A randomised controlled trial of treatment for idiopathic intracranial hypertension

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.

Incidence of Cerebellar Tonsillar Ectopia in Idiopathic Intracranial Hypertension: A Mimic of the Chiari I Malformation

“…2,5 Treatment consists of weight reduction, acetazolamide, and surgical intervention, including CSF shunt surgery. [6][7][8] Although IIH is a clinical diagnosis based on normal CSF composition with an elevated opening pressure (Ͼ20 cm H 2 O in nonobese patients and Ͼ25 cm H 2 O in obese patients with body mass index Ͼ30), supportive neuroimaging findings have been described. These include flattening of the posterior sclera, tortuosity of the optic nerve sheath, empty sella syndrome, and stenosis of the transverse venous sinuses.…”

Headache prevalence and clinical features in patients with idiopathic intracranial hypertension (IIH)

“…In fact, clear data on this topic are not available as only recently some reports have focused on the evaluation of the possible correlations between some clinical and instrumental aspects in the follow up of IIH patients [8][9][10][11].…”

Optic Neuropathy in Idiopathic Intracranial Hypertension

“…(34) Studies so far, could neither support nor deny the longtermbenefit of acetazolamide or any other type of medical therapy in the treatment of IIH. (35,36) …”