Randomized placebo-controlled study of lovastatin in children with neurofibromatosis type 1
| Autor: | Jonathan M, Payne, Belinda, Barton, Nicole J, Ullrich, Alan, Cantor, Stephen J C, Hearps, Gary, Cutter, Tena, Rosser, Karin S, Walsh, Gerard A, Gioia, Pamela L, Wolters, James, Tonsgard, Elizabeth, Schorry, David, Viskochil, Laura, Klesse, Michael, Fisher, David H, Gutmann, Alcino J, Silva, Scott J, Hunter, Celiane, Rey-Casserly, Nancy L, Cantor, Anna W, Byars, Peter L, Stavinoha, Joseph D, Ackerson, Carol L, Armstrong, Jill, Isenberg, Sharon H, O'Neil, Roger J, Packer, Bruce, Korf, Maria T, Acosta, Kathryn N, North, Cynthia, Flanigan |
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| Rok vydání: | 2016 |
| Předmět: |
0301 basic medicine
Male Pediatrics Placebo-controlled study Neuropsychological Tests law.invention Executive Function 0302 clinical medicine Randomized controlled trial law Attention NF Clinical Trials Consortium Pediatric education.field_of_study Cambridge Neuropsychological Test Automated Battery Rehabilitation Mental Health 6.1 Pharmaceuticals Cognitive Sciences Female medicine.medical_specialty Neurofibromatosis 1 Test of everyday attention Clinical Trials and Supportive Activities Clinical Sciences Population Placebo Basic Behavioral and Social Science Article Neurofibromatosis 03 medical and health sciences Rare Diseases Double-Blind Method Clinical Research Behavioral and Social Science medicine Humans Learning Lovastatin education Neurology & Neurosurgery business.industry Neurosciences Evaluation of treatments and therapeutic interventions Confidence interval Clinical trial 030104 developmental biology Attention Deficit Disorder with Hyperactivity Quality of Life Neurology (clinical) business 030217 neurology & neurosurgery |
| Zdroj: | Neurology, vol 87, iss 24 |
| ISSN: | 1526-632X |
| Popis: | 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. ClinicalTrials.gov identifier: This study was registered at ClinicalTrials.gov (NCT00853580) and Australian New Zealand Clinical Trials Registry (ACTRN12607000560493). Classification of evidence: This study provides Class I evidence that for children with NF1, lovastatin does not improve visuospatial learning or attention deficits. |
| Databáze: | OpenAIRE |
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