Reducing risks of surgeries and complications in von Hippel-Lindau disease with belzutifan treatment in China.

Abstract

e23063 Background: Von Hippel-Lindau (VHL) disease is a rare hereditary disorder causing neoplasms in multiple organs. Prevalence estimates range from 1:36,000 to 1:91,000 in different populations. Treatment options are limited, with active surveillance often leading to repeated surgeries due to metastasis recurrence and surgical complications that seriously affect quality of life. Belzutifan, the first HIF-2α inhibitor, was approved by the National Medical Products Administration (NMPA) in November 2024 in China, for VHL associated renal cell carcinoma (RCC), CNS hemangioblastomas (Hb), and pancreatic neuroendocrine tumors (pNET). This study aimed to quantify the impact of belzutifan on annual rate and costs of VHL-related surgeries and surgical complications. Methods: A previously published cost-consequence model was adapted for the Chinese setting to estimate the frequency of VHL-related surgeries and surgical complications before and after belzutifan treatment. Parameters included annual rates of VHL-related RCC and other tumor surgeries, risks of complications, and related costs. Surgery rates for before and after belzutifan were sourced from the LITESPARK-004 trial (as of April 1, 2022), which were validated with local clinical experts. Costs were collected through published literatures, government published prices and survey with local experts consultation. Costs were estimated in 2024 Chinese Yuan (¥) from a healthcare system perspective. Results: The initiation of belzutifan led to an 83% reduction (from 0.337 to 0.059 per person-year) and 84% reduction (from 0.217 to 0.035 per person-year) in the annual number of VHL-related surgeries and surgical complications, respectively. The annual number of VHL-related RCC and non-RCC surgeries were decreased by 66% (from 0.108 to 0.037 per person-year) and 91% (from 0.229 to 0.021 surgeries per person-year), respectively. Among these non-RCC surgeries, the rate of CNS Hb surgeries was reduced the most (from 0.115 to 0.011 surgeries per person-year). Belzutifan was estimated to reduce the annual costs of surgeries (from ¥18,274 to ¥2,692 per patient) and surgical complications (from ¥2,877 to ¥633 per patient). This resulted in a total cost decrease from ¥21,151 to ¥3,325, representing an 84% reduction. Conclusions: The cost-consequence analysis demonstrated that belzutifan initiation could substantially reduce healthcare system resource use and costs associated with VHL-related surgeries and surgical complications, by more than 80% based on trial-observed reductions in VHL-related surgeries.