez-Alejandro, Koji Tomiyama","doi":"10.1016/j.ajt.2024.10.023","DOIUrl":null,"url":null,"abstract":"<p><p>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 a FGFR2 rearrangement and was treated with pemigatinib, a competitive inhibitor for FGFR1/2/3. This resulted in complete radiographic and metabolic response after two months of treatment. He was considered eligible for LDLT after 6 months of observation on treatment with sustained response. He underwent an uncomplicated LDLT (including an uncomplicated donor surgery) and at one year follow-up is without evidence of disease recurrence. We believe this is the first report of LDLT for this indication.</p>","PeriodicalId":123,"journal":{"name":"American Journal of Transplantation","volume":" ","pages":""},"PeriodicalIF":8.9000,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"American Journal of Transplantation","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.ajt.2024.10.023","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"SURGERY","Score":null,"Total":0}
引用次数: 0
Abstract
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 a FGFR2 rearrangement and was treated with pemigatinib, a competitive inhibitor for FGFR1/2/3. This resulted in complete radiographic and metabolic response after two months of treatment. He was considered eligible for LDLT after 6 months of observation on treatment with sustained response. He underwent an uncomplicated LDLT (including an uncomplicated donor surgery) and at one year follow-up is without evidence of disease recurrence. We believe this is the first report of LDLT for this indication.
期刊介绍:
The American Journal of Transplantation is a leading journal in the field of transplantation. It serves as a forum for debate and reassessment, an agent of change, and a major platform for promoting understanding, improving results, and advancing science. Published monthly, it provides an essential resource for researchers and clinicians worldwide.
The journal publishes original articles, case reports, invited reviews, letters to the editor, critical reviews, news features, consensus documents, and guidelines over 12 issues a year. It covers all major subject areas in transplantation, including thoracic (heart, lung), abdominal (kidney, liver, pancreas, islets), tissue and stem cell transplantation, organ and tissue donation and preservation, tissue injury, repair, inflammation, and aging, histocompatibility, drugs and pharmacology, graft survival, and prevention of graft dysfunction and failure. It also explores ethical and social issues in the field.