Parent-defined target symptoms respond to risperidone in RUPP Autism study: Customer approach to clinical trials

L. Eugene Arnold, Benedetto Vitiello, Christopher McDougle, Larry Scahill, Bhavik Shah, Nilda M. Gonzalez, Shirley Chuang, Mark Davies, Jill Hollway, Michael G. Aman, Pegeen Cronin, Kathleen Koenig, Arlene E. Kohn, Donald J. McMahon, Elaine Tierney

Research output: Contribution to journalArticlepeer-review

151 Scopus citations


Objective: A consumer-oriented efficacy assessment in clinical trials should measure changes in chief complaint and consumer request (symptoms of most concern to patient/caregiver), which may be diluted in change scores of multisymptom scales. Method: In the Research Units on Pediatric Psychopharmacology (RUPP) Autism Network 8-week double-blind trial of risperidone versus placebo, the chief concerns of parents were collected at 0, 4, and 8 weeks (endpoint), in addition to standardized primary measures. Blinded clinical judges rated change from baseline to 4 and 8 weeks on a 9-point scale (1 = normalized, 5 = unchanged, 9 = disastrous); 94 participants had usable data. Results: The most common symptoms identified by parents were tantrums, aggression, and hyperactivity. Interrater reliability was excellent. Mean ratings at endpoint were 2.8 ± 1.2 on risperidone and 4.5 ± 1.3 on placebo (p < .001). Ratings were collinear with Clinical Global Impression-Improvement and Aberrant Behavior Checklist Irritability subscale (primary dimensional measure). Effect size d was 1.4, compared to 1.2 on the Aberrant Behavior Checklist Irritability subscale. Effect sizes varied twofold by symptom category, largest for self-injury (2.11) and tantrums (1.95). Conclusions: Risperidone was superior to placebo in reducing symptoms of most concern to parents of autistic children with irritable behavior. Rating individualized participant-chosen target symptoms seems a reliable, sensitive, efficient, and consumer-friendly way to assess treatment effect and might have clinical application.

Original languageEnglish (US)
Pages (from-to)1443-1450
Number of pages8
JournalJournal of the American Academy of Child and Adolescent Psychiatry
Issue number12
StatePublished - Dec 2003
Externally publishedYes


  • Assessment
  • Autism
  • Instruments
  • Pervasive developmental disorder
  • Treatment

ASJC Scopus subject areas

  • Developmental and Educational Psychology
  • Psychiatry and Mental health


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