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 JC Hearps, Melissa Rouel, Stephanie Malarbi, Kathryn N North, Jonathan M Payne
BMJ OPEN | BMJ PUBLISHING GROUP | Published : 2018
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 thera..View full abstract
Awarded by Children's Tumor Foundation
This work is supported by the Children's Tumor Foundation (grant number 2012-10-003).