Neurosurgical Implications of Targeting Hypoxia-Inducible Factor 2α in Hemangioblastomas with Belzutifan.

Autor: Pumford AD; Mayo Clinic Alix School of Medicine, Mayo Clinic, Rochester, Minnesota, USA. Electronic address: pumford.andrew@mayo.edu@mayo.edu., Bauman M; Mayo Clinic Alix School of Medicine, Mayo Clinic, Rochester, Minnesota, USA., Bouchal S; Mayo Clinic Alix School of Medicine, Mayo Clinic, Rochester, Minnesota, USA., Riviere-Cazaux C; Mayo Clinic Alix School of Medicine, Mayo Clinic, Rochester, Minnesota, USA., Jusue-Torres I; Department of Neurologic Surgery, Mayo Clinic, Rochester, Minnesota, USA., Hong S; Department of Neurologic Surgery, Mayo Clinic, Rochester, Minnesota, USA., Neth BJ; Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA., Sener U; Department of Neurology, Mayo Clinic, Rochester, Minnesota, USA., Parney IF; Department of Neurologic Surgery, Mayo Clinic, Rochester, Minnesota, USA.
Jazyk: angličtina
Zdroj: World neurosurgery [World Neurosurg] 2024 Oct; Vol. 190, pp. 291-296. Date of Electronic Publication: 2024 Aug 02.
DOI: 10.1016/j.wneu.2024.07.175
Abstrakt: Objective: To highlight the neurosurgical implications of the hypoxia-inducible factor-2α- targeting agent belzutifan in the management of both von-Hippel Lindau (VHL)-associated and sporadic hemangioblastomas (HBLs).
Methods: The literature was queried for VHL, HBLs, and belzutifan. A summary of recent uses of belzutifan and currently ongoing clinical trials that are investigating the use of belzutifan in the treatment of HBLs is presented.
Results: VHL disease occurs as a result of germline mutations in the VHL tumor suppressor gene on chromosome 3p25-p26, leading to growth of benign and malignant tumors such as HBLs. The possibility of intermittent growth in HBLs indicates that it is important to avoid hasty surgical interventions. Belzutifan is the first nonsurgical food and drug administration-approved treatment for VHL disease-related tumors that may delay or circumvent the need for surgery or radiation therapy by inhibiting HIF-2α, an important component of cellular hypoxic response. There is limited real-world experience of belzutifan in patients with HBLs as a primary indication, though there are 2 phase II clinical trials investigating the use of belzutifan in the treatment of HBLs.
Conclusions: There is limited experience regarding the use of belzutifan for CNS hemangioblastoma. While its application has been limited to a small group of clinical cases, it has exhibited significant efficacy in reducing the size and consequences of HBLs. Based on the promising outcomes observed in individual patient experiences and ongoing clinical trials, we infer that further exploration and integration of belzutifan into neurosurgical treatment plans for both sporadic and VHL-associated HBLs are warranted.
(Copyright © 2024 Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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