[Treatment of von Hippel-Lindau Disease and Hemangioblastoma]

Von Hippel-Lindau (VHL) disease is a hereditary tumor syndrome characterized by the development of neoplasms in multiple organs. Hemangioblastoma is one of the most common tumors associated with VHL disease. In 2024, the "Clinical Practice Guidelines for VHL Disease" were established in Japan to provide guidance for the diagnosis, treatment, and management of VHL disease. Furthermore, in 2025, the HIF-2αinhibitor belzutifan was approved for insurance coverage in Japan for VHL-associated tumors, including hemangioblastomas. In this article, we first provide an overview of VHL disease and the "Clinical Practice Guidelines for VHL Disease," and then discuss the diagnosis and treatment of hemangioblastomas, including surgery, endovascular therapy, and radiotherapy. Finally, we review the role of the HIF-2αinhibitor belzutifan. In the future, management of VHL disease and hemangioblastomas will likely require not only individual treatment modalities, but also optimal multimodal strategies combining surgery, radiotherapy, and systemic drug therapy.