Apalutamide efficacy, safety and wellbeing in older patients with advanced prostate cancer from Phase 3 randomised clinical studies TITAN and SPARTAN.

BACKGROUND: Apalutamide plus androgen-deprivation therapy (ADT) improved outcomes in metastatic castration-sensitive prostate cancer (mCSPC) and non-metastatic castration-resistant PC (nmCRPC) in the Phase 3 randomised TITAN and SPARTAN studies, respectively, and maintained health-related quality of life (HRQoL). Apalutamide treatment effect by patient age requires assessment. METHODS: Post-hoc analysis assessed patients receiving 240 mg/day apalutamide (525 TITAN and 806 SPARTAN) or placebo (527 TITAN and 401 SPARTAN) with ongoing ADT, stratified by age groups. Prostate-specific antigen declines, radiographic progression-free survival, metastasis-free survival, overall survival (OS), HRQoL and safety were assessed using descriptive statistics, Kaplan-Meier method, Cox proportional-hazards model and mixed-effects model for repeated measures. RESULTS: Hazard ratios (95% confidence intervals) generally favoured apalutamide plus ADT versus ADT alone across all endpoints regardless of age; e.g., OS values were 0.57 (0.40-0.80), 0.70 (0.54-0.91) and 0.74 (0.40-1.39) (TITAN) and 0.39 (0.19-0.78), 0.89 (0.69-1.16) and 0.81 (0.58-1.15) (SPARTAN) in patients aged <65, 65-79 and ≥80 years. Regardless of age, apalutamide also maintained HRQoL and was tolerated well with a potential trend in rates of adverse events increasing with age. Limitations include post-hoc nature and variability in sample size of age groups. CONCLUSIONS: Apalutamide plus ADT was an effective and well-tolerated option maintaining HRQoL in patients with mCSPC and nmCRPC regardless of age. CLINICAL TRIAL REGISTRATION: TITAN (NCT02489318); SPARTAN (NCT01946204).

Prostate-specific antigen and health-related quality of life in individuals with advanced prostate cancer treated with apalutamide: a plain language summary of the SPARTAN and TITAN studies.

WHAT IS THIS SUMMARY ABOUT?: This is a summary of a paper that describes the results of the SPARTAN and TITAN studies, which looked at whether a treatment called apalutamide can help treat individuals with advanced prostate cancer.The SPARTAN study included 1207 participants with nonmetastatic castration-resistant prostate cancer (or nmCRPC). The TITAN study included 1052 participants with metastatic castration-sensitive prostate cancer (or mCSPC). Treatment with apalutamide was compared with treatment with placebo. In both studies, all participants were also given androgen deprivation therapy (or ADT), which has been used for many years for the treatment of prostate cancer.The results showed that treatment with apalutamide plus ADT increased participants' survival time while their health-related quality of life stayed the same, compared with placebo plus ADT. Also, apalutamide plus ADT increased the length of time that the cancer did not spread to other parts of the body (metastasize) or did not continue to grow. In both studies, treatment with apalutamide plus ADT was associated with a deep decline in blood prostate-specific antigen (or PSA) levels (called a deep PSA decline). This additional analysis of the SPARTAN and TITAN studies was performed to understand whether the deep PSA decline in participants who received apalutamide plus ADT was linked to their overall health-related quality of life. WHAT WERE THE RESULTS OF THE ADDITIONAL ANALYSIS?: In participants who received apalutamide plus ADT, those who achieved a deep PSA decline after the start of treatment had a greater chance that their health-related quality of life would remain stable. When participants achieved a deep PSA decline at 3 months after the start of treatment, the benefit to their health-related quality of life, including physical wellbeing, was even greater. WHAT DO THESE RESULTS MEAN FOR INDIVIDUALS WITH ADVANCED PROSTATE CANCER?: For individuals with advanced prostate cancer, it is important to monitor both PSA decline and any impacts on health-related quality of life. These results will help doctors and other healthcare professionals have a better understanding of patients' cancer experience and the impact of their treatment.Clinical Trial Registration: NCT01946204 (SPARTAN) and, NCT02489318 (TITAN) (ClinicalTrials.gov).

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.

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.

Apalutamide plus abiraterone acetate and prednisone versus placebo plus abiraterone and prednisone in metastatic, castration-resistant prostate cancer (ACIS): a randomised, placebo-controlled, double-blind, multinational, phase 3 study.

BACKGROUND: The majority of patients with metastatic castration-resistant prostate cancer (mCRPC) will have disease progression of a uniformly fatal disease. mCRPC is driven by both activated androgen receptors and elevated intratumoural androgens; however, the current standard of care is therapy that targets a single androgen signalling mechanism. We aimed to investigate the combination treatment using apalutamide plus abiraterone acetate, each of which suppresses the androgen signalling axis in a different way, versus standard care in mCRPC. METHODS: ACIS was a randomised, placebo-controlled, double-blind, phase 3 study done at 167 hospitals in 17 countries in the USA, Canada, Mexico, Europe, the Asia-Pacific region, Africa, and South America. We included chemotherapy-naive men (aged ≥18 years) with mCRPC who had not been previously treated with androgen biosynthesis signalling inhibitors and were receiving ongoing androgen deprivation therapy, with an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1, and a Brief Pain Inventory-Short Form question 3 (ie, worst pain in the past 24 h) score of 3 or lower. Patients were randomly assigned (1:1) via a centralised interactive web response system with a permuted block randomisation scheme (block size 4) to oral apalutamide 240 mg once daily plus oral abiraterone acetate 1000 mg once daily and oral prednisone 5 mg twice daily (apalutamide plus abiraterone-prednisone group) or placebo plus abiraterone acetate and prednisone (abiraterone-prednisone group), in 28-day treatment cycles. Randomisation was stratified by presence or absence of visceral metastases, ECOG performance status, and geographical region. Patients, the investigators, study team, and the sponsor were masked to group assignments. An independent data-monitoring committee continually monitored data to ensure ongoing patient safety, and reviewed efficacy data. The primary endpoint was radiographic progression-free survival assessed in the intention-to-treat population. Safety was reported for all patients who received at least one dose of study drug. This study is completed and no longer recruiting and is registered with ClinicalTrials.gov, number NCT02257736. FINDINGS: 982 men were enrolled and randomly assigned from Dec 10, 2014 to Aug 30, 2016 (492 to apalutamide plus abiraterone-prednisone; 490 to abiraterone-prednisone). At the primary analysis (median follow-up 25·7 months [IQR 23·0-28·9]), median radiographic progression-free survival was 22·6 months (95% CI 19·4-27·4) in the apalutamide plus abiraterone-prednisone group versus 16·6 months (13·9-19·3) in the abiraterone-prednisone group (hazard ratio [HR] 0·69, 95% CI 0·58-0·83; p<0·0001). At the updated analysis (final analysis for overall survival; median follow-up 54·8 months [IQR 51·5-58·4]), median radiographic progression-free survival was 24·0 months (95% CI 19·7-27·5) versus 16·6 months (13·9-19·3; HR 0·70, 95% CI 0·60-0·83; p<0·0001). The most common grade 3-4 treatment-emergent adverse event was hypertension (82 [17%] of 490 patients receiving apalutamide plus abiraterone-prednisone and 49 [10%] of 489 receiving abiraterone-prednisone). Serious treatment-emergent adverse events occurred in 195 (40%) patients receiving apalutamide plus abiraterone-prednisone and 181 (37%) patients receiving abiraterone-prednisone. Drug-related treatment-emergent adverse events with fatal outcomes occurred in three (1%) patients in the apalutamide plus abiraterone-prednisone group (2 pulmonary embolism, 1 cardiac failure) and five (1%) patients in the abiraterone-prednisone group (1 cardiac failure and 1 cardiac arrest, 1 mesenteric arterial occlusion, 1 seizure, and 1 sudden death). INTERPRETATION: Despite the use of an active and established therapy as the comparator, apalutamide plus abiraterone-prednisone improved radiographic progression-free survival. Additional studies to identify subgroups of patients who might benefit the most from combination therapy are needed to further refine the treatment of mCRPC. FUNDING: Janssen Research & Development.

Using nationally representative percentiles to interpret PROMIS pediatric measures.

PURPOSE: This study's aim was to use a representative sample of the US pediatric population to estimate percentiles for several PROMIS pediatric measures: Anger, Anxiety, Depressive Symptoms, Family Relationships, Fatigue, Global Health, Life Satisfaction, Meaning and Purpose, Pain Behavior, Pain Interference, Physical Activity, Physical Function Mobility, Physical Function Upper Extremity, Physical Stress Experiences, Positive Affect, Psychological Stress Experiences, Sleep Disturbance, Sleep Impairment, and Peer Relationships. METHODS: We used two separate, nationally representative samples of parents and children aged 5-17 years drawn in different years from the GfK Knowledge Panel, a dual-frame online probability panel. RESULTS: All measures that were developed using a representative sample had a median at or near the expected value of 50. For the other measures, the 50th percentile was often 10 points or more from 50. Several domains had high floors or low ceilings. No domain's percentiles completely corresponded to the percentiles associated with a normal distribution with a mean of 50 and standard deviation of 10. CONCLUSIONS: This work allows users to interpret a child's self-reported quality of life relative to children in the US general population. When attempting to evaluate whether a child falls above or below other children in the US, one should use the values presented in this study. In addition, we recommend that users should focus on whether a child's score falls into one of a few broad severity groups rather than on specific percentile scores.

Development and Validation of the Pediatric Sleep Practices Questionnaire: A Self-Report Measure for Youth Ages 8-17 Years.

Objective: To develop and evaluate the validity of a self-report measure of sleep practices for youth 8-17 years. Methods: Following recommended guidelines for the development of patient reported outcomes (PROs), sleep practice concepts were identified through expert (n = 8) and child (n = 28) concept elicitation interviews and a systematic literature review. Items were developed based on these concepts and tested in cognitive interviews with children (n = 32). Psychometric analyses were applied to item response data collected from a diverse sample of youth 8-17 years (n = 307). Construct validity was evaluated through tests of associations between sleep practices and sleep disturbance and sleep-related impairment. Finally, clinical validity of the tool was assessed by comparing scores of youth with and without a parent-identified sleep problem. Results: The final Pediatric Sleep Practices Questionnaire (PSPQ) included 15 items that were used to identify 5 sleep practices: sleep timing, sleep routines and consistency, technology use before bedtime, sleep environment, and the need for parental presence to fall asleep. A confirmatory factor analysis supported the hypothesized structure (all factor loadings ≥ 0.72) and PSPQ indices were significantly associated with self-reported sleep disturbances and sleep-related impairment. Finally, children with parent-reported sleep problems had shorter sleep opportunity, later bedtimes, greater need for parental presence, poorer bedtime routines, and more technology use than children without parent-reported sleep problems. Conclusions: The PSPQ was developed using best-practice PRO development methodology. The PSPQ can be used in clinical settings and for research assessment to capture modifiable sleep practices that may promote or interfere with healthy sleep.

Clinical Validity of the PROMIS Pediatric Sleep Measures across Populations of Children with Chronic Illnesses and Neurodevelopment Disorders.

OBJECTIVE: To examine the clinical validity of the Patient Reported Outcome Measurement Information System (PROMIS) Pediatric Sleep Disturbance (SD) and Sleep-Related Impairment (SRI) short forms. METHODS: Youth (8-17 years) from clinical populations with known SDs (sleep clinic n = 126, autism n = 276, asthma n = 82, asthma + eczema n = 68) and the general population (n = 902) completed the PROMIS Pediatric SD and SRI 8-item short forms, along with established measures of sleep (Children's Report of Sleep Patterns, Sleep Habits Survey), PROMIS Pediatric Fatigue, and parent-reported clinical indicators (does child have sleep problem, use melatonin, use prescription sleep medication). RESULTS: Confirmatory factor analyses demonstrated factorial invariance for all clinical groups. Significant differences between the general population and clinical groups were found for SD and SRI (medium to large effect sizes). Convergent validity was demonstrated through separate hierarchical regression models that showed significant associations between parent-reported clinical indicators and SD and SRI, above and beyond clinical group, as well as moderate to strong correlations between the PROMIS sleep measures and both established measures of sleep and fatigue. CONCLUSIONS: The PROMIS Pediatric SD and SRI short forms provide clinicians and researchers a brief, accurate, and valid way to measure patient-reported sleep outcomes in pediatric populations.

Investigating child self-report capacity: a systematic review and utility analysis.

PURPOSE: To identify and evaluate methods for assessing pediatric patient-reported outcome (PRO) data quality at the individual level. METHODS: We conducted a systematic literature review to identify methods for detecting invalid responses to PRO measures. Eight data quality indicators were applied to child-report data collected from 1780 children ages 8-11 years. We grouped children with similar data quality patterns and tested for between-group differences in factors hypothesized to influence self-report capacity. RESULTS: We identified 126 articles that described 494 instances in which special measures or statistical techniques were applied to evaluate data quality at the individual level. We identified 22 data quality indicator subtypes: 9 direct methods (require administration of special items) and 13 archival techniques (statistical procedures applied to PRO data post hoc). Application of archival techniques to child-report PRO data revealed 3 distinct patterns (or classes) of the data quality indicators. Compared to class 1 (56%), classes 2 (36%) and 3 (8%) had greater variation in their PRO item responses. Three archival indicators were especially useful for differentiating plausible item response variation (class 2) from statistically unlikely response patterns (class 3). Neurodevelopmental conditions, which are associated with a range of cognitive processing challenges, were more common among children in class 3. CONCLUSION: A multi-indicator approach is needed to identify invalid PRO responses. Once identified, assessment environments and measurement tools should be adapted to best support these individuals' self-report capacity. Individual-level data quality indicators can be used to gauge the effectiveness of these accommodations.

Psychometric Evaluation of the Participation and Sensory Environment Questionnaire-Home Scale (PSEQ-H).

IMPORTANCE: The impact of sensory processing challenges on occupational participation is underrepresented by existing measurement tools even though these outcomes are highly prioritized by families. The Participation and Sensory Environment Questionnaire-Home Scale (PSEQ-H) is a parent-report assessment designed to evaluate the impact of the sensory environment on young children's participation in home-based activities. OBJECTIVE: To describe the psychometric evaluation of the PSEQ-H, including the tool's structural validity; item difficulty, discrimination, and bias; reliability; and construct validity. DESIGN: Psychometric field study. SETTING: Community. PARTICIPANTS: Three hundred four parents of children ages 2-7 yr (167 parents of children with autism spectrum disorder). METHOD: Parent-report PSEQ-H data were factor analyzed, calibrated using Item Response Theory, and evaluated for construct validity. RESULTS: The final PSEQ-H is a reliable and valid 15-item parent-report measure of the sensory environment's impact on children's dressing, self-care, sleep, and social and play activities. CONCLUSIONS AND RELEVANCE: The PSEQ-H can be used to plan and evaluate the effectiveness of interventions for reducing the impact of the sensory environment on children's participation in home-based tasks and activities. WHAT THIS ARTICLE ADDS: The PSEQ-H measures how young children's sensory environments influence their participation at home. The measure can be used to plan and evaluate occupational therapy interventions that aim to reduce sensory processing-related barriers to children's completion of developmentally salient activities.

Assessing Children's Eudaimonic Well-Being: The PROMIS Pediatric Meaning and Purpose Item Banks.

OBJECTIVE: To describe the development of the Patient-Reported Outcome Measurement Information System (PROMIS) Pediatric Meaning and Purpose item banks, child-report and parent-proxy editions. METHODS: Data were collected from two samples. The first comprised 1,895 children (8-17 years old) and 927 parents of children 5-17 years old recruited from an Internet panel, medical clinics, and schools. The second comprised a nationally representative sample of 990 children 8-17 years old and 1,292 parents of children 5-17 years old recruited from a different Internet panel. Item pool evaluation was done with Sample 1 and analyses were used to support decisions about item retention. The combined sample was used for item response theory (IRT) calibration of the item bank. Both samples were used in validation studies. RESULTS: Eleven items were deleted from the item pool because of poor psychometric performance. The final versions of the scales showed excellent reliability (>0.90). Short form scales (4 or 8 items) had a high degree of precision across over 4 SD units of the latent variable. The item bank positively correlated with extant measures of positive psychological functioning, and negatively correlated with measures of emotional distress, pessimism, and pain. Lower meaning and purpose scores were associated with adolescence and presence of a special healthcare need. CONCLUSION: The PROMIS Pediatric Meaning and Purpose item banks and their short forms are ready for use in clinical research and practice. They are measures of children's eudaimonic well-being and indicative of children's hopefulness, optimism, goal-directedness, and feelings that life is worth living.

Qualitative Development and Content Validation of the PROMIS Pediatric Sleep Health Items.

Objective: To develop the Patient Reported Outcome Measurement Information System (PROMIS) Pediatric Sleep Health item pool and evaluate its content validity. Participants: Participants included 8 expert sleep clinician-researchers, 64 children ages 8-17 years, and 54 parents of children ages 5-17 years. Methods: We started with item concepts and expressions from the PROMIS Sleep Disturbance and Sleep Related Impairment adult measures. Additional pediatric sleep health concepts were generated by expert (n = 8), child (n = 28), and parent (n = 33) concept elicitation interviews and a systematic review of existing pediatric sleep health questionnaires. Content validity of the item pool was evaluated with item translatability review, readability analysis, and child (n = 36) and parent (n = 21) cognitive interviews. Results: The final pediatric Sleep Health item pool includes 43 items that assess sleep disturbance (children's capacity to fall and stay asleep, sleep quality, dreams, and parasomnias) and sleep-related impairments (daytime sleepiness, low energy, difficulty waking up, and the impact of sleep and sleepiness on cognition, affect, behavior, and daily activities). Items are translatable and relevant and well understood by children ages 8-17 and parents of children ages 5-17. Conclusions: Rigorous qualitative procedures were used to develop and evaluate the content validity of the PROMIS Pediatric Sleep Health item pool. Once the item pool's psychometric properties are established, the scales will be useful for measuring children's subjective experiences of sleep.

Psychometric Evaluation of the PROMIS® Pediatric Psychological and Physical Stress Experiences Measures.

Objective: To provide psychometric evaluation of the PROMIS® Pediatric Psychological and Physical Stress Experiences measures. Methods: Across two studies, Psychological and Physical Stress Experiences items were administered to 2,875 children aged 8-17 years and 2,212 parents of children aged 5-17 years. Analyses included descriptive statistics, reliability, factor analysis, differential item functioning (DIF), and assessment of construct validity. Items were calibrated using item response theory to estimate item parameters representative of the United States. Recommended eight- and four-item short forms were constructed for child- and parent-report versions of the Psychological and Physical Stress Experiences item banks. Results: Final item banks were unidimensional and items were locally independent and free from impactful DIF. Psychological Stress banks include 19 child-report and 12 parent-proxy items. Physical Stress banks include 26 child-report and 26 parent-proxy items. All instruments have strong internal consistency and retest-reliability, and provide precise estimates of varying stress levels. The instruments' construct validity was evidenced by known-group comparisons and convergence with legacy measures. Conclusions: The Patient Reported Outcome Measurement Information System (PROMIS) Pediatric Psychological and Physical Stress item banks and short forms provide efficient, precise, and valid assessments of children's stress experiences.

Development and psychometric evaluation of the PROMIS Pediatric Life Satisfaction item banks, child-report, and parent-proxy editions.

PURPOSE: To describe the psychometric evaluation and item response theory calibration of the PROMIS Pediatric Life Satisfaction item banks, child-report, and parent-proxy editions. METHODS: A pool of 55 life satisfaction items was administered to 1992 children 8-17 years old and 964 parents of children 5-17 years old. Analyses included descriptive statistics, reliability, factor analysis, differential item functioning, and assessment of construct validity. Thirteen items were deleted because of poor psychometric performance. An 8-item short form was administered to a national sample of 996 children 8-17 years old, and 1294 parents of children 5-17 years old. The combined sample (2988 children and 2258 parents) was used in item response theory (IRT) calibration analyses. RESULTS: The final item banks were unidimensional, the items were locally independent, and the items were free from impactful differential item functioning. The 8-item and 4-item short form scales showed excellent reliability, convergent validity, and discriminant validity. Life satisfaction decreased with declining socio-economic status, presence of a special health care need, and increasing age for girls, but not boys. After IRT calibration, we found that 4- and 8-item short forms had a high degree of precision (reliability) across a wide range (>4 SD units) of the latent variable. CONCLUSIONS: The PROMIS Pediatric Life Satisfaction item banks and their short forms provide efficient, precise, and valid assessments of life satisfaction in children and youth.

Development and Evaluation of the PROMIS® Pediatric Positive Affect Item Bank, Child-Report and Parent-Proxy Editions.

OBJECTIVE: The purpose of this study is to describe the psychometric evaluation and item response theory calibration of the PROMIS Pediatric Positive Affect item bank, child-report and parent-proxy editions. METHODS: The initial item pool comprising 53 items, previously developed using qualitative methods, was administered to 1,874 children 8-17 years old and 909 parents of children 5-17 years old. Analyses included descriptive statistics, reliability, factor analysis, differential item functioning, and construct validity. A total of 14 items were deleted, because of poor psychometric performance, and an 8-item short form constructed from the remaining 39 items was administered to a national sample of 1,004 children 8-17 years old, and 1,306 parents of children 5-17 years old. The combined sample was used in item response theory (IRT) calibration analyses. RESULTS: The final item bank appeared unidimensional, the items appeared locally independent, and the items were free from differential item functioning. The scales showed excellent reliability and convergent and discriminant validity. Positive affect decreased with children's age and was lower for those with a special health care need. After IRT calibration, we found that 4 and 8 item short forms had a high degree of precision (reliability) across a wide range of the latent trait (>4 SD units). CONCLUSION: The PROMIS Pediatric Positive Affect item bank and its short forms provide an efficient, precise, and valid assessment of positive affect in children and youth.

Development and Content Validation of the Transition Readiness Inventory Item Pool for Adolescent and Young Adult Survivors of Childhood Cancer.

Objective: The development of the Transition Readiness Inventory (TRI) item pool for adolescent and young adult childhood cancer survivors is described, aiming to both advance transition research and provide an example of the application of NIH Patient Reported Outcomes Information System methods. Methods: Using rigorous measurement development methods including mixed methods, patient and parent versions of the TRI item pool were created based on the Social-ecological Model of Adolescent and young adult Readiness for Transition (SMART). Results: Each stage informed development and refinement of the item pool. Content validity ratings and cognitive interviews resulted in 81 content valid items for the patient version and 85 items for the parent version. Conclusions: TRI represents the first multi-informant, rigorously developed transition readiness item pool that comprehensively measures the social-ecological components of transition readiness. Discussion includes clinical implications, the application of TRI and the methods to develop the item pool to other populations, and next steps for further validation and refinement.

Children's family experiences: development of the PROMIS® pediatric family relationships measures.

PURPOSE: To describe the development of pediatric family relationships measures, with versions for child self-report (8-17 years) and parent-report for children 5-17 years old. Measures were created for integration into the Patient Reported Outcomes Measurement Information System (PROMIS®). METHODS: Semi-structured interviews with 10 experts, 24 children, and 8 parents were conducted to elicit and clarify essential elements of family relationships. A systematic literature review was conducted to identify item concepts representative of each element. The concepts were transformed into items that were iteratively revised based on cognitive interviews (n = 43 children) and item translatability review. Psychometric studies involving 2846 children and 2262 parents were conducted to further refine and validate the instruments. RESULTS: Qualitative procedures supported the development of content valid Family Relationships item banks. Final child- and parent-report item banks each contain 47 items. Unidimensional item banks were calibrated using IRT-modeling to estimate item parameters representative of the US population and to enable computerized adaptive test administration. Four- and eight-item short forms were constructed for standard fixed format administration. All instruments have strong internal consistency, retest-reliability, and provide precise estimates of various levels of family relationship quality. Preliminary evidence of the instruments' validity was provided by known-group comparisons and convergence with legacy measures. CONCLUSION: The PROMIS pediatric Family Relationships measures can be applied in research focused on determinants, outcomes, and the protective effects of children's subjective family relationship experiences.

Concurrent validity of the PROMIS® pediatric global health measure.

PURPOSE: To evaluate the concurrent validity of the PROMIS Pediatric Global Health measure (PGH-7), child-report and parent-proxy versions. METHODS: Surveys were administered via home computer on two separate occasions (December, 2011 and August/September, 2012) to a convenience sample of 4636 children 8-17 years old and 2609 parents who participated in a national Internet panel. Data analysis included: (1) evaluations of differences in PGH-7 scores between groups defined by sociodemographics, clinical characteristics, and access to health care; (2) associations with 15 PROMIS pediatric measures; and (3) correlations with two health-related quality-of-life instruments, the KIDSCREEN-10 and PedsQL-15. RESULTS: PGH-7 scores were lower for children with chronic conditions, Hispanic ethnicity, low socioeconomic status, and barriers to accessing health care. The PGH-7 showed excellent convergent and discriminant validity with PROMIS pediatric measures of physical, mental, and social health. The PGH-7 was strongly correlated with the KIDSCREEN-10, which assesses positive health, and moderately correlated with the PedsQL-15, which assesses problems with a child's health. CONCLUSIONS: The PGH-7 measures global health, summarizing a child's physical, mental, and social health into a single score. These properties make it a useful clinical, population health, and research tool for applications that require an efficient, precise, and valid summary measure of a children's self-reported health status. Future research should prospectively evaluate the PGH-7's capacity to detect change that results from alterations in clinical status, transformations of the healthcare delivery system, and children's health development.

Development, validation, and utility of an instrument to assess core competencies in the Leadership Education in Neurodevelopmental and Related Disabilities (LEND) program.

To describe the development and psychometric evaluation of the Core Competency Measure (CCM), an instrument designed to assess professional competencies as defined by the Maternal Child Health Bureau (MCHB) and targeted by Leadership Education in Neurodevelopmental and Related Disabilities (LEND) programs. The CCM is a 44-item self-report measure comprised of six subscales to assess clinical, interdisciplinary, family-centered/cultural, community, research, and advocacy/policy competencies. The CCM was developed in an iterative fashion through participatory action research, and then nine cohorts of LEND trainees (N = 144) from 14 different disciplines completed the CCM during the first week of the training program. A 6-factor confirmatory factor analysis model was fit to data from the 44 original items. After three items were removed, the model adequately fit the data (comparative fit indices = .93, root mean error of approximation = .06) with all factor loadings exceeding .55. The measure was determined to be quite reliable as adequate internal consistency and test-retest reliability were found for each subscale. The instrument's construct validity was supported by expected differences in self-rated competencies among fellows representing various disciplines, and the convergent validity was supported by the pattern of inter-correlations between subscale scores. The CCM appears to be a reliable and valid measure of MCHB core competencies for our sample of LEND trainees. It provides an assessment of key training areas addressed by the LEND program. Although the measure was developed within only one LEND Program, with additional research it has the potential to serve as a standardized tool to evaluate the strengths and limitations of MCHB training, both within and between programs.