Trial reportOphthalmology2024
Oral Hypoxia-Inducible Factor 2α Inhibitor Belzutifan in Ocular von Hippel-Lindau Disease: Subgroup Analysis of the Single-Arm Phase 2 LITESPARK-004 Study.
Trial report in Ophthalmology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 12 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
12 citing papers in PubMed.
- From Somatic Driver to Molecular Therapy in Retinal, Choroidal and Periocular Vascular Disease: Mosaicism, Targetable Pathways and Ocular Toxicity.International journal of molecular sciences · 2026Review
- Oral belzutifan treatment in von hippel-lindau-associated retinal hemangioblastoma: a promising approach.International journal of retina and vitreous · 2026Article
- Clinical outcomes of HIF-2α inhibition in Von Hippel-Lindau disease: a multicenter real-world study.The oncologist · 2026Article
- Von Hippel-Lindau disease: pathophysiological and clinical advances.Familial cancer · 2026Review
- Very rapid response to belzutifan of VHL-related intracranial and retinal hemangioblastomas.American journal of ophthalmology case reports · 2026Article
- Hypoxic tumor microenvironment and immune cell dynamics: From metabolic reprogramming to therapeutic innovation.Iranian journal of basic medical sciences · 2026Review
- Belzutifan for Ocular Tumors in Patients with von Hippel-Lindau Disease.Ocular oncology and pathology · 2025Article
- From surgery to systemic therapy in von Hippel-Lindau disease: insights from extended follow-up of LITESPARK-004.Translational andrology and urology · 2025Article
- The clinical features and real world treatment outcomes in von Hippel Lindau related retinal capillary hemangioblastomas.International journal of retina and vitreous · 2025Article
- Genetics and current research models of Mendelian tumor predisposition syndromes with ocular involvement.Progress in retinal and eye research · 2025Review
- Linking hypoxia-inducible factor (HIF)-1α and HIF-2α expression in head and neck cancer.Bioinformation · 2025Article
- Risk Analysis of Retinal Hemangioblastomas in Nonadvanced Stages of von Hippel-Lindau Syndrome Using Ultra-widefield Imaging: The ULTRA von Hippel-Lindau Study.Ophthalmology scienceArticle
Corrections and comments
- Erratum issuedCorrigendum.2025
Authors and funding
19 authors.
Funding
Abstract
purposeTo report the efficacy of the oral hypoxia-inducible factor 2α inhibitor belzutifan in participants with von Hippel-Lindau disease-associated retinal hemangioblastomas in the LITESPARK-004 study.
designSubgroup analysis of the phase 2, single-arm, open-label LITESPARK-004 study.
participantsAdults with 1 or more von Hippel-Lindau disease-associated measurable renal cell carcinoma tumors not requiring immediate surgical intervention were eligible.
methodsParticipants received oral belzutifan 120 mg once daily until disease progression or unacceptable treatment-related toxicity.
main outcome measuresEfficacy of belzutifan in retinal hemangioblastomas was a secondary end point, measured as response (improved, stable, or progressed) by independent reading center-certified graders based on color fundus imaging performed every 12 weeks using the investigator's preferred imaging standards. Additional assessments, where available, included OCT and ultra-widefield fluorescein angiography.
resultsAmong 61 participants in LITESPARK-004, 12 had 1 or more evaluable active retinal hemangioblastomas in 16 eyes at baseline per independent reading center. As of April 1, 2022, the median follow-up for participants with ocular von Hippel-Lindau disease at baseline was 37.3 months. All 16 eyes were graded as improved, with a response rate of 100.0% (95% confidence interval, 79.4%-100%). No new retinal hemangioblastomas or ocular disease progression were reported as of data cutoff date. Eight participants underwent additional multimodal eye assessments performed at the National Institutes of Health study site. Among this subgroup, 10 of 24 hemangioblastomas in 8 eyes of 6 participants measured 500 μm or more in greatest linear dimension at baseline and were analyzed further. All 10 hemangioblastomas had a mean area reduction of 15% or more by month 12 and of 30% or more by month 24.
conclusionsBelzutifan showed promising activity against ocular von Hippel-Lindau disease, including capacity to control retinal hemangioblastomas, with effects sustained for more than 2 years while treatment is ongoing. FINANCIAL DISCLOSURE(S): Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.
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