Assessment in multisite randomized clinical trials of patients with autistic disorder: The autism RUPP network

L. Eugene Arnold, Michael G. Aman, Andres Martin, Angie Collier-Crespin, Benedetto Vitiello, Elaine Tierney, Robert Asarnow, Felicia Bell-Bradshaw, Betty Jo Freeman, Patricia Gates-Ulanet, Ami Klin, James T. McCracken, Christopher J. McDougle, James J. McGough, David J. Posey, Lawrence Scahill, Naomi Swiezy, Louise Ritz, Fred Volkmar

Research output: Contribution to journalArticle

91 Citations (Scopus)

Abstract

Assessment of autistic disorder (autism) symptoms, primary and secondary, poses more challenging problems than ordinarily found in multisite randomized clinical trial (RCT) assessments. For example, subjects may be uncommunicative and extremely heterogeneous in problem presentation, and current pharmacological treatments are not likely to alter most core features of autism. The Autism Research Units on Pediatric Psychopharmacology (RUPP Autism Network) resolved some of these problems during the design of a risperidone RCT in children/adolescents. The inappropriateness of the usual anchors for a Clinical Global Impression of Severity (CGI-S) was resolved by defining uncomplicated autism without secondary symptoms as a CGI-S of 3, mildly ill. The communication problems, compromising use of the patient as an informant, were addressed by several strategies, including careful questioning of care providers, rating scales, laboratory tests, and physical exams. The broad subject heterogeneity requires outcome measures sensitive to individual change over a wide spectrum of treatment response and side effects. The problems of neuropsychologically testing nonverbal, lower functioning, sometimes noncompliant subjects requires careful instrument selection/adaptation and flexible administration techniques. The problems of assessing low-end IQs, neglected by most standardized test developers, was resolved by an algorithm of test hierarchy. Scarcity of other autism-adapted cognitive and neuropsychological tests and lack of standardization required development of a new, specially adapted battery. Reliability on the Autism Diagnostic Interview (currently the most valid diagnostic instrument) and other clinician instruments required extensive cross-site training (in- person, videotape, and teleconference sessions). Definition of a treatment responder required focus on individually relevant target symptoms, synthesis of possible modest improvements in many domains, and acceptance of attainable though imperfect goals. The assessment strategy developed is implemented in a RCT of risperidone (McDougle et al., 2000) for which the design and other methodological challenges are described elsewhere (Scahill et al., 2000). Some of these problems and solutions are partially shared with RCTs of other treatments and other disorders.

Original languageEnglish
Pages (from-to)99-111
Number of pages13
JournalJournal of Autism and Developmental Disorders
Volume30
Issue number2
DOIs
StatePublished - 2000
Externally publishedYes

Fingerprint

Autistic Disorder
Randomized Controlled Trials
Risperidone
Telecommunications
Psychopharmacology
Videotape Recording
Neuropsychological Tests
Therapeutics
Communication
Outcome Assessment (Health Care)
Pharmacology
Interviews
Pediatrics
Research

Keywords

  • Autism
  • Clinical trials
  • Risperidone

ASJC Scopus subject areas

  • Psychology(all)
  • Developmental and Educational Psychology

Cite this

Arnold, L. E., Aman, M. G., Martin, A., Collier-Crespin, A., Vitiello, B., Tierney, E., ... Volkmar, F. (2000). Assessment in multisite randomized clinical trials of patients with autistic disorder: The autism RUPP network. Journal of Autism and Developmental Disorders, 30(2), 99-111. https://doi.org/10.1023/A:1005451304303

Assessment in multisite randomized clinical trials of patients with autistic disorder : The autism RUPP network. / Arnold, L. Eugene; Aman, Michael G.; Martin, Andres; Collier-Crespin, Angie; Vitiello, Benedetto; Tierney, Elaine; Asarnow, Robert; Bell-Bradshaw, Felicia; Freeman, Betty Jo; Gates-Ulanet, Patricia; Klin, Ami; McCracken, James T.; McDougle, Christopher J.; McGough, James J.; Posey, David J.; Scahill, Lawrence; Swiezy, Naomi; Ritz, Louise; Volkmar, Fred.

In: Journal of Autism and Developmental Disorders, Vol. 30, No. 2, 2000, p. 99-111.

Research output: Contribution to journalArticle

Arnold, LE, Aman, MG, Martin, A, Collier-Crespin, A, Vitiello, B, Tierney, E, Asarnow, R, Bell-Bradshaw, F, Freeman, BJ, Gates-Ulanet, P, Klin, A, McCracken, JT, McDougle, CJ, McGough, JJ, Posey, DJ, Scahill, L, Swiezy, N, Ritz, L & Volkmar, F 2000, 'Assessment in multisite randomized clinical trials of patients with autistic disorder: The autism RUPP network', Journal of Autism and Developmental Disorders, vol. 30, no. 2, pp. 99-111. https://doi.org/10.1023/A:1005451304303
Arnold, L. Eugene ; Aman, Michael G. ; Martin, Andres ; Collier-Crespin, Angie ; Vitiello, Benedetto ; Tierney, Elaine ; Asarnow, Robert ; Bell-Bradshaw, Felicia ; Freeman, Betty Jo ; Gates-Ulanet, Patricia ; Klin, Ami ; McCracken, James T. ; McDougle, Christopher J. ; McGough, James J. ; Posey, David J. ; Scahill, Lawrence ; Swiezy, Naomi ; Ritz, Louise ; Volkmar, Fred. / Assessment in multisite randomized clinical trials of patients with autistic disorder : The autism RUPP network. In: Journal of Autism and Developmental Disorders. 2000 ; Vol. 30, No. 2. pp. 99-111.
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