Wedekind Mary Frances, Ranalli Mark, Shah Nilay
Division of Pediatric Hematology/Oncology/BMT, Nationwide Children's Hospital, Columbus, Ohio.
Department of Pediatrics, Ohio State University College of Medicine, Columbus, Ohio.
Pediatr Blood Cancer. 2017 Nov;64(11). doi: 10.1002/pbc.26586. Epub 2017 Apr 18.
Advanced-stage renal cell carcinoma (RCC) carries a dismal prognosis for pediatric patients with few studied therapeutic options. Cabozantinib is a small molecule tyrosine kinase inhibitor against the oncoprotein MET. It is currently approved by the U.S. Food and Drug administration for second-line treatment of RCC in adults. There is no published data on its use in children with RCC. We report here two pediatric patients with recurrent metastatic RCC whose tumors expressed MET and were treated with cabozantinib. Both patients had significant disease regression and symptomatic improvement for over 15 months with tolerable adverse effects. Cabozantinib can maintain prolonged disease control in pediatric RCC and warrants further study.
晚期肾细胞癌（RCC）对儿科患者的预后不佳，可供研究的治疗选择很少。卡博替尼是一种针对癌蛋白MET的小分子酪氨酸激酶抑制剂。它目前已获美国食品药品监督管理局批准，用于成人RCC的二线治疗。尚无关于其在儿童RCC中应用的公开数据。我们在此报告两名复发性转移性RCC儿科患者，其肿瘤表达MET，并接受了卡博替尼治疗。两名患者均出现显著的疾病消退和症状改善，持续超过15个月，且不良反应可耐受。卡博替尼可在儿科RCC中维持长期疾病控制，值得进一步研究。
