title: Response to treatment in a prospective national infantile spasms cohort.
abstract: 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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Departments of Pediatrics and Neurology, School of Medicine, University of Colorado Anschutz Medical Campus, Aurora, CO.
authors.forename: Kelly G
authors.initials: KG
authors.lastname: Knupp
authors.affiliation: Departments of Pediatrics and Neurology, School of Medicine, Oregon Health & Sciences University, Portland, OR.
authors.forename: Jason
authors.initials: J
authors.lastname: Coryell
authors.affiliation: Departments of Neurology and Pediatrics, Mayo Clinic, Rochester, MN.
authors.forename: Katherine C
authors.initials: KC
authors.lastname: Nickels
authors.affiliation: Division of Neurology, Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
authors.forename: Nicole
authors.initials: N
authors.lastname: Ryan
authors.affiliation: Colorado School of Public Health, Department of Biostatistics and Informatics, University of Colorado Anschutz Medical Campus, Aurora, CO.
authors.forename: Erin
authors.initials: E
authors.lastname: Leister
authors.affiliation: Division of Epilepsy and Clinical Neurophysiology, Department of Neurology, Boston Children's Hospital, Boston, MA.
authors.forename: Tobias
authors.initials: T
authors.lastname: Loddenkemper
authors.affiliation: Weill Cornell Medical Center, New York, NY.
authors.forename: Zachary
authors.initials: Z
authors.lastname: Grinspan
authors.affiliation: Departments of Neurology and Pediatrics, Johns Hopkins Hospital, Baltimore, MD.
authors.forename: Adam L
authors.initials: AL
authors.lastname: Hartman
authors.affiliation: Departments of Neurology and Pediatrics, Johns Hopkins Hospital, Baltimore, MD.
authors.forename: Eric H
authors.initials: EH
authors.lastname: Kossoff
authors.affiliation: Center for Neuroscience, Children's National Health System, Dallas, TX.
authors.forename: William D
authors.initials: WD
authors.lastname: Gaillard
authors.affiliation: Department of Pediatrics, Division of Pediatric Neurology, Ohio State University, Nationwide Children's Hospital, Columbus, OH.
authors.forename: John R
authors.initials: JR
authors.lastname: Mytinger
authors.affiliation: Department of Pediatrics & Communicable Diseases (Division of Pediatric Neurology), University of Michigan, Ann Arbor, MI.
authors.forename: Sucheta
authors.initials: S
authors.lastname: Joshi
authors.affiliation: Department of Pediatrics & Communicable Diseases (Division of Pediatric Neurology), University of Michigan, Ann Arbor, MI.
authors.forename: Renée A
authors.initials: RA
authors.lastname: Shellhaas
authors.affiliation: Departments of Pediatrics and Neurology, University of San Francisco, San Francisco, CA.
authors.forename: Joseph
authors.initials: J
authors.lastname: Sullivan
authors.affiliation: Division of Neurology, Children's Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA.
authors.forename: Dennis
authors.initials: D
authors.lastname: Dlugos
authors.affiliation: Department of Neurology, University of Washington, Seattle, WA.
authors.forename: Lorie
authors.initials: L
authors.lastname: Hamikawa
authors.affiliation: Ann & Robert H. Lurie Children's Hospital of Chicago and Departments of Pediatrics and Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL.
authors.forename: Anne T
authors.initials: AT
authors.lastname: Berg
authors.affiliation: Ann & Robert H. Lurie Children's Hospital of Chicago and Departments of Pediatrics and Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL.
authors.forename: John
authors.initials: J
authors.lastname: Millichap
authors.affiliation: Ann & Robert H. Lurie Children's Hospital of Chicago and Departments of Pediatrics and Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL.
authors.forename: Douglas R
authors.initials: DR
authors.lastname: Nordli
authors.affiliation: Departments of Neurology and Pediatrics, Mayo Clinic, Rochester, MN.
authors.forename: Elaine
authors.initials: E
authors.lastname: Wirrell
mesh_terms: D000284:Administration, Oral; D000305:Adrenal Cortex Hormones / Q000008:administration & dosage*; D000324:Adrenocorticotropic Hormone / Q000627:therapeutic use*; D000927:Anticonvulsants / Q000008:administration & dosage*; D002675:Child, Preschool; D015331:Cohort Studies; D005260:Female; D006801:Humans; D007223:Infant; D008297:Male; D015995:Prevalence; D011446:Prospective Studies; D012307:Risk Factors; D013036:Spasms, Infantile / Q000175:diagnosis / Q000188:drug therapy* / Q000453:epidemiology*; D014481:United States / Q000453:epidemiology; D020888:Vigabatrin / Q000627:therapeutic use*
chemical_list: D000305:Adrenal Cortex Hormones; D000927:Anticonvulsants; D000324:Adrenocorticotropic Hormone; D020888:Vigabatrin