Coco Danilo, Leanza Silvana
Department of General, Robotic and Oncologic Surgery, Giglio Foundation Hospital, Cefalù, Palermo, Italy.
J Kidney Cancer VHL. 2025 Sep 5;12(3):16-24. doi: 10.15586/jkc.v12i3.396. eCollection 2025.
The von Hippel-Lindau (VHL) syndrome is a rare autosomal dominant disorder caused by mutations in the VHL tumor suppressor gene, leading to the development of benign and malignant tumors in multiple organs, including the kidneys, brain, spine, retina, and pancreas. Since its initial description in the early 20th century, significant progress has been made in understanding its pathogenesis, genetic basis, and clinical management. This narrative review provides a comprehensive overview of VHL syndrome, from its discovery to the latest medical and surgical therapies. A systematic literature review was conducted following the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, incorporating the Egger test to assess publication bias. The review highlights the evolution of diagnostic criteria, the role of genetic testing, and the development of targeted therapies such as hypoxia-inducible factor 2-alpha (HIF-2α) inhibitors. Surgical interventions, including nephron-sparing surgery and minimally invasive techniques, are also discussed. This review emphasizes the importance of a multidisciplinary approach to managing VHL syndrome and explores emerging therapies that hold promise for improving patient outcomes.
冯·希佩尔-林道(VHL)综合征是一种罕见的常染色体显性疾病,由VHL肿瘤抑制基因突变引起,可导致多个器官(包括肾脏、大脑、脊柱、视网膜和胰腺)出现良性和恶性肿瘤。自20世纪初首次描述以来,在了解其发病机制、遗传基础和临床管理方面已取得重大进展。这篇叙述性综述全面概述了VHL综合征,从其发现到最新的医学和外科治疗方法。按照系统评价和Meta分析的首选报告项目(PRISMA)指南进行了系统的文献综述,并采用Egger检验评估发表偏倚。该综述重点介绍了诊断标准的演变、基因检测的作用以及靶向治疗(如缺氧诱导因子2-α(HIF-2α)抑制剂)的发展。还讨论了手术干预措施,包括保留肾单位手术和微创技术。本综述强调了多学科方法管理VHL综合征的重要性,并探索了有望改善患者预后的新兴治疗方法。