What is the treatment for pseudotumor cerebri (idiopathic intracranial hypertension)?

Treatment of Pseudotumor Cerebri (Idiopathic Intracranial Hypertension)

Weight loss is the first-line treatment for overweight patients with pseudotumor cerebri, while acetazolamide should be initiated simultaneously as the primary medical therapy for patients with mild visual loss. 1

Initial Diagnostic Confirmation

Before initiating treatment, confirm the diagnosis with:

• MRI of the head and orbits (with and without contrast) to exclude secondary causes and evaluate for signs of elevated intracranial pressure including posterior globe flattening, optic nerve tortuosity, and empty sella 1, 2, 3
• MR venography or CT venography to exclude cerebral venous sinus thrombosis or stenosis 1, 3
• Lumbar puncture demonstrating opening pressure >250 mm H₂O (or >280 mm H₂O in children) with normal CSF composition 1, 3
• Ophthalmologic examination documenting papilledema and baseline visual function 3

Treatment Algorithm

Step 1: Identify and Remove Causative Factors

Discontinue medications that may cause or exacerbate intracranial hypertension, including tetracyclines, vitamin A, retinoids, steroids, growth hormone, thyroxine, and lithium 1

Step 2: Weight Management (Disease-Modifying Therapy)

For patients with BMI >30 kg/m²:

• Counsel about weight management at the earliest opportunity with a target of 5-15% weight loss, which can induce remission 3
• Refer to community or hospital-based weight management programs 3
• Consider bariatric surgery for sustained weight loss in appropriate candidates 3
• Topiramate may assist with weight loss through appetite suppression while also reducing intracranial pressure via carbonic anhydrase inhibition 1

Step 3: Medical Therapy

Acetazolamide is the first-line medication for patients with mild visual loss:

• Start at a low dose and gradually increase as needed and tolerated 1
• The Idiopathic Intracranial Hypertension Treatment Trial established acetazolamide as proven first-line therapy 4
• Zonisamide may be an alternative where topiramate causes excessive side effects 1

Important caveat: Serial lumbar punctures are NOT recommended for long-term management of IIH 1

Step 4: Surgical Intervention

Surgical treatment is indicated for:

• Imminent risk of visual loss or rapidly progressive visual decline requiring urgent intervention 3
• Failure of medical management with progressive vision loss 1

Surgical options in order of preference:

1. Ventriculoperitoneal (VP) shunt is the preferred CSF diversion procedure due to lower reported revision rates compared to lumboperitoneal shunts 1, 3

2. Optic Nerve Sheath Fenestration (ONSF) is effective and safe with fewer complications than CSF diversion procedures, but should only be performed by experienced clinicians trained in this technique 1

3. Neurovascular stenting may improve symptoms in patients with venous sinus stenosis, though potential complications include headache, stent-adjacent stenosis, and rare serious events 1

Temporizing measure: A lumbar drain may protect vision while planning definitive surgery in acute cases 3

Headache Management

Recognize that patients often develop migrainous headaches superimposed on headaches from raised intracranial pressure, requiring specific migraine treatment beyond pressure-lowering therapies 1

Important limitation: CSF shunting to exclusively treat headache has limited evidence, with 68% of patients continuing to have headaches at 6 months and 79% by 2 years 1

Monitoring and Follow-up

• Regular ophthalmologic assessments to monitor papilledema and visual function 3
• Be aware of high treatment failure rates: worsening vision after stabilization occurs in 34% at 1 year and 45% at 3 years 1
• Once papilledema resolves, visual monitoring within hospital services may no longer be required, but exercise caution with asymptomatic patients who may remain asymptomatic during recurrence 1
• If significant deterioration of visual function occurs, consider diagnostic lumbar puncture 3

Critical Pitfalls to Avoid

• Do NOT use acetazolamide combined with furosemide in preterm infants, as this combination increases mortality and neurological morbidity 5
• Do NOT perform ventricular puncture routinely, as it increases risk of CSF infection and loculated hydrocephalus 5
• Patients must inform the relevant driving agency following VP shunt placement 1
• Patients should not donate blood during or for at least 1 year following discontinuation of acetazolamide therapy if they were on retinoid-class medications 5

Special Populations

For non-obese patients, thoroughly investigate secondary causes including medications, endocrine disorders, and cerebral venous abnormalities 3