Infantile Spasms: Diagnosis and Treatment

Table 2 – Treatment Options for Infantile Spasms^1,2

Treatment	Time To Effect	Duration Of Therapy	Response Rate	Comments
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
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%	• 1^st 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

Treatment

Time To Effect

Duration Of Therapy

Response Rate

Comments

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

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%

• 1^st 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)

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