Kitamura Satoshi, Hara Takuto, Okamura Yasuyoshi, Terakawa Tomoaki, Chiba Koji, Teishima Jun, Nakano Yuzo, Miyake Hideaki
Department of Urology, Kobe University Graduate School of Medicine, 7-51 Kusunoki-cho, Kobe, Japan.
Int Cancer Conf J. 2024 Oct 1;14(1):17-20. doi: 10.1007/s13691-024-00727-w. eCollection 2025 Jan.
Hereditary hemorrhagic telangiectasia (HHT) is a rare genetic disorder characterized by refractory recurrent epistaxis and gastrointestinal bleeding. Recent studies have reported the hemostatic effects of tyrosine kinase inhibitors on HHT-related bleeding. A 67-year-old man with HHT underwent laparoscopic radical nephrectomy for right renal cell carcinoma discovered during an investigation of anemia. Five years after surgery, pancreatic metastasis with biliary dilatation was found on computed tomography. After a biliary stent was inserted, the patient was treated with cabozantinib plus nivolumab. His hemoglobin level significantly improved from 4.8 g/dL to a maximum of 14.7 g/dL, and transfusion frequency reduced from five to one per 5 months. Despite tumor reduction after 6 months of treatment, the patient developed acute cholangitis because of biliary hemorrhage, which ultimately resulted in hepatic failure and death. This case is the first to indicate the potential of the tyrosine kinase inhibitor cabozantinib to control bleeding and tumor progression in patients with metastatic renal cell carcinoma with HHT-related bleeding.
遗传性出血性毛细血管扩张症（HHT）是一种罕见的遗传性疾病，其特征为难治性反复鼻出血和胃肠道出血。最近的研究报道了酪氨酸激酶抑制剂对HHT相关出血的止血作用。一名67岁的HHT男性患者因在贫血检查中发现右肾细胞癌而接受了腹腔镜根治性肾切除术。术后5年，计算机断层扫描发现胰腺转移伴胆管扩张。插入胆管支架后，患者接受了卡博替尼联合纳武单抗治疗。他的血红蛋白水平从4.8 g/dL显著提高到最高14.7 g/dL，输血频率从每5个月5次降至1次。尽管治疗6个月后肿瘤缩小，但患者因胆道出血并发急性胆管炎，最终导致肝衰竭死亡。该病例首次表明酪氨酸激酶抑制剂卡博替尼在控制伴有HHT相关出血的转移性肾细胞癌患者出血和肿瘤进展方面的潜力。
