Simvastatin for cognitive deficits and behavioural problems in patients with neurofibromatosis type 1 (NF1-SIMCODA): a randomised, placebo-controlled trial

van der Vaart, Thijs; Plasschaert, Ellen; Rietman, André B; Renard, Marleen; Oostenbrink, Rianne; Vogels, Annick; de Wit, Marie-Claire Y; Descheemaeker, Mie-Jef; Vergouwe, Yvonne; Catsman-Berrevoets, Coriene E; Legius, Eric; Elgersma, Ype; Moll, Henriëtte A

doi:10.1016/S1474-4422(13)70227-8

Simvastatin for cognitive deficits and behavioural problems in patients with neurofibromatosis type 1 (NF1-SIMCODA): a randomised, placebo-controlled trial

Author:

van der Vaart, Thijs

Plasschaert, Ellen ; Rietman, André B ; Renard, Marleen ; Oostenbrink, Rianne ; Vogels, Annick ; de Wit, Marie-Claire Y ; Descheemaeker, Mie-Jef ; Vergouwe, Yvonne ; Catsman-Berrevoets, Coriene E ; Legius, Eric ; Elgersma, Ype ; Moll, Henriëtte A

Keywords:

Science & Technology, Life Sciences & Biomedicine, Clinical Neurology, Neurosciences & Neurology, DOUBLE-BLIND, MOUSE MODEL, HYPERACTIVITY DISORDER, CHILDREN, ATTENTION, LOVASTATIN, CHOLESTEROL, PERFORMANCE, PHENOTYPE, FREQUENCY, Adolescent, Child, Child Behavior Disorders, Cognition Disorders, Double-Blind Method, Drug Administration Schedule, Female, Humans, Hydroxymethylglutaryl-CoA Reductase Inhibitors, Male, Neurofibromatosis 1, Placebos, Simvastatin, Time Factors, Treatment Outcome, 1103 Clinical Sciences, 1109 Neurosciences, Neurology & Neurosurgery, 3202 Clinical sciences, 3209 Neurosciences

Abstract:

Neurofibromatosis type 1 is a common genetic disorder characterised by neurocutaneous manifestations and cognitive and behavioural problems. Statins were shown to reduce analogous learning deficits in a mouse model of the disease, but a short-term trial in humans was inconclusive. We aimed to assess the use of simvastatin for the improvement of cognitive and behavioural deficits in children with neurofibromatosis type 1 for 12 months.