Journal of Neurology

, Volume 258, Issue 5, pp 874–881

A randomised controlled trial of treatment for idiopathic intracranial hypertension

  • Alexandra K. Ball
  • Andrew Howman
  • Keith Wheatley
  • Michael A. Burdon
  • Timothy Matthews
  • Andrew S. Jacks
  • Mark Lawden
  • Arul Sivaguru
  • Alexandra Furmston
  • Steven Howell
  • Basil Sharrack
  • M. Brendan Davies
  • Alexandra J. Sinclair
  • Carl E. Clarke
Original Communication

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 

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Copyright information

© Springer-Verlag 2010

Authors and Affiliations

  • Alexandra K. Ball
    • 1
    • 10
  • Andrew Howman
    • 2
  • Keith Wheatley
    • 3
  • Michael A. Burdon
    • 4
  • Timothy Matthews
    • 4
  • Andrew S. Jacks
    • 4
  • Mark Lawden
    • 5
  • Arul Sivaguru
    • 6
  • Alexandra Furmston
    • 2
  • Steven Howell
    • 7
  • Basil Sharrack
    • 7
  • M. Brendan Davies
    • 8
  • Alexandra J. Sinclair
    • 9
  • Carl E. Clarke
    • 1
    • 6
  1. 1.Department of Neurology, School of Experimental Medicine, College of Medical and Dental SciencesUniversity of BirminghamBirminghamUK
  2. 2.Birmingham Clinical Trials UnitUniversity of BirminghamBirminghamUK
  3. 3.Cancer Research UK Clinical Trials UnitUniversity of BirminghamBirminghamUK
  4. 4.Birmingham and Midlands Eye CentreSandwell and Birmingham NHS TrustBirminghamUK
  5. 5.Department of Neurology, Leicester General HospitalUniversity Hospitals of LeicesterLeicesterUK
  6. 6.Department of Neurology, City HospitalSandwell and West Birmingham Hospitals NHS TrustBirminghamUK
  7. 7.Department of Neurology, Royal Hallamshire HospitalSheffield Teaching Hospitals NHS Foundation TrustSheffieldUK
  8. 8.Department of NeurologyUniversity Hospital of North Staffordshire NHS TrustStoke-on-TrentUK
  9. 9.Academic Unit of Ophthalmology, School of Immunology, Infection and Inflammation, College of Medical and Dental SciencesUniversity of BirminghamBirminghamUK
  10. 10.North Staffordshire Rehabilitation CentreUniversity Hospital of North Staffordshire NHS TrustStoke-on-TrentUK

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