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A Risk-Benefit Assessment of Treatments for Infantile Spasms

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Abstract

Infantile spasms are a devastating epileptic encephalopathy of the young child. The continuing spasms and hypsarrhythmia have a deleterious effect on brain maturation and further cognitive development.

Corticotropin (adrenocorticotropic hormone) or corticosteroids have been the gold standard treatment for the last 40 years, but there is little agreement on the best agent to use, or the dosage and duration of the treatment. Despite this empirical approach, corticotropin or corticosteroids are effective in controlling spasms and normalising electroencephalograms in about 60% of cases. The major concern with this treatment is the occurrence of frequent and severe adverse effects.

The introduction of vigabatrin in the 1990s improved the outcome of infantile spasms. Vigabatrin shows an efficacy at least equal to that of corticosteroids, and even higher in specific groups such as those with tuberous sclerosis. The major advantages of vigabatrin are the ability to initiate treatment at the full dosage, rapid efficacy, suitability for outpatient treatment and particularly good tolerability with only minor adverse effects. Recently, however, the safety of vigabatrin has caused concern since a specific visual field loss has been reported in treated adults.

The current problem is determining the risk-benefit ratio of vigabatrin and corticosteroids/corticotropin in children with infantile spasms, and to specify the groups where their use could be optimal. Visual field loss is usually asymptomatic and can be detected only by perimetric visual field studies. In children, especially in the young or disabled, it is difficult if not impossible to detect the visual field loss and it is not yet known if children are at higher or lower risk for this adverse effect. Until a clear answer about the occurrence of this adverse effect in children has been established through randomised study, vigabatrin may still be considered first-line therapy in infantile spasms. Children who do not achieve a good response to vigabatrin should be switched to corticotropin/corticosteroid therapy.

Despite the efficacy of corticosteroids and vigabatrin, the use of the conventional antiepileptic drugs, the newly developed antiepileptic drugs and some promising results with ketogenic diet, 25 to 30% of patients with infantile spasms continue to have spasms and experience psychomotor regression. These drug-resistant patients could be candidates for surgery.

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Acknowledgements

The author wishes to thank Dr Catherine Chiron and Professor Olivier Dulac for their advice.

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  1. Department of Neuropediatrics, Hôpital St Vincent de Paul, 74-72 avenue Denfert Rochereau, 75014, Paris, France

    Rima Nabbout

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Nabbout, R. A Risk-Benefit Assessment of Treatments for Infantile Spasms. Drug-Safety 24, 813–828 (2001). https://doi.org/10.2165/00002018-200124110-00003

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