Commentary: Determining Economic Factors That Matter to People With Intellectual and Developmental Disabilities and Their Caregivers: A Process Framework.

BACKGROUND: The 2020-2029 strategic plan for the Patient-Centered Outcomes Research Trust Fund calls for addressing data infrastructure gaps that are critical for studying issues around intellectual and developmental disabilities (I/DD). Specifically, the plan calls for data collection on economic factors that affect person-centered approaches to health care decision-making. Among people with I/DD and their caregivers, such economic factors may include financial costs of care, decreased opportunities for leisure and recreation, income losses associated with caregiving, and foregone opportunities for skill acquisition or other human capital investments. OBJECTIVE: This commentary supports responsiveness to the Patient-Centered OutcomesResearch Trust Fund (PCORTF) calls by conceptualizing and operationalizing a framework for identifying preferences on economic factors that are relevant to people with I/DD and their caregivers. MAIN ARGUMENTS: The framework outlined in this commentary addresses barriers to data collection that hinder measure development in the study of I/DD. This work is significant and timely given the continued movement to integrate and maintain people with I/DD within communities and recent methodological advances for eliciting preferences among people with I/DD. RELEVANCE TO THE SPECIAL ISSUE: Readers will be introduced to a framework for building data capacity in the study of economic outcomes among a population that is a high research priority for federal funding agencies. This commentary aims to be useful to researchers in planning, developing, and initiating projects in this area.

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.

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.

Comparative effectiveness of paediatric kidney stone surgery (the PKIDS trial): study protocol for a patient-centred pragmatic clinical trial.

INTRODUCTION: The strength of the evidence base for the comparative effectiveness of three common surgical modalities for paediatric nephrolithiasis (ureteroscopy, shockwave lithotripsy and percutaneous nephrolithotomy) and its relevance to patients and caregivers are insufficient. We describe the methods and rationale for the Pediatric KIDney Stone (PKIDS) Care Improvement Network Trial with the aim to compare effectiveness of surgical modalities in paediatric nephrolithiasis based on stone clearance and lived patient experiences. This protocol serves as a patient-centred alternative to randomised controlled trials for interventions where clinical equipoise is lacking. METHODS AND ANALYSIS: The PKIDS is a collaborative learning organisation composed of 26 hospitals that is conducting a prospective pragmatic clinical trial comparing the effectiveness of ureteroscopy, shockwave lithotripsy and percutaneous nephrolithotomy for youth aged 8-21 years with kidney and/or ureteral stones. Embedded within clinical care, the PKIDS trial will collect granular patient-level, surgeon-level and institution-level data, with a goal enrolment of 1290 participants over a 21-month period. The primary study outcome is stone clearance, defined as absence of a residual calculus of >4 mm on postoperative ultrasound. Secondary outcomes include patient-reported physical, emotional and social health outcomes (primarily using the Patient-Reported Outcome Measurement Information System), analgesic use and healthcare resource use. Timing and content of secondary outcomes assessments were set based on feedback from patient partners. Heterogeneity of treatment effect for stone clearance and patient-reported outcomes by participant and stone characteristics will be assessed. ETHICS AND DISSEMINATION: This study is approved by the central institutional review board with reliance across participating sites. Participating stakeholders will review results and contribute to development dissemination at regional, national and international meetings. TRIAL REGISTRATION NUMBER: NCT04285658; Pre-results.

Sleep Well! An adapted behavioral sleep intervention implemented in urban primary care.

STUDY OBJECTIVES: To describe the adaptation, feasibility, and initial outcomes of Sleep Well!, an intervention for early childhood insomnia and insufficient sleep, designed for families from lower-socioeconomic status backgrounds presenting to large metropolitan primary care sites. METHODS: Fifteen caregiver-child dyads (caregivers: 92.3% mothers, 80.0% Black, 53.3% ≤ 125% US poverty level; children: 73.3% female, 86.7% Black, mean age = 3.0 years) participated in this multimethod, single-arm trial. A family advisory board of caregivers (n = 4) and a clinician advisory board of sleep experts, primary care clinicians, and psychologists (n = 13) provided intervention feedback throughout the pilot. Most adaptations were related to intervention delivery methods, with some related to sleep strategies. At postintervention, caregivers completed surveys on intervention acceptability and cultural humility (primary outcomes) and completed semistructured interviews. Caregivers also reported on child sleep pre- and postintervention. RESULTS: Thirteen (86.6%) families completed Sleep Well! and 12 (80.0%) completed pre- and postintervention measures. Caregivers reported strong intervention acceptability and cultural humility. There were preintervention to postintervention reductions in child sleep problems, bedroom electronics, sleep onset latency, and night awakening frequency and duration. Nighttime sleep duration and overall insufficient sleep also improved. Qualitative data also showed strong intervention acceptability and perceived flexibility, with few participation barriers. CONCLUSIONS: A brief, early childhood behavioral sleep intervention delivered in primary care with families from primarily lower-socioeconomic status backgrounds and/or racially minoritized backgrounds is feasible to implement, with strong retention rates, acceptability, and perceptions of cultural humility. Child sleep improvements are positive and warrant replication in a randomized controlled trial. CLINICAL TRIAL REGISTRATION: Registry: ClinicalTrials.gov; Name: Implementing Behavioral Sleep Intervention in Urban Primary Care; URL: https://clinicaltrials.gov/ct2/show/NCT04046341; Identifier: NCT04046341. CITATION: Williamson AA, Okoroji C, Cicalese O, et al. Sleep Well! An adapted behavioral sleep intervention implemented in urban primary care. J Clin Sleep Med. 2022;18(4):1153-1166.

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.