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2024 Top Story in von Hippel-Lindau Disease: Belzutifan for Treatment of Pancreatic Neuroendocrine Tumors
Approximately 20% of patients with von Hippel–Lindau (VHL) disease will develop pancreatic neuroendocrine tumors (vPNET). Of them, about 10% may develop metastatic disease, and many will undergo surgical resection, which will often include resection of the distal pancreas or a Whipple procedure for pancreatic head/neck lesions. In addition to the surgical risk, these major surgeries affect the patient's quality of life in terms of malabsorption and food tolerability for life.
On this background, the efficacy of belzutifan in shrinking these lesions or healing patients with these tumors is a major advance in VHL-related clinical management.
In 2024, a subanalysis of the LITESPARK-004 study was published, providing more data on the efficacy of belzutifan for vPNET.1
Within the LITESPARK-004 cohort, all patients had at least one pancreatic lesion (although this was not an inclusion criterion), and 22 patients (36%) had at least one measurable pNET at baseline. The objective response rate for any pancreatic lesion (pNET or cyst) was 84%, achieved in 51 of the 61 patients. The objective response rate for pNET was even higher, reached in 20/22 patients (91%), with seven lesions reaching complete response.
Furthermore, only 18% of patients had grade 3 adverse events, with no grade 4/5 adverse events, compared with the known impact of surgical intervention.
These data support the use of belzutifan for vPNET requiring intervention and add further evidence for its groundbreaking impact on managing patients with VHL and its effects on renal cell carcinoma and central nervous system hemangioblastomas.