title: Off-label drug use in hospitalized children.
abstract: OBJECTIVE
  To describe the magnitude of off-label drug use, to identify drugs most commonly used off-label, and to identify factors associated with off-label drug use in children hospitalized in the United States.
  
  
  METHODS
  Retrospective cohort study.
  
  
  METHODS
  Administrative database containing inpatient resource utilization data from January 1 to December 31, 2004, from 31 tertiary care pediatric hospitals in the United States.
  
  
  METHODS
  Hospitalized patients 18 years or younger.
  
  
  METHODS
  Institution and patient characteristics.
  
  
  METHODS
  Off-label drug use was defined as use of a specific drug in a patient younger than the Food and Drug Administration-approved age range for any indication of that drug.
  
  
  RESULTS
  At least 1 drug was used off-label in 297 592 (78.7%) of 355 409 patients discharged during the study. Off-label use accounted for $270 275 849 (40.5%) of the total dollars spent on these medications. Medications classified as central or autonomic nervous system agents or as fluids or nutrients, or gastrointestinal tract agents were most commonly used off-label, whereas antineoplastic agents were rarely used off-label. Factors associated with off-label use in multivariate analysis were as follows: undergoing a surgical procedure, age older than 28 days, greater severity of illness, and all-cause in-hospital mortality.
  
  
  CONCLUSIONS
  Most patients hospitalized at tertiary care pediatric institutions receive at least 1 medication outside the terms of the Food and Drug Administration product license. Substantial variation in the frequency of off-label use was observed across diagnostic categories and drug classes. Despite the frequent off-label use of drugs, using an administrative database, we cannot determine which of these treatments are unsafe or ineffective and which treatments result in substantial benefit to the patient.
authors.affiliation: Division of Infectious Diseases, Children's Hospital of Philadelphia, 34th Street & Civic Center Boulevard, Philadelphia, PA 19104, USA. shahs@email.chop.edu
authors.forename: Samir S
authors.initials: SS
authors.lastname: Shah
authors.forename: Matthew
authors.initials: M
authors.lastname: Hall
authors.forename: Denise M
authors.initials: DM
authors.lastname: Goodman
authors.forename: Pamela
authors.initials: P
authors.lastname: Feuer
authors.forename: Vidya
authors.initials: V
authors.lastname: Sharma
authors.forename: Crayton
authors.initials: C
authors.lastname: Fargason
authors.forename: Daniel
authors.initials: D
authors.lastname: Hyman
authors.forename: Kathy
authors.initials: K
authors.lastname: Jenkins
authors.forename: Marjorie L
authors.initials: ML
authors.lastname: White
authors.forename: Fiona H
authors.initials: FH
authors.lastname: Levy
authors.forename: James E
authors.initials: JE
authors.lastname: Levin
authors.forename: David
authors.initials: D
authors.lastname: Bertoch
authors.forename: Anthony D
authors.initials: AD
authors.lastname: Slonim
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D004348:Drug Labeling; D004363:Drug Utilization / Q000592:standards* / Q000706:statistics & numerical data*; D005260:Female; D006760:Hospitalization; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D012189:Retrospective Studies