Luc Lecavalier - US grants

DESCRIPTION (provided by applicant): Autism is a condition of early childhood onset marked by social delay, communication delay and repetitive behavior affecting 2 per 1,000 children (Fombonne, 2003). If the wider spectrum is included (PDD-NOS and Asperger's disorder), the prevalence is 67 per 1000 children (Autism and Developmental Disorders Monitoring Network, 2007). Given the severity and the prevalence, pervasive developmental disorders (PDDs) have enormous public health impact. There is general agreement that intervention in the preschool years can reduce the long-term disability of PDD, but the required components and optimal intensity of these programs remain unclear (National Research Council, 2001). In addition, recommended behavioral and educational treatments for young children with PDD are not available in all communities (Lord et al., 2005). Moreover, preschool interventions often focus on the child (Smith, 1999) leaving parents to manage the child's behavior on their own (Jacobson &Mulick, 2000). Thus, although the role of parents as partners in the treatment team is universally acknowledged, few studies have tested parent-based interventions and large-scale interventions are non-existent (Smith et al., 2007). In response to PA 07-092 (Collaborative R01s for Mental Disorders) and PA 07-085 (Research on Autism and Autism Spectrum Disorders), this application describes a five-site study to compare parent training to psychoeducation in 180 preschool-age children with PDD. Eligible subjects will be randomly assigned to receive either Parent Training (PT) or Psychoeducational Intervention (PEP) over 6 months. The primary outcomes are the change in the child's irritable and noncompliant behavior rated by the parent. Overall improvement and gains in adaptive functioning (social, communication and daily living skill domains) will be assessed by a rater who is blind to treatment assignment. Parent-child interaction will be assessed in a semi-structured lab sequence by blinded raters. The effects of PT on parental stress and the possible moderating effects of concurrent interventions will also be explored. Training and quality assurance services will be shared across sites;Yale University will provide data management and statistical services. This application is congruent with recommendations of an NIMH advisory council (Research in Psychosocial and Behavioral Interventions in Autism), which called for large-scale, randomized trials to evaluate the efficacy and promote the dissemination of psychosocial interventions in children with PDD (Lord et al., 2005;Smith et al., 2007). To conduct this study, we have assembled a consortium of experienced investigators from leading autism research programs including Yale, University of Rochester, University of Pittsburgh, Ohio State University, and Indiana University. These centers have extensive experience with conducting multisite trials (e.g., Research Units on Pediatric Psychopharmacology (RUPP) Autism Network) and with parenting training for children with autism. PUBLIC HEALTH RELEVANCE: There is an urgent need for large-scale, methodologically rigorous studies to develop evidence-based interventions for children with autism. This trial is designed to address this need by providing information on optimal practices for educating parents to be effective teachers and advocates for their young children.

Project Summary / Abstract. This application, written in response to PAR-18-039, will advance outcome measurement for sleep problems in children (age 3-12 years) with Autism Spectrum Disorder (ASD). Sleep problems (e.g., sleep disturbances and sleep-related impairments) in children with ASD range from mild to severe and affect as many as 80% in this age group (Katz et al, 2018).There is no accepted outcome measure for sleep problems in children with ASD. The absence of a relevant, reliable and valid outcome measure is a barrier to rigorous testing of behavioral or pharmacological treatments targeting sleep in this population. Using a mixed method design, this multisite, transdisciplinary project will build a new parent rating for sleep disturbances and related impairments in children with ASD. The study is guided by methods described in Patient Reported Outcome Measures (www.Fda.Gov/Cder/Guidance, 2009). This monograph compels investigators to include the patient population in measure development. Given the age range of this study and likelihood of cognitive or language delays in children with ASD, we rely on parents as primary informants. We have an outstanding team of investigators and consultants with expertise in measure development and sleep problems in ASD. In Year 1, up to six focus groups with parents will explore the onset and behavioral manifestations of sleep problems in children with ASD. The focus group material will be reviewed and coded to generate a bank of items (Bearss et al. 2016). These items will be reviewed for clarity and reading level by parents and an expert panel. In Year 2, the bank of items (version 1 of ASD-Sleep Questionnaire; ASDSQ) will be placed on a secure website to collect parent-reported data on sleep disturbances and related impairments in 1200 children with ASD. Items will be scored 0-3 (none, mild, moderate or severe). Factor analysis and item response theory will be applied to establish the factor structure and prune the item pool toward the final version (Scahill et al. in press). In Year 3 and early Year 4, an in-person clinical assessment of 135 children with ASD (age 3-12 years) will be conducted to collect reliability and validity data on the final ASDSQ version. A subsample of children will be invited to return for test-retest on ASDSQ, 2 weeks and 4 weeks later. In Year 4, data from the clinical assessment will be used to evaluate convergent validity, divergent validity and test-retest reliability of the ASDSQ. Also in Year 3 and early Year 4, we will collect five consecutive nights of actigraphy data in a total of 75 children with ASD (age 3 to 12) to evaluate sleep efficiency (percentage of time sleeping divided by total time in bed with lights off). Actigraphy-measured sleep efficiency (ASE) will be used to conduct exploratory analyses comparing ASDSQ scores in children with impaired ASE values (poor sleepers) to those with acceptable ASE values (good sleepers) (Goldman et al. 2009; Moore et al. 2017). Receiver operating characteristic curves will be used to explore the sensitivity and specificity of SDSQ on ASE classifications.