ArticlePediatric research2010
2-hydroxypropyl-beta-cyclodextrin raises hearing threshold in normal cats and in cats with Niemann-Pick type C disease.
Article in Pediatric research, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 70 papers.
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.
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.
Who cites it
70 citing papers in PubMed, 112 citations in OpenAlex.
- Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial.Lancet (London, England) · 2017Trial
- Cholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann-Pick C1 (NPC1).Human molecular genetics · 2014Trial
- Enhancing the Water Solubility and Efficacy of Anticancer Drugs Using Hydroxypropyl-β-Cyclodextrin.International journal of molecular sciences · 2026Review
- Differently increased volumes of multiple brain areas inFrontiers in neuroanatomy · 2024Article
- Advances in research on potential therapeutic approaches for Niemann-Pick C1 disease.Frontiers in pharmacology · 2024Review
- Lysosomal cholesterol accumulation is commonly found in most peroxisomal disorders and reversed by 2-hydroxypropyl-β-cyclodextrin.Science China. Life sciences · 2023Article
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- Experimental animal models of drug-induced sensorineural hearing loss: a narrative review.Annals of translational medicine · 2021Review
- Neurodegeneration in Niemann-Pick Type C Disease: An Updated Review on Pharmacological and Non-Pharmacological Approaches to Counteract Brain and Cognitive Impairment.International journal of molecular sciences · 2021Review
- Spatiotemporal Developmental Upregulation of Prestin Correlates With the Severity and Location of Cyclodextrin-Induced Outer Hair Cell Loss and Hearing Loss.Frontiers in cell and developmental biology · 2021Article
- 2-Hydroxypropyl-β-cyclodextrin Ototoxicity in Adult Rats: Rapid Onset and Massive Destruction of Both Inner and Outer Hair Cells Above a Critical Dose.Neurotoxicity research · 2020Article
- Beyond the Typical Syndrome: Understanding Non-motor Features in Niemann-Pick Type C Disease.Cerebellum (London, England) · 2020Review
- 7-Ketocholesterol in disease and aging.Redox biology · 2020Review
- Pharmacokinetic Properties of Fluorescently Labelled Hydroxypropyl-Beta-Cyclodextrin.Biomolecules · 2019Article
- Current Challenges in Understanding the Cellular and Molecular Mechanisms in Niemann-Pick Disease Type C1.International journal of molecular sciences · 2019Review
- ROS Scavenger, Ebselen, Has No Preventive Effect in New Hearing Loss Model Using a Cholesterol-Chelating Agent.Journal of audiology & otology · 2019Article
- Main Olfactory and Vomeronasal Epithelium Are Differently Affected in Niemann-Pick Disease Type C1.International journal of molecular sciences · 2018Article
- The susceptibility of cochlear outer hair cells to cyclodextrin is not related to their electromotile activity.Acta neuropathologica communications · 2018Article
- Linear Cyclodextrin Polymer Prodrugs as Novel Therapeutics for Niemann-Pick Type C1 Disorder.Scientific reports · 2018Article
10 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 1 institution in 1 country.
Funding
Abstract
2-hydroxypropyl-beta-cyclodextrin (HPbetaCD) is a promising experimental therapy for Niemann-Pick type C disease that improved intracellular cholesterol transport, substantially reduced neurodegeneration and hepatic disease, and increased lifespan in npc1 mice. On the basis of favorable treatment outcome in mice, HPbetaCD is being evaluated as a therapy in children with Niemann-Pick type C (NPC) disease. We evaluated the efficacy of HPbetaCD in the feline model of NPC disease and recognized a dose-dependent increase in hearing threshold associated with therapy as determined by brain stem auditory evoked response (BAER) testing. To further assess the effect of HPbetaCD on hearing threshold, normal cats were administered the drug s.c. at either 4000 mg/kg or 8000 mg/kg body weight, or intrathecally at a dose of 4000 mg/kg brain weight. HPbetaCD caused a significant increase in hearing threshold following one dose of 8000 mg/kg s.c. or 120 mg intrathecally, and the effect was maintained for at least 12 weeks. Repeated weekly s.c. administration of 4000 mg/kg HPbetaCD resulted in a similar increase in hearing threshold. These studies are the first to describe a specific negative effect of HPbetaCD on the auditory system and suggest the need for auditory testing in patients receiving similar doses of HPbetaCD.
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Registered trials
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.