Table 2 – Treatment Options for Infantile Spasms1,2
Treatment | Time To Effect | Duration Of Therapy | Response Rate | Comments | ||||||||||||||||||||
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ACTH |
14 days |
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 |
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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 |
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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 |
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Topiramate |
>1 month |
Unclear |
10–48% |
• Limited data available • Side effects: anorexia, hypohidrosis, nephrolithiasis |
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Zonisamide |
Up to 3 weeks |
Unclear |
26–41% |
• Limited data available |
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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) |