Journal article
Randomized placebo-controlled study of lovastatin in children with neurofibromatosis type 1
Neurology, Vol.87(24), pp.2575-2584
13/12/2016
PMCID: PMC5207004
PMID: 27956565
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Source: InCites
Abstract
Objective: To assess the efficacy of lovastatin on visuospatial learning and attention for treating cognitive and behavioral deficits in children with neurofibromatosis type 1 (NF1).
Methods: A multicenter, international, randomized, double-blind, placebo-controlled trial was conducted between July 2009 and May 2014 as part of the NF Clinical Trials Consortium. Children with NF1 aged 8–15 years were screened for visuospatial learning or attention deficits (n = 272); 146 children demonstrated deficits at baseline and were randomly assigned to lovastatin (n = 74; 40 mg/d) or placebo (n = 70). Treatment was administered once daily for 16 weeks. Primary outcomes were total errors on the Cambridge Neuropsychological Test Automated Battery Paired Associate Learning task (visuospatial learning) and the Score subtest from the Test of Everyday Attention for Children (sustained attention). Secondary outcomes measured executive function, attention, visuospatial skills, behavior, and quality of life. Primary analyses were performed on the intention-to-treat population.
Results: Lovastatin had no significant effect on primary outcomes after 16 weeks of treatment: visuospatial learning (Cohen d = −0.15, 95% confidence interval −0.47 to 0.18) or sustained attention (Cohen d = 0.19, 95% confidence interval −0.14 to 0.53). Lovastatin was well tolerated, with no increase in reported adverse events compared to placebo.
Conclusions: Lovastatin administered once daily for 16 weeks did not improve visuospatial learning or attention in children with NF1 and is not recommended for amelioration of cognitive deficits in this population.
Details
- Title
- Randomized placebo-controlled study of lovastatin in children with neurofibromatosis type 1
- Creators
- Jonathan M Payne - Murdoch Children's Research InstituteBelinda Barton - University of SydneyNicole J Ullrich - Harvard UniversityAlan Cantor - University of Alabama at BirminghamStephen J C HearpsGary Cutter - University of Alabama at BirminghamTena Rosser - University of Southern CaliforniaKarin S Walsh - Children’s National Health SystemGerard A Gioia - Children’s National Health SystemPamela L Wolters - National Institutes of HealthJames Tonsgard - University of ChicagoElizabeth Schorry - Cincinnati Children's Hospital Medical CenterDavid Viskochil - University of UtahLaura Klesse - The University of Texas at AustinMichael Fisher - University of PennsylvaniaDavid H Gutmann - Washington University in St. LouisAlcino J Silva - University of California at Los AngelesScott J Hunter - University of ChicagoCeliane Rey-Casserly - Harvard UniversityNancy L Cantor - Primary Children's Medical CenterAnna W Byars - Cincinnati Children's Hospital Medical CenterPeter L Stavinoha - The University of Texas MD Anderson Cancer CenterJoseph D Ackerson - University of Alabama at BirminghamCarol L Armstrong - University of PennsylvaniaJill Isenberg - Washington University in St. LouisSharon H O'Neil - University of Southern CaliforniaRoger J Packer - Children’s National Health SystemBruce Korf - University of Alabama at BirminghamMaria T Acosta - Children’s National Health SystemKathryn N North - University of MelbourneNF Clinical Trials Consortium
- Publication Details
- Neurology, Vol.87(24), pp.2575-2584
- Publisher
- Wolters Kluwer Health
- Grant note
- U01 NS082320 / NINDS NIH HHS R01 NS065840 / NINDS NIH HHS U54 HD090257 / NICHD NIH HHS
- Identifiers
- 991013092666702368
- Copyright
- © 2016 American Academy of Neurology.
- Academic Unit
- Faculty of Health
- Language
- English
- Resource Type
- Journal article