Neoadjuvant Pemigatinib as a Bridge to Living Donor Liver Transplantation for Intrahepatic Cholangiocarcinoma with FGFR2 Rearrangement.

IF 8.9 2区 医学 Q1 SURGERY American Journal of Transplantation Pub Date : 2024-11-06 DOI:10.1016/j.ajt.2024.10.023
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
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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.

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新辅助佩米加替尼作为FGFR2重排肝内胆管癌活体肝移植的桥梁
我们报告了一例患有肝内胆管癌(iCCA)的55岁男性患者,他在接受二线培米加替尼治疗后出现完全放射学反应,随后接受了活体肝移植(LDLT)。LDLT治疗iCCA尚存争议,但最近的报道指出,LDLT对无法切除的患者,尤其是接受6个月全身治疗后病情稳定的患者有潜在益处。与此同时,基因组图谱研究也发现了 iCCA 潜在的可治疗肿瘤靶点。该患者的肿瘤基因组图谱显示其存在表皮生长因子受体 2 重排,并接受了表皮生长因子受体 1/2/3 竞争性抑制剂派米加替尼的治疗。治疗两个月后,患者出现了完全的放射学和代谢反应。经过 6 个月的持续反应治疗观察后,他被认为符合 LDLT 治疗条件。他接受了不复杂的 LDLT(包括不复杂的供体手术),随访一年后无疾病复发迹象。我们相信,这是首例针对这一适应症的 LDLT 报告。
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来源期刊
CiteScore
18.70
自引率
4.50%
发文量
346
审稿时长
26 days
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.
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