Brian A. Boyd - US grants

DESCRIPTION (provided by applicant): This RO1 application requests funds to develop and validate a novel outcome measure targeting the behavioral inflexibility in children with intellectual and developmental disabilities (IDD). This behavioral domain encompasses a host of closely related clinical sequelae that can be profoundly disabling and are common in IDD such as (a) an overall inflexible temperament or behavioral style that underlies discrete idiosyncratic forms of repetitive and rigid behavior manifestations (e.g., inability to tolerate changes in the environment, stereotyped uses of objects); (b) associated mood and behavior problems that arise when this inflexible temperament and its manifestations cannot be accommodated; and (c) interference with learning and adaptation that stems from rigidity / inflexibility as even treatmen itself can represent intrusion and change for an inflexible child (e.g., a new pill, a new therapy exercise). In recent years there have been large-scale drug trials and small behavioral trials targeting discrete behaviors that fall under the domain of repetitive inflexible behavior. However, the measures included in many of these clinical trials have little to no evidence of their ability o detect change over time. In fact, while several instruments exist that measure the discrete behavioral endpoints of this overall construct of rigidity / inflexibility, no instrument exists tht adequately extends much beyond a behavior checklist approach. The overall objective of this application is to develop and validate a new measure, the Repetitive and Inflexible Behavior Inventory (RIBI), with the primary goal of developing a measure that is sensitive to change over time. The RIBI is designed as a parent report measure for children (ages 3 -18) with IDD. The rationale is that a novel measure is needed to (a) capture the range of repetitive inflexible behaviors that are found in children with IDD across a variety of idiopathic and genetic disorders, (b) measure the functional impact of these behaviors on the family's daily life, and (c) detect change over time when these behaviors are the target of intervention. Thus, the specific aims of this project are to (1) examine the psychometric properties of the RIBI for children and adolescents with autism spectrum disorder (ASD), establishing reliability, factor structure, content and construct validity; (2) evaluate the RIBI's ability to measure change over time in children with ASD; and (3) examine the psychometric stability of the RIBI when applied to children and adolescents with Fragile-X and Prader-Willi syndromes. To accomplish these aims, a multisite study will be conducted using recommended instrument development methodologies, such as focus groups, expert panels, and online surveys (n=1500). This team is in an ideal position to develop the RIBI because of our prior experience with measure development, and the conduct of clinical trials targeting repetitive inflexible behavior. The expected deliverable i an outcome measure that is appropriate for IDD populations and minimally burdensome to complete in order to be useful for multisite clinical trials.

Abstract The majority of validated social-communication measures for children with autism spectrum disorder (ASD) were designed to behaviorally phenotype or diagnose this core symptom domain and not to detect change over time. The lack of validated outcome measures has somewhat stifled intervention research efforts as well as early interventionists? ability to monitor the effects of their programs on children?s social-communicative outcomes. It has become difficult to sort out whether non-significant treatment effects are a result of truly ineffective interventions, or rather poorly validated measures for this population. We know that the early, foundational social-communication and language skills of children with ASD predict their functioning into adolescence and even adulthood, with functional use of language by age 5 being one of the best predictors of long-term prognosis. As such, clinicians have begun to focus their efforts on improving children?s social- communication and language skills using both developmental and behavioral interventions as well as pharmaceutical treatments. In reviewing these treatment trials, it is clear there is a range of intervention success and even more clear that we lack a consistent set of outcome measures for the social-communication symptom domain. Our group has been working to validate measures of key social-communication and language skills and are able to draw from a wealth of prior psychometric data to adapt and validate a brief observational measure that can be easily used within clinical trials as well as within routine care and practice. We propose to adapt and psychometrically validate a measure of social-communication and language skills for young children, ages 12 ? 60 months, preliminarily or formally diagnosed with ASD. Specifically, we plan to validate the Early Communication Indicator for Autism Spectrum Disorder (ECI-ASD) using a robust and representative multisite sample of well-characterized children with ASD (n = 400). The current version of the ECI is norm-referenced and allows for progress monitoring, which means the measure can be used in a formative, data-driven fashion to monitor children?s intervention progress and make changes if children are not improving, but it also can provide summative outcome data. In addition, the current measure can be scored live using a mobile app. Currently, no other current social-communication measure specifically designed for children with ASD easily allows for ongoing intervention progress monitoring, data visualization, and live scoring, which will make the adapted ECI-ASD a unique outcome measurement tool. Further, the ECI-ASD will provide the ability to enter data into an online platform to compare the progress of children with ASD to normative data and this represents a sorely needed and clear innovation above current measurement approaches. The expected deliverable is a novel outcome measure of key social-communication and language skills that is psychometrically sound, minimally burdensome to administer and score, and sensitive to the incremental change expected for young children with ASD.

PROJECT SUMMARY Families are facing significant challenges during the COVID-19 pandemic, especially those caring for a child with autism spectrum disorder (ASD). A recent SPARK survey1 (N = 8,000) found that approximately 95% of parents reported increases in their child?s challenging behaviors as a result of COVID-19, and the restricted, repetitive behaviors and interests (RRBI) of children likely fall within this category. Since only 35% of parents who took the SPARK survey indicated their child is receiving therapy services remotely, the COVID-19 pandemic will likely exacerbate this core symptom of ASD. RRBIs are often the target for clinical treatment, as they can significantly interfere with learning, and are associated with long-term functional impairment, and psychiatric comorbidities (e.g., anxiety). Given children with ASD are currently out of routine and experiencing a severe reduction in therapeutic and educational services, the RRBIs and comorbidities of children are likely magnified at this time. Yet, there are no effective therapies to treat the full range of repetitive, inflexible behaviors found in ASD, and there have been no large-scale studies of behavioral interventions specifically developed to target this core symptom domain. This has resulted in a significant treatment gap between the number of effective behavioral therapies used to target atypical social-communication behaviors in autism when compared to those designed to treat repetitive behaviors. Thus, our team developed and pilot-tested the Family-Implemented Treatment for Behavioral Inflexibility (FITBI), derived from neurobehavioral models of reward and learning. The overall goal of FITBI is to teach parents how to identify high probability cues in the environment that can elicit RRBI symptoms and teach their child to inhibit repetitive behaviors and instead replace them with flexible and more functional behaviors. Since our initial pilot studies, the need for technology-based interventions has certainly increased. While the effectiveness of telehealth interventions for children with ASD is emerging, early research suggests interventions delivered via telehealth for children with ASD may be just as effective as in-person interventions. Yet, we currently have no understanding of the effectiveness of RRBI interventions delivered remotely. Therefore, the overall goal of this project is to conduct a parallel group randomized controlled trial (RCT) comparing remote delivery of FITBI (12-week intervention + 3 booster sessions over 6 months) to remote delivery of a parent education only (PE) control condition in a final sample size of 100 (3-9 years) children with ASD and high rates of ritualistic repetitive behaviors. We will use TORSH, a comprehensive secure online platform that enhances therapist-parent coaching via telehealth. Further, an important objective of this proposal is to examine child and parent factors associated with treatment response and uptake in order to advance translational research and knowledge on personalized intervention approaches.