Lawrence Scahill1,2, Karen Bearss1,2, Rena Sarhangian3, Christopher J McDougle4,5, L Eugene Arnold6, Michael G Aman6, James T McCracken7, Elaine Tierney8, Scott Gillespie1,2, Valentina Postorino1,2, Benedetto Vitiello9. 1. 1 School of Medicine, Emory University , Atlanta, Georgia . 2. 2 Marcus Autism Center , Atlanta, Georgia . 3. 3 David Geffen School of Medicine, University of California Los Angeles , Los Angeles, California. 4. 4 Harvard Medical School, Massachusetts General Hospital , Boston, Massachusetts. 5. 5 Lurie Center for Autism , Boston, Massachusetts. 6. 6 Nisonger Center, Ohio State University , Columbus, Ohio. 7. 7 Division of Child Psychiatry, University of California at Los Angeles , Los Angeles, California. 8. 8 Kennedy Krieger Institute , Baltimore, Maryland. 9. 9 National Institute of Mental Health , Bethesda, Maryland.
Abstract
OBJECTIVES: Parent rating scales are commonly used to evaluate change in clinical trials. Despite advantages, these measures may not capture parental impression of the child's most salient problems. We examine the use of parent target problems (PTPs) in a randomized trial of methylphenidate (MPH) in children with autism spectrum disorder and symptoms of attention-deficit/hyperactivity disorder. METHODS: This multisite, 4-week, randomized crossover trial compared three dose levels (low, medium, and high) of MPH with placebo. At baseline, the independent evaluator (IE) asked parents to nominate the child's two biggest problems. For each problem, the IE and parent coconstructed a brief narrative of the behavior and the impact on family life. The IE and parents reviewed and revised the narratives at subsequent visits. A panel of four judges, blind to treatment condition, independently reviewed the narratives to rate change from baseline on a 9-point scale: 1, normal; 2, markedly improved; 3, definitely improved; 4, equivocally improved; 5, no change; 6, possibly worse; 7, definitely worse; 8, markedly worse; 9, disastrously worse. The mean of the four raters was compared with primary and key secondary ratings from the original study. RESULTS: Two PTPs were recorded at baseline for 60 participants. The inter-rater reliability of the four judges across all PTPs and time points was excellent (intraclass correlation = 0.95). On the primary outcome measure (Aberrant Behavior Checklist Hyperactivity subscale), the medium and high-dose levels were superior to placebo. On the mean PTP rating, only the high dose was superior to placebo. We also compared PTP cutoff scores 3.0 (definitely improved), 3.25, and 3.5 with the rate of positive response on the Improvement item of the Clinical Global Impressions scale in the original study. Sensitivities ranged from 68% to 88%. CONCLUSIONS: The parent target problem method offers a systematic way to identify and track patient-centered outcomes.
RCT Entities:
OBJECTIVES: Parent rating scales are commonly used to evaluate change in clinical trials. Despite advantages, these measures may not capture parental impression of the child's most salient problems. We examine the use of parent target problems (PTPs) in a randomized trial of methylphenidate (MPH) in children with autism spectrum disorder and symptoms of attention-deficit/hyperactivity disorder. METHODS: This multisite, 4-week, randomized crossover trial compared three dose levels (low, medium, and high) of MPH with placebo. At baseline, the independent evaluator (IE) asked parents to nominate the child's two biggest problems. For each problem, the IE and parent coconstructed a brief narrative of the behavior and the impact on family life. The IE and parents reviewed and revised the narratives at subsequent visits. A panel of four judges, blind to treatment condition, independently reviewed the narratives to rate change from baseline on a 9-point scale: 1, normal; 2, markedly improved; 3, definitely improved; 4, equivocally improved; 5, no change; 6, possibly worse; 7, definitely worse; 8, markedly worse; 9, disastrously worse. The mean of the four raters was compared with primary and key secondary ratings from the original study. RESULTS: Two PTPs were recorded at baseline for 60 participants. The inter-rater reliability of the four judges across all PTPs and time points was excellent (intraclass correlation = 0.95). On the primary outcome measure (Aberrant Behavior Checklist Hyperactivity subscale), the medium and high-dose levels were superior to placebo. On the mean PTP rating, only the high dose was superior to placebo. We also compared PTP cutoff scores 3.0 (definitely improved), 3.25, and 3.5 with the rate of positive response on the Improvement item of the Clinical Global Impressions scale in the original study. Sensitivities ranged from 68% to 88%. CONCLUSIONS: The parent target problem method offers a systematic way to identify and track patient-centered outcomes.
Authors: Benjamin L Handen; Michael G Aman; L Eugene Arnold; Susan L Hyman; Rameshwari V Tumuluru; Luc Lecavalier; Patricia Corbett-Dick; Xueliang Pan; Jill A Hollway; Kristin A Buchan-Page; Laura B Silverman; Nicole V Brown; Robert R Rice; Jessica Hellings; Daniel W Mruzek; Sarah McAuliffe-Bellin; Elizabeth A Hurt; Melissa M Ryan; Lynne Levato; Tristram Smith Journal: J Am Acad Child Adolesc Psychiatry Date: 2015-09-03 Impact factor: 8.829
Authors: Lawrence Scahill; James T McCracken; Bryan H King; Carol Rockhill; Bhavik Shah; Laura Politte; Roy Sanders; Mendy Minjarez; Jennifer Cowen; Jennifer Mullett; Chris Page; Denise Ward; Yanhong Deng; Sandra Loo; James Dziura; Christopher J McDougle Journal: Am J Psychiatry Date: 2015-08-28 Impact factor: 18.112
Authors: Victoria Hallett; Luc Lecavalier; Denis G Sukhodolsky; Noreen Cipriano; Michael G Aman; James T McCracken; Christopher J McDougle; Elaine Tierney; Bryan H King; Eric Hollander; Linmarie Sikich; Joel Bregman; Evdokia Anagnostou; Craig Donnelly; Lily Katsovich; Kimberly Dukes; Benedetto Vitiello; Kenneth Gadow; Lawrence Scahill Journal: J Autism Dev Disord Date: 2013-10
Authors: 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 Journal: J Am Acad Child Adolesc Psychiatry Date: 2003-12 Impact factor: 8.829
Authors: Karen Bearss; Cynthia Johnson; Tristram Smith; Luc Lecavalier; Naomi Swiezy; Michael Aman; David B McAdam; Eric Butter; Charmaine Stillitano; Noha Minshawi; Denis G Sukhodolsky; Daniel W Mruzek; Kylan Turner; Tiffany Neal; Victoria Hallett; James A Mulick; Bryson Green; Benjamin Handen; Yanhong Deng; James Dziura; Lawrence Scahill Journal: JAMA Date: 2015-04-21 Impact factor: 157.335
Authors: Elisabeth Sheridan; Scott Gillespie; Cynthia R Johnson; Luc Lecavalier; Tristram Smith; Naomi Swiezy; Kylan Turner; Jill Pritchett; Daniel W Mruzek; Andrea N Evans; Karen Bearss; Lawrence Scahill Journal: Res Child Adolesc Psychopathol Date: 2021-07-02