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Objective: Idiopathic intracranial hypertension (IIH) is a neurometabolic disease with an increasing incidence. The pathophysiology is unknown, but improvement of diagnosis and management requires discovery of novel biomarkers. Our objective was to identify such candidate biomarkers in IIH, and secondarily, test for associations between identified metabolites and disease severity. Methods: This is a prospective case–control study with collection of cerebrospinal fluid (CSF), serum, and clinical data from new‐onset, treatment‐naïve patients with IIH (n = 60). Patients were included consecutively from 2 tertiary headache centers in Denmark, and age, sex, and body mass index (BMI) ‐matched healthy controls (n = 35) were recruited. Clinical data were retrieved at ocular remission (n = 55). Samples were analyzed using non‐targeted mass spectrometry. Results: Serum sphingosine 1‐phosphate (S1P), adenosine, and glutamate were 0.46‐fold (q < 0.0001), 0.25‐fold (q = 0.0048), and 0.44‐fold (q < 0.0001) lower, respectively, in IIH. CSF stearoyl‐lysophosphatidylcholine (LysoPC‐18) and 2‐palmitoyl‐lysophosphatidylcholine (LysoPC‐16) were 0.42 (q = 0.0025) and 0.37 (q < 0.001) ‐fold lower. LysoPC‐18 was higher in patients with moderate–severe versus mild papilledema (p = 0.022). LysoPC‐18 correlated positively with retinal nerve fiber layer thickness (p = 0.0012, r = 0.42) and inversely with mean deviation on automated perimetry (p = 0.01, r = −0.35). Higher baseline serum S1P (p = 0.018) and lower CSF LysoPC‐16 (p = 0.003) were associated with optic nerve atrophy at ocular remission. Pathway analysis suggests dysregulated lipid metabolism and redox disturbances in new‐onset IIH. Interpretation: We identify perturbed metabolism in new‐onset IIH. S1P and LysoPC‐16 demonstrate potential prognostic value due to association with subsequent optic nerve atrophy. This association between specific, differential metabolites and outcome provides substantial evidence for novel biomarkers of clinical significance that should be the focus of further targeted studies. ANN NEUROL 2024;96:595–607 [ABSTRACT FROM AUTHOR]

We aimed to systematically examine the optic nerve head anatomy in patients with idiopathic intracranial hypertension (IIH) using a standardized optical coherence tomography (OCT) protocol. The study retrospectively included 32 patients diagnosed from 2014 to 2021 with IIH. Using OCT, in accordance with a standardized scanning protocol for patients with optic disc drusen, the presence of optic disc drusen, prelaminar hyperreflective lines, peripapillary hyperreflective ovoid mass-like structures, the retinal nerve fiber layer thickness, and macular ganglion cell layer volume was obtained. Optic disc drusen were found in 3.1%, hyperreflective lines in 31.3%, and peripapillary hyperreflective ovoid mass-like structures in 81.3% of all IIH patients at least three months after the time of diagnosis. We found no significant differences in retinal nerve fiber layer thickness or macular ganglion cell layer volume in patients with hyperreflective lines or PHOMS respectively compared to patients without hyperreflective lines (p = 0.1285 and p = 0.1835). In conclusion, the prevalence of optic disc drusen in IIH patients is similar to the reported prevalence in the general population. The high prevalence of hyperreflective lines and peripapillary hyperreflective ovoid mass-like structures in IIH patients suggest these structures be a result of crowding in the optic nerve head caused by papilledema. [ABSTRACT FROM AUTHOR]