The Preserving Kidney Function in Children With CKD (PRESERVE) Study: Rationale, Design, and Methods.

Rationale & Objective: PRESERVE seeks to provide new knowledge to inform shared decision-making regarding blood pressure (BP) management for pediatric chronic kidney disease (CKD). PRESERVE will compare the effectiveness of alternative strategies for monitoring and treating hypertension on preserving kidney function; expand the National Patient-Centered Clinical Research Network (PCORnet) common data model by adding pediatric- and kidney-specific variables and linking electronic health record data to other kidney disease databases; and assess the lived experiences of patients related to BP management. Study Design: Multicenter retrospective cohort study (clinical outcomes) and cross-sectional study (patient-reported outcomes [PROs]). Setting & Participants: PRESERVE will include approximately 20,000 children between January 2009-December 2022 with mild-moderate CKD from 15 health care institutions that participate in 6 PCORnet Clinical Research Networks (PEDSnet, STAR, GPC, PaTH, CAPRiCORN, and OneFlorida+). The inclusion criteria were ≥1 nephrologist visit and ≥2 estimated glomerular filtration rate (eGFR) values in the range of 30 to <90 mL/min/1.73 m2 separated by ≥90 days without an intervening value ≥90 mL/min/1.73 m2 and no prior dialysis or kidney transplant. Exposures: BP measurements (clinic-based and 24-hour ambulatory BP); urine protein; and antihypertensive treatment by therapeutic class. Outcomes: The primary outcome is a composite event of a 50% reduction in eGFR, eGFR of <15 mL/min/1.73 m2, long-term dialysis or kidney transplant. Secondary outcomes include change in eGFR, adverse events, and PROs. Analytical Approach: Longitudinal models for dichotomous (proportional hazards or accelerated failure time) and continuous (generalized linear mixed models) clinical outcomes; multivariable linear regression for PROs. We will evaluate heterogeneity of treatment effect by CKD etiology and degree of proteinuria and will examine variation in hypertension management and outcomes based on socio-demographics. Limitations: Causal inference limited by observational analyses. Conclusions: PRESERVE will leverage the PCORnet infrastructure to conduct large-scale observational studies that address BP management knowledge gaps for pediatric CKD, focusing on outcomes that are meaningful to patients. Plain-Language Summary: Hypertension is a major modifiable contributor to loss of kidney function in chronic kidney disease (CKD). The purpose of PRESERVE is to provide evidence to inform shared decision-making regarding blood pressure management for children with CKD. PRESERVE is a consortium of 16 health care institutions in PCORnet, the National Patient-Centered Clinical Research Network, and includes electronic health record data for >19,000 children with CKD. PRESERVE will (1) expand the PCORnet infrastructure for research in pediatric CKD by adding kidney-specific variables and linking electronic health record data to other kidney disease databases; (2) compare the effectiveness of alternative strategies for monitoring and treating hypertension on preserving kidney function; and (3) assess the lived experiences of patients and caregivers related to blood pressure management.

Leveraging Serologic Testing to Identify Children at Risk For Post-Acute Sequelae of SARS-CoV-2 Infection: An Electronic Health Record-Based Cohort Study from the RECOVER Program.

Using an electronic health record-based algorithm, we identified children with Coronavirus disease 2019 (COVID-19) based exclusively on serologic testing between March 2020 and April 2022. Compared with the 131 537 polymerase chain reaction-positive children, the 2714 serology-positive children were more likely to be inpatients (24% vs 2%), to have a chronic condition (37% vs 24%), and to have a diagnosis of multisystem inflammatory syndrome in children (23% vs <1%). Identification of children who could have been asymptomatic or paucisymptomatic and not tested is critical to define the burden of post-acute sequelae of severe acute respiratory syndrome coronavirus 2 infection in children.

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.

Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.

OBJECTIVES: To evaluate whether Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome (PRO) measures can serve as valid endpoints in a clinical trial of a chronic pediatric illness. STUDY DESIGN: We evaluated the responsiveness of PROMIS pediatric measures collected through the Clinical Outcomes of Methotrexate Binary Therapy in Practice (COMBINE) trial, a multicenter, randomized, double-blind, placebo-controlled, pragmatic clinical trial in pediatric patients with Crohn's disease (CD). We examined the relationships between changes in PROMIS pediatric measures and changes in disease activity by evaluating PRO score changes among patients who did and patients who did not experience improvement in disease activity. RESULTS: Participants included 266 children and adolescents with CD from a total of 35 institutions. Over the course of follow-up, participants showed improvement in most PRO domains, with the largest effect sizes observed for the clinically improved group. Patients who maintained steroid-free remission showed significantly lower PRO scores for the Pain Interference, Fatigue, and inflammatory bowel disease (IBD) Symptoms domains and higher scores for the Positive Affect domain. CONCLUSIONS: This study demonstrates the responsiveness of the PROMIS pediatric measures of Fatigue and Pain Interference as study endpoints in a large, multicenter pragmatic trial in pediatric CD, extending a growing body of research supporting the use of PROMIS pediatric measures as reliable PRO endpoints for clinical trials.

A Longitudinal Comparison of Alternatives to Body Mass Index Z-Scores for Children with Very High Body Mass Indexes.

OBJECTIVE: The current Centers for Disease Control and Prevention (CDC) body mass index (BMI) z-scores are inaccurate for BMIs of ≥97th percentile. We, therefore, considered 5 alternatives that can be used across the entire BMI distribution: modified BMI-for-age z-score (BMIz), BMI expressed as a percentage of the 95th percentile (%CDC95th percentile), extended BMIz, BMI expressed as a percentage of the median (%median), and %median adjusted for the dispersion of BMIs. STUDY DESIGN: We illustrate the behavior of the metrics among children of different ages and BMIs. We then compared the longitudinal tracking of the BMI metrics in electronic health record data from 1.17 million children in PEDSnet using the intraclass correlation coefficient to determine if 1 metric was superior. RESULTS: Our examples show that using CDC BMIz for high BMIs can result in nonsensical results. All alternative metrics showed higher tracking than CDC BMIz among children with obesity. Of the alternatives, modified BMIz performed poorly among children with severe obesity, and %median performed poorly among children who did not have obesity at their first visit. The highest intraclass correlation coefficients were generally seen for extended BMIz, adjusted %median, and %CDC95th percentile. CONCLUSIONS: Based on the examples of differences in the BMI metrics, the longitudinal tracking results and current familiarity BMI z-scores and percentiles. Both extended BMIz and extended BMI percentiles may be suitable replacements for the current z-scores and percentiles. These metrics are identical to those in the CDC growth charts for BMIs of <95th percentile and are superior for very high BMIs. Researchers' familiarity with the current CDC z-scores and clinicians with the CDC percentiles may ease the transition to the extended BMI scale.

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.

Psychometric Findings for the SCAR-Q Patient-Reported Outcome Measure Based on 731 Children and Adults with Surgical, Traumatic, and Burn Scars from Four Countries.

BACKGROUND: Each year, millions of individuals develop scars secondary to surgery, trauma, and/or burns. Scar-specific patient-reported outcome measures to evaluate outcomes are needed. To address the gap in available measures, the SCAR-Q was developed following international guidelines for patient-reported outcome measure development. This study field tested the SCAR-Q and examined its psychometric properties. METHODS: Patients aged 8 years and older with a surgical, traumatic, and/or burn scar anywhere on their face or body were recruited between March of 2017 and April of 2018 at seven hospitals in four countries. Participants answered demographic and scar questions, the Fitzpatrick Skin Typing Questionnaire, the Patient and Observer Scar Assessment Scale (POSAS), and the SCAR-Q. Rasch measurement theory was used for the psychometric analysis. Cronbach's alpha, test-retest reliability, and concurrent validity were also examined. RESULTS: Consent was obtained from 773 patients, and 731 completed the study. Participants were aged 8 to 88 years, and 354 had surgical, 184 had burn, and 199 had traumatic scars. Analysis led to refinement of the SCAR-Q Appearance, Symptoms, and Psychosocial Impact scales. Reliability was high, with person separation index values of 0.91, 0.81, and 0.79; Cronbach alpha values of 0.96, 0.91, and 0.95; and intraclass correlation coefficient values of 0.92, 0.94, and 0.88, respectively. As predicted, correlations between POSAS scores and the Appearance and Symptom scales were higher than those between POSAS and Psychosocial Impact scale scores. CONCLUSIONS: With increasing scar revisions, a scar-specific patient-reported outcome measure is needed to measure outcomes that matter to patients from their perspective. The SCAR-Q represents a rigorously developed, internationally applicable patient-reported outcome measure that can be used to evaluate scars in research, clinical care, and quality improvement initiatives.

Primary Care Accessibility Effects on Health Care Utilization Among Urban Children.

OBJECTIVE: Evidence suggests that spatial accessibility to primary care is a contributing factor to appropriate health care utilization, with limited primary care access resulting in avoidable hospitalizations and emergency department visits which are burdensome on individuals and our health care system. Limited research, however, has examined the effects on children. METHODS: We evaluated associations of spatial accessibility to primary care on health care utilization among a sample of 16,709 children aged 0 to 3 years in Philadelphia who were primarily non-White and publicly insured. Log-Poisson models with generalized estimating equations were used to estimate incidence rate ratios (RR) and 95% confidence intervals (CI), while accounting for 3 levels of clustering (within individual, within primary care practice, within neighborhood). RESULTS: In age-adjusted models, the lowest level of spatial accessibility was associated with 7% fewer primary care visits (RR 0.93, 95% CI 0.91, 0.95), 15% more emergency department visits (RR 1.15, 95% CI 1.09, 1.22), and 18% more avoidable hospitalizations (RR 1.18, 95% CI 1.01, 1.37). After adjustment for individual- (race/ethnicity, sex, number of chronic conditions, insurance status) and neighborhood-level (racial composition and proportion of housing units with no vehicle), spatial accessibility was not significantly associated with rate of health care utilization. CONCLUSIONS: Individual-level predisposing factors, such as age, race, and need, attenuate the association between accessibility to primary care and use of primary care, emergency department visits, and avoidable hospitalization. Given the possibility of modifying access to primary care unlike immutable individual factors, a focus on spatial accessibility to primary care may promote appropriate health care utilization.

Assessing Performance in Simulated Cleft Palate Repair Using a Novel Video Recording Setup.

OBJECTIVE: To test the feasibility of implementing a high-fidelity cleft palate simulator during a workshop in Santiago, Chile, using a novel video endoscope to assess technical performance. DESIGN: Sixteen cleft surgeons from South America participated in a 2-day cleft training workshop. All 16 participants performed a simulated repair, and 13 of them performed a second simulated repair. The repairs were recorded using a low-cost video camera and a newly designed camera mouth retractor attachment. Twenty-nine videos were assessed by 3 cleft surgeons using a previously developed cleft palate objective structured assessment of technical skill (CLOSATS with embedded overall score assessment) and global rating scale. The reliability of the ratings and technical performance in relation to minimum acceptable scores and previous experience was assessed. RESULTS: The video setup provided acceptable recording quality for the purpose of assessment. Average intraclass correlation coefficient for the CLOSATS, global, and overall performance score was 0.69, 0.75, and 0.82, respectively. None of the novice surgeons passed the CLOSATS and global score for both sessions. One participant in the intermediate group, and 2 participants in the advanced group passed the CLOSATS and global score for both sessions. There were highly experienced participants who failed to pass the CLOSATS and global score for both sessions. CONCLUSIONS: The cleft palate simulator can be practically implemented with video-recording capability to assess performance in cleft palate repair. This technology may be of assistance in assessing surgical competence in cleft palate repair.

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 validation of the PROMIS Pediatric Sleep Disturbance and Sleep-Related Impairment item banks.

Study Objectives: To develop and evaluate the measurement properties of child-report and parent-proxy versions of the Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Sleep Disturbance and Sleep-Related Impairment item banks. Methods: A national sample of 1104 children (8-17 years old) and 1477 parents of children 5-17 years old was recruited from an internet panel to evaluate the psychometric properties of 43 sleep health items. A convenience sample of children and parents recruited from a pediatric sleep clinic was obtained to provide evidence of the measures' validity; polysomnography data were collected from a subgroup of these children. Results: Factor analyses suggested two dimensions: sleep disturbance and daytime sleep-related impairment. The final item banks included 15 items for Sleep Disturbance and 13 for Sleep-Related Impairment. Items were calibrated using the graded response model from item-response theory. Of the 28 items, 16 are included in the parallel PROMIS adult sleep health measures. Reliability of the measures exceeded 0.90. Validity was supported by correlations with existing measures of pediatric sleep health and higher sleep disturbance and sleep-related impairment scores for children with sleep problems and those with chronic and neurodevelopmental disorders. The sleep health measures were not correlated with results from polysomnography. Conclusions: The PROMIS Pediatric Sleep Disturbance and Sleep-Related Impairment item banks provide subjective assessments of child's difficulty falling and staying asleep as well as daytime sleepiness and its impact on functioning. They may prove useful in the future for clinical research and practice. Future research should evaluate their responsiveness to clinical change in diverse patient populations.

Development of the Learning Health System Researcher Core Competencies.

OBJECTIVE: To develop core competencies for learning health system (LHS) researchers to guide the development of training programs. DATA SOURCES/STUDY SETTING: Data were obtained from literature review, expert interviews, a modified Delphi process, and consensus development meetings. STUDY DESIGN: The competencies were developed from August to December 2016 using qualitative methods. DATA COLLECTION/EXTRACTION METHODS: The literature review formed the basis for the initial draft of a competency domain framework. Key informant semi-structured interviews, a modified Delphi survey, and three expert panel (n = 19 members) consensus development meetings produced the final set of competencies. PRINCIPAL FINDINGS: The iterative development process yielded seven competency domains: (1) systems science; (2) research questions and standards of scientific evidence; (3) research methods; (4) informatics; (5) ethics of research and implementation in health systems; (6) improvement and implementation science; and (7) engagement, leadership, and research management. A total of 33 core competencies were prioritized across these seven domains. The real-world milieu of LHS research, the embeddedness of the researcher within the health system, and engagement of stakeholders are distinguishing characteristics of this emerging field. CONCLUSIONS: The LHS researcher core competencies can be used to guide the development of learning objectives, evaluation methods, and curricula for training programs.

Preliminary Validation of the PROMIS Parent-Proxy Peer Relationships Measure in Children with Autism Spectrum Disorder: A DBPNet Study.

OBJECTIVE: To evaluate the content and construct validity of the existing PROMIS Pediatric Parent-Proxy Peer Relationships Measure in 5- to 12-year-old children with autism spectrum disorder (ASD). METHOD: Parents of 121 children aged 5 to 12 years who met DSM-IV criteria for ASD completed the Peer Relationships Measure using computerized adaptive testing (CAT). Parents also completed the Social Responsiveness Scale, Second Edition (SRS-2) and a demographic form. Intelligence quotient test results were extracted from clinical or research records. Five parents participated in semi-structured interviews about their child's peer relationships and the item content on the Peer Relationships Measure. RESULTS: The children in the sample were primarily male (87%). The sample was racially and ethnically diverse, and parents were predominantly highly educated. The mean T-score (SD) on the Peer Relationships Measure was 36 (8), with a range from 15 to 62. For 98% of subjects, the CAT required administration of 5 items to reach a standard error of measurement of less than 4 T-score units. The Peer Relationships Measure demonstrated a large correlation with the SRS-2 (r = -0.60, p < .0001). In semi-structured interviews, parents reported that the items on the Peer Relationships Measure were relevant to the peer relationships of their child with ASD, but they reported a few challenges related to variability in their children's peer relationships over time and to somewhat limited knowledge of relationships in school. CONCLUSION: The PROMIS Pediatric Parent-Proxy Peer Relationships Measure may be an efficient, precise, and valid measure of peer relationships for 5- to 12-year-old children with ASD.

Concept and measurement of pediatric value.

In the new health care marketplace, families will be making important decisions concerning choice of health plan, health provider, and even accountable care organizations. Ideally, they would make these decisions using information on health care value, which comprises the relationships between patient/family-centered outcomes (the outputs of health care services) and costs of providing care to achieve these outcomes. Providing information on pediatric value will require new investments in data collection systems that include outcomes that matter to children and families and costs measured at the level of the child. The analysis of these data must account for the perspective of the user of the information. In the case of families, direct standardization should be used to contrast care in one health care system with another according to the unique characteristics of each family and child.

Individual and Instructional Determinants of Student Engagement in Physical Education.

This study was conducted to identify student characteristics and instructional factors that impact student engagement in physical education (PE). Data were derived from the systematic observation of 124 sessions taught by 31 physical educators and the administration of health and PE engagement questionnaires to 2,018 students in grades 5-8. Physical activity was directly affected by student engagement and perceived competence in PE and indirectly affected by students' body image through its association with PE engagement. Multilevel analyses revealed that the proportion of class time devoted to game play was negatively associated with student engagement in PE. Although less frequently used during PE sessions, skill practice was positively associated with student engagement and inactive instruction was negatively associated with student engagement. These effects were particularly pronounced among students with poor competence beliefs. Implications for PE instructional practice and future research are presented.