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Response to treatment in a prospective national infantile spasms cohort
Kelly G Knupp1, Jason Coryell2, Katherine C Nickels3
1Departments of Pediatrics and Neurology, School of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO.
Insights
Standard treatments like ACTH are effective for infantile spasms. Early and sustained response rates varied by treatment choice, with ACTH showing higher efficacy than other options.
Area of Science:
- Pediatric Neurology
- Epileptology
Background:
- Infantile spasms (IS) are a severe epileptic encephalopathy in infants under two years.
- Optimal treatment strategies for IS remain a subject of debate.
Purpose of the Study:
- To evaluate early and sustained treatment response in infantile spasms.
- To compare clinical remission and electrographic resolution of hypsarrhythmia.
- To assess if treatment response differs by etiology or developmental status.
Main Methods:
- A multicenter, prospective database of infants with new IS diagnoses was established.
- Response was defined as sustained clinical remission and hypsarrhythmia resolution at 3 months.
- Standard therapies (ACTH, oral corticosteroids, vigabatrin) were analyzed individually; nonstandard therapies collectively.
- Etiology and developmental status were assessed; response rates compared using chi-square and logistic regression.
Main Results:
- Overall, 46% of infants on standard therapy responded versus 9% on nonstandard therapy (p < 0.001).
- Response rates for initial treatments: ACTH (55%), oral corticosteroids (39%), vigabatrin (36%), other (9%) (p < 0.001).
- Etiology or developmental status did not significantly alter treatment response patterns.
Conclusions:
- Treatment choice significantly impacts infantile spasms response rates.
- Standard therapies, including ACTH, oral corticosteroids, and vigabatrin, are recommended as initial treatments for IS.
- Adrenocorticotropic hormone (ACTH) demonstrated superior efficacy compared to other standard therapies for infantile spasms.
Objective:
Infantile spasms are seizures associated with a severe epileptic encephalopathy presenting in the first 2 years of life, and optimal treatment continues to be debated. This study evaluates early and sustained response to initial treatments and addresses both clinical remission and electrographic resolution of hypsarrhythmia. Secondarily, it assesses whether response to treatment differs by etiology or developmental status.
Methods:
The National Infantile Spasms Consortium established a multicenter, prospective database enrolling infants with new diagnosis of infantile spasms. Children were considered responders if there was clinical remission and resolution of hypsarrhythmia that was sustained at 3 months after first treatment initiation. Standard treatments of adrenocorticotropic hormone (ACTH), oral corticosteroids, and vigabatrin were considered individually, and all other nonstandard therapies were analyzed collectively. Developmental status and etiology were assessed. We compared response rates by treatment group using chi-square tests and multivariate logistic regression models.
Results:
Two hundred thirty infants were enrolled from 22 centers. Overall, 46% of children receiving standard therapy responded, compared to only 9% who responded to nonstandard therapy (p < 0.001). Fifty-five percent of infants receiving ACTH as initial treatment responded, compared to 39% for oral corticosteroids, 36% for vigabatrin, and 9% for other (p < 0.001). Neither etiology nor development significantly modified the response pattern by treatment group.
Interpretation:
Response rate varies by treatment choice. Standard therapies should be considered as initial treatment for infantile spasms, including those with impaired development or known structural or genetic/metabolic etiology. ACTH appeared to be more effective than other standard therapies.
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