title: Modafinil for excessive daytime sleepiness in myotonic dystrophy type 1--the patients' perspective.
abstract: Excessive daytime sleepiness (EDS), of very similar pattern to that seen in narcolepsy syndrome, is extremely common in myotonic dystrophy type 1 (DM1). In a significant minority it has a profound disabling effect on employment, social functioning and activities of daily living. Limited published studies have shown inconsistent results from use of the psychostimulant drug modafinil. A recent European Medicines Agency (EMA) review concluded that on current evidence regarding safety and efficacy, modafinil's use should be restricted to the treatment of narcolepsy. In other conditions (although DM1 was not specifically considered) it was concluded that there was insufficient evidence of benefit to outweigh potentially serious side-effects, including severe skin reactions and cardiac arrhythmia. Clinicians with extensive experience in the management of DM1 have found modafinil to be extremely effective in appropriately selected patients with a very low incidence of serious side-effects. Given the recent EMA review, patients have expressed concern about the potential restriction of the use of modafinil in DM1. This brief review is an audit of the experience of a large group of patients and their clinicians concerning EDS and DM1 and concludes that despite the limited literature there is strong evidence to support the use of modafinil in carefully selected patients.
authors.affiliation: Department of Neurology, West Wing, John Radcliffe Hospital, Oxford OX3 9DU, UK. david.hilton-jones@clneuro.ox.ac.uk
authors.forename: D
authors.initials: D
authors.lastname: Hilton-Jones
authors.forename: M
authors.initials: M
authors.lastname: Bowler
authors.forename: H
authors.initials: H
authors.lastname: Lochmueller
authors.forename: C
authors.initials: C
authors.lastname: Longman
authors.forename: R
authors.initials: R
authors.lastname: Petty
authors.forename: M
authors.initials: M
authors.lastname: Roberts
authors.forename: M
authors.initials: M
authors.lastname: Rogers
authors.forename: C
authors.initials: C
authors.lastname: Turner
authors.forename: D
authors.initials: D
authors.lastname: Wilcox
mesh_terms: D001559:Benzhydryl Compounds / Q000627:therapeutic use*; D000697:Central Nervous System Stimulants / Q000627:therapeutic use*; D006970:Disorders of Excessive Somnolence / Q000188:drug therapy / Q000209:etiology / Q000523:psychology; D005260:Female; D006801:Humans; D008297:Male; D000077408:Modafinil; D009223:Myotonic Dystrophy / Q000150:complications* / Q000453:epidemiology; D011795:Surveys and Questionnaires; D006113:United Kingdom / Q000453:epidemiology
chemical_list: D001559:Benzhydryl Compounds; D000697:Central Nervous System Stimulants; D000077408:Modafinil