Original Communication

Journal of Neurology

, Volume 258, Issue 5, pp 874-881

First online:

A randomised controlled trial of treatment for idiopathic intracranial hypertension

  • Alexandra K. BallAffiliated withDepartment of Neurology, School of Experimental Medicine, College of Medical and Dental Sciences, University of BirminghamNorth Staffordshire Rehabilitation Centre, University Hospital of North Staffordshire NHS Trust Email author 
  • , Andrew HowmanAffiliated withBirmingham Clinical Trials Unit, University of Birmingham
  • , Keith WheatleyAffiliated withCancer Research UK Clinical Trials Unit, University of Birmingham
  • , Michael A. BurdonAffiliated withBirmingham and Midlands Eye Centre, Sandwell and Birmingham NHS Trust
  • , Timothy MatthewsAffiliated withBirmingham and Midlands Eye Centre, Sandwell and Birmingham NHS Trust
  • , Andrew S. JacksAffiliated withBirmingham and Midlands Eye Centre, Sandwell and Birmingham NHS Trust
  • , Mark LawdenAffiliated withDepartment of Neurology, Leicester General Hospital, University Hospitals of Leicester
  • , Arul SivaguruAffiliated withDepartment of Neurology, City Hospital, Sandwell and West Birmingham Hospitals NHS Trust
  • , Alexandra FurmstonAffiliated withBirmingham Clinical Trials Unit, University of Birmingham
    • , Steven HowellAffiliated withDepartment of Neurology, Royal Hallamshire Hospital, Sheffield Teaching Hospitals NHS Foundation Trust
    • , Basil SharrackAffiliated withDepartment of Neurology, Royal Hallamshire Hospital, Sheffield Teaching Hospitals NHS Foundation Trust
    • , M. Brendan DaviesAffiliated withDepartment of Neurology, University Hospital of North Staffordshire NHS Trust
    • , Alexandra J. SinclairAffiliated withAcademic Unit of Ophthalmology, School of Immunology, Infection and Inflammation, College of Medical and Dental Sciences, University of Birmingham
    • , Carl E. ClarkeAffiliated withDepartment of Neurology, School of Experimental Medicine, College of Medical and Dental Sciences, University of BirminghamDepartment of Neurology, City Hospital, Sandwell and West Birmingham Hospitals NHS Trust

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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.

Keywords

Idiopathic intracranial hypertension Randomised controlled clinical trials