Vigabatrin for the treatment of infantile spasms: final report of a randomized trial

Area of Science:

• Pediatric Neurology
• Clinical Pharmacology

Background:

• Infantile spasms are a severe form of epilepsy in infants.
• Vigabatrin is a first-line treatment for infantile spasms, but optimal dosing is debated.

Purpose of the Study:

• To compare the efficacy of high-dose versus low-dose oral vigabatrin in achieving spasm cessation in infants with newly diagnosed infantile spasms.
• To evaluate the long-term safety and maintenance of spasm cessation with vigabatrin treatment.

Main Methods:

• A large, randomized, single-blind study involving 221 infants with newly diagnosed infantile spasms.
• Patients received either high-dose (100-148 mg/kg/d) or low-dose (18-36 mg/kg/d) oral vigabatrin for 14-21 days, followed by open-label treatment up to 3 years.
• Spasm cessation was defined as 7 consecutive days of spasm freedom confirmed by video-electroencephalogram within the first 14 days.

Main Results:

• Significantly more infants in the high-dose vigabatrin group achieved spasm cessation compared to the low-dose group (15.9% vs 7.0%; P = .0375).
• During follow-up, 23% of subjects relapsed, but 72% of those who relapsed regained spasm freedom.
• Adverse events were generally mild to moderate.

Conclusions:

• High-dose vigabatrin is more effective than low-dose vigabatrin in achieving rapid spasm cessation in infants with infantile spasms.
• Vigabatrin demonstrates a dose-dependent effect on spasm reduction and maintains efficacy in the majority of infants.
• The treatment is generally well-tolerated, with most adverse events being mild to moderate.