This infographic summarises the design and results of the Idiopathic Intracranial Hypertension (IIH) Pressure Medicine Study. This was a human experimental medicine study that utilised a randomised, sequential, crossover design. Women with active IIH were treated, in randomised order, for two weeks with acetazolamide, amiloride, furosemide, spironolactone and topiramate; with assessments at baseline and two weeks with a minimum one-week drug washout between drugs [1]. The trial was a landmark publication as it deployed highly accurate telemetric ICP devices [2]. It demonstrated ICP reduction with all drugs, except amiloride, but the magnitude of change was small. Patients reported significant side effects with acetazolamide and topiramate including cognitive decline. Cognitive measures were impaired by acetazolamide and topiramate.
References
Mitchell JL, Lyons HS, Walker JK, Yiangou A, Thaller M, Grech O, et al. A randomized sequential cross-over trial evaluating five purportedly ICP-lowering drugs in idiopathic intracranial hypertension. Headache. 2025;65:258–68. https://doi.org/10.1111/head.14897.
Mollan SP, Momin SNA, Khatkar PS, Grech O, Sinclair AJ, Tsermoulas G. A neuro-ophthalmologist’s guide to advances in intracranial pressure measurements. Eye Brain. 2023;15:113–24. https://doi.org/10.2147/EB.S404642.
Funding
This trial was funded by Enterprising Birmingham, University of Birmingham, UK, from 1 August 2016. Further funding was sought and from 1 August 2019 an investigator-led grant was obtained from Invex Therapeutics. AS is supported by a Sir Jules Thorn Award for Biomedical Research.
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Design and drafting – GB, ML. Review – JLM, AJS, SPM. All authors approved the final version.
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ML reports speaker fees (Pfizer); travel (AbbVie, Teva, Lundbeck). AS reports consultancy fees from Novartis, AbbVie, Vertex and Orion Pharma and speaker fees from Teva UK and Novartis. She was previously a Director and Chief Scientific Officer with shares at Invex Therapeutics. SM reports consultancy fees (Invex Therapeutics); advisory board fees (Ocular Therapeutix); speaker fees (Teva); Travel (AbbVie and European Alliance of Associations for Rheumatology); Receipt of equipment (Heidelberg Engineering). SM is a section editor at Eye Journal. Authors declare no other financial relationships with any organisations that might have an interest in the submitted work; and no other relationships or activities that could appear to have influenced the submitted work.
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Berman, G., Mitchell, J.L., Lowe, M. et al. Infographic: Landmark trials in Neuro-Ophthalmology —results of the randomised sequential cross-over trial evaluating five purportedly ICP lowering drugs in idiopathic intracranial hypertension. Eye (2025). https://doi.org/10.1038/s41433-025-03946-4
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DOI: https://doi.org/10.1038/s41433-025-03946-4
