Life satisfaction for adolescents with developmental and behavioral disabilities during the COVID-19 pandemic.

BACKGROUND: This study aimed to identify contextual factors associated with life satisfaction during the COVID-19 pandemic for adolescents with mental, emotional, behavioral, and developmental (MEBD) disabilities. METHODS: Data were collected from a sample of 1084 adolescents aged 11-21 years from April 2020 to August 2021. This cross-sectional study used a sequential machine learning workflow, consisting of random forest regression and evolutionary tree regression, to identify subgroups of adolescents in the Environmental influences on Child Health Outcomes (ECHO) consortium who demonstrated enhanced vulnerability to lower life satisfaction as described by intersecting risk factors, protective factors, and MEBD disabilities. RESULTS: Adolescents with a history of depression, anxiety, autism, and attention-deficit/hyperactivity disorder were particularly susceptible to decreased life satisfaction in response to unique combinations of stressors experienced during the COVID-19 pandemic. These stressors included decreased social connectedness, decreased family engagement, stress related to medical care access, pandemic-related traumatic stress, and single-caregiver households. CONCLUSION: Findings from this study highlight the importance of interventions aimed specifically at increasing adolescent social connectedness, family engagement, and access to medical support for adolescents with MEBD disabilities, particularly in the face of stressors, such as a global pandemic. IMPACT: Through a machine learning process, we identified contextualized risks associated with life satisfaction among adolescents with neurodevelopmental disabilities during the COVID-19 pandemic. The COVID-19 pandemic resulted in large-scale social disruptions for children and families. Such disruptions were associated with worse mental health outcomes in the general pediatric population, but few studies have examined specific subgroups who may be at heightened risk. We endeavored to close that gap in knowledge. This study highlights the importance of social connectedness, family engagement, and access to medical support as contributing factors to life satisfaction during the COVID-19 pandemic for adolescents with neurodevelopmental disabilities.

Combining developmental and sleep health measures for autism spectrum disorder screening: an ECHO study.

BACKGROUND: Sleep problems are reported for up to 80% of autistic individuals. We examined whether parsimonious sets of items derived from the Modified Checklist for Autism in Toddlers, Revised (M-CHAT-R) and the Brief Infant Sleep Questionnaire (BISQ) are superior to the standard M-CHAT-R in predicting subsequent autism spectrum disorder (ASD) diagnoses. METHODS: Participants from 11 Environmental influences on Child Health Outcomes (ECHO) cohorts were included. We performed logistic LASSO regression models with 10-fold cross-validation to identify whether a combination of items derived from the M-CHAT-R and BISQ are superior to the standard M-CHAT-R in predicting ASD diagnoses. RESULTS: The final sample comprised 1552 children. The standard M-CHAT-R had a sensitivity of 44% (95% CI: 34, 55), specificity of 92% (95% CI: 91, 94), and AUROC of 0.726 (95% CI: 0.663, 0.790). A higher proportion of children with ASD had difficulty falling asleep or resisted bedtime during infancy/toddlerhood. However, LASSO models revealed parental reports of sleep problems did not improve the accuracy of the M-CHAT-R in predicting ASD diagnosis. CONCLUSION: While children with ASD had higher rates of sleep problems during infancy/toddlerhood, there was no improvement in ASD developmental screening through the incorporation of parent-report sleep metrics. IMPACT: Parental-reported sleep problems are common in autism spectrum disorder (ASD). We investigated whether the inclusion of parental-reports of infant/toddler sleep patterns enhanced the effectiveness of developmental screening for autism. We reported higher rates of difficulty falling asleep and resisting bedtime during infancy and toddlerhood among children later diagnosed with ASD; however, we did not find an improvement in ASD developmental screening through the incorporation of parent-report sleep metrics. In our sample, the standard M-CHAT-R had a sensitivity of 39% among children of mothers with government insurance compared with a sensitivity of 53% among children of mothers with employer-based insurance.

Assessing the interchangeability of linked scores in multivariable statistical analyses.

PURPOSE: Using the lens of classical test theory, we examine a linkage's generalizability with respect to use in multivariable analyses, including multiple regression and structural equation modeling, rather than comparison of established subpopulations as is most common in the literature. METHODS: To aid in this evaluation, we present a structural-equation-modeling based statistical method to examine the suitability of a given linkage for use cases involving continuous and categorical variables external to the linkage itself. RESULTS: Using the PROMIS® Parent Proxy and Early Childhood Global Health measures, we show that, although a high correlation between the scores (here, r = .829) may imply a general suitability for linking, a more detailed investigation of content, measurement structure, and results of the proposed methodology reveal important differences between the measures which can compromise interchangeability in certain use cases. CONCLUSION: In addition to the statistical quality of a linkage, users of linking methodology should also assess the question of whether the linkage is appropriate to apply to particular use cases of interest.

Development of the PROMIS pediatric stigma and extension to the PROMIS pediatric stigma: skin item banks.

PURPOSE: To develop the PROMIS Pediatric Stigma (PPS) and Skin (PPS-Skin) by constructing a common metric for measuring stigma in children with various conditions, while capturing the unique features of each condition. METHODS: Data from 860 children, ages 8-17, with a diagnosis of epilepsy, pNF (neurofibromatosis type 1 associated neurofibroma plexform), MD (muscular dystrophy), cancer, or skin conditions recruited from three projects were analyzed. Children with epilepsy, pNF and MD (sample-1) completed the original 18-item Neuro-QoL Stigma, while children with cancer and skin conditions (e.g., atopic dermatitis, psoriasis, and genetic skin disorders; sample-2) completed a 16-item version and 6 additional skin related items. Exploratory factor analysis (EFA) and confirmatory analysis (CFA) were used to evaluate unidimensionality of 24 stigma items. Differential item functioning (DIF) was used to evaluate measurement equivalence on group, gender, age, and conditions. Item response theory model (IRT) was used to construct the final measure. RESULTS: Sufficient unidimensionality was supported by both EFA and CFA. No items showed significant DIF indicating stable measurement properties across groups of comparison. All items fit the IRT model and were able to be calibrated together to form the PPS which consists of 18 core items. The PPS-Skin (18 cores items + 6 skin items) was developed by calibrating 6 skin items onto the common metric as the PPS. CONCLUSIONS: We used IRT techniques to successfully develop the PPS and the PPS-Skin, which share a common metric and account for unique and common concerns related to chronic conditions.

Prevalence and associations of fatigue in childhood atopic dermatitis: A cross-sectional study.

BACKGROUND: Fatigue is a symptom that can negatively impact patients' quality of life. However, the relationship of AD with fatigue has not been fully studied, especially in children. OBJECTIVE: To determine the prevalence of fatigue in AD patients, and whether AD severity, demographics and comorbidities are associated with increased fatigue in children. METHODS: A cross-sectional observational study was performed among 248 children with AD. Paediatric patients (ages 8-17 years) and parents (of children ages 0-17 years) completed a questionnaire, including demographics, history of atopic comorbidities and validated severity measures of AD, itch, pain, sleep disturbance, sleep-related impairment and fatigue. AD severity was also assessed by clinician-reported Eczema Area and Severity Index (EASI), Scoring AD (SCORAD) and Investigator's Global Assessment (IGA). Fatigue was assessed using Patient Reported Outcome Measurement Information System (PROMIS) Pediatric Fatigue T-score. RESULTS: Most children with AD had no (38.6%) or mild (32.1%) fatigue, with fewer having moderate (27.2%) or severe (2%) fatigue. Moderate/severe PROMIS Pediatric fatigue T-scores were increased with moderate (25.7%/1.4%) and severe (39.3%/5.4%) IGA vs. mild IGA (18.0%/0.0%) and those with 5-6 (44.4%/0.0%) and 7 (44.2%/5.2%) nights of SD from eczema. Moderate-severe PROMIS Pediatric Fatigue T-scores were associated with history of hay fever (adjusted OR [95% Cl]: 2.803 [1.395-5.632]) and family income (<$100,000: 3.049 [1.294-7.181]), but inversely with Black (0.40 [0.168-0.969]) and AAPI (0.285 [0.094-0.859]) race. In multivariable regression models controlling for demographic factors, PROMIS Pediatric Fatigue T-score was significant with more severe scores for IGA, POEM, EASI, SCORAD, NRS-itch, SCORAD-itch, average itch in the past 7 days, PROMIS Pediatric Pain severity, PROMIS Pediatric SD, PROMIS Pediatric SRI, SCORAD-sleep and more frequent SD from AD. CONCLUSIONS: Fatigue is a common yet underappreciated symptom in children with AD, particularly those with moderate-severe AD, and warrants more attention in clinical practice and trials.

Self-Reported Health Outcomes of Children and Youth with 10 Chronic Diseases.

OBJECTIVES: To identify pediatric patient-reported outcomes (PROs) that are associated with chronic conditions and to evaluate the effects of chronic disease activity on PROs. STUDY DESIGN: Participants (8-24 years old) and their parents were enrolled into 14 studies that evaluated Patient-Reported Outcome Measurement Information System PROs across 10 chronic conditions-asthma, atopic dermatitis, cancer, cancer survivors, chronic kidney disease, Crohn's disease, juvenile idiopathic arthritis, lupus, sickle cell disease, and type 1 diabetes mellitus. PRO scores were contrasted with the US general population of children using nationally representative percentiles. PRO-specific coefficients of variation were computed to illustrate the degree of variation in scores within vs between conditions. Condition-specific measures of disease severity and Cohen d effect sizes were used to examine PRO scores by disease activity. RESULTS: Participants included 2975 child respondents and 2392 parent respondents who provided data for 3409 unique children: 52% were 5-12 years old, 52% female, 25% African American/Black, and 14% Hispanic. Across all 10 chronic conditions, children reported more anxiety, fatigue, pain, and mobility restrictions than the general pediatric population. Variation in PRO scores within chronic disease cohorts was equivalent to variation within the general population, exceeding between-cohort variation by factors of 1.9 (mobility) to 5.7 (anxiety). Disease activity was consistently associated with poorer self-reported health, and these effects were weakest for peer relationships. CONCLUSIONS: Chronic conditions are associated with symptoms and functional status in children and adolescents across 10 different disorders. These findings highlight the need to complement conventional clinical evaluations with those obtained directly from patients themselves using PROs.

Measuring PROMIS® Physical Activity and Sleep Problems in Early Childhood.

OBJECTIVE: Physical activity (PA) and sleep are leading health indicators for individuals of all ages. Monitoring young children's PA and sleep using psychometrically sound instruments could help facilitate timely interventions to promote healthy development. This article describes the development of the PROMIS® Early Childhood (EC) Parent Report Physical Activity (PA) and Sleep Problems (SP) measures for children aged 1-5 years. METHODS: Item pools were generated by interviewing parents, input from content experts, and literature review. Data from a U.S. general population sample were used to determine factor structures of item pools via factor analytic approaches, estimate item parameters via item response theory (IRT) models, and establish norms. Pearson correlations were used to evaluate across-domain associations. Analysis of variance was used and known-groups' validity of PA and SP by comparing their scores to PROMIS EC Parent Report Global Health: child's physical, emotional, and mental conditions. RESULTS: Initial item pools consisted of 19 and 26 items for PA and SP, respectively. Factor analyses' results supported unidimensionality of 5 and 16 items measuring PA and SP, respectively, which were then calibrated using IRT. Norms were established by centering to a probability-based U.S. general population. Computerized adaptive testing algorithms were established. Some analyses supported initial measure validity. CONCLUSIONS: The PROMIS EC PA calibrated scale and SP item banks are user-friendly and brief, yet produce precise scores. Both measures enable psychometrically sound assessment of PA behavior and sleep problems. Future studies to comprehensively evaluate the validity of these two measures are warranted.

Psychometric Considerations in Developing PROMIS® Measures for Early Childhood.

OBJECTIVE: The early expression of lifespan health and disease states can often be detected in early childhood. Currently, the Patient-Reported Outcome Measurement Information System (PROMIS®) includes over 300 measures of health for individuals ages 5 years and older. We extended PROMIS to early childhood by creating developmentally appropriate, lifespan coherent parent-report measures for 1-5-year-olds. This paper describes the psychometric approaches used for these efforts. METHODS: 2 waves of data from parents of children ages 1-5 were collected via 2 internet panel companies. Wave 1 data (n = 1,400) were used to evaluate item pool unidimensionality, model fit, and initial item parameters. Combined data from wave 1 and wave 2 (reference sample; n = 1,057) were used to estimate final item parameters. Using item response theory methods, we developed and tested 12 item pools: Global Health, Physical Activity, Sleep Disturbance, Sleep-related Impairment, Anger/Irritability, Anxiety, Depressive Symptoms, Positive Affect, Self-Regulation, Engagement, Family Relationships, and Peer Relationships. RESULTS: Wave 1 analyses supported the unidimensionality of Physical Activity, Positive Affect, Anger/Irritability, Anxiety, Depressive Symptoms, and Global Health. Family Relationships and Peer Relationships were combined to form "Social Relationships"; Sleep Disturbance and Sleep-related Impairment were combined to form "Sleep Problems." Self-Regulation was divided into "Flexibility" and "Frustration Tolerance"; Engagement was divided into "Curiosity" and "Persistence." Short forms were developed for item banks with more than 10 items; and. CONCLUSIONS: Using rigorous mixed-methods, we successfully extended PROMIS to early childhood (1-5-year-olds). Measures are now publicly available in English and Spanish (www.healthmeasures.net).

Measuring PROMIS® Well-Being in Early Childhood.

OBJECTIVE: Expand the current Patient-Reported Outcome Measurement Information System (PROMIS®) well-being measures to early childhood (1-5 years) using best practices from PROMIS and developmental science. METHODS: Qualitative methods included expert input, literature and measure review, and parent interviews to confirm measure frameworks, item understandability, and developmental appropriateness. Quantitative methods included two waves of field testing and item response theory (IRT)-based psychometric evaluation of reliability and validity, as well as IRT centering and item calibration. Correlational analyses with other PROMIS Early Childhood (EC) Parent Report measures and known-group differences analyses by health status were conducted to evaluate construct validity. All measures were normed to the general U.S. population. RESULTS: Qualitative results suggested three primary early childhood well-being domains: Positive Affect, Engagement, and Self-Regulation. Quantitative results revealed a unidimensional factor structure for Positive Affect and multidimensional factor structures for Engagement and Self-Regulation, both of which had two factors accounting for >10% of modeled variance reflecting unique unidimensional domains. This resulted in five final PROMIS EC well-being measures: Positive Affect, Engagement-Curiosity, Engagement-Persistence, Self-Regulation-Flexibility, and Self-Regulation-Frustration Tolerance. Correlations and known-groups differences analyses showed robust construct validity across a range of chronic health conditions. CONCLUSIONS: The new PROMIS EC Parent Report well-being measures offer clinicians and researchers a brief, efficient, and precise way to evaluate young children's well-being. All five measures include only positively valanced item content, which pushes the field to evaluate the presence of children's positive assets rather than the absence of problems.

Measuring PROMIS® Social Relationships in Early Childhood.

OBJECTIVE: Apply the Patient-Reported Outcome Measurement Information System (PROMIS®) mixed-methods approach to develop and validate new parent-report measures of young children's (1-5 years) family and peer relationships that conceptually align to those for 5-17 year olds. METHODS: Expert input, parent interviews, and reviews of theoretical and empirical literature were used to develop draft item pools, which were administered in two waves of panel surveys (N = 1,750). Psychometric evaluation was conducted using item response theory-based methods. Scores were normed to the general U.S. population. Initial validation analyses were conducted using Pearson's correlations and analysis of variance to examine known-group differences between children with various health conditions. RESULTS: Experts and parents confirmed the content validity of existing PROMIS family and peer relationships domain frameworks and suggested adding child-caregiver interactions and empathic behaviors, respectively. Bi-factor model analysis supported sufficient unidimensionality where family and peer relationships were modeled as distinct subdomains of a broader concept, Social Relationships. The new measure was robust in discriminating young children with poor social relationships. Correlational and known-group analyses revealed positive associations with general health and well-being and negative associations with emotional and physical distress. CONCLUSIONS: The PROMIS Early Childhood Parent-Report Social Relationships item bank enables clinicians and researchers a brief, efficient, and precise way to evaluate early relational health. Subdomain short forms also offer the ability to assess specific components (i.e., child-caregiver, family, and peer) for more targeted interventions and analyses.

Measuring PROMIS® Global Health in Early Childhood.

OBJECTIVE: Assessing general ("global") health is important to clinicians caring for patients, researchers studying patient subgroups, and epidemiologists tracking population trends. The Patient-Reported Outcomes Measurement Information System® (PROMIS®) introduced an adult self-report Global Health measure (ages 18+) in 2009 and pediatric versions (ages 5-17 years) in 2014. Our aim was to extend global health assessment to 1-5-year olds. METHODS: We used the PROMIS mixed-methods approach to develop PROMIS Early Childhood (EC) Global Health, emphasizing qualitative measure development guidance utilizing input from experts and parents. Quantitatively, we conducted two data collection waves with parents of 1-5-year olds and applied state-of-the-science measure development methods, including exploratory, confirmatory, and bi-factor analytics, particularly regarding potentially multi-dimensional Global Health item content. We conducted a series of hypothesis-based across-domain association analyses, which were more exploratory in nature, and known-groups validity analyses. RESULTS: Experts emphasized the physical, mental, and social facets of global health, and parents described the broader, overarching construct. Using Waves 1 (N = 1,400) and 2 (N = 1,057) data, we retained six items directly sourced from the age 5-17 version and two new items. The resulting 8-item PROMIS EC Global Health was sufficiently unidimensional, so we fit item responses to the graded response model for parameter estimation. This produced an 8-item scale with one total score. Across-domain associations and known-groups validity analyses largely supported our hypotheses. CONCLUSIONS: We achieved our aim to extend global health assessment to 1-5-year olds and to thereby expand the range of PROMIS life course global health assessment from children aged 1-17 years, to adults of all ages.

Measuring PROMIS® Emotional Distress in Early Childhood.

OBJECTIVE: Create and validate developmentally sensitive parent-report measures of emotional distress for children ages 1-5 years that conceptually align with the Patient-Reported Outcome Measurement Information System (PROMIS®) pediatric measures. METHODS: Initial items were generated based on expert and parent input regarding core components of emotional distress in early childhood and review of theoretical and empirical work in this domain. Items were psychometrically tested using data from two waves of panel surveys. Item response theory (IRT) was applied to develop item calibration parameters (Wave 1), and scores were centered on a general U.S. population sample (Wave 2). Final PROMIS early childhood (EC) instruments were compared with existing measures of related constructs to establish construct validity. RESULTS: Experts and parents confirmed the content validity of the existing PROMIS Pediatric emotional distress domains (i.e., anger, anxiety, and depressive symptoms) as developmentally salient for young children. Existing items were adapted and expanded for early childhood by employing best practices from developmental measurement science. Item banks as well as 4- and 8-item short forms, free from differential item functioning across sex and age, were constructed for the three domains based on rigorous IRT analyses. Correlations with subscales from previously validated measures provided further evidence of construct validity. CONCLUSIONS: The PROMIS EC Anger/Irritability, Anxiety, and Depressive Symptoms measures demonstrated good reliability and initial evidence of validity for use in early childhood. This is an important contribution to advancing brief, efficient measurement of emotional distress in young children, closing a developmental gap in PROMIS pediatric emotional distress assessment.

Psychometric validation of a patient-reported experience measure of obstetric racism© (The PREM-OB Scale™ suite).

BACKGROUND: Perinatal quality improvement lacks valid tools to measure adverse hospital experiences disproportionately impacting Black mothers and birthing people. Measuring and mitigating harm requires using a framework that centers the lived experiences of Black birthing people in evaluating inequitable care, namely, obstetric racism. We sought to develop a valid patient-reported experience measure (PREM) of Obstetric Racism© in hospital-based intrapartum care designed for, by, and with Black women as patient, community, and content experts. METHODS: PROMIS© instrument development standards adapted with cultural rigor methodology. Phase 1 included item pool generation, modified Delphi method, and cognitive interviews. Phase 2 evaluated the item pool using factor analysis and item response theory. RESULTS: Items were identified or written to cover 7 previously identified theoretical domains. 806 Black mothers and birthing people completed the pilot test. Factor analysis concluded a 3 factor structure with good fit indices (CFI = 0.931-0.977, RMSEA = 0.087-0.10, R2  > .3, residual correlation < 0.15). All items in each factor fit the IRT model and were able to be calibrated. Factor 1, "Humanity," had 31 items measuring experiences of safety and accountability, autonomy, communication, and empathy. A 12-item short form was created to ease respondent burden. Factor 2, "Racism," had 12 items measuring experiences of neglect and mistreatment. Factor 3, "Kinship," had 7 items measuring hospital denial and disruption of relationships between Black mothers and their child or support system. CONCLUSIONS: The PREM-OB Scale™ suite is a valid tool to characterize and quantify obstetric racism for use in perinatal improvement initiatives.

Parent report of sleep health and attention regulation in a cross-sectional study of infants and preschool-aged children with atopic dermatitis.

BACKGROUND/OBJECTIVES: Older children with atopic dermatitis (AD) suffer from poor sleep and attention problems. However, until recently, the dearth of developmentally sensitive assessment tools impeded characterization in younger children. We aimed to characterize sleep and attention problems in young children with AD and identify modifiable factors. METHODS: A cross-sectional study of children with AD aged 1-4 years was stratified by disease severity (Patient-Oriented Eczema Measure), age, and racial/ethnic groups. Developmentally sensitive surveys assessed attention (Multidimensional Assessment Profile of Attention Regulation), sleep, and itch (Patient-Reported Outcomes Measurement Information System). Linear regression models identified predictors of sleep health and attention dysregulation. RESULTS: Parents (n = 60) of children aged 2.78 ± 0.98 years with severe (n = 25), moderate (n = 25), or mild (n = 10) AD were recruited across the United States. Significantly reduced sleep health (T-score ≥ 60) was reported in 86% of children with moderate/severe disease (n = 43), and 50% had ≥5 nights of disturbed sleep per week. A suboptimal sleep environment was identified with 32% of children with too much light, noise, or electronic device usage. With regard to attention regulation, in children with severe AD, 80% had trouble sitting still and 72% of children had trouble paying attention no matter their surroundings. In fully adjusted models, AD severity was a significant predictor of poor sleep health (B = 0.79 [0.31-1.28], p < .01) and attention dysregulation (B = 1.22 [0.51-1.93], p < .01). CONCLUSIONS: More severe AD correlates with poor sleep health and attention dysregulation. In addition to aggressive treatment of AD, clinicians should advise on modifiable sleep hygiene practices and consider screening for attention dysregulation in young children.

Enhancing the Content Validity of Self-Reported Physical Activity Self-Efficacy in Adolescents: A Qualitative Study.

PURPOSE: This study's primary purpose was to enhance the content validity of a self-reported measure of self-efficacy for physical activity (PA) in adolescents. This was addressed through assessment of younger and older adolescents' understanding of the construct of self-efficacy for PA, coupled with assessment of the content coverage and comprehensibility of items derived from existing measures. METHODS: Participants completed individual semistructured and cognitive debriefing interviews as well as 3 PA self-efficacy questionnaires. RESULTS: Thematic analysis identified personal and environmental facilitators and barriers to PA self-efficacy. The major categories were physical; psychological; interaction with surroundings; support and relationships; attitudes; and services, systems, and natural environments. Cognitive interviews resulted in the retention of 52 final items: 24 for self-efficacy with perceived facilitators and 28 for self-efficacy to overcome barriers. CONCLUSIONS: This study provides a PA self-efficacy instrument with expanded content coverage that is relevant to adolescents as young as 11 years. With further validation testing in future studies, this instrument will enable pediatric physical therapists and researchers to assess PA self-efficacy and design effective intervention strategies to improve PA.

Considerations to Support Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures in Ambulatory Clinics.

OBJECTIVE: To identify challenges to the use of Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric measures in the ambulatory pediatric setting and possible solutions to these challenges. STUDY DESIGN: Eighteen semistructured telephone interviews of health system leaders, measurement implementers, and ambulatory pediatric clinicians were conducted. Five coders used applied thematic analysis to iteratively identify and refine themes in interview data. RESULTS: Most interviewees had roles in leadership or the implementation of patient-centered outcomes; 39% were clinicians. Some had experience using PROMIS clinically (44%) and 6% were considering this use. Analyses yielded 6 themes: (1) selection of PROMIS measures, (2) method of administration, (3) use of PROMIS Parent Proxy measures, (4) privacy and confidentiality of PROMIS responses, (5) interpretation of PROMIS scores, and (6) using PROMIS scores clinically. Within the themes, interviewees illuminated specific unique considerations for using PROMIS with children, including care transitions and privacy. CONCLUSIONS: Real-world challenges continue to hamper PROMIS use. Ongoing efforts to disseminate information about the integration of PROMIS measures in clinical care is critical to impacting the health of children.

Longitudinal Change in Quality of Life in Neurological Disorders Measures Over 3 Years in Patients with Early Parkinson's Disease.

BACKGROUND: The Quality of Life in Neurological Disorders (Neuro-QoL) is a publicly available health-related quality-of-life measurement system. OBJECTIVE: The aim of this study was to evaluate the utility of Neuro-QoL item banks as outcome measures for clinical trials in Parkinson's disease. METHODS: An analysis of Neuro-QoL responsiveness to change and construct validity was performed in a multicenter clinical trial cohort. RESULTS: Among 310 participants over 3 years, changes in five of eight Neuro-QoL domains were significant (P < 0.05) but very modest. The largest effect sizes were seen in the cognition and mobility domains (0.35-0.39). The largest effect size for change over the year in which levodopa was initiated was -0.19 for lower extremity function-mobility. For a similarly designed clinical trial, estimated sample size required to demonstrate a 50% reduction in worsening ranged from 420 to more than 1000 participants per group. CONCLUSIONS: More sensitive tools will be required to serve as an outcome measure in early Parkinson's disease. © 2021 International Parkinson and Movement Disorder Society.

Minimal important change (MIC): a conceptual clarification and systematic review of MIC estimates of PROMIS measures.

We define the minimal important change (MIC) as a threshold for a minimal within-person change over time above which patients perceive themselves importantly changed. There is a lot of confusion about the concept of MIC, particularly about the concepts of minimal important change and minimal detectable change, which questions the validity of published MIC values. The aims of this study were: (1) to clarify the concept of MIC and how to use it; (2) to provide practical guidance for estimating methodologically sound MIC values; and (3) to improve the applicability of PROMIS by summarizing the available evidence on plausible PROMIS MIC values. We discuss the concept of MIC and how to use it and provide practical guidance for estimating MIC values. In addition, we performed a systematic review in PubMed on MIC values of any PROMIS measure from studies using recommended approaches. A total of 50 studies estimated the MIC of a PROMIS measure, of which 19 studies used less appropriate methods. MIC values of the remaining 31 studies ranged from 0.1 to 12.7 T-score points. We recommend to use the predictive modeling method, possibly supplemented with the vignette-based method, in future MIC studies. We consider a MIC value of 2-6 T-score points for PROMIS measures reasonable to assume at this point. For surgical interventions a higher MIC value might be appropriate. We recommend more high-quality studies estimating MIC values for PROMIS.

Rasch Analysis of Social Attitude Barriers and Facilitators to Participation for Individuals with Disabilities.

OBJECTIVES: To develop item banks of social attitude barriers and facilitators to participation and validate them with established instruments. DESIGN: We used the Rasch model to identify misfitting items and rating scale problems, calibrate items, and develop KeyForms and short forms. Correlations between the Social Attitude Barriers and Facilitators item banks with the Patient-Reported Outcomes Measurement Information System (PROMIS) Social Health domain and National Institutes of Health Toolbox Emotional Battery Social Relationships domain were computed to evaluate convergent and divergent validity. SETTING: Community-dwelling individuals traveled to 3 academic medical centers for testing. PARTICIPANTS: Participants (N=558) who had a primary impairment of stroke, spinal cord injury, or traumatic brain injury (mean age, 47.0±16.0y) completed 31 social attitude facilitator and 51 barrier items using a 5-point rating scale. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Item banks to measure social attitude barriers and facilitators for individuals with disabilities. RESULTS: After combining the "never" and "rarely" rating scale categories, 30 Facilitator items fit the Rasch model and demonstrated person reliability of 0.93. After collapsing the "never" and "rarely" rating scale categories, 45 Barrier items fit the Rasch model and demonstrated person reliability of 0.95. Ceiling and floor effects were negligible for both item banks. Facilitators and Barriers item banks were negatively correlated, and these banks were moderately correlated with PROMIS and Toolbox measures, providing evidence of convergent and divergent validity. CONCLUSIONS: Findings support the reliability and validity of the Social Attitude Facilitators and Barriers item banks. These item banks allow investigators and clinicians to measure perceptions of social attitudes, providing information that can guide individual interventions to reduce barriers and promote facilitators. Moderate correlations between the Social Attitude banks and PROMIS and Toolbox variables provide support for the measurement and theory of environmental influences on social health and participation.

Symptom burden trajectories experienced by patients with brain tumors.

BACKGROUND: Survivors of childhood brain tumors experience persistent health concerns across their lifespan. In the current study, the authors evaluated changes in symptom burden over the course of 12 months using pediatric Patient-Reported Outcomes Measurement Information System (PROMIS) measures. METHODS: Data from 202 survivors aged 8 to 21 years and 262 parents of survivors who were aged 5 to 21 years were analyzed. All completed a PROMIS Cognition short form and computerized adaptive tests of pediatric Anxiety, Depressive Symptoms, Fatigue, Mobility, Upper Extremity Function, and Peer Relationships. Approximately one-half of participants (223 participants: 97 survivors of childhood brain tumors and 126 parents) completed the 12-month follow-up. Linear mixed-effects models evaluated group-level symptoms over time. Cox proportional hazard models explored whether symptoms predicted survival, and latent class growth analysis investigated patterns of individual-level symptom changes over time. RESULTS: Linear mixed-effects models demonstrated that patient-reported Cognition and parent-reported Anxiety worsened over time. Latent class growth analysis results indicated that patient and parent reports diverged, both with regard to the number of classes identified and in the trends of these classes. Parents and patients reported similar patterns of depression over time. For the other areas, parents either were more likely to observe different patterns (Peer Relationships and Mobility) or less likely to observe different patterns (Upper Extremity Function, Cognition, Anxiety, and Fatigue). Baseline patient-reported Mobility and Upper Extremity Function were found to be associated with survival. CONCLUSIONS: Survivors of childhood brain tumors demonstrated different trajectory patterns of symptom burden. Along with baseline functioning status and days since treatment, patient-reported Mobility and Upper Extremity Function were associated with survival, suggesting a possible role for patient-reported outcomes in clinical care, especially individualized, tailored assessments such as PROMIS.