In 2014, the NIMH’s Autism Spectrum Disorder Pediatric, Early Detection, Engagement and Services (ASD PEDS) Network was formed to develop and test systems innovations that rapidly engage young children with ASD in diagnostic and treatment services1. Five studies across nine states were funded, each testing a different model of care (i.e., family navigation, enhanced community identification, enhanced primary care identification, online screening and identification with provider training, identification in Part C early intervention). The current paper represents an effort to learn from the collective experience of the five diverse ASD PEDS Network projects. Specifically, we evaluate where each study falls on the pragmatic (i.e., “real-world”) to explanatory (i.e., “ideal condition”) continuum in order to inform future implementation efforts and identify research priorities for early ASD identification and intervention services.

The Pragmatic Explanatory Continuum Indicator Summary–2 (PRECIS-2) was used to assess the five ASD PEDS Network studies2. First, investigator teams from each study completed the Template for Intervention Description and Replication (TIDieR) checklist for their specific study3. Second, a group of independent reviewers with expertise in community ASD services and trial design (n=3) reviewed the checklists and rated each study on the nine PRECIS constructs (i.e., eligibility, recruitment, setting, organization, delivery, adherence, follow-up, outcomes, analysis) from 1 (most “explanatory”) to 5 (most “pragmatic”). Third, a modified Delphi approach was used to reach agreement for each study in each domain.

The domains rated as most pragmatic (i.e., most reflective of usual care) were those measuring the inclusiveness of data in the analyses (M= 5.0, SD=0) and the flexibility allowed in providers’ adherence to the intervention protocol (M=4.34, SD=1.19). Domains rated as most explanatory (i.e., least reflective of usual care) included the time and effort devoted to follow-up data collection (M=2.23, SD=0.66) and the resources and expertise needed to deliver the intervention (M=2.32, SD=0.81).

Overall, there was considerable variability across ASD PEDS Network studies and PRECIS-2 domains. The least pragmatic aspects of these studies were the settings in which they were conducted and the manner in which outcome data were collected, suggesting that these areas may pose particular challenges for community-based trials focused on early detection and service access for ASD. To achieve the goal of increasing pragmatic research trials and “real-world” applicability for ASD research, future research might benefit from using the PRECIS-2 during initial trial design, developing more pragmatic outcome measures, and improving methods for integration of research into “real-world” settings.

Cluster-randomized and stepped-wedge are pragmatic trial designs that have become increasingly popular in recent years. Due to feasibility or logistical constraints, individual-level randomization is often not possible, and interventions must be implemented at the cluster (e.g., site, clinic) level. Power/sample size calculations are used to identify whether a proposed design s feasible for detecting a clinically meaningful effect of an intervention. Tools that perform these calculations are thus essential in the planning of an effective study and for assessing various design options.

Power calculations for cluster-randomized and stepped-wedge designs incorporate the correlation between multiple observations in the same cluster. They also require additional consideration such as the number of clusters, and individuals per cluster. There are trade-offs when evaluating each of these two designs, and often one is considered when the other is not feasible and/or does not provide sufficient power. With a free, web-based applet, we unify the power/sample size calculations for these two clustered study designs in a single application, allowing for easy comparison and evaluation of alternative designs.

Using an R Shiny application, we implement methodology developed for cluster-randomized and stepped-wedge designs for sample size/power calculations. We incorporate recent extensions, such as cluster auto-correlation, washout effects, and hybrid designs. The application will use a guided step-by-step process, where users will specify the parameters of their trial design. Users will provide inputs related to the outcome of interest and study design, such as number of clusters, individuals per cluster, desired power, type I error rate, outcome distribution, effect size, and the intraclass correlation coefficient. Outputs will include a visualization of the study design, and a summary statement describing the design, assumptions, and power/sample size values.

The R Shiny calculator will be hosted online as a web applet that can be used by clinicians and statisticians to help plan their trial design. A range of examples will be presented to demonstrate the use of the calculator. Documentation for the methods and references be provided. Code for the application and power calculations will be shared using Github, where users can provide feedback and request modifications or extensions.

With this online calculator, we aim to increase the accessibility of current and emerging sample size methodology for researchers who are considering pragmatic design alternatives to answer their research question.

KATE GUASTAFERRO, PhD
Assistant Research Professor, Pennsylvania State University

Child sexual abuse (CSA) affects about 60,000 children per year in the United States and has estimated societal costs in excess of $9.3 billion. Despite the proliferation of parent education programs that have successfully reduced the risk for physical abuse and neglect, these programs are not designed to prevent CSA specifically and have not affected rates of CSA. While parents are not the most common perpetrators of CSA, they are an important agent of change within the family context and they control access to children by creating a protective and safe environment. This study sought to examine the knowledge, attitude, and behavioral changes attributable to the addition of a newly-created, parent-focused CSA prevention module to existing evidence-based parent education programs commonly prescribed for parents who are deemed ‘at risk’ within the child welfare system.

A cluster randomized controlled trial was conducted among six community-based organizations randomized to provide Parents as Teachers plus a one-hour CSA module (PAT+CSA) or Parents as Teachers delivered as usual (PAT Only). CSA related-knowledge, attitudes, and protective behaviors (i.e., involvement, positive parenting, and inconsistent discipline) were assessed at four time points (baseline, post-PAT Only, post-PAT+CSA, and a one month follow-up).

CSA related knowledge and attitudes were significantly higher in the PAT+CSA condition than in the PAT Only condition (p = 0.032) at post-intervention. Behaviors also increased from baseline to post-intervention (p < 0.05) and remained increased at the one month follow-up assessment (p < 0.001).

A singular added session focused on CSA prevention can significantly improve parents’ ability to demonstrate CSA preventive knowledge, attitudes, and behavioral skills which can be maintained over time. Parents are critical in preventing CSA and these findings indicate it is possible to augment current approaches to parent education with CSA-specific curricula to impact rates of CSA.