This study investigated the effectiveness of belzutifan, a daily oral medication, in treating central nervous system (CNS) haemangioblastomas associated with VHL disease, usually in the brain and along the spine. Researchers followed patients for over three years to assess how well the treatment reduced tumour size and its safety profile. Among the 50 patients with evaluable CNS haemangioblastomas, 44% showed a meaningful reduction in tumour size when considering all types of lesions, including solid and cystic components. When focusing only on solid lesions of measurable size, 76% of patients experienced tumour shrinkage. These effects were sustained for more than three years.
While the treatment was effective for many, nearly half of the patients experienced severe side effects (grade 3 or higher), with anaemia being the most common, affecting 12% of participants. Two patients experienced grade 4 adverse events, and two deaths were reported, though these were not attributed to belzutifan.
Overall, belzutifan demonstrated significant and long-lasting antitumour activity, supporting its role as a systemic treatment option for VHL-associated CNS haemangioblastomas. However, careful monitoring is necessary to manage potential side effects.
Here is the original article: Iliopoulos O, Iversen AB, Narayan V, Maughan BL, Beckermann KE, Oudard S, Else T, Maranchie JK, Goldberg CM, Fu W, Perini RF, Liu Y, Linehan WM, Srinivasan R, Jonasch E. Belzutifan for patients with von Hippel-Lindau disease-associated CNS haemangioblastomas (LITESPARK-004): a multicentre, single-arm, phase 2 study. Lancet Oncol. 2024 Oct;25(10):1325-1336. doi: 10.1016/S1470-2045(24)00389-9. Epub 2024 Sep 13. Erratum in: Lancet Oncol. 2024 Nov;25(11):e542. doi: 10.1016/S1470-2045(24)00587-4. PMID: 39284337.
https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(24)00389-9/abstract