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, and acetazolamide should be initiated 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 (most useful imaging modality) to ensure normal brain parenchyma without hydrocephalus, mass, or structural lesions 1, 2
• CT or MR venography to exclude cerebral venous sinus thrombosis 1, 2
• Lumbar puncture in lateral decubitus position showing opening pressure >200 mm H₂O (or >250 mm H₂O for definitive diagnosis) with normal CSF composition 1, 2
• Fundoscopic examination confirming papilledema 2
• Formal visual field testing to establish baseline and guide treatment intensity 3

Treatment Algorithm Based on Severity

For All Patients (Regardless of Severity)

Identify and discontinue causative medications including tetracyclines, vitamin A, retinoids, steroids, growth hormone, thyroxine, and lithium 1

Mild Visual Loss

Start acetazolamide as first-line medical therapy with gradual dose escalation as tolerated 1, 3

Initiate weight loss program targeting 5-10% weight reduction with low-sodium diet in overweight patients 1, 3

• Weight loss alone can induce remission in some patients 4
• Topiramate may be considered as it suppresses appetite and provides carbonic anhydrase inhibition 1
• Zonisamide serves as an alternative when topiramate causes excessive side effects 1

Furosemide may be added as a second-line agent if acetazolamide alone is insufficient 5

Severe or Rapidly Progressive Visual Loss

Surgical intervention is required when visual function is declining acutely 2, 4

Optic Nerve Sheath Fenestration (ONSF):

• Effective and safe procedure that may be repeated if initially unsuccessful 1
• Fewer complications than CSF diversion procedures 1
• Must be performed only by experienced clinicians trained in this technique 1

Ventriculoperitoneal (VP) shunt:

• Preferred CSF diversion procedure due to lower reported revision rates compared to lumboperitoneal shunts 1
• Patients must inform relevant driving agencies following VP shunt placement 1
• Limited effectiveness for headache alone (68% continue having headaches at 6 months, 79% at 2 years) 1

Neurovascular stenting:

• May improve symptoms but carries risks including headache, stent-adjacent stenosis, and rare serious complications 1

Headache Management

Recognize that IIH patients often develop migrainous headaches superimposed on raised intracranial pressure headaches, requiring specific migraine treatment approaches beyond ICP-lowering therapies 1, 6

Mild headaches can be treated with NSAIDs or migraine prophylactic agents 5

What NOT to Do

Avoid serial lumbar punctures for management of IIH 1

Never use acetazolamide combined with furosemide in preterm infants as this combination increases mortality and neurological morbidity 1

Do not perform ventricular puncture routinely as it increases risk of CSF infection and loculated hydrocephalus 1

Do not allow patients to donate blood during or for at least 1 year following discontinuation of acetazolamide if they were on retinoid-class medications 1

Monitoring and Expected Outcomes

Serial visual field testing and fundoscopic examinations are essential to detect treatment failure 3

Be aware that treatment failure rates are substantial:

• 34% experience worsening vision after stabilization at 1 year 1
• 45% at 3 years 1
• One-third to one-half fail to improve headache despite treatment 1

Once papilledema resolves, hospital-based visual monitoring may no longer be required, but exercise caution with asymptomatic patients who may remain asymptomatic during recurrence 1

Special Populations

Pregnant women and children require special treatment considerations, though acetazolamide and weight management remain foundational 5

Severely obese patients may benefit from bariatric surgery as an effective long-term treatment, though it is not useful for acute intervention 5