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Background Assessment of bone health in young children has been hampered by limited reference values for bone mineral content (BMC) and areal bone mineral density (aBMD) by dual energy X-ray absorptiometry (DXA). Objectives To identify age, sex, and population ancestry effects on BMC and aBMD and develop smoothed reference ranges for BMC and aBMD in young children. To quantify precision of bone measurements and influence of height-for-age Z-scores on bone Z-scores. Methods We recruited 484 healthy children ages 1 to 2 years or 4.5 to 5 years at 2 clinical centers, who were seen once or up to 7 times over a 3-year period. Lumbar spine, distal forearm, and whole-body subtotal (ages ≥ 3 years) BMC and aBMD were measured by DXA. These data were combined with data from the Bone Mineral Density in Childhood Study from children ages 5 to 8.9 years to create the smoothed reference curves. Results For 1- to 5-year-olds, BMC and aBMD at all skeletal sites increased with age. Age trends differed by sex for BMC and aBMD of the spine, distal one-third radius, ultradistal radius, and by ancestry (Black vs non-Black) for all measures. BMC and aBMD precision (% coefficient of variation) ranged from 1.0% to 4.4%. Height Z-scores were positively associated with bone Z-scores and accounted for 4% to 45% of the variance. Conclusions We demonstrate the feasibility of bone density measurements in young children and provide robust reference ranges and stature adjustments for calculation of bone Z-scores at multiple skeletal sites to enable bone health assessments. more...

Objective We conducted a pre-post pilot trial to determine the feasibility and acceptability of a multi-component intervention (pre-clinic letter, shared decision making cards and follow-up phone call) designed to facilitate SDM in pediatric inflammatory bowel disease (IBD). Methods We recruited physicians (n = 11) caring for IBD patients and families (n = 36) expected to discuss anti-tumor necrosis treatment. We measured feasibility and acceptability of the intervention, observed SDM, perceived SDM, decision conflict, and regret. Medical records were used to assess clinical outcomes, time to decision and adherence. We compared all outcomes between the usual care and intervention study arms. Results Two out of three intervention components were feasible. Visit length increased significantly in the intervention arm. Parents and patients rated the intervention as acceptable, as did most physicians. The intervention was associated with a higher-level of observed SDM. There was no difference perceived SDM, decision conflict, regret or quality of life outcomes between arms. Physician global assessment improved over time in the intervention arm. Conclusions This pilot trial provides important guidance for developing a larger scale trial of a modified intervention. Practice implications Overall, our intervention shows promise in supporting SDM and engaging both parents and patients in pediatric IBD decisions. more...

To describe the scope and nature of health concerns, functional impairments, and quality of life issues among adults with brachial plexus birth injury (BPBI). A mixed methods study was conducted by surveying two social media networks of adults with BPBI using a combination of closed-ended and open-ended questions regarding the role of BPBI on ones’ health, function, and quality of life. Closed-ended responses were compared across ages and genders. Open-ended responses were qualitatively analyzed to expand upon the close-ended responses. Surveys were completed by 183 respondents (83% female, age range 20–87 years). BPBI was reported to impact hand and arm use in 80% of participants (including affected and unaffected limbs and bimanual tasks), overall health in 60% (predominantly pain), activity participation in 79% (predominantly activities of daily living and leisure), life roles in 76% (predominantly occupation and parenting), and overall quality of life in 73% (predominantly self-esteem, relationships, and appearance). Significantly more females than males reported other medical conditions and an impact on hand and arm use and life roles. No other responses varied by age or gender. BPBI affects many facets of health related quality of life in adulthood with variability among affected individuals.IMPLICATIONS FOR REHABILITATIONBrachial plexus birth injury’s scope of impact in adulthood is broad, covering every aspect of HRQoL.The focus of rehabilitation for brachial plexus birth injury (BPBI) through the lifespan should extend beyond improving physical musculoskeletal function and include comprehensive support for physical, emotional, social, and life role concerns.Brachial plexus birth injury’s nature of impact in adulthood varies among individuals within each aspect of health-related quality of life.The variability of BPBI’s impact in adulthood underscores the need for individualized, patient-centered assessment and rehabilitative care. Brachial plexus birth injury’s scope of impact in adulthood is broad, covering every aspect of HRQoL. The focus of rehabilitation for brachial plexus birth injury (BPBI) through the lifespan should extend beyond improving physical musculoskeletal function and include comprehensive support for physical, emotional, social, and life role concerns. Brachial plexus birth injury’s nature of impact in adulthood varies among individuals within each aspect of health-related quality of life. The variability of BPBI’s impact in adulthood underscores the need for individualized, patient-centered assessment and rehabilitative care. more...

We investigated the incidence and predictors of failure to undergo the Fontan in children with hypoplastic left heart syndrome who survived superior cavopulmonary connection.The cohort consists of all patients with hypoplastic left heart syndrome who survived to hospital discharge after superior cavopulmonary connection between 1988 and 2017. The primary outcome was attrition, which was defined as death, nonsuitability for the Fontan, or cardiac transplantation before the Fontan. Subjects were excluded if they were awaiting the Fontan, were lost to follow-up, or underwent biventricular repair. The study period was divided into 4 eras based on changes in operative or medical management. Attrition was estimated with 95% confidence intervals, and predictors were identified using adjusted, logistic regression models.Of the 856 hospital survivors after superior cavopulmonary connection, 52 died, 7 were deemed unsuitable for Fontan, and 12 underwent or were awaiting heart transplant. Overall attrition was 8.3% (71/856). Attrition rate did not change significantly across eras. A best-fitting multiple logistic regression model was used, adjusting for superior cavopulmonary connection year and other influential covariates: right ventricle to pulmonary artery shunt at Norwood (P .01), total support time at superior cavopulmonary connection (P .01), atrioventricular valve reconstruction at superior cavopulmonary connection (P = .02), performance of other procedures at superior cavopulmonary connection (P = .01), and length of stay after superior cavopulmonary connection (P .01).In this study spanning more than 3 decades, 8.3% of children with hypoplastic left heart syndrome failed to undergo the Fontan after superior cavopulmonary connection. This attrition rate has not decreased over 30 years. Use of a right ventricle to pulmonary artery shunt at the Norwood procedure was associated with increased attrition. more...

Background Children with congenital heart defects have an increased risk of neurodevelopmental disability. The impact of environmental chemical exposures during daily life on neurodevelopmental outcomes in toddlers with congenital heart defects is unknown. Methods This prospective study investigated the impacts of early childhood exposure to mixtures of environmental chemicals on neurodevelopmental outcomes after cardiac surgery. Outcomes were assessed at 18 months of age using The Bayley Scales of Infant and Toddler Development-III. Urinary concentrations of exposure biomarkers of pesticides, phenols, parabens, and phthalates, and blood levels of lead, mercury, and nicotine were measured at the same time point. Bayesian profile regression and weighted quantile sum regression were utilized to assess associations between mixtures of biomarkers and neurodevelopmental scores. Results One-hundred and forty infants were enrolled, and 110 (79%) returned at 18 months of age. Six biomarker exposure clusters were identified from the Bayesian profile regression analysis; and the pattern was driven by 15 of the 30 biomarkers, most notably 13 phthalate biomarkers. Children in the highest exposure cluster had significantly lower adjusted language scores by -9.41 points (95%CI: -17.2, -1.7) and adjusted motor scores by -4.9 points (-9.5, -0.4) compared to the lowest exposure. Weighted quantile sum regression modeling for the overall exposure-response relationship showed a significantly lower adjusted motor score (β = -2.8 points [2.5th and 97.5th percentile: -6.0, -0.6]). The weighted quantile sum regression index weights for several phthalates, one paraben, and one phenol suggest their relevance for poorer neurodevelopmental outcomes. Conclusions Like other children, infants with congenital heart defects are exposed to complex mixtures of environmental chemicals in daily life. Higher exposure biomarker concentrations were associated with significantly worse performance for language and motor skills in this population. more...

OBJECTIVES The objective of this study was to estimate hospital mortality and length of stay (LOS) for children with hypoplastic left heart syndrome undergoing superior cavopulmonary connection (SCPC). METHODS All hypoplastic left heart syndrome interstage survivors who underwent SCPC between 1 January 1988 and 31 December 2017 were included. The study period was divided into 4 eras based on changes in operative or medical management. Mortality rates were estimated using standard binomial proportions. Adjusted and unadjusted logistic regression models were used to identify risk factors for mortality and LOS. RESULTS The most common procedures for the cohort (n = 958) were Hemi-Fontan (57.3%) or Bidrectional Glenn shunt (35.7%). The mortality was 4.1% overall and decreased in all 3 later eras compared to era 1. Factors associated with mortality in a multiple covariate model included longer total support time, earlier gestational age, longer LOS at the Norwood Procedure and need for additional procedures. Overall, the median LOS was 7.0 days with a decrease from eras 1 to 2 and plateaued in eras 3 and 4. Predictors of longer LOS included genetic anomaly, longer Norwood LOS, additional procedures, lower weight at surgery and longer total support time. The type of SCPC was not associated with mortality or LOS. CONCLUSIONS In this large cohort of patients with hypoplastic left heart syndrome undergoing SCPC, hospital mortality has decreased significantly. LOS initially declined but plateaued in recent eras. The risk factors for mortality and longer LOS are related to patient and procedural complexity, especially the need for additional procedures at the time of SCPC. more...

Aim: Digital media use is prevalent among children and linked to potential developmental and health risks, but validated measures of children's digital media use are lacking. The aim of this study was to validate the Portuguese version of the ScreenQ with three distinct children's age groups. Methods: Parents of children living in Portugal completed an online survey including the 16-item version of the ScreenQ and items related to home activities and digital media use. A combination of classical and modern theory (Rasch) methods was used for analysis. Results: A total of 549 mothers and 51 fathers of 325 girls and 322 boys from 6 months to 9 years and 11 months old responded to the survey. Point-measure correlations were all positive and endorsement of item values were within acceptable ranges. Cronbach's coefficient α was acceptable for a new measure, and test–retest reliability was high. Statistically significant correlations were found between ScreenQ total scores and relevant demographic, play-related, parenting and digital media use items. Conclusion: The Portuguese version of the ScreenQ exhibited sound psychometric properties, including internal consistency and concurrent validity referenced to external items. Higher ScreenQ scores were correlated with higher digital media multitasking, lower parent–child interaction, and higher concerns regarding child's learning and behaviour. more...

Introductory article on JSCDM's Special Issue on the Practice of Clinical Data Management Worldwide.

Screen-based media use is prevalent in children and is associated with health risks. American Academy of Pediatrics (AAP) recommendations involve access to screens, frequency, content, and co-viewing. The aim of this study was to test the ScreenQ, a composite measure of screen-based media use. ScreenQ is a 15-item parent report measure reflecting AAP recommendations. Range is 0–26, higher scores reflecting greater non-adherence. With no “gold standard” available, four validated measures of skills and parenting practices cited as influenced by overuse were applied as the external criteria, including expressive language, speed of processing, emergent literacy, and cognitive stimulation at home. Psychometric analyses involved Rasch methods and Spearman’s ρ correlations. Sixty-nine families were administered ScreenQ. Child age ranged from 36 to 63 months old (52 ± 8; 35 girls). Mean ScreenQ score was 9.6 (±5.0; 1–22). Psychometric properties were strong (rCo-α = 0.74). ScreenQ scores were negatively correlated with CTOPP-2 (Comprehensive Test of Phonological Processing, Second Edition) (rρ = −0.57), EVT-2 (Expressive Vocabulary Test, Second Edition) (rρ = −0.45), GRTR (Get Ready to Read!) (rρ = −0.30) and StimQ-P (rρ = −0.42) scores (all p ≤ 0.01). ScreenQ shows potential as a composite measure of screen-based media use in young children in the context of AAP recommendations. ScreenQ scores were correlated with lower executive, language and literacy skills, and less stimulating home cognitive environment. more...

Background: The consent process used in clinical research today falls markedly short of the ideal process envisioned nearly 30 years ago. Critics have suggested that the informed consent pr...

Little is known about the number of minors enrolled in clinical research today. IRB administrators at leading pediatric medical centers were surveyed regarding studies with minors. Analyses were descriptive in nature with adaptive Bayesian bootstrap imputation used with missing data. Officials from 17/41 (41.5%) pediatric research centers responded: 74,204 active studies were estimated, 29,078 (39%) included minors, and 6574 (23%) were “more than minimal risk.” Minors accounted for 0.7–2.87M research subjects. Pediatric medicine desperately needs a more accurate and reliable reporting system for tracking the recruitment, retention, and involvement of minors in clinical research. more...

Clinical data management and its contemporary analogs, clinical research informatics and clinical data science constitute a small but rapidly growing specialty within the broader field of biomedical science. As such, the literature base is just evolving, drawing heavily from the larger and more established disciplines of biomedical informatics, biostatistics, clinical operations, and regulatory affairs. The purpose of this paper is to offer suggestions to individuals interested in submitting their research to the Journal of the Society for Clinical Data Management: Informatics and Data Science in Clinical Research.&nbsp more...

Objectives:The objective of this study was to investigate changes in serum biomarkers of acute brain injury, including white matter and astrocyte injury during chronic foetal hypoxaemia. We have previously shown histopathological changes in myelination and neuronal density in fetuses with chronic foetal hypoxaemia at a level consistent with CHD.Methods:Mid-gestation foetal sheep (110 ± 3 days gestation) were cannulated and attached to a pumpless, low-resistance oxygenator circuit, and incubated in a sterile fluid environment mimicking the intrauterine environment. Fetuses were maintained with an oxygen delivery of 20–25 ml/kg/min (normoxemia) or 14–16 ml/kg/min (hypoxaemia). Myelin Basic Protein and Glial Fibrillary Acidic Protein serum levels in the two groups were assessed by ELISA at baseline and at 7, 14, and 21 days of support.Results:Based on overlapping 95% confidence intervals, there were no statistically significant differences in either Myelin Basic Protein or Glial Fibrillary Acidic Protein serum levels between the normoxemic and hypoxemic groups, at any time point. No statistically significant correlations were observed between oxygen delivery and levels of Myelin Basic Protein and Glial Fibrillary Acidic Protein.Conclusion:Chronic foetal hypoxaemia during mid-gestation is not associated with elevated serum levels of acute white matter (Myelin Basic Protein) or astrocyte injury (Glial Fibrillary Acidic Protein), in this model. In conjunction with our previously reported findings, our data support the hypothesis that the brain dysmaturity with impaired myelination found in fetuses with chronic hypoxaemia is caused by disruption of normal developmental pathways rather than by direct cellular injury. more...

The Social and Emotional Assets and Resilience Scale (SEARS) is a promising instrument for prediction of resilience in youth; however, there is limited data to support its use. The purpose of the current study was to examine the factor structure, measurement invariance, internal consistency, and validity of the SEARS-Adolescent Report in youth 8 to 20 years of age. Two hundred and twenty-five childhood cancer survivors ( Mage = 15.9, SD = 4.2; 51.4% male; 74.5% White) and 122 student controls without history of significant health problems ( Mage = 14.2, SD = 3.5; 54.1% female; 79.5% White) 8 to 20 years of age completed the SEARS-A. The SEARS-A was found to have an adequate factor structure and model fit (χ2 = 1215.5, p < .001; root mean square error of approximation = .057; comparative fit index = .95; standardized root mean square residual = .06) and demonstrated invariance across domains of age, health status, gender, race, and socioeconomic status (Δ comparative fit index < −0.01). It also demonstrated excellent internal reliability, criterion validity, and current validity when compared with another well-established measure of psychological adjustment. As such, the SEARS-A has potential to be a useful, valid, and psychometrically sound tool for predicting social–emotional adjustment outcomes among at-risk youth 8 to 20 years of age. more...

The AAP recommends "shared" reading from early infancy for healthy development. However, many families are uncertain how to read most enjoyably and effectively with infants, especially from underserved backgrounds. Shared reading quality (interactivity) moderates benefits yet is challenging to measure. SHARE/STEP is a new model of shared reading quality at this age incorporating evidence-based behaviors.To test the SharePR parent-report measure of caregiver-infant reading quality.This study involved mother-infant dyads in two unrelated trials in an obstetric (0-2 months old) and pediatric (6-9 months old) clinic. SharePR is a 10-item measure based on the SHARE/STEP model. Analyses involved descriptive statistics, measures of psychometric integrity, and correlations with home literacy environment (HLE).There were 99 dyads in the younger (1.2 + 0.5 months) and 108 dyads in the older groups (6.6 + 1.1 months). A majority were of non-white race (73%, 96%) and low-socioeconomic status (56%, 44% in-poverty). SharePR administration time was under 2 min and scores were normally distributed at each age. Psychometric properties were strong in terms of internal consistency and reliability. Scores were positively correlated with HLE for the older group (p 0.05).SharePR may be an efficient tool to quantify shared reading quality with infants, warranting further investigation.Data for these analyses were collected via two unrelated trials led by the lead author (J.S.H.). For the younger cohort, this is registered on the ClinicalTrials.gov website, ID# NCT04031235. For the older cohort, this is registered on the ClinicalTrials.gov website, ID# 2017-6856.The AAP recommends caregiver-child ("shared") reading beginning in infancy, yet many families are uncertain how to do so. Verbal and social-emotional interactivity during shared reading ("quality") moderates benefits and is often low in families from disadvantaged backgrounds, yet is challenging to measure. SharePR is a 10-item parent-report measure of shared reading quality based on a novel conceptual model incorporating evidence-based behaviors (SHARE/STEP). SharePR exhibited promising psychometric properties in two separate samples of mothers of younger and older infants. SharePR is a potentially useful measure of shared reading quality at this formative age, for research and to frame early reading guidance. more...

BACKGROUND AND OBJECTIVES: The American Academy of Pediatrics recommends literacy and school readiness promotion during well visits. The Reading House (TRH) is a children’s book–based screener of emergent literacy skills in preschool-aged children. Vocabulary, rhyming, and rapid naming are core emergent skills, and reading abilities are associated with thicker cortex in the left hemisphere. Our objective was to expand validity of TRH relative to these skills and explore association with cortical thickness. METHODS: Healthy preschool-aged children completed MRI including a T1-weighted anatomic scan. Before MRI, TRH and assessments of rapid naming (Comprehensive Test of Phonological Processing, Second Edition), rhyming (Pre-Reading Inventory of Phonological Awareness), vocabulary (Expressive Vocabulary Test, Second Edition), and emergent literacy (Get Ready to Read!) were administered. Analyses included Spearman-ρ correlations (rρ)accounting for age, sex, and socioeconomic status (SES). MRI analyses involved whole-brain measures of cortical thickness relative to TRH scores, accounting for covariates. RESULTS: Seventy children completed assessments (36–63 months old; 36 female) and 52 completed MRI (37–63 months; 29 female). TRH scores were positively correlated with Comprehensive Test of Phonological Processing, Second Edition (rρ = 0.61), Expressive Vocabulary Test, Second Edition (rρ = 0.54), Get Ready to Read! (rρ = 0.87), and Pre-Reading Inventory of Phonological Awareness scores (rρ = 0.64; all P < .001). These correlations remained statistically significant across age, sex, and SES groups. TRH scores were correlated with greater thickness in left-sided language and visual cortex (P–family-wise error CONCLUSIONS: These findings expand validation evidence for TRH as a screening tool for preschool-aged children, including associations with emergent skills and cortical thickness, and suggest important differences related to SES. more...

Background:The contribution of neonatal cyanosis, inherent to cyanotic congenital heart disease, to the magnitude of neurologic injury during deep hypothermic circulatory arrest has not been fully delineated. This study investigates the impact of cyanosis and deep hypothermic circulatory arrest on brain injury.Methods:Neonatal piglets were randomised to placement of a pulmonary artery to left atrium shunt to create cyanosis or sham thoracotomy. At day 7, animals were randomised to undergo deep hypothermic circulatory arrest or sham. Arterial oxygen tension and haematocrit were obtained. Neurobehavioural performance was serially assessed. The animals were sacrificed on day 14. Brain tissue was assessed for neuronal necrosis using a 5-point histopathologic score.Results:Four experimental groups were analysed (sham, n = 10; sham + deep hypothermic circulatory arrest, n = 8; shunt, n = 9; shunt + deep hypothermic circulatory arrest, n = 7). Cyanotic piglets had significantly higher haematocrit and lower partial pressure of oxygen at day 14 than non-cyanotic piglets. There were no statistically significant differences in neurobehavioural scores at day 1. However, shunt + deep hypothermic circulatory arrest piglets had evidence of greater neuronal injury than sham animals (median (range): 2 (0–4) versus 0 (0–0), p = 0.02).Discussion:Cyanotic piglets undergoing deep hypothermic circulatory arrest had increased neuronal injury compared to sham animals. Significant injury was not seen for either cyanosis or deep hypothermic circulatory arrest alone relative to shams. These findings suggest an interaction between cyanosis and deep hypothermic circulatory arrest and may partially explain the suboptimal neurologic outcomes seen in children with cyanotic heart disease who undergo deep hypothermic circulatory arrest. more...

Objective To estimate feasibility, usability and efficacy of a mobile parenting app (Rx for Success; RxS) to enhance reading guidance provided to parents of young children during well-visits. Methods This trial was conducted at a clinic serving primarily families of Hispanic ethnicity and low-socioeconomic status (SES) where Reach Out and Read (ROR) is standard practice. It involved 252 parent-child dyads in 2 age groups (~6-months old, ~18-months old) randomized during well-visits to receive RxS or a children's book modeling alternatives to screen time (Control) by research coordinators. RxS involves videos, activities and “push” messages. Follow-up assessments were conducted approximately 6 months later, including impression and use, shared reading behaviors, child language and screen time. Results A total of 217 dyads completed both visits (110 RxS, 107 Control). Time to introduce RxS was under 3 minutes and 32% of parents experienced largely minor performance issues. Parent impression of RxS was favorable for both age groups at baseline and follow-up, though use was infrequent, attributable to a desire for more relevant and updated content. Significant findings favoring RxS included shared reading as a favorite activity, more frequent shared reading reported at 12 months and higher language scores at 24 months. Screen time was equivalent between cohorts, exceeding American Academy of Pediatrics guidelines. Conclusions A mobile app introduced to parents of young children from low-SES backgrounds was feasible during well-visits, rated as helpful, and effective to enhance shared reading at younger and language at older ages. While a potentially impactful enhancement to ROR, features needing improvement were identified. more...

s constitute an important part of the scientific process. They allow readers to assess important attributes of a given project or study in a quick and efficient manner. Each year the Society of Clinical Data Management (SCDM), similar to other professional societies, issues a ‘Call for Abstracts’ in which data professionals are encouraged to submit summaries of their work for possible presentation at the annual meeting. If an abstract is accepted, authors will be asked to share their work at the meeting through a podium, poster, panel, or round-table presentation. The purpose of this article is to educate SCDM members about the fundamentals of writing an abstract, including its overarching purpose, key components, and potential for contributions to both the profession and science, more generally. Recommendations for writing abstracts are also provided. more...

Introduction: Resilience is the ability to recover after stressful events and is believed to help reduce the long-term adverse effects. The role of resilience as a determinant of neurodevelopmental (ND) outcomes and quality of life (QOL) in young adults with congenital heart disease (CHD) has not been fully delineated. Hypothesis: Higher resilience in young adults with CHD is associated with better QOL and can be predicted by patient factors and ND status in the preschool period. Methods: Prospective evaluation of subjects ≥ 18 years of age enrolled in a study of genetic determinants of ND outcome after repair of CHD at age ≤ 6 months. The subjects had previously undergone a ND evaluation (executive function, attention/impulsivity and social skills) at 4 years of age. For this study, subjects completed the RS-14, a questionnaire that captures the constructs of resilience; and the SF-36, a functional health status questionnaire. Factors associated with resilience were determined using generalized linear (gamma) regression. Results: The RS-14 and SF-36 were completed by 151 subjects (74 male and 77 female, mean age 19.5 ± 1.1 years). The median resilience score of 79 (CI 0.95 = 77, 82) was significantly greater than the young adult population median of 76.0, p = 0.006. Resilience was graded as moderate or higher in 103 subjects (68%). Higher resilience scores were significantly correlated with higher QOL assessed by the SF-36 Mental Component Summary, r = 0.40, p < 0.001. By simple regression, Caucasian race was associated with higher resilience scores, p = 0.03. Prematurity, low birth weight, presence of a genetic anomaly, and severity of CHD were not associated with higher or lower scores, all p > 0.2. No operative management factors were associated with higher or lower scores, all p > 0.28. Better performance for executive function ( p = 0.03) and social skills ( p = 0.02) at the 4-year ND evaluation were associated with higher resilience. Hyperactivity was associated with lower resilience scores, p = 0.02. Conclusions: Many young adults with CHD demonstrate good resilience. Higher resilience is associated with better QOL. Performance on ND testing in the preschool period may identify patients with lower resilience and offer an opportunity for intervention. more...

The American Academy of Pediatrics recommends literacy and school readiness promotion during well visits.Healthy preschool-aged children completed MRI including a T1-weighted anatomic scan. Before MRI, TRH and assessments of rapid naming (Comprehensive Test of Phonological Processing, Second Edition), rhyming (Pre-Reading Inventory of Phonological Awareness), vocabulary (Expressive Vocabulary Test, Second Edition), and emergent literacy (Seventy children completed assessments (36-63 months old; 36 female) and 52 completed MRI (37-63 months; 29 female). TRH scores were positively correlated with Comprehensive Test of Phonological Processing, Second Edition (These findings expand validation evidence for TRH as a screening tool for preschool-aged children, including associations with emergent skills and cortical thickness, and suggest important differences related to SES. more...

Background The superior cavo‐pulmonary connection was introduced at our institution in 1988 for infants undergoing surgery for hypoplastic left heart syndrome. Patients with hypoplastic left heart syndrome remain at high risk for mortality in the time period between the Norwood procedure and the superior cavo‐pulmonary connection. The primary objectives of this study were to compare interstage mortality across 4 eras and analyze factors that may impact interstage mortality. Methods and Results Patients with hypoplastic left heart syndrome who underwent the Norwood procedure, were discharged from the hospital, and were eligible for superior cavo‐pulmonary connection between January 1, 1988, and December 31, 2017, were included. The study period was divided into 4 eras based on changes in operative or medical management. Mortality rates were estimated with 95% CIs. Adjusted and unadjusted logistic regression models were used to identify risk factors for mortality. There were 1111 patients who met the inclusion criteria. Overall, interstage mortality was 120/1111 (10.8%). Interstage mortality was significantly lower in era 4 relative to era 1 (4.6% versus 13.4%; P =0.02) during the time that age at the superior cavo‐pulmonary connection was the lowest (135 days; P P =0.02) and was more routinely practiced in era 4. Conclusions During this 30‐year experience, the risk of interstage mortality decreased significantly in the most recent era. Factors that coincide with this finding include younger age at superior cavo‐pulmonary connection, introduction of an interstage monitoring program, and increased use of the right ventricle to pulmonary artery shunt. more...

Objective Juvenile-onset fibromyalgia (JFM) is a chronic debilitating pain condition that negatively impacts physical, social and academic functioning. Cognitive-behavioral therapy (CBT) is beneficial in reducing functional disability among adolescents with JFM but has only a modest impact on pain reduction and does not improve physical exercise participation. This randomized controlled trial (RCT) aims to test whether a novel intervention that combines CBT with specialized neuromuscular exercise training (the Fibromyalgia Integrative Training program for Teens “FIT Teens”) is superior to CBT alone or a graded aerobic exercise (GAE) program. Design/Methods This 3-arm multi-site RCT will examine the efficacy of the FIT Teens intervention in reducing functional disability (primary outcome) and pain intensity (secondary outcome), relative to CBT or GAE. All interventions are 8-weeks (16 sessions) in duration and are delivered in small groups of 4–6 adolescents with JFM. A total of 420 participants are anticipated to be enrolled across seven sites with approximately equal allocation to each treatment arm. Functional disability and average pain intensity in the past week will be assessed at baseline, post-treatment and at 3-, 6-, 9- and 12-month follow-up. The 3-month follow-up is the primary endpoint to evaluate treatment efficacy; longitudinal assessments will determine maintenance of treatment gains. Changes in coping, fear of movement, biomechanical changes and physical fitness will also be evaluated. Conclusions This multi-site RCT is designed to evaluate whether the combined FIT Teens intervention will have significantly greater effects on disability and pain reduction than CBT or GAE alone for youth with JFM. Clinical trials.gov registration: NCT 03268421. more...

BackgroundVeno-arterial extracorporeal membrane oxygenation is frequently used in patients with cardiac disease. We evaluated short-term outcomes and identified factors associated with hospital mortality in cardiac patients supported with veno-arterial extracorporeal membrane oxygenation.MethodsA retrospective review of patients supported with veno-arterial extracorporeal membrane oxygenation at a university-affiliated children’s hospital was performed.ResultsA total of 253 patients with cardiac disease managed with extracorporeal membrane oxygenation were identified; survival to discharge was 48%, which significantly improved from 39% in an earlier era (1995–2001) (p=0.01). Patients were categorised into surgical versus non-surgical groups on the basis of whether they had undergone cardiac surgery before or not, respectively. The most common indication for extracorporeal membrane oxygenation was extracorporeal cardiopulmonary resuscitation: 96 (51%) in the surgical group and 45 (68%) in the non-surgical group. In a multiple covariate analysis, single-ventricle physiology (p=0.01), duration of extracorporeal membrane oxygenation (pConclusionWe report improvement in survival compared with an earlier era in cardiac patients supported with extracorporeal membrane oxygenation. Single-ventricle physiology continues to negatively impact survival, along with evidence of organ dysfunction during extracorporeal membrane oxygenation, duration of extracorporeal membrane oxygenation, and length of stay. more...

Background The American Academy of Pediatrics (AAP) recommends shared reading beginning as soon as possible after birth to promote healthy development. Shared reading quality can strongly influence outcomes, especially in children from low-SES households. Dialogic reading is a method developed to enhance verbal interactivity and engagement through book sharing, advocated by the AAP and clinic-based programs such as Reach Out and Read. There is no brief, validated, caregiver report measure of dialogic reading or shared reading quality currently available. Methods This cross-sectional study involved 49 healthy mother-child dyads (mean child age 4.5 yrs., SD = 0.6 yrs.) from 2 separate MRI-based studies. The DialogPR was administered by trained research coordinators following MRI, along with the READ subscale of the validated StimQ-P measure of home cognitive environment. The DialogPR consists of eight items developed in consultation with experts in early literacy, based on the PEER/CROWD dialogic reading conceptual model. Estimated reading level is 6th grade. Descriptive statistics were computed at both the item and scale levels. Modern theory Rasch methods were used to analyze all eight DialogPR items along with preliminary estimates of reliability and validity. Results Our combined sample involved 15 boys and 34 girls, and was diverse in terms of age, household income, and maternal education. DialogPR administration time was less than 2 min, with no problems reported. The DialogPR demonstrated strong internal consistency and reliability (Cronbach’s alpha = 0.82), and criterion-related validity with the StimQ-P READ (Spearman’s rho coefficient = 0.53). Rasch analysis revealed strong psychometric properties in terms of reliability, variability in item difficulty, and inter-item and item-measure correlations. Conclusions Preliminary evidence suggests that the DialogPR may be an efficient means to assess shared reading quality and dialogic reading via caregiver report for clinical and research purposes, warranting further investigation. more...

OBJECTIVE: National health organizations and expert committees have issued recommendations for health behaviors related to obesity risk. Behavioral and family-based weight management interventions for preschoolers often target improving adherence to these recommendations, but it is unknown how the health behaviors of preschoolers with obesity enrolled in weight control treatments (WCTs) compare with these guidelines. In this study, the dietary intake, activity, and sleep behaviors of preschoolers with obesity enrolled in a family-based behavioral WCT are described and compared with national health behavior recommendations. METHODS: Health behaviors of 151 preschoolers with obesity (M age = 4.60, SD = 0.93) enrolled in a clinical trial of a weight management program were measured at baseline through caregiver-report questionnaires, three 24-hr dietary recalls, and accelerometers. RESULTS: In total, 70% of the sample exceeded daily caloric recommendations, only 10 and 5% met recommendations for fruit and vegetable intake, respectively, and only 30% met the recommendation of consuming no sugar-sweetened beverages. The majority of the sample met the daily recommendations for 60 min of moderate-to-vigorous activity (80%) more...

Screen-based media use is prevalent in children and is associated with health risks. American Academy of Pediatrics (AAP) recommendations involve access to screens, frequency, content, and co-viewing. The aim of this study was to test the ScreenQ, a composite measure of screen-based media use.ScreenQ is a 15-item parent report measure reflecting AAP recommendations. Range is 0-26, higher scores reflecting greater non-adherence. With no "gold standard" available, four validated measures of skills and parenting practices cited as influenced by overuse were applied as the external criteria, including expressive language, speed of processing, emergent literacy, and cognitive stimulation at home. Psychometric analyses involved Rasch methods and Spearman's ρ correlations.Sixty-nine families were administered ScreenQ. Child age ranged from 36 to 63 months old (52 ± 8; 35 girls). Mean ScreenQ score was 9.6 (±5.0; 1-22). Psychometric properties were strong (rScreenQ shows potential as a composite measure of screen-based media use in young children in the context of AAP recommendations. ScreenQ scores were correlated with lower executive, language and literacy skills, and less stimulating home cognitive environment. more...

BACKGROUND: The American Academy of Pediatrics recommends literacy promotion and developmental assessment during well-child visits. Emergent literacy skills are well defined, and the use of early screening has the potential to identify children at risk for reading difficulties and guide intervention before kindergarten. METHODS: The Reading House (TRH) is a children’s book designed to screen emergent literacy skills. These are assessed by sharing the book with the child and using a 9-item, scripted scoring form. Get Ready to Read! (GRTR) is a validated measure shown to predict reading outcomes. TRH and GRTR were administered in random order to 278 children (mean: 43.1 ± 5.6 months; 125 boys, 153 girls) during well-child visits at 7 primary care sites. Parent, child, and provider impressions of TRH were also assessed. Analyses included Rasch methods, Spearman-ρ correlations, and logistic regression, including covariates age, sex, and clinic type. RESULTS: Psychometric properties were strong, including item difficulty and reliability. Internal consistency was good for new measures (rCo-α = 0.68). The mean TRH score was 4.2 (±2.9; range: 0–14), and mean GRTR was 11.1 (±4.4; range: 1–25). TRH scores were positively correlated with GRTR scores (rs = 0.66; high), female sex, private practice, and child age (P < .001). The relationship remained significant controlling for these covariates (P < .05). The mean TRH administration time was 5:25 minutes (±0:55; range: 3:34–8:32). Parent, child, and provider impressions of TRH were favorable. CONCLUSIONS: TRH is a feasible, valid, and enjoyable means by which emergent literacy skills in 3- and 4-year-old children can be directly assessed during primary care. more...

Background: Shared decision making (SDM) between families and physicians may facilitate informed, timely decisions to proceed with biologic therapy in children with inflammatory bowel disease (IBD). Our team previously developed an SDM tool to aid communication between physicians and families when considering biologic therapy for children with IBD. Objective: We are conducting a prospective, pre-post pilot trial of a new SDM tool. The primary aim of the study is to assess feasibility of both the intervention and trial procedures for a future large-scale trial. Methods: We are enrolling physicians with experience prescribing biologic therapy in the past year and families of children with IBD. Families in the intervention arm receive a 3-step intervention including a letter sent before trial consent or clinic appointment, an in-clinic decision tool and a follow-up phone call. Our primary trial outcome is a measure of feasibility, with measures of clinical and decision outcomes secondary. We seek to enroll 27 families in each of 2 arms (usual-care and intervention) and plan data collection at the time of the initial visit or hospital stay, and at 1 week, 3 months, and 6 months after the initial visit. Conclusion: This study protocol is designed to demonstrate that integrating novel consent procedures, including timing and multiple versions of written consent, may increase trial feasibility while maintaining scientific rigor and full protection of study participants. Keywords: Pediatrics, Inflammatory bowel disease, Pilot trials, Consent more...

Background Family-based obesity treatment interventions can successfully reduce energy intake in preschoolers. An implicit goal of obesity treatment interventions is to improve diet quality, but diet quality has been less examined as a treatment outcome in studies of preschoolers. The purpose of this study was to conduct a secondary analysis comparing the change in diet quality and home food environment in preschoolers assigned to a behavioral family-based obesity intervention (LAUNCH), motivational interviewing (MI) condition, or standard care (STC) condition. Methods Three 24-h dietary recalls were completed at baseline and 6-months and were analyzed using NDS-R software; diet quality was assessed using the Healthy Eating Index-2010 (HEI-2010). Availability of foods and beverages in the home was assessed through direct observation using the Home Health Environment tool that classifies foods and beverages as ‘red’ or ‘green’ based upon fat and sugar content. Repeated measures linear mixed effects models were used to examine changes in diet quality and home food environment between conditions (LAUNCH, MI, STC). Results At 6-months, preschoolers in the LAUNCH condition had a higher HEI-2010 total score (62.8 ± 13.7) compared to preschoolers in the MI (54.7 ± 13.4, P = 0.022) and STC (55.8 ± 11.6, P = 0.046) conditions. Regarding the home food environment, families in LAUNCH had significantly less ‘red’ foods in their home at 6-months (12.5 ± 3.4 ‘red’ foods) compared to families in MI (14.0 ± 3.7 ‘red’ foods, P = 0.030), and STC (14.3 ± 3.4 ‘red’ foods, P = 0.006). There were no statistically significant differences across home food environments for number of ‘green’ foods. Conclusion Family-based obesity treatment interventions for preschoolers can improve overall diet quality and alter the home food environment through reductions in ‘red’ foods. Trial registration Clinicaltrials.gov, NCT01546727. Registered March 7, 2012. more...

Objective Hypoplastic left heart syndrome is one of the most common and challenging lesions requiring surgical intervention in the neonatal period. The Norwood procedure for hypoplastic left heart syndrome was first reported in 1983. The objective of this study was to describe early outcomes after the Norwood procedure at a single institution over 30 years. Methods This retrospective cohort study included all patients with hypoplastic left heart syndrome (and variants) who underwent the Norwood procedure between January 1984 and May 2014 at a single institution. The study period was divided into 6 eras: era 1, 1984 to 1988; era 2, 1989 to 1993; era 3, 1994 to 1998; era 4, 1999 to 2003; era 5, 2004 to 2008; and era 6, 2009 to 2014. The primary outcome was in-hospital mortality after the Norwood procedure. Binomial point estimates complete with 95% confidence intervals (CL0.95) were computed for the entire cohort and by era. Results During the study period, 1663 infants underwent the Norwood procedure. Overall in-hospital mortality was 25.9% (CL0.95, 23.8-28.0). Mortality by chronologic era was 40.4% (CL0.95, 34.9-45.9), 33.6% (CL0.95, 29.2-37.9), 28.7% (CL0.95, 22.8-34.6), 14.9% (CL0.95, 10.4-19.3), 11.2% (CL0.95, 7.4-15.0), and 15.7% (CL0.95, 10.3-21.1). Survival was improved in eras 4 to 6 compared with eras 1 to 3 (P all Conclusions Survival after the Norwood procedure has plateaued despite improvements in diagnosis, perioperative care, and surgical techniques. Nonmodifiable patient characteristics are important determinants of the risk of mortality. more...

Medication nonadherence is a significant health care issue requiring regular behavioral treatment. Lack of sufficient health care resources and patient/family time commitment for weekly treatment are primary barriers to receiving appropriate self-management support. We describe the methodology of the Telehealth Enhancement of Adherence to Medication (TEAM) trial for medication nonadherence in pediatric inflammatory bowel disease (IBD). For this trial, participants 11-18 years of age will be recruited from seven pediatric hospitals and will complete an initial 4-week run in to assess adherence to a daily medication. Those who take less than 90% of their prescribed medication will be randomized. A total of 194 patients with IBD will be randomized to either a telehealth behavioral treatment (TBT) arm or education only (EO) arm. All treatments will be delivered via telehealth video conferencing. The patients will be assessed at baseline, post-treatment, 3, 6, and 12 months. We anticipate that participants in the TBT arm will demonstrate a statistically significant improvement at post-treatment and 3-, 6-, and 12-month follow-up compared to participants in the EO arm for both medication adherence and secondary outcomes (i.e., disease severity, patient quality of life, and health care utilization). If efficacious, the TEAM intervention could be disseminated broadly and reduce health care access barriers so that the patients could receive much needed self-management intervention. more...

OBJECTIVES: Pregnancies with congenital heart disease in the foetus have an increased prevalence of pre-eclampsia, small for gestational age and preterm birth, which are evidence of an impaired maternal–foetal environment (MFE). METHODS: The impact of an impaired MFE, defined as pre-eclampsia, small for gestational age or preterm birth, on outcomes after cardiac surgery was evaluated in neonates (n = 135) enrolled in a study evaluating exposure to environmental toxicants and neuro-developmental outcomes. RESULTS: The most common diagnoses were transposition of the great arteries (n = 47) and hypoplastic left heart syndrome (n = 43). Impaired MFE was present in 28 of 135 (21%) subjects, with small for gestational age present in 17 (61%) patients. The presence of an impaired MFE was similar for all diagnoses, except transposition of the great arteries (P more...

Extracorporeal membrane oxygenation is an important form of short-term mechanical support in children with cardiac disease, but information on long-term outcomes and quality of life is limited. The primary objective of this study was to determine the long-term outcomes of children previously supported by extracorporeal membrane oxygenation for cardiac etiologies.A retrospective analysis was performed on patients with cardiac disease managed with extracorporeal membrane oxygenation between January 1, 1995, and December 31, 2012, at the Children's Hospital of Philadelphia. Survivors completed patient- and parent-reported verbal and written surveys, and univariate analyses assessed risk factors for long-term outcomes.Tertiary-care children's hospital.Patients with cardiac disease managed with extracorporeal membrane oxygenation.None.Over 18 years, 396 patients were managed with extracorporeal membrane oxygenation with 43% survival to discharge. The median age at cannulation was 78 days. The majority had congenital heart disease (86%), surgery prior to extracorporeal membrane oxygenation (71%), and cardiopulmonary arrest as the primary extracorporeal membrane oxygenation indication (53%). With 6-year median follow-up, 66% are known to be deceased, including 38 deaths after hospital discharge. Among survivors at discharge, 65 (38%) completed the phone survey, and 33 (19%) completed the written survey. Negative clinical outcomes, defined as having at least significant physical limitations or "fair" or "poor" health, were present in 18% of patients. No patient- or extracorporeal membrane oxygenation-related variables were associated with negative outcomes in univariate analyses. There were significantly lower self-reported and parent-reported written Pediatric Quality of Life Inventory quality of life scores in children compared with healthy individual normative data but no differences in adolescents.In this series of pediatric cardiac patients supported by extracorporeal membrane oxygenation, mortality was 66% with 6-year median follow-up. The majority reported positive outcomes with respect to health and physical limitations, but children reported lower quality of life compared with healthy individuals. more...

The paper-based pedigree is the current standard for family health history (FHH) documentation in genetic counseling. Several tools for electronic capture of family health data have been developed to improve re-use and accessibility, data quality and standardization, ease of updating, and integration with electronic medical records. One such tool, the tablet-based Proband application, provides a flexible approach to data capture in dynamic and diverse clinical settings. This study compared Proband FHH collection to paper-based methods and investigated the usability of Proband in a clinical setting. After one use by 23 genetic counselors and students, Proband had 91% accuracy with a FHH audio scenario, which was significantly less (p more...

This study aimed to provide insight into the development of postural control abilities in youth. A total of 276 typically developing adolescents (155 males, 121 females) with a mean age of 13.23 years (range of 7.11–18.80) were recruited for participation. Subjects performed two-minute quiet standing trials in bipedal stance on a force plate. Center of pressure (COP) trajectories were quantified using Sample Entropy (SampEn) in the anterior-posterior direction (SampEn-AP), SampEn in the medial–lateral direction (SampEn-ML), and Path Length (PL) measures. Three separate linear regression analyses were conducted to predict the relationship between age and each of the response variables after adjusting for individuals’ physical characteristics. Linear regression models showed an inverse relationship between age and entropy measures after adjusting for body mass index. Results indicated that chronological age was predictive of entropy and path length patterns. Specifically, older adolescents exhibited center of pressure displacement (smaller path length) and less complex, more regular center of pressure displacement patterns (lower SampEn-AP and SampEn-ML values) compared to the younger children. These findings support prior studies suggesting that developmental changes in postural control abilities may continue throughout adolescence and into adulthood. more...

The African American Heart Failure Trial (A-HeFT) and the FDA approval of BiDil for race-specific prescription have stirred the debate about the scientific and medical status of race. Yet there is no assessment of the potential fallouts of this dispute on physicians' willingness to prescribe the drug. We present here an analysis of the factors influencing physicians' prescription of BiDil and investigate whether exposure to the controversy has an impact on their therapeutic judgments about the drug.We conducted an electronic survey with physicians in the department of internal medicine at the University of Cincinnati. Participants were randomly assigned to two groups, with one group receiving information about the controversy over BiDil. We used various statistical tests, including a linear mixed effects model, to analyze the results.27% of the participants reported using patients' race as a major factor in making treatment decisions. 33% reported the inefficacy of standard therapies, 25% the severity of the disease, and 15% other unspecified factors as primary determining criteria in prescribing BiDil. With respect to the controversy, 68% of physicians reported that they were not aware of any controversy surrounding BiDil. Physicians' willingness to prescribe BiDil as a therapy was associated with their awareness of the controversy surrounding A-HeFT (Overall, physicians prescribe and are willing to prescribe BiDil more to black patients than to white patients. However, physicians' lack of awareness about the controversial scientific status of A-HeFT suggests the need for more efficient ways to convey scientific information about BiDil to clinicians. Furthermore, the uncertainties about the determination of clinical utility of BiDil for the individual patient raise questions about whether this specific race-based therapy is in patients' best interest. more...

The aim of this study is to determine the contribution of strain ε cc in mid left ventricular (LV) segments to the reduction of composite LV circumferential ε cc in assess severity of duchenne muscular dystrophy (DMD) heart disease as assessed by cardiac magnetic resonance imaging (CMR). DMD patients and control subjects were stratified by age, LV ejection fraction, and late gadolinium enhancement (LGE) status. Tagged CMR images were analyzed for global ventricular function, LGE imaging, and composite and segmental ε cc. The relationship between changes in segmental ε cc changes and LGE across patient groups was assessed by a statistical step-down model. LV ε cc exhibited segmental heterogeneity; in control subjects and young DMD patients, ε cc was greatest in LV lateral free wall segments. However, with increasing age and cardiac disease severity as demonstrated by decreased EF and development of myocardial strain the segmental differences diminished. In subjects with advanced heart disease as evidenced by reduced LV ejection fraction and presence of LGE, very little segmental heterogeneity was present. In control subjects and young DMD patients, ε cc was greatest in LV lateral free wall segments. Increased DMD heart disease severity was associated with reduced composite; ε cc diminished regional ε cc heterogeneity and positive LGE imaging. Taken together, these findings suggest that perturbation of segmental, heterogeneous ε cc is an early biomarker of disease severity in this cross-section of DMD patients. more...