title: The effect of methylphenidate on neurofibromatosis type 1: a randomised, double-blind, placebo-controlled, crossover trial.
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.
  
  
  BACKGROUND
  ClinicalTrials.gov NCT00169611.
authors.forename: Laurence
authors.initials: L
authors.lastname: Lion-François
authors.forename: François
authors.initials: F
authors.lastname: Gueyffier
authors.forename: Catherine
authors.initials: C
authors.lastname: Mercier
authors.forename: Daniel
authors.initials: D
authors.lastname: Gérard
authors.forename: Vania
authors.initials: V
authors.lastname: Herbillon
authors.forename: Isabelle
authors.initials: I
authors.lastname: Kemlin
authors.forename: Diana
authors.initials: D
authors.lastname: Rodriguez
authors.forename: Tiphanie
authors.initials: T
authors.lastname: Ginhoux
authors.forename: Emeline
authors.initials: E
authors.lastname: Peyric
authors.forename: Virginie
authors.initials: V
authors.lastname: Coutinho
authors.forename: Valentine
authors.initials: V
authors.lastname: Bréant
authors.forename: Vincent
authors.initials: V
authors.lastname: des Portes
authors.forename: Stéphane
authors.initials: S
authors.lastname: Pinson
authors.forename: Patrick
authors.initials: P
authors.lastname: Combemale
authors.forename: Behrouz
authors.initials: B
authors.lastname: Kassaï
mesh_terms: D002648:Child; D018592:Cross-Over Studies; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008774:Methylphenidate / Q000627:therapeutic use*; D009456:Neurofibromatosis 1 / Q000175:diagnosis* / Q000188:drug therapy* / Q000523:psychology; D016896:Treatment Outcome
chemical_list: D008774:Methylphenidate