Belzutifan for patients with Von Hippel-Lindau (VHL) disease-associated heterogeneous tumors - a retrospective single center analysis.

ABSTRACT:

Background

Von Hippel-Lindau (VHL) disease is a hereditary tumor predisposition syndrome characterized by benign and malignant tumors affecting multiple organ systems. Standard treatment primarily involves surgical resection and tumor ablation, which can lead to progressive organ dysfunction. Belzutifan, an oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, has emerged as a novel therapeutic option for VHL-associated tumors. However, data on its efficacy and safety outside of clinical trials remain limited. This study evaluates the outcomes of off-label belzutifan treatment in patients with advanced VHL-related organ manifestations, where standard therapeutic options were limited or contraindicated.

Results

A retrospective analysis was conducted on eight patients with

SUBMITTER: Rhein K 

PROVIDER: S-EPMC12579424 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature