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.

中文翻译：

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新辅助 pemigatinib 作为 FGFR2 基因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，他在接受二线 pemigatinib 完全放射学反应后接受了活体肝移植 （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 pemigatinib 治疗，pemigatinib 是成纤维细胞生长因子受体 1、2 和 3 的竞争性抑制剂。这导致治疗 2 个月后出现完全的影像学和代谢反应。在观察治疗 6 个月后，他被认为适合 LDLT 并伴有持续反应。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。