Multinational phase 2 trial of belzutifan for von Hippel-Lindau disease-associated RCC: 50 months follow-up of LITESPARK-004

The multinational phase 2 LITESPARK-004 trial evaluated the hypoxia-inducible factor (HIF)-2α for von Hippel-Lindau disease-associated renal cell carcinoma. Long-term follow-up of the trial have now been published. At a median duration of follow-up of 49.9 months (IQR 48.9-52.2), 36 of 61 patients (59%) were continuing treatment. Forty-one of 61 patients (67%; 95% CI 54-79) had an objective response; among these, seven (11%) had a complete response. Thirteen grade 3 treatment-related adverse events occurred in 11 patients; none of the patients had a grade 4 or 5 treatment-related adverse event. Four (7%) patients had serious treatment-related adverse events. The authors conclude that these results support the use of belzutifan as systemic treatment for von Hippel-Lindau disease-associated renal cell carcinoma.