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Outcome Measures for Clinical Drug Trials in Autism

Published online by Cambridge University Press:  07 November 2014

Michael G. Aman ,
Sherie Novotny ,
Carole Samango-Sprouse ,
Luc Lecavalier ,
Elizabeth Leonard ,
Kenneth D. Gadow ,
Bryan H. King ,
Deborah A. Pearson ,
Morton Ann Gernsbacher  and
Michael Chez
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Abstract

This paper identifies instruments and measures that may be appropriate for randomized clinical trials in participants with autism spectrum disorders (ASDs). The Clinical Global Impressions scale was recommended for all randomized clinical trials. At this point, however, there is no “perfect” choice of outcome measure for core features of autism, although we will discuss five measures of potential utility. Several communication instruments are recommended, based in part on suitability across the age range. In trials where the intention is to alter core features of ASDs, adaptive behavior scales are also worthy of consideration. Several “behavior complexes” common to ASDs are identified, and instruments are recommended for assessment of these. Given the prevalence of cognitive impairment in ASDs, it is important to assess any cognitive effects, although cognitive data from ASD randomized clinical trials, thus far, are minimal. Guidance from trials in related pharmacologic areas and behavioral pharmacology may be helpful. We recommend routine elicitation of side effects, height and weight, vital signs, and (in the case of antipsychotics) extrapyramidal side-effects assessment. It is often appropriate to include laboratory tests and assessments for continence and sleep pattern.

Type
Review Article
Information
CNS Spectrums , Volume 9 , Issue 1 , January 2004 , pp. 36 - 47
Copyright
Copyright © Cambridge University Press 2004

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References

REFERENCES

1.Guy, W. ECDEU Assessment Manuel for Psychopharmacology, Revised. Rockville, MD: National Institute of Mental Health. US Department Health Education and Welfare Pub (ADM). 1976;76–338.Google Scholar
2.Lord, C, Rutter, M, DiLavre, PC. Autism Diagnostic Observation Schedule-Generic (ADOS-G). San Antonio, Tex: Psychological Corp; 1998.Google Scholar
3.Schopler, E, Reichler, RJ, Devellis, RF, Daly, K. Toward objective classification of childhood autism. J Autism Dev Disord. 1980;10:91–103.CrossRefGoogle ScholarPubMed
4.Schopler, E, Reichler, RJ, Renner, BR. The Childhood Autism Rating Scale. Los Angeles, Calif: Western Psychological Services; 1988.Google Scholar
5.Constantino, JN, Davis, SA, Reich, W, et al.Validation of a brief quantitative measure of autistic traits: comparison of the Social Responsiveness Scale with the Autism Diagnostic Interview-Revised. J Autism Dev Disord. In press.Google Scholar
6.Matson, JL. Matson Evaluation of Social Skills with Youngsters. Worthington, Ohio: IDS Publishing; 1990.Google Scholar
7.Gilliam, JE. Gilliam Autism Rating Scale. Austin, Tex: Pro-Ed; 1995.Google Scholar
8.Freeman, BJ, Ritvo, ER, Yokota, A, Ritvo, A. A scale for rating symptoms of patients with the syndrome of autism in real life settings. J Am Acad Child Adolesc Psychiatry. 1986;25:130–136.CrossRefGoogle ScholarPubMed
9.Krug, DA, Arick, J, Almond, P. Autism Screening Instrument for Educational Planning. Portland, Or: ASIEP Educational Co.; 1980.Google Scholar
10.Belsito, KM, Law, PA, Kirk, KS, Landa, RJ, Zimmerman, AW. Lamotrigine therapy for autistic disorder: a randomized, double-blind, placebo controlled trial. J Autism Dev Disord. 2001;31:175–181.CrossRefGoogle ScholarPubMed
11.Owley, T, Steele, E, Corsello, C, et al.A double-blind, placebo-controlled trial of secretin for the treatment of autistic disorder. Med Gen Med. 1999;E2.Google Scholar
12.Owley, T, McMahon, W, Cook, EH, et al.Multisite, double-blind, placebo-controlled trial of porcine secretin in autism. J Am Acad Child Adolesc Psychiatry. 2001;40:1293–1299.CrossRefGoogle ScholarPubMed
13.Mesibov, GB, Schopler, E, Schaffer, B, Michal, N. Use of the Childhood Autism Rating Scale with autistic adolescents and adults. J Am Acad Child Adolesc Psychiatry. 1989;28:538–541.CrossRefGoogle ScholarPubMed
14.Tobin, LE, Glenwick, DS. Relation of the Childhood Autism Rating Scale-Parent version to diagnosis, stress, and age. Res Dev Disabil. 2002;23:211–223.CrossRefGoogle Scholar
15.DiLalla, DL, Rodgers, SJ. Domains of the Childhood Autism Rating Scale: relevance for diagnosis and treatment. J Aut Dev Disord. 1994;24:115–128.CrossRefGoogle ScholarPubMed
16.Eaves, RC, Milner, B. The criterion-related validity of the Childhood Autism Rating Scale and the Autism Behavior Checklist. J Abnorm Child Psychol. 1993;21:481–491.CrossRefGoogle ScholarPubMed
17.Pilowsky, T, Yirmiya, N, Shulman, C, Ronit, D. The Autism Diagnostic Interview-Revised and the Childhood Autism Rating Scale: differences between diagnostic systems and comparison between genders. J Aut Dev Disord. 1998;28:143–151.CrossRefGoogle ScholarPubMed
18.Sevin, JA, Matson, JL, Coe, DA, Fee, VE, Sevin, BM. A comparison and evaluation of three commonly used autism scales. J Aut Dev Disord. 1991;21:417–432.CrossRefGoogle ScholarPubMed
19.Constantino, JN, Hudziak, JJ, Todd, RD. Deficits in reciprocal social behavior in male twins: evidence for a genetically independent domain of psychopathology. J Am Acad Child Adolesc Psychiatry. 2003;42:458–467.CrossRefGoogle ScholarPubMed
20.Constantino, JN, Przybeck, T, Friesen, D, Todd, RD. Reciprocal social behavior in children with and without pervasive developmental disorders. J Dev Behav Pediatr. 2000;21:2–11.CrossRefGoogle ScholarPubMed
21.South, M, Williams, BJ, McMahon, WM, et al.Utility of the Gilliam Autism Rating Scale in research and clinical populations. J Autism Dev Disord. 2002;32:593–599.CrossRefGoogle ScholarPubMed
22.Matson, JL, Compton, LS, Sevin, JA. Comparison and item analysis of the MESSY for autistic and normal children. Res Dev Disabil. 1991;12:361–369.CrossRefGoogle ScholarPubMed
23.Njardvik, U, Matson, JL, Cherry, KE. A comparison of social skills in adults with autistic disorder, pervasive developmental disorder not otherwise specified, and mental retardation. J Autism Dev Disord. 1999;29:287–295.CrossRefGoogle Scholar
24.Krug, DA, Arick, J, Almond, P. Behavior checklist for identifying severely handicapped individuals with high levels of autistic behavior. J Child Psychol Psychiatry. 1980;21:221–229.CrossRefGoogle ScholarPubMed
25.Kelley, E, Jones, G, Fein, D. Language assessment in children. In: Goldstein, G, Beers, SR, eds. The Comprehensive Handbook of Psychological Assessment, Volume 1: Intellectual and Neuropsychological Assessment. New York, NY: Wiley & Sons; 2003.Google Scholar
26.Filipek, PA, Accardo, PJ, Baranek, GT, et al.The screening and diagnosis of autistic spectrum disorders. J Aut Dev Disord. 1999;29:439–484.CrossRefGoogle ScholarPubMed
27.Mundy, P, Hogan, A. A preliminary manual for the abridged Early Social Communication Scales (ESCS). Coral Gables, Fla: University of Miami Press; 1996.Google Scholar
28.Dunn, LM, Dunn, LA. Peabody Picture Vocabulary Test. 3rd ed. Circle Pines, Minn: American Guidance Service; 1997.Google Scholar
29.Williams, KT. Expressive Vocabulary Test. Circle Pines, Minn: American Guidance Service; 1997.Google Scholar
30.Carrow-Woolfolk, E. Comprehensive Assessment of Spoken Language. Circle Pines, Minn: American Guidance Service; 1999.Google Scholar
31.Semmel, E, Wiig, EH, Secord, WA. Manual for the Clinical Evaluation of Language Fundamentals. 3rd ed. San Antonio, Tex: The Psychological Corporation; 1995.Google Scholar
32.Secord, WA & Semel, E. Manual for the Preschool Clinical Evaluation of Language Fundamentals. 3rd ed. San Antonio, Tex: The Psychological Corporation; 1995.Google Scholar
33.Sparrow, S, Balla, D, Ciccetti, D. Vineland Adaptive Behavior Scales. Circle Pines, Minn: American Guidance Service; 1984.Google Scholar
34.Bruininks, RH, Woodcock, RW, Weatherman, RF, Hill, BK. Scales of Independent Behavior. rev ed. Manual. Boston, Mass: Riverside Publishing Company; 1996.Google Scholar
35.Nihira, K, Leland, H, Lambert, N. AAMR Adaptive Behavior Scale-Residential Community: Second Edition. Austin, Tex: PRO-ED; 1993.Google Scholar
36.Carter, AS, Volkmar, FR, Sparrow, SS, et al.The Vineland Adaptive Behavior Scales: supplementary norms for individuals with autism. J Aut Dev Disord. 1998;28:287–302.CrossRefGoogle ScholarPubMed
37.Kern, JK, Miller, VS, Cauller, PL, Kendall, PR, Mehta, PJ, Dodd, M. Effectiveness of N,N-dimethylglycine in autism and pervasive developmental disorder. J Child Neurol. 2001;16:169–173.CrossRefGoogle Scholar
38.Partington, JW, Sundberg, ML. Assessment of Basic Language and Learning Skills. Pleasant Hill, Calif: Behavior Analysts, Inc; 1998.Google Scholar
39.Gadow, KD, Sprafkin, J. Early Childhood Symptom Inventory Manual. Stony Brook, NY: Checkmate Plus; 1997.Google Scholar
40.Gadow, KD, Sprafkin, J. Child Symptom Inventories Manual. Stony Brook, NY: Checkmate Plus; 1994.Google Scholar
41.Gadow, KD, Sprafkin, J. Adolescent Symptom Inventory Manual. Stony Brook, NY: Checkmate Plus; 1997.Google Scholar
42.Diagnostic and Statistical Manual of Mental Disorders. 4th ed. Washington, DC: American Psychiatric Press; 1994.Google Scholar
43.Aman, MG, Assessing Psychopathology and Behavior Problems in Persons with Mental Retardation: A Review of Available Instruments. DHHS Publication No. [ADM] 91-1712. Rockville, MD: U.S. Department of Health and Human Services; 1991.Google Scholar
44.Aman, MG. Review and evaluation of instruments for assessing emotional and behavioural disorders. Australia and New Zealand Journal of Developmental Disabilities. 1991;17:127–145.CrossRefGoogle Scholar
45.Lecavalier, L, Aman, MG. Rating instruments. In: Matson, JL, Matson, ML, eds. Behavior Modification for Persons with Developmental Disabilities: Empirically Supported Treatments. Kingston, NY: National Association for the Dually Diagnosed Press; In press.Google Scholar
46.Aman, MG, Singh, NN. Aberrant Behavior Checklist-Community. Supplementary Manual. East Aurora, NY: Slosson Educational Publications; 1994.Google Scholar
47.Scahill, L, Riddle, MA, McSwiggin-Hardin, M, et al.Children's Yale-Brown Obsessive Compulsive Scale: reliability and validity. J Am Acad Child Adolesc Psychiatry. 1997;36:844–852.CrossRefGoogle ScholarPubMed
48.Esbensen, AJ, Rojahn, J, Aman, MG, Ruedrich, S. The reliability and validity of an assessment instrument for anxiety, depression and mood among individuals with mental retardation. J Aut Dev Disord. 2003;33:617–629.CrossRefGoogle ScholarPubMed
49.Rojahn, J, Matson, JL, Lott, D, Esbensen, AJ, Smalls, Y. The Behavior Problems Inventory: an instrument for the assessment of self-injury, stereotyped behavior, and aggression/destruction in individuals with developmental disabilities. J Aut Dev Disord. 2001;31:577–588.CrossRefGoogle ScholarPubMed
50.Goodman, WK, Price, LH, Rasmussen, SA, et al.The Yale-Brown Obsessive Compulsive Scale (Y-BOCS): Part I. Development, use, and reliability. Arch Gen Psychiatry. 1989;46:1006–1011.CrossRefGoogle Scholar
51.Overall, JE. Campbell, M. Behavioral assessment of psychopathology in children: infantile autism. J Clin Psychol. 1988;44:708–716.3.0.CO;2-T>CrossRefGoogle ScholarPubMed
52.Einfeld, SL, Tonge, BJ. Manual for the Developmental Behavior Checklist. Sydney, Australia: Monash University Center for Developmental Psychiatry and School of Psychiatry, University of New South Wales; 2002.Google Scholar
53.Matson, JL. Manual for the Diagnostic Assessment for the Severely Handicapped-II. Baton Rouge, La: Louisiana State University Press; 1995.Google Scholar
54.Feinstein, C, Kaminer, Y, Barrett, RP, Tylenda, B. The assessment of mood and affect in developmentally disabled children and adolescents: The Emotional Disorders Rating Scale. Res Dev Disabil. 1988;9:109–121.CrossRefGoogle ScholarPubMed
55.Ollendick, TH. Reliability and validity of the Revised Fear Survey Schedule for Children (FSSC-R). Behav Res Ther. 1983;21:685–692.CrossRefGoogle Scholar
56.Sarphare, G, Aman, MG. Parent- and self-ratings of anxiety in children with mental retardation: agreement levels and test-retest reliability. Res Dev Disabil. 1996;17:27–39.CrossRefGoogle ScholarPubMed
57.Ramirez, SZ, Kratochwill, TR. Development of the Fear Survey Schedule for Children with and without mental retardation. Behav Assess. 1990;12:457–470.Google Scholar
58.Aman, MG, Tassé, MJ, Rojahn, J, Hammer, D. The Nisonger CBRF: a child behavior rating form for children with developmental disabilities. Res Dev Disabil. 1996;17:41–57.CrossRefGoogle ScholarPubMed
59.Tassé, MD, Aman, MG, Hammer, D, Rojahn, J. The Nisonger Child Behavior Rating Form: age and gender effects and norms. Res Dev Disord. 1996;17:59–75.CrossRefGoogle ScholarPubMed
60.Behar, LB, Stringfield, SA. A behavior rating scale for the preschool child. Dev Psychol. 1974;10:601–610.CrossRefGoogle Scholar
61.Aman, MG, Rojahn, J. The psychometric characteristics of the Preschool Behavior Questionnaire in preschoolers with developmental handicaps. Journal of Developmental and Physical Disabilities. 1994;6:311–325.CrossRefGoogle Scholar
62.Bodfish, JW, Symons, FJ, Parker, DE, Lewis, MH. Varieties of repetitive behavior in autism. J Autism Dev Disord. 2000;30:237–243.CrossRefGoogle ScholarPubMed
63.Schroeder, SR, Rojahn, J, Reese, RM. Reliability and validity of instruments for assessing psychotropic medication effects on selfinjurious behavior in mental retardation. J Autism Dev Disord. 1997;27:89–102.CrossRefGoogle ScholarPubMed
64.Rojahn, J, Matlock, ST, Tassé, MJ. The Stereotyped Behavior Scale: Psychometric properties and norms. Res Dev Disabil. 2000;21:437–454.CrossRefGoogle ScholarPubMed
65.Zelazo, PR. Infant-toddler information processing assessment for children with pervasive developmental disorder and autism: Part I. Infants Young Child. 1997:10:1–14.Google Scholar
66.Arnold, LE, Aman, MG, Martin, A, et al.Assessment in multisite randomized clinical trials of patients with autistic disorder: the Autism RUPP Network. J Autism Dev Disord. 2000;30:99–111.CrossRefGoogle ScholarPubMed
67.Roid, GH, Miller, LJ. Later International Performance Scale. rev. Wood Dale, Ill: Stoelting; 1997.Google Scholar
68.Aman, MG. Applications of computerized cognitive-motor measures to the assessment of psychoactive drugs. In: Dodson, WE, Kinsbourne, M, Hiltbrunner, B, eds. The Assessment of Cognitive Function in Epilepsy. New York, NY: Demos Publications; 1991.Google Scholar
69.Pearson, DA, Yaffee, LS, Loveland, KA, Lewis, KR. A comparison of sustained and selective attention in children who have mental retardation with and without attention deficit hyperactivity disorder. Am J Ment Retard. 1996;100:92–607.Google ScholarPubMed
70.Anderson, TT, Campbell, M, Grega, DM, Perry, R, Small, AM, Green, WH. Haloperidol in the treatment of infantile autism: Effects on learning and behavioral symptoms. Am J Psychiatry. 1984;141:1195–1202.Google ScholarPubMed
71.Handen, BL, Breaux, AM, Gosling, A, Ploof, DL, Feldman, H. Efficacy of Ritalin among mentally retarded children with ADHD. Pediatrics. 1990;86:922–930.CrossRefGoogle Scholar
72.Kalachnik, JE. Measuring side effects of psychopharmacologic medication in individuals with mental retardation and developmental disabilities. Ment Retard Dev Disabil Res Rev. 1999;5:348–359.3.0.CO;2-N>CrossRefGoogle Scholar
73.Barnes, TR. A rating scale for drug-induced akathisia. Br J Psychiatry. 1989;154:672–676CrossRefGoogle ScholarPubMed
74.Simpson, GM, Angus, JW. A rating scale for extrapyramidal side effects. Acta Psychiatr Scand 1970;212:11–19.CrossRefGoogle ScholarPubMed
75.National Institute of Mental Health. Abnormal Involuntary Movement Scale (AIMS). Psychopharmacol Bull. 1985;21:1077–1080.Google Scholar