Evidence map›Paper›PMID 37450072›Full record

ArticleJournal of neuro-oncology2023

Belzutifan in adults with VHL-associated central nervous system hemangioblastoma: a single-center experience.

Bryan J Neth, Mason J Webb, Jessica White, Joon H Uhm, Pavel N Pichurin, Ugur Sener

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Article in Journal of neuro-oncology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed
3.8field-weighted citation impact, top 6% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

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.

2 · The registry

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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

12 citing papers in PubMed, 16 citations in OpenAlex.

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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

6 authors at 3 institutions in 1 country.

Bryan J NethDepartment of Neurology, Mayo Clinic, Rochester, MN, 55905, USA.
Mason J WebbDepartment of Medical Oncology, Mayo Clinic, 200 First Street SW, Rochester, MN, 55905, USA.
Jessica WhiteDepartment of Neurology, Mayo Clinic, Rochester, MN, 55905, USA.
Joon H UhmDepartment of Neurology, Mayo Clinic, Rochester, MN, 55905, USA.
Pavel N PichurinDepartment of Clinical Genomics, Mayo Clinic, Rochester, MN, 55905, USA.
Ugur SenerDepartment of Neurology, Mayo Clinic, Rochester, MN, 55905, USA. Sener.Ugur@mayo.edu.
Mayo Clinic · USMayo Clinic in Arizona · USMayo Clinic in Florida · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

purposeBelzutifan is a selective inhibitor of hypoxia-inducible factor 2 alpha (HIF-2a) that has emerged as a targeted therapy option for Von Hippel-Lindau (VHL) syndrome-associated tumors with recent FDA approval. There is limited real-world evidence regarding safety and efficacy in CNS hemangioblastoma. Our objective was to report on our clinical experience with belzutifan in adult patients with VHL-associated CNS hemangioblastoma.

methodsWe retrospectively reviewed our institutional experience of belzutifan in adult patients (> 18 years of age at time of therapy) with VHL and craniospinal CNS hemangioblastomas not amenable to surgical resection. The period for study review was October 2021 to March 2023.

results4 patients (all female) with a median age of 36 years at time of belzutifan initiation were included. Median duration of therapy at last follow-up was 11 months (6-17 months). All patients had radiographic response to therapy after a median of 3 months (2-5 months), with maximal response to therapy after a median of 8 months (3-17 months). Therapy was well tolerated, with the most common adverse effect being anemia. No patients had treatment pauses or dose adjustments due to belzutifan-related toxicity. No patients experienced hypoxia.

conclusionWe showed that belzutifan is safe and well-tolerated with strong disease response for CNS hemangioblastoma in adults with VHL, supporting continued use of belzutifan in this patient population. Future studies should assess duration of treatment, effects of cessation after long-term use, and markers of therapeutic response.

Indexed as

Central Nervous System NeoplasmsHemangioblastomavon Hippel-Lindau DiseaseAdultCentral Nervous SystemFemaleHumansIndenesRetrospective StudiesVon Hippel-Lindau Tumor Suppressor ProteinbelzutifanIndenesVHL protein, humanVon Hippel-Lindau Tumor Suppressor ProteinBelzutifanCraniospinalHemangioblastomaReal worldToxicityVon Hippel–Lindau

Identifiers

PMID37450072
OpenAlexW4384298797

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.