ArticleAnnals of neurology2026
Idiopathic Intracranial Hypertension Is Characterized by a Distinct Proteomic Profile.
Article in Annals of neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
objectivesThe pathophysiology of idiopathic intracranial hypertension (IIH) is poorly understood and disease-specific biomarkers are lacking. We aimed to shed light on IIH pathophysiology and identify disease-specific biomarkers.
methodsThis prospective cross-sectional cohort study included patients with new-onset IIH and age-, body mass index-, and sex-matched healthy controls from 2 tertiary Danish Headache Centers (discovery cohort). Liquid chromatography coupled to tandem mass spectrometry analysis was used to measure reproducible proteomic profiles for paired serum and cerebrospinal fluid (CSF) samples. Results were validated using 3 validation cohorts comprising patients with IIH (pwIIH), cerebral sinus venous thrombosis (pwCSVT), and normal pressure hydrocephalus (pwNPH).
resultsWe included 53 pwIIH and 35 controls. PwIIH presented with papilledema and increased median lumbar puncture opening pressure (interquartile range [IQR]) (38.0 [33-46] vs 19.0 [17.0-23.0] cmH
interpretationWe identified a panel of independently validated IIH-specific candidate biomarkers. The serum candidate biomarkers pointed toward CA2 driven CSF hypersecretion in IIH providing a possible explanation for the therapeutic efficacy of CA inhibitors. The regulation of markers associated with neuronal impairment (APP, S100P/S100A12) confirmed the non-benign character of IIH. ANN NEUROL 2026;100:255-267.
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