LITESPARK-004: long-term belzutifan in VHL disease-associated neoplasms

In this video, Prof Dr Sylvie Rottey discusses the 6-year follow-up of LITESPARK-004, a phase II study evaluating belzutifan in patients with von Hippel-Lindau (VHL) disease-associated neoplasms.¹

Belzutifan is a first-in-class HIF-2α inhibitor. In this study, it was evaluated in patients with VHL disease-associated renal cell carcinoma, central nervous system (CNS) haemangioblastomas or pancreatic neuroendocrine tumours who did not require immediate surgery.

After extended follow-up, belzutifan continued to show durable antitumour activity. Objective response rates were 70% for renal cell carcinoma, 52% for CNS haemangioblastomas and 91% for pancreatic neuroendocrine tumours. More than half of patients remained on treatment after six years, and 64% had not undergone any VHL disease-related surgical procedure after starting belzutifan.

The safety profile remained manageable with long-term treatment. Grade 3 treatment-related adverse events occurred in 20% of patients, most commonly anaemia, and no grade 4 or 5 treatment-related adverse events were reported.

Overall, these data support the long-term use of belzutifan in selected patients with VHL disease-associated neoplasms, with durable disease control and potential reduction in the need for repeated surgical interventions.