Patient-Reported Impact of Symptoms in Fibromyalgia (PRISM-FM).

J Rheumatol 2024; doi: 10.3899/jrheum.2023-0698 Lynne Kennedy Matallana, MS, was missing from the author byline and footnotes. Her affiliation is National Fibromyalgia Association, Newport Beach, California, USA. She declares no conflicts of interest relevant to the article. This correction applies only to the February 15 2024 First Release. The correct author byline and footnotes appear in the print and online issues.

Development and validation of an episodic memory measure in the Mobile Toolbox (MTB): Arranging Pictures.

INTRODUCTION: Arranging Pictures is a new episodic memory test based on the NIH Toolbox (NIHTB) Picture Sequence Memory measure and optimized for self-administration on a personal smartphone within the Mobile Toolbox (MTB). We describe evidence from three distinct validation studies. METHOD: In Study 1, 92 participants self-administered Arranging Pictures on study-provided smartphones in the lab and were administered external measures of similar and dissimilar constructs by trained examiners to assess validity under controlled circumstances. In Study 2, 1,021 participants completed the external measures in the lab and self-administered Arranging Pictures remotely on their personal smartphones to assess validity in real-world contexts. In Study 3, 141 participants self-administered Arranging Pictures remotely twice with a two-week delay on personal iOS smartphones to assess test-retest reliability and practice effects. RESULTS: Internal consistency was good across samples (ρxx = .80 to .85, p < .001). Test-retest reliability was marginal (ICC = .49, p < .001) and there were significant practice effects after a two-week delay (ΔM = 3.21 (95% CI [2.56, 3.88]). As expected, correlations with convergent measures were significant and moderate to large in magnitude (ρ = .44 to .76, p < .001), while correlations with discriminant measures were small (ρ = .23 to .27, p < .05) or nonsignificant. Scores demonstrated significant negative correlations with age (ρ = -.32 to -.21, p < .001). Mean performance was slightly higher in the iOS compared to the Android group (MiOS = 18.80, NiOS = 635; MAndroid = 17.11, NAndroid = 386; t(757.73) = 4.17, p < .001), but device type did not significantly influence the psychometric properties of the measure. Indicators of potential cheating were mixed; average scores were significantly higher in the remote samples (F(2, 850) = 11.415, p < .001), but there were not significantly more perfect scores. CONCLUSION: The MTB Arranging Pictures measure demonstrated evidence of reliability and validity when self-administered on personal device. Future research should examine the potential for cheating in remote settings and the properties of the measure in clinical samples.

Measurement Invariance in Intellectual and Developmental Disability Research.

Measurement invariance (MI) is a psychometric property of an instrument indicating the degree to which scores from an instrument are comparable across groups. In recent years, there has been a marked uptick in publications using MI in intellectual and developmental disability (IDD) samples. Our goal here is to provide an overview of why MI is important to IDD researchers and to describe some challenges to evaluating it, with an eye towards nudging our subfield into a more thoughtful and measured interpretation of studies using MI.

Development and Validation of a Vocabulary Measure in the Mobile Toolbox.

OBJECTIVE: We describe the development of a new computer adaptive vocabulary test, Mobile Toolbox (MTB) Word Meaning, and validity evidence from 3 studies. METHOD: Word Meaning was designed to be a multiple-choice synonym test optimized for self-administration on a personal smartphone. The items were first calibrated online in a sample of 7,525 participants to create the computer-adaptive test algorithm for the Word Meaning measure within the MTB app. In Study 1, 92 participants self-administered Word Meaning on study-provided smartphones in the lab and were administered external measures by trained examiners. In Study 2, 1,021 participants completed the external measures in the lab and Word Meaning was self-administered remotely on their personal smartphones. In Study 3, 141 participants self-administered Word Meaning remotely twice with a 2-week delay on personal iPhones. RESULTS: The final bank included 1363 items. Internal consistency was adequate to good across samples (ρxx = 0.78 to 0.81, p < .001). Test-retest reliability was good (ICC = 0.65, p < .001), and the mean theta score was not significantly different upon the second administration. Correlations were moderate to large with measures of similar constructs (ρ = 0.67-0.75, p < .001) and non-significant with measures of dissimilar constructs. Scores demonstrated small to moderate correlations with age (ρ = 0.35 to 0.45, p < .001) and education (ρ = 0.26, p < .001). CONCLUSION: The MTB Word Meaning measure demonstrated evidence of reliability and validity in three samples. Further validation studies in clinical samples are necessary.

Developmental Associations between Cognition and Adaptive Behavior in Intellectual and Developmental Disability.

Background: Intellectual and developmental disabilities (IDDs) are associated with both cognitive challenges and difficulties in conceptual, social, and practical areas of living (DSM-5). Individuals with IDD often present with an intellectual disability in addition to a developmental disability such as autism or Down syndrome. Those with IDD may present with deficits in intellectual functioning as well as adaptive functioning that interfere with independence and living skills. The present study sought to examine associations of longitudinal developmental change in domains of cognition (NIH Toolbox Cognition Battery, NIHTB-CB) and adaptive behavior domains (Vineland Adaptive Behavior Scales-3; VABS-3) including Socialization, Communication, and Daily Living Skills (DLS) over a two-year period. Methods: Eligible participants for this multisite longitudinal study included those who were between 6 and 26 years at Visit 1, and who had a diagnosis of, or suspected intellectual disability (ID), including borderline ID. Three groups were recruited, including those with fragile X syndrome, Down syndrome, and other/idiopathic intellectual disability. In order to examine the association of developmental change between cognitive and adaptive behavior domains, bivariate latent change score (BLCS) models were fit to compare change in the three cognitive domains measured by the NIHTB-CB (Fluid, Crystallized, Composite) and the three adaptive behavior domains measured by the VABS-3 (Communication, DLS, and Socialization). Results: Over a two-year period, change in cognition (both Crystalized and Composite) was significantly and positively associated with change in daily living skills. Also, baseline cognition level predicted growth in adaptive behavior, however baseline adaptive behavior did not predict growth in cognition in any model. Conclusions: The present study demonstrated that developmental improvements in cognition and adaptive behavior are associated in children and young adults with IDD, indicating the potential for cross-domain effects of intervention. Notably, improvements in Daily Living Skills on the VABS-3 emerged as a primary area of adaptive behavior that positively related to improvements in cognition. This work provides evidence for the clinical, "real life" meaningfulness of the NIHTB-CB in IDD, and important empirical support for the NIHTB-CB as a fit-for-purpose performance-based outcome measure for this population.

The Crohn's Disease-Health Index: Development and Evaluation of a Novel Outcome Measure.

OBJECTIVE: We sought to develop and validate the Crohn's Disease-Health Index (CD-HI), a disease-specific, patient-reported outcome measure that serially measures Crohn's disease (CD) symptomatic burden in adults with CD. BACKGROUND: As therapeutic interventions are tested among patients with CD, responsive outcome measures are needed to track disease progression and therapeutic gain during clinical trials. PATIENTS AND METHODS: We conducted a national cross-sectional study of individuals with CD to identify the most prevalent and impactful symptoms of CD. The most relevant symptoms were included in the CD-HI. We used factor analysis, qualitative patient interviews, test-retest reliability evaluation, and known group validity testing to evaluate and optimize the CD-HI. RESULTS: The CD-HI contains 12 subscales that comprehensively measure CD burden using the patient's perspective. Fifteen adults with CD beta tested the CD-HI and found the instrument to be clear, easy to use, and relevant to them. Twenty-three adults with CD participated in an assessment of test-retest reliability, which indicated high reliability of individual questions, subscales, and the full instrument (intraclass correlation coefficient = 0.84 for the full instrument). The CD-HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.98 for the full instrument). The CD-HI distinguished between groups of individuals with CD known to differ in disease severity. CONCLUSIONS: This research supports the use of the CD-HI as a valid, sensitive, reliable, and relevant patient-reported outcome to determine the multifactorial disease burden of those with CD, assess the relevance and merit of future CD therapies, and support drug labeling claims.

Using the Mobile Toolbox in child and adolescent samples: A feasibility study.

Cognitive research with developmental samples requires improved methods that support large-scale, diverse, and open science. This paper offers initial evidence to support the Mobile Toolbox (MTB), a self-administered remote smartphone-based cognitive battery, in youth populations, from a pilot sample of 99 children (Mage = 11.79 years; 36% female; 53% White, 33% Black or African American, 9% Asian, and 15% Hispanic). Completion rates (95%-99%), practice performance (96%-100%), internal consistency (0.60-0.98), and correlations with similar NIHTB measures (0.55-0.77) provide the first evidence to support the MTB in a youth sample, although there were some inconsistencies across measures. Preliminary findings provide promising evidence of the MTB in developmental populations, and further studies are encouraged.

Patient-Reported Impact of Symptoms in Fibromyalgia (PRISM-FM).

OBJECTIVE: To identify the frequency and relative importance of symptoms experienced by adults with fibromyalgia (FM) and determine factors associated with a higher disease burden. METHODS: We conducted semistructured interviews with 15 participants with FM, collecting 1479 quotes regarding the symptomatic burden of FM. We then performed an international cross-sectional study involving 1085 participants with FM to determine the prevalence and relative importance (scale 0-4) of 149 symptoms representing 14 symptomatic themes. We performed subgroup analysis to determine how age, sex, disease duration, medication use, employment status, change in employment status, missing work due to FM, and ability level are related to symptomatic theme prevalence. RESULTS: The symptomatic themes with the highest prevalence in FM were pain (99.8%), muscle tenderness (99.8%), and fatigue (99.3%). The symptomatic themes that had the greatest effect on patients' lives were related to fatigue (2.88), pain (2.85), muscle tenderness (2.79), and impaired sleep and daytime sleepiness (2.70). Symptomatic theme prevalence was most strongly associated with the modified Rankin Scale level of disability, disability status, and change in employment status (on disability vs not on disability). CONCLUSION: Participants with FM identify a variety of symptoms that significantly affect their daily lives. Many of these symptoms, such as fatigue, sleep disturbance, and activity limitation, are life-altering and not related to traditional diagnostic criteria. Symptom prevalence in this population varies across subgroups based on demographic categories and disability status.

How Are Age, Gender, and Country Differences Associated With PROMIS Physical Function, Upper Extremity, and Pain Interference Scores?

BACKGROUND: The interpretation of patient-reported outcomes requires appropriate comparison data. Currently, no patient-specific reference data exist for the Patient-Reported Outcome Measurement Information System (PROMIS) Physical Function (PF), Upper Extremity (UE), and Pain Interference (PI) scales for individuals 50 years and older. QUESTIONS/PURPOSES: (1) Can all PROMIS PF, UE, and PI items be used for valid cross-country comparisons in these domains among the United States, the United Kingdom, and Germany? (2) How are age, gender, and country related to PROMIS PF, PROMIS UE, and PROMIS PI scores? (3) What is the relationship of age, gender, and country across individuals with PROMIS PF, PROMIS UE, and PROMIS PI scores ranging from very low to very high? METHODS: We conducted telephone interviews to collect custom PROMIS PF (22 items), UE (eight items), and PI (eight items) short forms, as well as sociodemographic data (age, gender, work status, and education level), with participants randomly selected from the general population older than 50 years in the United States (n = 900), United Kingdom (n = 905), and Germany (n = 921). We focused on these individuals because of their higher prevalence of surgeries and lower physical functioning. Although response rates varied across countries (14% for the United Kingdom, 22% for Germany, and 12% for the United States), we used existing normative data to ensure demographic alignment with the overall populations of these countries. This helped mitigate potential nonresponder bias and enhance the representativeness and validity of our findings. We investigated differential item functioning to determine whether all items can be used for valid crosscultural comparisons. To answer our second research question, we compared age groups, gender, and countries using median regressions. Using imputation of plausible values and quantile regression, we modeled age-, gender-, and country-specific distributions of PROMIS scores to obtain patient-specific reference values and answer our third research question. RESULTS: All items from the PROMIS PF, UE, and PI measures were valid for across-country comparisons. We found clinically meaningful associations of age, gender, and country with PROMIS PF, UE, and PI scores. With age, PROMIS PF scores decreased (age ß Median = -0.35 [95% CI -0.40 to -0.31]), and PROMIS UE scores followed a similar trend (age ß Median = -0.38 [95% CI -0.45 to -0.32]). This means that a 10-year increase in age corresponded to a decline in approximately 3.5 points for the PROMIS PF score-a value that is approximately the minimum clinically important difference (MCID). Concurrently, we observed a modest increase in PROMIS PI scores with age, reaching half the MCID after 20 years. Women in all countries scored higher than men on the PROMIS PI and 1 MCID lower on the PROMIS PF and UE. Additionally, there were higher T-scores for the United States than for the United Kingdom across all domains. The difference in scores ranged from 1.21 points for the PROMIS PF to a more pronounced 3.83 points for the PROMIS UE. Participants from the United States exhibited up to half an MCID lower T-scores than their German counterparts for the PROMIS PF and PROMIS PI. In individuals with high levels of physical function, with each 10-year increase in age, there could be a decrease of up to 4 points in PROMIS PF scores. Across all levels of upper extremity function, women reported lower PROMIS UE scores than men by an average of 5 points. CONCLUSION: Our study provides age-, gender-, and country-specific reference values for PROMIS PF, UE, and PI scores, which can be used by clinicians, researchers, and healthcare policymakers to better interpret patient-reported outcomes and provide more personalized care. These findings are particularly relevant for those collecting patient-reported outcomes in their clinical routine and researchers conducting multinational studies. We provide an internet application ( www.common-metrics.org/PROMIS_PF_and_PI_Reference_scores.php ) for user-friendly accessibility in order to perform age, gender, and country conversions of PROMIS scores. Population reference values can also serve as comparators to data collected with other PROMIS short forms or computerized adaptive tests. LEVEL OF EVIDENCE: Level II, diagnostic study.

Autistic Children's Irritability During Social Communication Assessments.

PURPOSE: Accurate measurement of autistic children's social communication is critical for assessing skills, setting intervention goals, evaluating change over time, determining service eligibility, and determining classroom placement. There are various types of assessments, some of which use specific tasks to elicit social communication. Structured tasks may frustrate children, inadvertently elicit irritability, and have a cascading effect on their ability to communicate. To date, no studies have evaluated how differing types of social communication assessments may exacerbate children's irritability and impact assessment scores. We examined the extent to which (a) social communication assessment type (structured vs. naturalistic) impacts autistic children's irritability and (b) child irritability is associated with social communication scores. METHOD: Autistic toddlers (n = 114, Mage = 33.09 months, SD = 6.15) completed the Communication and Symbolic Behavior Scales (CSBS; structured) and a 10-min play-based mother-child interaction (MCX; naturalistic). Child irritability was scored on both assessments using a global rating scale of 0-15. RESULTS: Child irritability during the CSBS was significantly higher than during the MCX (V = 4892, p < .001, r = .68). Higher irritability was associated with lower CSBS social communication scores (B = -0.05, p = .03), but not MCX scores (B = 0.04, p = .13; Theil's F = 6.92, p = .009). CONCLUSIONS: Our findings suggest that the CSBS may pose unique challenges for autistic children, as it led to higher rates of irritability and negatively affected children's social communication scores. Evaluating the association between assessment type and irritability supports the complete characterization of autistic children's experience during assessments and clinicians in obtaining a more representative measure of social communication.

Characterizing mechanisms of caregiver-mediated naturalistic developmental behavioral interventions for autistic toddlers: A randomized clinical trial.

LAY ABSTRACT: Caregiver-mediated early interventions support caregivers' use of strategies to improve their young autistic child's communication. In the current clinical trial, we sought to isolate the most effective strategies to improve short-term and long-term child communication outcomes. Results demonstrated how children may benefit from caregiver prompts to facilitate long-term language outcomes. In conclusion, the current study improves our understanding of how early intervention facilitates child communication outcomes.

A psychometric evaluation of the NIH Toolbox fluid cognition tests adapted for Swahili and Dholuo languages in Kenyan children and adolescents.

OBJECTIVE: Our objective was to evaluate the psychometric properties of the culturally adapted NIH Toolbox African Languages® when used in Swahili and Dholuo-speaking children in western Kenya. METHOD: Swahili-speaking participants were recruited from Eldoret and Dholuo-speaking participants from Ajigo; all were <14 years of age and enrolled in primary school. Participants completed a demographics questionnaire and five fluid cognition tests of the NIH Toolbox® African Languages program, including Flanker, Dimensional Change Card Sort (DCCS), Picture Sequence Memory, Pattern Comparison, and List Sorting tests. Statistical analyses examined aspects of reliability, including internal consistency (in both languages) and test-retest reliability (in Dholuo only). RESULTS: Participants included 479 children (n = 239, Swahili-speaking; n = 240, Dholuo-speaking). Generally, the tests had acceptable psychometric properties for research use within Swahili- and Dholuo-speaking populations (mean age = 10.5; SD = 2.3). Issues related to shape identification and accuracy over speed limited the utility of DCCS for many participants, with approximately 25% of children unable to match based on shape. These cultural differences affected outcomes of reliability testing among the Dholuo-speaking cohort, where accuracy improved across all five tests, including speed. CONCLUSIONS: There is preliminary evidence that the NIH Toolbox ® African Languages potentially offers a valid assessment of development and performance using tests of fluid cognition in Swahili and Dholuo among research settings. With piloting underway across other diverse settings, future research should gather additional evidence on the clinical utility and acceptability of these tests, specifically through the establishment of norming data among Kenyan regions and evaluating these psychometric properties.

What is typical: Atypical in young children's attention regulation?: Characterizing the developmental spectrum with the Multidimensional Assessment Profiles-Attention Regulation Infant-Toddler (MAPS-AR-IT) Scale.

While attention dysregulation is a promising early indicator of neurodevelopmental risk, in particular attention-deficit/hyperactivity disorder (ADHD), it is difficult to characterize clinical concern due to its developmental expectability at the transition to toddlerhood. Thus, explicating the typical:atypical continuum of risk indicators is among the key future directions for research to promote early identification and intervention, and prevent decrements in the attainment of developmental milestones into early childhood. In this paper, we present the Multidimensional Assessment Profiles-Attention Regulation Infant-Toddler (MAPS-AR-IT) Scale, a novel parent-report survey of dimensional, developmentally specified indicators of attention (dys)regulation. Item Response Theory was employed to characterize the typical:atypical spectrum of both normative and more concerning dysregulation (including the contexts in which behavior occurs). We provide evidence of the validity of this measure in capturing the full typical:atypical spectrum via a longitudinal sample of typically developing children at 12-18 months of age (baseline) via concurrent scores on well-validated temperament and clinical measures. We also examine longitudinal stability and predictive validity if the MAPS-AR-IT via a clinical interview of ADHD symptoms at 24-30 months (follow-up). While not diagnostic, we present evidence of the utility of the MAPS-AR-IT in explicating individual neurodevelopmental risk and elucidating the broader typicality of behaviors related to attention (dys)regulation.

Aunque la desregulación de la atención es un prometedor indicador temprano del riesgo neural de desarrollo, en particular el trastorno de déficit en la atención/hiperactividad (ADHD), es difícil caracterizar las preocupaciones clínicas debido al factor de expectativa de desarrollo al momento de la transición a la temprana niñez. De manera que explicar la progresión típica:atípica de indicadores de riesgo está entre las futuras directrices claves para la investigación con el fin de promover la temprana identificación e intervención, y prevenir disminuciones en el alcance de hitos críticos hacia la temprana niñez. En este ensayo, presentamos la Escala de Perfiles de Evaluación Multidimensional - Regulación de la Atención del Infante-Niño Pequeñito (MAPS-AR-IT) una novedosa encuesta de reporte del progenitor, acerca de la (des)regulación de la atención, dimensional y específica para el desarrollo. Aportamos evidencia de la validez de esta medida para captar la completa gama típica:atípica por medio de una muestra longitudinal de niños típicamente en desarrollo, a los 12-18 meses de edad (edad base) por medio de puntajes concurrentes sobre el temperamento bien validado y las medidas clínicas, así como también la estabilidad longitudinal y la validez de predicción por medio de una entrevista clínica de síntomas de ADHD a los 24-30 meses (seguimiento). Se empleó la Teoría de Respuesta al Asunto para caracterizar la gama típica:atípica tanto de la desregulación normativa como de la más preocupante (incluyendo los contextos en los cuales ocurre el comportamiento). Aunque no se trata de diagnóstico, presentamos evidencia de la utilidad de MAPS-AR-IT para explicar el riesgo individual de desarrollo neural y elucidar el más amplio aspecto típico de comportamientos relacionados con la (des)regulación de la atención.

Bien que la dysrégulation de l'attention soit un indicateur précoce prometteur du risque neurodéveloppemental, en particulier le trouble déficitaire de l'attention/hyperactivité (TDHA) il est difficile de caractériser la préoccupation clinique du fait de sa prévisibilité développementale à la transition à la petite enfance. Par conséquent, expliquer le continuum typique:atypique des indicateurs de risque s'avère être une des directions futures de recherches clé pour promouvoir l'identification et l'intervention précoce, et prévenir les baisses dans la réalisation d'étapes développementales importantes jusque dans la petite enfance. Dans cet article nous présentons l'Echelle Multidimensional Assessment Profiles - Attention Regulation Infant-Toddler (MAPS-AR-IT) (échelle de profils d'évaluation multidimensionnelle - régulation de l'attention bébé-petit enfant, abrégée selon l'anglais MAP-AR-IT), une étude nouvelle basée sur les rapports faits par les parents de la (dys)régulation de l'attention dimensionnelle et spécifiée selon le développement. Nous démontrons la validité de cette mesure en capturant l'éventail total typique:atypique au moyen d'un échantillon longitudinal d'enfants se développement typiquement, à 12-18 mois (ligne de case) au moyen de scores concurrents de mesures cliniques et de tempérament bien validées, ainsi qu''une stabilité longitudinale et d'une validité prédictive au moyen d'un entretien Clinique des symptômes THHA à 24-30 mois (suivi). La Item Response Theory (IRT) a été employée pour caractériser l'éventail typique:atypique de la dysrégulation à la fois normative et celle plus inquiétante (y compris les contextes dans lesquels le comportement prend place). Bien que cela ne soit pas diagnostique, nous présentons la preuve de l'utilité de la MAPS-AR-IT en expliquant le risqué neurodéveloppemental individuel et en élucidant la typicalité plus large de comportements liés à la (dys)régulation de l'attention.

Codesign of remote data collection for chronic management of pediatric home mechanical ventilation.

INTRODUCTION: Outpatient monitoring of children using invasive home mechanical ventilation (IHMV) is recommended, but access to care can be difficult. This study tested if remote (home-based) data collection was feasible and acceptable in chronic IHMV management. METHODS: A codesign study was conducted with an IHMV program, home nurses, and English- and Spanish-speaking parent-guardians of children using IHMV (0-17 years; n = 19). After prototyping, parents used a remote patient monitoring (RPM) bundle to collect patient heart rate, respiratory rate (RR), oxygen saturation, end-tidal carbon dioxide (EtCO2 ), and ventilator pressure/volume over 8 weeks. User feedback was analyzed using qualitative methods and the System Usability Scale (SUS). Expected marginal mean differences within patient measures when awake, asleep, or after a break were calculated using mixed effects models. RESULTS: Patients were a median 2.9 years old and 11 (58%) took breaks off the ventilator. RPM data were entered on a mean of 83.7% (SD ± 29.1%) weeks. SUS scores were 84.8 (SD ± 10.5) for nurses and 91.8 (SD ± 10.1) for parents. Over 90% of parents agreed/strongly agreed that RPM data collection was feasible and relevant to their child's care. Within-patient comparisons revealed that EtCO2 (break-vs-asleep 2.55 mmHg, d = 0.79 [0.42-1.15], p < .001; awake-vs-break 1.48, d = -0.49 [0.13-0.84], p = .02) and RR (break-vs-asleep 16.14, d = 2.12 [1.71-2.53], p < .001; awake-vs-break 3.44, d = 0.45 [0.10-0.04], p = .03) were significantly higher during ventilator breaks. CONCLUSIONS: RPM data collection in children with IHMV was feasible, acceptable, and captured clinically meaningful vital sign changes during ventilator breaks, supporting the clinical utility of RPM in IHMV management.

A Comparison of Maternal and Clinician Perception of Communication in Autistic Toddlers.

PURPOSE: Parent instruction in communication facilitation strategies for autistic toddlers relies on assumptions that parents interpret child behaviors in alignment with clinician definitions of communication. The purpose of this study was to identify features of child behaviors that are predictive of alignment in identification of child communication between clinical researchers and mothers of young autistic children. METHOD: Participants were 33 mothers and their autistic children between 18 and 48 months of age. Mothers' and clinical researchers' perceptions of child communication were assessed using a procedure in which mothers and clinical researchers each independently identified child communication in the same ten 1-min video clips of each mother's child. Endorsed communicative acts were coded for the presence of conventional forms (e.g., vocalization) and potentially communicative forms (e.g., body movement). Multilevel binomial regressions, fit with Bayesian inference, were conducted to predict classification of maternal endorsements of child communication based on the presence of conventional and potentially communicative forms as either an aligned act (i.e., act endorsed by mother and clinical researcher as communicative) or a unique maternal endorsement (i.e., act endorsed by mother but not clinical researcher). RESULTS: The presence of vocalization, verbalization, and gesture each significantly predicted increased likelihood of alignment; the presence of eye contact did not. Although repetitive and sensory behaviors significantly increased the likelihood of unique maternal endorsement, affect shifts and body movements each significantly reduced the likelihood of unique maternal endorsement, and hand activity was not significantly predictive of unique maternal endorsement. CONCLUSIONS: Misalignment in mothers' and clinical researchers' identification of communication may be in part due to mothers' endorsement of behavioral forms that are not traditionally classified as part of a child's communication repertoire. Findings emphasize the need to work toward designing communication interventions that consider the ways in which clinicians and parents of autistic children each bring their own interpretive frameworks to the early intervention experience.

The amyotrophic lateral sclerosis-health index (ALS-HI): development and evaluation of a novel outcome measure.

Objective: The identification of effective therapeutics for ALS necessitates valid and responsive outcome measures to track disease progression and therapeutic gain in clinical trial settings. The Amyotrophic Lateral Sclerosis-Health Index (ALS-HI) is a multifaceted, disease-specific patient-reported outcome measure (PRO) designed to measure ALS symptomatic disease burden in adults with ALS. Methods: Through a national cross-sectional study of individuals with ALS, we identified the most important symptoms in ALS. These symptoms were incorporated into the ALS-HI, a measure that quantifies the multifaceted disease burden in ALS. We performed factor analysis, qualitative patient interviews, test-retest reliability assessment, and known groups analysis to evaluate and validate the ALS-HI. Results: The cross-sectional study included 497 participants with ALS who identified the most important symptoms to include in the ALS-HI. Fifteen participants beta tested the ALS-HI and found it to be clear, easy to use, and relevant. Twenty-one participants engaged in a test-retest reliability study, which indicated the reliability of the instrument (intraclass correlation coefficient = 0.952 for full instrument). The final ALS-HI and its subscales demonstrated a high internal consistency (Cronbach's α = 0.981 for full instrument) and an ability to differentiate between groups with dissimilar disease severity. Conclusions: This research supports use of the ALS-HI as a valid, sensitive, reliable, and relevant PRO to assess the multifactorial disease burden faced by adults with ALS. The ALS-HI has potential as a mechanism to track disease progression and treatment efficacy during therapeutic trials.

Using bookmarking methods with orthopedic clinicians and patients with fractures produces score interpretation labels for patient-reported outcome measures.

OBJECTIVE: The objective of this study was to determine the patient-reported outcome measure (PROM) score ranges associated with descriptive labels (i.e., within normal limits, mild, moderate, severe) by using bookmarking methods with orthopedic clinicians and patients who have experienced a bone fracture. STUDY DESIGN AND SETTING: We created vignettes comprised of six items and responses from the Patient-Reported Outcomes Measurement Information System (PROMIS) Upper Extremity Function, Physical Function, and Pain Interference item banks reflecting different levels of severity. Two groups of patients with fractures (n = 11) and two groups of orthopedic clinicians (n = 16) reviewed the vignettes and assigned descriptive labels independently and then discussed as a group until reaching consensus via a videoconference platform. RESULTS: PROMIS Physical Function and Pain Interference thresholds (T = 50, 40, 25/30 and T = 50/55, 60, 65/70, respectively) for patients with bone fractures were consistent with the results from other patient populations. Upper Extremity thresholds were about 10 points (1 SD) more severe (T = 40, 30, 25/20) compared to the other measures. Patient and clinician perspectives were similar. CONCLUSION: Bookmarking methods generated meaningful score thresholds for PROMIS measures. These thresholds between severity categories varied by domain. Threshold values for severity represent important supplemental information to interpret PROMIS scores clinically.

Modifying the social responsiveness scale for adaptive administration.

PURPOSE: The social responsiveness scale (SRS) is frequently used to quantify the autism-related phenotype and is gaining use in health outcomes research. However, it has a high respondent burden (65 items) for large-scale studies. Further, most evaluations of it have focused on the school-age form, not the preschool form. More validity evidence of shortened forms is necessary in the general population to support the broader health outcomes context of use. METHODS: We evaluated the psychometrics of the SRS in 7030 individuals from multiple predominantly neurotypical samples in order to shorten it based on non-autistic sample metrics. Analyses included item factor analysis, differential item functioning (DIF), and multiple-group item response theory (IRT) to place the SRS items on a comparable scale, which was then simulated via computer adaptive testing (CAT) administration. RESULTS: The SRS was broadly unidimensional with few methodological residual dependencies. On average, males had more autistic characteristics than females, and preschoolers had fewer characteristics than school-age children. The final IRT calibration included 45 items equated across forms, and each form had 11 with significant wording discrepancies and 9 items with near-identical wording that exhibited form-related DIF. The CAT simulation suggested a median of 14 items was sufficient to reach a reliable score, demonstrating its feasibility across the range of impairments. CONCLUSION: IRT allows practitioners the ability to get highly reliable scores with fewer items than the full-length SRS. This supports the future application of the SRS in a computer adaptive testing mode in both neurotypical and ASD samples.

Comparing ability and norm-referenced scores as clinical trial outcomes for neurodevelopmental disabilities: a simulation study.

BACKGROUND: For genetic conditions associated with neurodevelopmental disorder (GCAND), developmental domains such as motor ability, thinking and learning, social abilities, and communication are potential intervention targets. Performance on measures of developmental concepts can be expressed using several types of scores. Norm-referenced scores are intended for the diagnostic context, allowing for the identification of impairment relative to age-based expectations, and can exhibit dramatic floor effects when used in individuals with more significant limitations. Person ability scores, which are derived via Rasch analysis or item response theory, are available on many standardized tests and are intended to measure within-person change. However, they have not been used or evaluated as primary endpoints in GCAND clinical trials. In this study, we simulated a series of parallel-arm clinical trials under several chronological age and impairment conditions, to compare empirically the power and type I error rate of operationalizing test performance using ability scores rather than norm-referenced scores. RESULTS: Using the Vineland Adaptive Behavior Scales as the example, we demonstrated an advantage in statistical power of ability scores over norm-referenced scores at extreme levels of impairment. This advantage was at least partially driven by floor effects in norm-referenced scores. For simulated conditions where impairment was less severe, ability scores outperformed norm-referenced scores, but they were more similar. The type I error rate closely approximated the nominal type I error rate of 5% for both scores. CONCLUSION: The results of this simulation demonstrate a substantial power and interpretative advantage of ability scores over norm-referenced scores for studies of GCAND that will enroll participants with high levels of impairment. These results are expected to generalize to studies of developmental concepts, regardless of the etiology or specific test. However, the relative advantage of ability scores is expected to be even greater for tests with a higher floor than the Vineland.

Teaching Caregivers to Support Social Communication: Results From a Randomized Clinical Trial of Autistic Toddlers.

BACKGROUND: Studies of early caregiver-mediated interventions targeting social communication of young autistic children have yielded variable child outcomes. This study examined the effects of combining two caregiver-mediated interventions on caregiver strategy use and child social communication and language outcomes. METHOD: This was a multisite parallel randomized controlled trial. Participants included 120 caregivers and their autistic children between 24 and 36 months of age. Dyads were randomly assigned to receive a hybrid intervention that combined Enhanced Milieu Teaching (EMT) and Joint Attention, Symbolic Play, Engagement, and Regulation (JASPER) or to a behavior management control condition, each delivered over 6 months. Caregivers in the JASP-EMT group received twice-weekly, in-home, and hour-long sessions. Outcomes were measured at baseline, the end of intervention (T1), and 6 months later (T2) and included a naturalistic language sample procedure, standardized measures, and caregiver report measures. This trial was registered at clinicaltrials.gov (NCT02595697). RESULTS: Child outcomes did not differ between conditions at T1 or T2 for child primary (social communication) or secondary (language, play, and autism symptoms) outcomes. Relative to control group caregivers, intervention group caregivers demonstrated significantly higher use of JASP-EMT strategies at T1 and T2, with the exception of two strategies (Responsiveness and Matched Responsiveness), which were used significantly more by control group caregivers. Neither autism severity nor baseline caregiver responsiveness moderated outcomes. Post hoc analyses revealed significant correlations between specific strategies and all child outcomes. CONCLUSIONS: Twice-weekly caregiver-mediated intervention that taught caregivers of autistic children to use social communication support strategies did not yield significant child outcomes. Future studies should examine possible sources for the lack of main effects including unexpected differences in linguistic features of caregiver input, changes in control group caregiver behavior, and insufficient intervention dosage. SUPPLEMENTAL MATERIAL: https://doi.org/10.23641/asha.21714278.