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Identifying MicroRNA Biomarkers in Idiopathic Intracranial Hypertension: A Pilot Study

Hill LJ, Begum G, Thomas CN, Hubbard JC, Mugo C, Alimajstorovic Z, Botfield HF, Lyons HS et al.

Neurology, 2026

12 participants

Abstract

Background and objectives: Idiopathic intracranial hypertension (IIH) is characterized by raised intracranial pressure (ICP). It predominantly affects women with obesity and can cause disabling headaches and permanent visual loss. IIH is misdiagnosed in up to 40% of cases. Diagnosis and monitoring often rely on frequent invasive lumbar puncture. This pilot study aimed to identify serum microRNA (miRNA) markers associated with a diagnosis of IIH and disease activity and to assess their relevance in the CNS through identification in CSF.

Methods: Serum and CSF samples from participants in the IIH-Weight Trial (a randomized controlled trial comparing the effectiveness of bariatric surgery with that of a community weight management intervention in lowering ICP, recruited 2014-2017) were analyzed at baseline (active disease) and 12 months (participants in remission with no papilledema). Eligible participants were adult women with active IIH (those without papilledema were excluded). Disease activity was assessed by quantifying the degree of papilledema using Spectralis spectral-domain optical coherence tomography. A panel of 40 candidate miRNAs was assessed. Comparator groups included participants with obesity or migraine. Analyses used Student t tests and one-way ANOVA followed by Tukey multiple comparisons test. Differentially expressed miRNAs were evaluated in relation to clinical disease activity and metabolic profiles, using linear regression analysis.

Results: Five of 40…

How strong is the evidence?

Evidence Hub reads this as a randomized trial, done in people. Its evidence level is 3 of 5 (Moderate · trial / cohort / secondary analysis). Trials without a large randomized design, cohorts and secondary analyses give moderate evidence. Reported size: 12 participants or records.

How the five levels of evidence work. The level is assigned automatically from the abstract and can be wrong, so check the paper.

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