The effect of methylphenidate on neurofibromatosis type 1: a randomised, double-blind, placebo-controlled, crossover tri
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RESEARCH
Open Access
The effect of methylphenidate on neurofibromatosis type 1: a randomised, double-blind, placebo-controlled, crossover trial Laurence Lion-François1,14,15*, François Gueyffier2, Catherine Mercier3,4,5, Daniel Gérard6, Vania Herbillon1, Isabelle Kemlin7, Diana Rodriguez7,8, Tiphanie Ginhoux9,10, Emeline Peyric1, Virginie Coutinho7, Valentine Bréant11, Vincent des Portes1,12, Stéphane Pinson13, Patrick Combemale14, Behrouz Kassaï9 and et Réseau NF1 Rhône Alpes Auvergne-France14
Abstract Background: Neurofibromatosis type 1 (NF1) is an autosomal dominant disorder with an estimated prevalence of about 1/3000, independent of ethnicity, race, or gender. Attention Deficit Hyperactivity like Disorder (ADHD)-like characteristics are often reported in patients with NF1. We hypothesised that learning disabilities in NF1 children were related to ADHD symptoms. Treatment with methylphenidate (MPD) has improved learning disabilities in ADHD by acting on neurotransmitters. Our objective was to evaluate its efficacy on ADHD-like symptoms in neurofibromatosis type 1 children (7–12 years). Methods: This was a randomised, double blind, placebo controlled, and crossover trial comparing 0.5 to 0.8 mg/kg/d of MPD as it is indicated for ADHD to placebo in NF1 children with ADHD-like symptoms. Children aged 7 to 12 years were eligible when their IQ was between 80 and 120. The total follow-up was 9 weeks including 4 weeks for each period and 1 week wash out. Fifty subjects (25 for each period) were required for testing the primary study hypothesis. The main outcome was an improvement in scores on the simplified Conners’ Parent Rating Scale. Results: Thirty-nine patients were included between April 2004 and December 2010. Twenty participants received MPD and 19 placebo during the first period. They all completed the trial. MPD decreased the simplified Conners by 3.9 points (±1.1, p = 0. 0003). Conclusions: This is the first randomised controlled trial showing the short-term benefit of MPD on simplified Conners scores in NF1 children. Trial registration: ClinicalTrials.gov NCT00169611. Keywords: Neurofibromatosis, Randomised controlled trials, Methylphenidate, Attention deficit hyperactivity disorder
Background Neurofibromatosis type 1 (NF1), also known as von Recklinghausen’s disease, is an autosomal dominant disorder with an estimated prevalence of about 1/3000 [1]. Caused by mutation in the NF1 gene, this condition is characterised by multiple café-au lait spots, benign neurofibromas and Lisch nodules [2]. * Correspondence: [email protected] 1 Department of Pediatric Neurology, Mother & Child Hospital, 59, boulevard Pinel, 69677 Bron Lyon, France 14 Réseau NF1 Rhône Alpes Auvergne IHOP, Lyon, France Full list of author information is available at the end of the article
The evolution of NF1 is unpredictable and highly variable. It may be undetectable by the untrained eye, lead to mild disfigurement or even life threatening conditions [3]. The physical features of NF1 are well characterised, facilitat
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