Effects of methylphenidate on cognition and behaviour in children with neurofibromatosis type 1: a study protocol for a randomised placebo-controlled crossover trial

Natalie A Pride; Belinda Barton; Paul Hutchins; David R Coghill; Mayuresh S Korgaonkar; Stephen J C Hearps; Melissa Rouel; Stephanie Malarbi; Kathryn N North; Jonathan M Payne

doi:10.1136/bmjopen-2018-021800

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Effects of methylphenidate on cognition and behaviour in children with neurofibromatosis type 1: a study protocol for a randomised placebo-controlled crossover trial

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Effects of methylphenidate on cognition and behaviour in children with neurofibromatosis type 1: a study protocol for a randomised placebo-controlled crossover trial

Natalie A Pride, Belinda Barton, Paul Hutchins, David R Coghill, Mayuresh S Korgaonkar, Stephen J C Hearps, Melissa Rouel, Stephanie Malarbi, Kathryn N North and Jonathan M Payne

BMJ open, Vol.8, e021800

30/08/2018

DOI: https://doi.org/10.1136/bmjopen-2018-021800

PMCID: PMC6119452

PMID: 30166301

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Abstract

Introduction Dopamine dysregulation has been identified as a key modulator of behavioural impairment in neurofibromatosis type 1 (NF1) and a potential therapeutic target. Preclinical research demonstrates reduced dopamine in the brains of genetically engineered NF1 mouse strains is associated with reduced spatial-learning and attentional dysfunction. Methylphenidate, a stimulant medication that increases dopaminergic and noradrenergic neurotransmission, rescued the behavioural and dopamine abnormalities. Although preliminary clinical trials have demonstrated that methylphenidate is effective in treating attention deficit hyperactivity disorder (ADHD) symptoms in children with NF1, its therapeutic effect on cognitive performance is unclear. The primary aim of this clinical trial is to assess the efficacy of methylphenidate for reducing attention deficits, spatial working memory impairments and ADHD symptoms in children with NF1. Methods and analysis A randomised, double-blind, placebo-controlled trial of methylphenidate with a two period crossover design. Thirty-six participants with NF1 aged 7–16 years will be randomised to one of two treatment sequences: 6 weeks of methylphenidate followed by 6 weeks of placebo or; 6 weeks of placebo followed by 6 weeks of methylphenidate. Neurocognitive and behavioural outcomes as well as neuroimaging measures will be completed at baseline and repeated at the end of each treatment condition (week 6, week 12). Primary outcome measures are omission errors on the Conners Continuous Performance Test-II (attention), between-search errors on the Spatial Working Memory task from the Cambridge Neuropsychological Test Automated Battery (spatial working memory) and the Inattentive and Hyperactivity/Impulsivity Symptom Scales on the Conners 3-Parent. Secondary outcomes will examine the effect of methylphenidate on executive functions, attention, visuospatial skills, behaviour, fine-motor skills, language, social skills and quality of life. Ethics and dissemination This trial has hospital ethics approval and the results will be disseminated through peer-reviewed publications and international conferences.

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Title: Effects of methylphenidate on cognition and behaviour in children with neurofibromatosis type 1: a study protocol for a randomised placebo-controlled crossover trial
Creators: Natalie A Pride - University of Sydney
Belinda Barton - Children's Hospital at Westmead
Paul Hutchins - Children's Hospital at Westmead
David R Coghill - Royal Children's Hospital
Mayuresh S Korgaonkar - University of Sydney
Stephen J C Hearps - Murdoch Children's Research Institute
Melissa Rouel - Children's Hospital at Westmead
Stephanie Malarbi - Royal Children's Hospital
Kathryn N North - University of Melbourne
Jonathan M Payne - University of Melbourne
Publication Details: BMJ open, Vol.8, e021800
Publisher: BMJ Group
Identifiers: 991013092667902368
Academic Unit: Faculty of Health
Language: English
Resource Type: Journal article

Effects of methylphenidate on cognition and behaviour in children with neurofibromatosis type 1: a study protocol for a randomised placebo-controlled crossover trial

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