Karen Bearss

Medication and Parent Training in Children With Pervasive Developmental Disorders and Serious Behavior Problems: Results From a Randomized Clinical Trial

OBJECTIVE Many children with pervasive developmental disorders (PDDs) have serious, functionally impairing behavioral problems. We tested whether combined treatment (COMB) with risperidone and parent training (PT) in behavior management is superior to medication alone (MED) in improving severe behavioral problems in children with PDDs. METHOD This 24-week, three-site, randomized, parallel-groups clinical trial enrolled 124 children, aged 4 through 13 years, with PDDs, accompanied by frequent tantrums, self-injury, and aggression. The children were randomized 3:2 to COMB (n = 75) or MED (n = 49). The participants received risperidone monotherapy from 0.5 to 3.5 mg/day (with switch to aripiprazole if risperidone was ineffective). Parents in the COMB group (n = 75; 60.5%) received a mean of 10.9 PT sessions. The primary measure of compliance was the Home Situations Questionnaire (HSQ) score. RESULTS Primary: intent-to-treat random effects regression showed that COMB was superior to MED on HSQ (p = .006) [effect size at week 24 (d) = 0.34]. The HSQ score declined from 4.31 (± 1.67) to 1.23 (± 1.36) for COMB compared with 4.16 (± 1.47) to 1.68 (± 1.36) for MED. Secondary: groups did not differ on Clinical Global Impressions-Improvement scores at endpoint; compared with MED, COMB showed significant reductions on Aberrant Behavior Checklist Irritability (d = 0.48; p = .01), Stereotypic Behavior (d = 0.23; p = .04), and Hyperactivity/Noncompliance subscales (d = 0.55; p = .04). Final risperidone mean dose for MED was 2.26 mg/day (0.071 mg/kg), compared with 1.98 mg/day for COMB (0.066 mg/kg) (p = .04). CONCLUSIONS Medication plus PT resulted in greater reduction of serious maladaptive behavior than MED in children with PDDs, with a lower risperidone dose.

Gastrointestinal Symptoms in a Sample of Children with Pervasive Developmental Disorders

Objective To evaluate gastrointestinal (GI) problems in a large, well-characterized sample of children with pervasive developmental disorders (PDDs). Methods One hundred seventy two children entering one of two trials conducted by the Research Units on Pediatric Psychopharmacology (RUPP) Autism Network were assessed comprehensively prior to starting treatment and classified with regard to GI symptoms. Results Thirty nine (22.7%) were positive for GI problems, primarily constipation and diarrhea. Those with GI problems were no different from subjects without GI problems in demographic characteristics, measures of adaptive functioning, or autism symptom severity. Compared to children without GI problems, those with GI problems showed greater symptom severity on measures of irritability, anxiety, and social withdrawal. Those with GI problems were also less likely to respond to treatment.

Effect of Parent Training vs Parent Education on Behavioral Problems in Children With Autism Spectrum Disorder: A Randomized Clinical Trial

IMPORTANCE Disruptive behavior is common in children with autism spectrum disorder. Behavioral interventions are used to treat disruptive behavior but have not been evaluated in large-scale randomized trials. OBJECTIVE To evaluate the efficacy of parent training for children with autism spectrum disorder and disruptive behavior. DESIGN, SETTING, AND PARTICIPANTS This 24-week randomized trial compared parent training (n = 89) to parent education (n = 91) at 6 centers (Emory University, Indiana University, Ohio State University, University of Pittsburgh, University of Rochester, Yale University). We screened 267 children; 180 children (aged 3-7 years) with autism spectrum disorder and disruptive behaviors were randomly assigned (86% white, 88% male) between September 2010 and February 2014. INTERVENTIONS Parent training (11 core, 2 optional sessions; 2 telephone boosters; 2 home visits) provided specific strategies to manage disruptive behavior. Parent education (12 core sessions, 1 home visit) provided information about autism but no behavior management strategies. MAIN OUTCOMES AND MEASURES Parents rated disruptive behavior and noncompliance on co-primary outcomes: the Aberrant Behavior Checklist-Irritability subscale (range, 0-45) and the Home Situations Questionnaire-Autism Spectrum Disorder (range, 0-9). On both measures, higher scores indicate greater severity and a 25% reduction indicates clinical improvement. A clinician blind to treatment assignment rated the Improvement scale of the Clinical Global Impression (range, 1-7), a secondary outcome, with a positive response less than 3. RESULTS At week 24, the Aberrant Behavior Checklist-Irritability subscale declined 47.7% in parent training (from 23.7 to 12.4) compared with 31.8% for parent education (23.9 to 16.3) (treatment effect, -3.9; 95% CI, -6.2 to -1.7; P < .001, standardized effect size = 0.62). The Home Situations Questionnaire-Autism Spectrum Disorder declined 55% (from 4.0 to 1.8) compared with 34.2% in parent education (3.8 to 2.5) (treatment effect, -0.7; 95% CI, -1.1 to -0.3; P < .001, standardized effect size = 0.45). Neither measure met the prespecified minimal clinically important difference. The proportions with a positive response on the Clinical Global Impression-Improvement scale were 68.5% for parent training vs 39.6% for parent education (P < .001). CONCLUSIONS AND RELEVANCE For children with autism spectrum disorder, a 24-week parent training program was superior to parent education for reducing disruptive behavior on parent-reported outcomes, although the clinical significance of the improvement is unclear. The rate of positive response judged by a blinded clinician was greater for parent training vs parent education. TRIAL REGISTRATION clinicaltrials.gov Identifier: NCT01233414.

Examination of aggression and self-injury in children with autism spectrum disorders and serious behavioral problems.

This study identified subtypes of aggression in a sample of 206 children with autism spectrum disorder (ASD) who participated in 2 risperidone trials. The narratives were derived from a parent interview about each childs 2 most pressing problems. Five subtypes of aggression emerged: hot aggression only, cold aggression only, self-injurious behavior (SIB) only, aggression and SIB, and nonaggressive. All groups showed a high rate of positive response to risperidone with no differences across subtypes. These study findings extend understanding of aggression in ASD and may be useful to guide further study on biological mechanisms and individualized treatment in ASD.

Factor structure and psychometric properties of the revised Home Situations Questionnaire for autism spectrum disorder: The Home Situations Questionnaire-Autism Spectrum Disorder:

Previously, we adapted the Home Situations Questionnaire to measure behavioral non-compliance in everyday settings in children with pervasive developmental disorders. In this study, we further revised this instrument for use in autism spectrum disorder and examined its psychometric properties (referred to as the Home Situations Questionnaire-Autism Spectrum Disorder). To cover a broader range of situations and improve reliability, we prepared seven new items describing situations in which children with autism spectrum disorder might display non-compliance. Parents completed ratings of 242 children with autism spectrum disorder with accompanying disruptive behaviors (ages 4–14 years) participating in one of two randomized clinical trials. Results from an exploratory factor analysis indicated that the Home Situations Questionnaire-Autism Spectrum Disorder consists of two 12-item factors: Socially Inflexible (α = 0.84) and Demand Specific (α = 0.89). One-to-two-week test–retest reliability was statistically significant for all scored items and also for subscale totals. The pattern of correspondence between the Home Situations Questionnaire-Autism Spectrum Disorder and parent-rated problem behavior, clinician-rated repetitive behavior, adaptive behavior, and IQ provided evidence for concurrent and divergent validity of the Home Situations Questionnaire-Autism Spectrum Disorder. Overall, the results suggest that the Home Situations Questionnaire-Autism Spectrum Disorder is an adequate measure for assessing non-compliance in a variety of situations in this population, and use of its two subscales will likely provide a more refined interpretation of ratings.

Predictors and moderators of parent training efficacy in a sample of children with autism spectrum disorders and serious behavioral problems.

The Research Units on Pediatric Psychopharmacology—Autism Network reported additional benefit when adding parent training (PT) to antipsychotic medication in children with autism spectrum disorders and serious behavior problems. The intent-to-treat analyses were rerun with putative predictors and moderators. The Home Situations Questionnaire (HSQ) and the Hyperactivity/Noncompliance subscale of the Aberrant Behavior Checklist were used as outcome measures. Candidate predictors and moderators included 21 demographics and baseline measures of behavior. Higher baseline HSQ scores predicted greater improvement on the HSQ regardless of treatment assignment, but no other predictors of outcome were observed. None of the variables measured in this study moderated response to PT. Antipsychotic medication plus PT appears to be equally effective for children with a wide range of demographic and behavioral characteristics.

Parent Training in Autism Spectrum Disorder: What’s in a Name?

Parent training (PT) is well understood as an evidence-based treatment for typically developing children with disruptive behavior. Within the field of autism spectrum disorder (ASD), the term parent training has been used to describe a wide range of interventions including care coordination, psychoeducation, treatments for language or social development, as well as programs designed to address maladaptive behaviors. As a result, the meaning of “parent training” in ASD is profoundly uncertain. This paper describes the need to delineate the variants of PT in ASD and offers a coherent taxonomy. Uniform characterization of PT programs can facilitate communication with families, professionals, administrators, and third-party payers. Moreover, it may also serve as a framework for comparing and contrasting PT programs. In support of the taxonomy, a purposive sampling of the literature is presented to illustrate the range of parent training interventions in ASD.

Using qualitative methods to guide scale development for anxiety in youth with autism spectrum disorder

Anxiety is common in youth with autism spectrum disorder. Despite this common co-occurrence, studies targeting anxiety in this population are hindered by the under-developed state of measures in youth with autism spectrum disorder. Content validity (the extent to which an instrument measures the domain of interest) and an instrument’s relevance to the patient population are key components of measurement development. This article describes the application of qualitative research methods in the initial development of a parent-rated instrument of anxiety symptoms in youth with autism spectrum disorder. Overall, 48 parents of 45 children (aged 3–17 years) with autism spectrum disorder and at least mild anxiety participated in one of six focus groups at two sites (three groups per site). Systematic coding of the focus group transcripts identified broad themes reflecting the situations and events that trigger anxiety in children with autism spectrum disorder, the behavioral manifestations of anxiety in children with autism spectrum disorder, the parent and the child’s own response to anxiety, and broad behavioral patterns that could be associated with anxiety. From the focus group data, investigators generated 52 candidate items for a parent-rating of anxiety in youth with autism spectrum disorder. This report provides a detailed description of these early steps in developing a patient-oriented outcome measure.

Individualized Assessments in Treatment Research: An Examination of Parent-Nominated Target Problems in the Treatment of Disruptive Behaviors in Youth with Tourette Syndrome

Youth with Tourette syndrome (TS) often exhibit disruptive behaviors. Although improvement data on rating scales support the efficacy of structured psychotherapeutic interventions, there is growing interest in personalized outcome assessments. This report examined parent-nominated target problems (PTPs) as an individualized outcome measure in 48 youth with TS and disruptive behaviors, who participated in one of two randomized psychotherapy trials. At baseline, parents described two primary problems to an independent evaluator who generated a structured narrative for each problem. These narratives were reviewed and updated at endpoint. When rated by five treatment-blind judges, the PTP rating demonstrated excellent reliability and good convergent validity with the Disruptive Behavior Rating Scale (DBRS). The PTP rating exhibited comparable treatment effects to the DBRS, and accounted for additional variance in global treatment outcome. The PTP rating serves a reliable, valid, and sensitive personalized assessment in research trials that provides complementary information to standardized rating scales.

Design and Subject Characteristics in the Federally-Funded Citalopram Trial in Children with Pervasive Developmental Disorders

The Studies to Advance Autism Research and Treatment Network conducted a randomized trial with citalopram in children with Pervasive developmental disorders (PDDs). We present the rationale, design and sample characteristics of the citalopram trial. Subjects (128 boys, 21 girls) had a mean age of 9.3 (±3.12) years; 132 (88.6%) were diagnosed with autistic disorder (4.7% with Asperger’s Disorder; 6.7% with PDD-not otherwise specified). Less than half of the subjects were intellectually disabled; 117 (78.5%) were rated Moderate or Marked on the Clinical Global Impression for Severity. Study measures were similar to previous Research Units on Pediatric Psychopharmacology trials. Subjects in this trial were slightly older and more likely to have complaints of repetitive behavior than participants in RUPP trials.