title: A randomised controlled trial of treatment for idiopathic intracranial hypertension.
abstract: The cause of idiopathic intracranial hypertension (IIH) remains unknown, and no consensus exists on how patients should be monitored and treated. Acetazolamide is a common treatment but has never been examined in a randomised controlled trial. The objectives of this pilot trial are to prospectively evaluate the use of acetazolamide, to explore various outcome measures and to inform the design of a definitive trial in IIH. Fifty patients were recruited from six centres over 23 months and randomised to receive acetazolamide (n = 25) or no acetazolamide (n = 25). Symptoms, body weight, visual function and health-related quality-of-life measures were recorded over a 12-month period. Recruited patients had typical features of mild IIH and most showed improvement, with 44% judged to have IIH in remission at the end of the trial. Difficulties with recruitment were highlighted as well as poor compliance with acetazolamide therapy (12 patients). A composite measure of IIH status was tested, and the strongest concordance with final disease status was seen with perimetry (Somers' D = 0.66) and optic disc appearance (D = 0.59). Based on the study data, a sample size of 320 would be required to demonstrate a 20% treatment effect in a substantive trial. Clinical trials in IIH require pragmatic design to involve sufficiently large numbers of patients. Future studies should incorporate weighted composite scores to reflect the relative importance of common outcome measures in IIH.
authors.affiliation: Department of Neurology, School of Experimental Medicine, College of Medical and Dental Sciences, University of Birmingham, Birmingham, UK. alexball@doctors.org.uk
authors.forename: Alexandra K
authors.initials: AK
authors.lastname: Ball
authors.forename: Andrew
authors.initials: A
authors.lastname: Howman
authors.forename: Keith
authors.initials: K
authors.lastname: Wheatley
authors.forename: Michael A
authors.initials: MA
authors.lastname: Burdon
authors.forename: Timothy
authors.initials: T
authors.lastname: Matthews
authors.forename: Andrew S
authors.initials: AS
authors.lastname: Jacks
authors.forename: Mark
authors.initials: M
authors.lastname: Lawden
authors.forename: Arul
authors.initials: A
authors.lastname: Sivaguru
authors.forename: Alexandra
authors.initials: A
authors.lastname: Furmston
authors.forename: Steven
authors.initials: S
authors.lastname: Howell
authors.forename: Basil
authors.initials: B
authors.lastname: Sharrack
authors.forename: M Brendan
authors.initials: MB
authors.lastname: Davies
authors.forename: Alexandra J
authors.initials: AJ
authors.lastname: Sinclair
authors.forename: Carl E
authors.initials: CE
authors.lastname: Clarke
mesh_terms: D000086:Acetazolamide / Q000627:therapeutic use*; D000293:Adolescent; D000328:Adult; D000368:Aged; D002257:Carbonic Anhydrase Inhibitors / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D018579:Patient Selection; D010865:Pilot Projects; D011559:Pseudotumor Cerebri / Q000188:drug therapy*; D011788:Quality of Life; D012107:Research Design; D016896:Treatment Outcome; D058609:Visual Field Tests; D055815:Young Adult
chemical_list: D002257:Carbonic Anhydrase Inhibitors; D000086:Acetazolamide