Table 2 – Treatment Options for Infantile Spasms1,2

Treatment Time To Effect Duration Of Therapy Response Rate Comments

ACTH

14 days


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14 days then taper

86.7%

• Most accepted 1st line option

• Recent study indicates superiority over low-dose prednisolone

• Effective in treating spasms and eliminating hypsarrhythmia in symptomatic and cryptogenic cases of IS

• High relapse rate

• Side effects: hypertension, infection, weight gain, irritability

Corticosteroids

14 days

14 days then taper

Low-dose prednisolone: 25–59%

High-dose prednisolone: 67–80%

• Prednisolone often used

• Data is limited on use and appropriate dosing

• Relapse rate similar to ACTH or possibly lower

• Side effects: same as ACTH, may be more tolerable

Vigabatrin

14 days

6–9 months then taper

26–76% overall

Symptomatic cases: 27–59%

Cryptogenic cases: 35–82%

Tuberous sclerosis: >90%

• 1st line for patients with IS and tuberous sclerosis

• Response seen within 2 weeks and not after 12 weeks; discontinue if no response seen in 14 days

• Discontinue medication after 6 months to prevent peripheral visual field constriction

• Relapse rate: 16–21%

• Side effects: visual field loss, MRI changes, irritability, drowsiness, hypotonia

Second-line options

Treatment Time To Effect Duration Of Therapy Response Rate Comments

Ketogenic diet

1–3 months

At least 6 months

14–65% of patients are spasm-free in 1–3 months

• Used alone or in combination with another IS medication

• Higher efficacy seen in patients <1 years old and with cryptogenic IS

• Ketogenic formula available

• Side effects: constipation, nephrolithiasis, acidosis

Topiramate

>1 month

Unclear

10–48%

• Limited data available

• Side effects: anorexia, hypohidrosis, nephrolithiasis

Zonisamide

Up to 3 weeks

Unclear

26–41%

• Limited data available

Pyridoxine

Immediate (IV); 1–2 weeks (PO)

Once only (IV); lifelong (PO)

15%

• Limited data available

• Side effects: cardiorespiratory depression (IV), neuropathy at high doses (PO)