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Purpose: Norm-referenced, standardized measures are tools designed to characterize a child's abilities relative to their same-age peers, but they also have been used to measure changes in skills during intervention. This study compared the psychometric properties of four types of available scores from one commonly used standardized measure, the Preschool Language Scales–Fifth Edition (PLS-5), to detect changes in children's language skills during and after a language intervention. Method: This study included data from 110 autistic children aged 18–48 months whose mother participated in an 8-week parent-mediated language intervention. Children's language skills were measured at 3 time points using the PLS-5. Changes in children's expressive and receptive language skills were calculated using raw scores, standard scores, age equivalents, and growth scale values (GSVs). Results: Analysis of raw scores, age equivalents, and GSVs indicated significant improvement in the scores of autistic children in both receptive and expressive language throughout the study (i.e., during the intervention period and in the 3-month period after the intervention). Standard scores suggested improvement only in the receptive language scale during the intervention period. Standard scores showed a floor effect for children who scored at −3 SD below the mean. Conclusions: Findings suggested that GSVs were not only psychometrically sound but also the most sensitive measure of direct changes in skills compared to raw, standard, and age-equivalent scores. Floor effects may limit the sensitivity of standard scores to detect changes in children's skills. Strengths, limitations, and interpretations of each of the scoring approaches in measuring changes in skills during intervention were discussed. Supplemental Material: https://doi.org/10.23641/asha.21498522

Background: When developed properly, disease-specific patient reported outcome measures have the potential to measure relevant changes in how a patient feels and functions in the context of a therapeutic trial. The Huntington’s Disease Health Index (HD-HI) is a multifaceted disease-specific patient reported outcome measure (PROM) designed specifically to satisfy previously published FDA guidance for developing PROMs for product development and labeling claims. Objective: In preparation for clinical trials, we examine the validity, reliability, clinical relevance, and patient understanding of the Huntington’s Disease Health Index (HD-HI). Methods: We partnered with 389 people with Huntington’s disease (HD) and caregivers to identify the most relevant questions for the HD-HI. We subsequently utilized two rounds of factor analysis, cognitive interviews with fifteen individuals with HD, and test-retest reliability assessments with 25 individuals with HD to refine, evaluate, and optimize the HD-HI. Lastly, we determined the capability of the HD-HI to differentiate between groups of HD participants with high versus low total functional capacity score, prodromal versus manifest HD, and normal ambulation versus mobility impairment. Results: HD participants identified 13 relevant and unique symptomatic domains to be included as subscales in the HD-HI. All HD-HI subscales had a high level of internal consistency and reliability and were found by participants to have acceptable content, relevance, and usability. The total HD-HI score and each subscale score statistically differentiated between groups of HD participants with high versus low disease burden. Conclusion: Initial evaluation of the HD-HI supports its validity and reliability as a PROM for assessing how individuals with HD feel and function.

Background and ObjectiveIndividuals with intellectual disability (ID) experience protracted cognitive development compared with typical youth. Sensitive measurement of cognitive change in this population is a critical need for clinical trials and other intervention studies, but well-validated outcome measures are scarce. This study's aim was to evaluate the sensitivity of the NIH Toolbox Cognition Battery (NIHTB-CB) to detect developmental changes in groups with ID—fragile X syndrome (FXS), Down syndrome (DS), and other ID (OID)—and to provide further support for its use as an outcome measure for treatment trials.MethodsWe administered the NIHTB-CB and a reference standard cross-validation measure (Stanford-Binet Intelligence Scales, Fifth Edition [SB5]) to 256 individuals with FXS, DS, and OID (ages 6–27 years). After 2 years of development, we retested 197 individuals. Group developmental changes in each cognitive domain of the NIHTB-CB and SB5 were assessed using latent change score models, and 2-year growth was evaluated at 3 age points (10, 16, and 22 years).ResultsOverall, effect sizes of growth measured by the NIHTB-CB tests were comparable with or exceeded those of the SB5. The NIHTB-CB showed significant gains in almost all domains in OID at younger ages (10 years), with continued gains at 16 years and stability in early adulthood (22 years). The FXS group showed delayed gains in attention and inhibitory control compared with OID. The DS group had delayed gains in receptive vocabulary compared with OID. Unlike the other groups, DS had significant growth in early adulthood in 2 domains (working memory and attention/inhibitory control). Notably, each group's pattern of NIHTB-CB growth across development corresponded to their respective pattern of SB5 growth.DiscussionThe NIHTB-CB is sensitive to developmental changes in individuals with ID. Comparison with levels and timing of growth on the cross-validation measure shows that the NIHTB-CB has potential to identify meaningful trajectories across cognitive domains and ID etiologies. Sensitivity to change within the context of treatment studies and delineation of clinically meaningful changes in NIHTB-CB scores, linked to daily functioning, must be established in future research to evaluate the battery more completely as a key outcome measure.

STUDY AIM To bolster Crohn’s disease (CD) clinical trial and research infrastructure through the development and validation of a sensitive, multifactorial, disease-specific, patient-reported outcome measure (PRO) for patient monitoring in therapeutic trials of CD. BACKGROUND As therapeutic advancements are made in the field of CD, it is vital to have a fully validated, sensitive, and reliable, disease-specific PRO to accurately measure changes in disease burden. Importantly, the U.S. Food and Drug Administration has encouraged the use of PROs to measure therapeutic benefit of new treatments on patient populations. This research describes the creation, testing, and validation of a new, multifactorial PRO: The Crohn’s Disease-Health Index (CD-HI). METHODS Questions were selected for the CD-HI based on their importance to patients, potential to respond to therapeutic benefit, reliability, internal consistency, and generalizability (Figure 1). We beta tested the CD-HI to evaluate its clarity, ease of use, and applicability to the CD patient population. We performed test-retest reliability, known groups validity, internal consistency, and area under the curve (AUC) analyses. RESULTS The final version of the CD-HI contains 12 subscales that measure disease burden in the following areas: 1) fatigue, 2) dietary restrictions, 3) gastrointestinal health, 4) sleep and daytime sleepiness, 5) bowel and bladder function, 6) emotional wellbeing, 7) joint health, 8) pain, 9) neck and back health, 10) activity participation, 11) social wellbeing, and 12) skin health. The CD-HI also provides a composite measure of total disease burden. Fifteen participants participated in beta testing and identified the CD-HI as easy to use, clear, relevant, and comprehensive. Twenty-three individuals with CD participated in test-retest reliability testing of the CD-HI, which indicated a high reliability of the individual questions, subscales, and full instrument (intraclass correlation coefficient = 0.84 for the full instrument). The CD-HI and its subscales demonstrated a high internal consistency (Cronbach’s α = 0.98 for the full instrument). The CD-HI total and subscale scores successfully distinguished between patient cohorts with differing disease severity. AUC analysis demonstrated a high sensitivity and specificity of the CD-HI in distinguishing between those with CD on disability vs. not on disability, with an AUC value of 0.9087 (Figure 2). CONCLUSIONS The CD-HI is a fully validated, sensitive, and accurate marker of disease burden in CD. This disease-specific PRO is available for use by researchers, clinicians, patient organizations, and companies to monitor patients and measure disease burden during therapeutic trials

The NIH Toolbox includes a cognitive battery that provides an Early Childhood Composite score for children age 3-7. However, very few studies have evaluated feasibility when it is used in the youngest segment of this age range-3-year-olds. The current study evaluated performance on the four cognitive subtests composing the early childhood composite, two of which assess executive function, in a large sample of 3-year-olds enrolled in a Vanguard pilot of the National Children's Study. Results found that in a cohort of 609 3-year-olds (mean age = 39.6 months, SD = 1.6, 53% male, 64% White, 87% Non-Hispanic) who were administered four subtests included in the Early Childhood Composite, up to approximately 30% were unable to pass practice items on the Flanker, Dimensional Change Card Sort, and Picture Sequence Memory, whereas only approximately 3% were unable to pass practice items on the Picture Vocabulary Test. Furthermore, of those that did pass practice and achieve scores on the subtests, approximately 70% and 80% performed at or below chance level on the executive function tasks (Flanker and Dimensional Change Card Sort) and Picture Sequence Memory, respectively. Ultimately, the average 3-year-old has difficulty with three of the four NIH Toolbox tasks composing the Early Childhood Composite and may not yet have developed the requisite skills. These findings indicate that changes compatible with the developmental level of preschoolers are recommended to increase the feasibility and effectiveness of the NIH Toolbox in measuring individual cognition differences in 3-year-old children.

Coaching parents to use language facilitation strategies improves long-term language outcomes for autistic children. To optimize parent-mediated interventions, more studies need to explore factors that influence parents’ learning. This study involved 119 autistic children (18–48 months) and their biological mothers enrolled in a single-site, factorial randomized clinical trial. Mothers were taught to use one of two types of language facilitation strategies (responsive or directive) during eight weekly, hour-long instructional sessions. We explored the impact of (a) type of language facilitation strategy, (b) maternal Broad Autism Phenotype (subclinical traits of autism spectrum disorder), and (c) preintervention strategy use on mothers’ outcomes measured immediately and 3 months after intervention sessions. At postintervention, mothers who learned responsive strategies demonstrated significantly greater use of taught strategies than mothers who learned directive strategies ( d = 0.90, 95% CI =[0.47, 1.32]). Mothers’ use of taught strategies did not differ by Broad Autism Phenotype status. However, a significant two-way interaction was found between preintervention strategy use and Broad Autism Phenotype status on taught strategy use ( F(1, 107) = 6.04, p = 0.016, Δ R2 = 0.053). Findings suggest that strategy type, maternal Broad Autism Phenotype status, and preintervention strategy use may be important factors to be considered to individualize parent-mediated interventions. Lay Abstract Parent-mediated interventions support parents’ use of language facilitation strategies to improve their autistic child’s communication and language development. To improve the effectiveness of parent-mediated interventions, it is important to individualize interventions. This article evaluates how different components of parent-mediated interventions and mothers’ learning styles influence the effectiveness of the intervention. In a randomized clinical trial, mothers were taught to use one of two types of language facilitation strategies: responsive and directive. Mothers’ learning styles were characterized by the Broad Autism Phenotype (BAP) and their natural tendency to use language facilitation strategies before intervention. Findings suggest that it was easier for all mothers (irrespective of learning style) to use responsive strategies compared to directive strategies. In addition, mothers with learning styles that were not consistent with the BAP were more likely to benefit from the intervention if they did not naturally use strategies before the intervention. In contrast, mothers with learning styles that were consistent with the BAP were more likely to benefit from the intervention if they did naturally use strategies before the intervention. Teaching mothers to use responsive strategies results in greater strategy use. Consideration of BAP and mothers’ natural use of language facilitation strategies may inform intervention individualization.

STUDY AIM The goal of this research is to develop and validate a novel, disease-specific patient-reported outcome measure (PROM) for use in clinical monitoring and therapeutic trials of Crohn’s Disease (CD). BACKGROUND With approximately 780,000 cases of CD in the U.S., it is important to have a comprehensive, sensitive, and reliable disease-specific PROM that accurately indicates patient function, perceived health, and quality of life. Moreover, as clinical trials are planned for individuals with CD, a valid PROM is vital to determine the overall effect of potential treatments on patient disease burden. This research describes the construction and testing of the Crohn’s Disease-Health Index (CD-HI) as an efficient mechanism to quantify how patients with CD feel and function, and to measure key changes in a patient’s multifactorial health. METHODS We conducted semi-structured, qualitative interviews with 16 CD patients to determine the most frequent and important symptoms in CD. Based on patient responses, we designed a survey which inquired about 148 symptoms from 17 symptomatic themes. This survey was used in a cross-sectional study with 403 patients with CD recruited through the Crohn’s and Colitis Foundation and IBD Partners. The collected data was used to generate the first version of the CD-HI, which contained symptom questions that had the highest impact to the population and the potential to respond to therapeutic intervention. Symptom questions were grouped into distinct symptomatic themes using factor analysis. We beta-tested the CD-HI with 15 patients with CD to evaluate its usability, clarity, and applicability. Based on patient feedback, we modified the instrument and evaluated its longitudinal reliability with 23 patients with CD. Known groups testing was performed with the final instrument. The final version of the health index is now available for use in clinical trials. RESULTS The final version of the CD-HI contains subscales that measure the following areas of CD health: 1) fatigue, 2) dietary restrictions, 3) gastrointestinal health, 4) sleep and daytime sleepiness, 5) bowel and bladder function, 6) emotional wellbeing, 7) joint health, 8) pain, 9) neck and back health, 10) activity participation, 11) social wellbeing, and 12) skin health. Total CD disease burden is measured using a weighted composite of these subscale scores. During beta interviews, patients with CD deemed the instrument to be easy to use, clear, relevant, and comprehensive. Statistical analyses confirmed the reliability, validity, sensitivity, specificity, and internal consistency of the instrument. CONCLUSIONS Our research demonstrates the validity of the CD-HI as a marker of disease burden in CD. This disease-specific PROM provides researchers and clinicians with a reliable tool to use in patient monitoring and clinical trials.

Background Patients with Crohn’s disease experience a wide variety of clinical symptoms that affect how they feel and function. As therapeutic trials are planned for patients with Crohn’s disease, it is important to better understand the symptoms that have the greatest impact on Crohn’s disease patient’s lives. Objective To identify the most common and important disease manifestations in Crohn’s disease in a large population of patients. To determine the modifying factors that are associated with these symptoms. Methods We conducted a national cross-sectional study of 415 patients from the IBD Partners patient registry sponsored by the Crohn’s & Colitis Foundation to identify the prevalence and relative importance of 148 individual symptoms across 17 unique symptomatic themes. These themes were previously identified through 16 semi-structured qualitative interviews with Crohn’s patients. Results Crohn’s disease participants provided over 55,000 symptom rating responses. The symptomatic themes with the highest prevalence in Crohn’s disease were gastrointestinal issues (93.0%), fatigue (86.4%), dietary restrictions (77.9%), impaired sleep or daytime sleepiness (75.6%), and inability to do activities (72.3%). Symptomatic theme prevalence was widely associated with having above the median number of stools per day, having above the median number of bowel movements per day, having perianal disease, having to miss work, and unemployment. Discussion/Conclusion: Crohn’s disease symptoms, some under-recognized, vary based on disease characteristics and demographic features. These symptoms represent targets for future therapeutic interventions and are potential areas of interest for an upcoming disease-specific patient-reported outcome measure for this population. Acknowledgements Funding for this project was provided by UR Ventures. Research activities were conducted in collaboration with the Crohn’s & Colitis Foundation.

Access to early intervention as early in development as possible is critical to maximizing long-term outcomes for children with autism spectrum disorders (ASD). However, despite the fact that ASD can be reliably diagnosed by 24 months, the average age of diagnosis is 2 years later. Waitlists for specialized developmental evaluations are one barrier to early diagnosis. The purpose of this study was to examine one potential approach to reducing wait time for an ASD diagnostic evaluation by examining the utility of using more than one threshold for an autism screening tool, the Screening Tool for Autism in Toddlers and Young Children (STAT). Participants included 171 children between 24 and 36 months of age who received a medical diagnostic evaluation through Illinois' Early Intervention Program. This study directly compared the performance of the STAT when scored: (a) using the original single threshold, (b) using seven equally weighted items using a single threshold, and (c) using all items differentially weighted based on how strongly that item predicts a later ASD diagnosis. In addition, this study explored the potential utility of using two thresholds rather than a single threshold for each scoring method. Results of this study suggest that using a two-threshold logistic regression method has potential psychometric advantages over a single threshold and categorical scoring. Using this approach may reduce the wait time for specialty ASD diagnostic evaluations by maximizing true negatives and true positives, such that specialty evaluations may be reserved for those cases that are more ambiguous or more complex. Autism Research 2019, 12: 112-122. © 2018 International Society for Autism Research, Wiley Periodicals, Inc. LAY SUMMARY: This study examined the benefits of using two versus one cutoff score when screening for autism. Results indicate that having two scores and weighting test items based on predictive association with an autism diagnosis is better than using a single score and weighting each item equally. Using such an approach may reduce the wait time for specialty autism diagnostic evaluations, such that specialty evaluations may be reserved for those cases that are more ambiguous or more complex.

BACKGROUND Patient-reported outcomes are increasingly utilized in routine orthopedic clinical care. Computer adaptive tests (CATs) from the Patient-Reported Outcomes Measurement Information System (PROMIS) offer a brief and precise assessment that is well suited for collection within busy clinical environments. However, software apps that support the administration and scoring of CATs, provide immediate access to patient-reported outcome (PRO) scores, and minimize clinician burden are not widely available. OBJECTIVE Our objective was to design, implement, and test the feasibility and usability of a Web-based system for collecting CATs in orthopedic clinics. METHODS AO Patient Outcomes Center (AOPOC) was subjected to 2 rounds of testing. Alpha testing was conducted in 3 orthopedic clinics to evaluate ease of use and feasibility of integration in clinics. Patients completed an assessment of PROMIS CATs and a usability survey. Clinicians participated in a brief semistructured interview. Beta-phase testing evaluated system performance through load testing and usability of the updated version of AOPOC. In both rounds of testing, user satisfaction, bugs, change requests, and performance of PROMIS CATs were captured. RESULTS Patient feedback supported the ease of use in completing an assessment in AOPOC. Across both phases of testing, clinicians rated AOPOC as easy to use but noted difficulties in integrating a Web-based software application within their clinics. PROMIS CATs performed well; the default assessment of 2 CATs was completed quickly (mean 9.5 items) with a satisfactory range of measurement. CONCLUSION AOPOC was demonstrated to be an easy-to-learn and easy-to-use software application for patients and clinicians that can be integrated into orthopedic clinical care. The workflow disruption in integrating any type of PRO collection must be addressed if patients’ voices are to be better integrated in clinical care.