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.

TRIAL REGISTRATION:

ClinicalTrials.gov NCT00169611.

• Authors: Laurence Lion-François, François Gueyffier, Catherine Mercier, Daniel Gérard, Vania Herbillon, Isabelle Kemlin, Diana Rodriguez, Tiphanie Ginhoux, Emeline Peyric, Virginie Coutinho, Valentine Bréant, Vincent des Portes, Stéphane Pinson, Patrick Combemale, Behrouz Kassaï, Réseau NF1 Rhône Alpes Auvergne-France
• Journal: Orphanet journal of rare diseases (Orphanet J Rare Dis) Vol. 9 Pg. 142 (Sep 10 2014) ISSN: 1750-1172 [Electronic] England
• PMID: 25205361 (Publication Type: Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Chemical References

• Methylphenidate

Topics

• Child
• Cross-Over Studies
• Dose-Response Relationship, Drug
• Double-Blind Method
• Female
• Follow-Up Studies
• Humans
• Male
• Methylphenidate (therapeutic use)
• Neurofibromatosis 1 (diagnosis, drug therapy, psychology)
• Treatment Outcome