• DOI:10.1097/00004583-200312000-00011
• Corpus ID: 8263593

Parent-defined target symptoms respond to risperidone in RUPP autism study: customer approach to clinical trials.

@article{Arnold2003ParentdefinedTS,
  title={Parent-defined target symptoms respond to risperidone in RUPP autism study: customer approach to clinical trials.},
  author={L. Eugene Arnold and Benedetto Vitiello and Christopher J. McDougle and Lawrence Scahill and Bhavik Shah and N M Gonzalez and Shirley Z Chuang and Mark Davies and Jill Hollway and Michael G. Aman and Pegeen Cronin and Kathleen Koenig and Arlene E. Kohn and Donald J. McMahon and Elaine Tierney},
  journal={Journal of the American Academy of Child and Adolescent Psychiatry},
  year={2003},
  volume={42 12},
  pages={
          1443-50
        }
}

• L. E. Arnold, B. Vitiello, +12 authors E. Tierney
• Published 1 December 2003
• Medicine, Psychology
• Journal of the American Academy of Child and Adolescent Psychiatry

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.