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Neoadjuvant Pemigatinib as a Bridge to Living Donor Liver Transplantation for Intrahepatic Cholangiocarcinoma with FGFR2 Rearrangement.
American Journal of Transplantation ( IF 8.9 ) 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

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.

中文翻译:


新辅助 Pemigatinib 作为 FGFR2 重排肝管癌活体供体肝移植的桥梁。



我们报告了一例 55 岁男性肝内胆管癌 (iCCA) 病例,他在二线 pemigatinib 完全放射学反应后接受了活体供体肝移植 (LDLT)。iCCA 的 LDLT 存在争议,但最近的报告指出了对不可切除疾病患者的潜在益处,尤其是那些在全身治疗 6 个月后病情稳定的患者。同时,基因组分析在 iCCA 中确定了可能可治疗的肿瘤靶点。该患者的肿瘤基因组图谱显示 FGFR2 重排,并接受了 FGFR1/2/3 竞争性抑制剂 pemigatinib 治疗。这导致治疗两个月后出现完全的放射学和代谢反应。在观察持续反应的治疗 6 个月后,他被认为适合 LDLT。他接受了无并发症的 LDLT (包括无并发症的供体手术),随访 1 年无疾病复发的证据。我们认为这是 LDLT 针对该适应症的首次报告。
更新日期:2024-11-06
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