Medical Specialties Commons^™

Cross-Cultural Differences Among Children And Youth With Special Health Care Needs: A Mixed-Methods Analysis Of Chinese, Spanish, And English-Speaking Families, Kathleen Mcgrath, Miriam Franco, Amy Morales-Lara, Karen Bonuck Phd, Joanne Siegel

Developmental Disabilities Network Journal

One in five U.S. children has a special health care need, with prevalence rates rising each year. Children and youth with special health care needs (CYSHCN) from immigrant and/or non-English speaking families face heightened barriers to care, unmet health needs, and worsened health outcomes. This mixed-methods study explores cross-cultural similarities and differences among English-, Spanish-, and Chinese-speaking families of CYSHCN across four key domains – (1) the diagnostic process, (2) access to essential services, (3) community integration, and (4) social supports. As part of a statewide, New York State Department of Health funded initiative implemented across three University Centers for …

Impact (Immune Monitoring And Phenotype Assessment Of Clinical Trajectory) For Patients With Inborn Errors Of Immunity From The Primary Immune Deficiency Treatment Consortium (Pidtc), Danielle E Arnold, Hesham Eissa, Elizabeth Dunn, Geoffrey D E Cuvelier, Nicola A M Wright, Rebecca A Marsh, Suhag Parikh, Blachy Davila Saldana, Mark T Vander Lugt, Rosa Bacchetta, Lisa Forbes Satter, Shanmuganathan Chandrakasan, Erinn Kellner, Eric J Allenspach, Erica G Schmitt, Brent Logan, Lauri Burroughs, Morton J Cowan, Linda M Griffith, Luigi Notarangelo, Sung-Yun Pai, Michael A Pulsipher, Christopher C Dvorak, Elie Haddad, Jennifer M Puck, Troy R Torgerson, Jennifer W Leiding, Jennifer Heimall, Alice Y Chan

Faculty, Staff and Students Publications

Background: Evaluating natural history and outcomes of rare inborn errors of immunity (IEIs) has been challenging due to the diverse disease manifestations and lack of standardized data.

Objective: To develop a set of standardized, quantitative, generalizable data collection modules to measure organ dysfunction in IEIs, modeled on the previously validated Common Terminology Criteria for Adverse Event system for grading treatment toxicity.

Methods: Disease conditions were organized by organ system. Experts from the Primary Immune Deficiency Treatment Consortium identified essential features of severe combined immunodeficiency, chronic granulomatous disease, Wiskott-Aldrich syndrome, and primary immune regulatory disorders and compared these to Common Terminology …

The Relationship Between Psychological Stress And Sars-Cov-2 Nucleocapsid-Antibody Response Following Natural Infection: Longitudinal Findings From The Aegis Study, Erika T Beidelman, Ashley Judge, David B Allison, Stephanie Dickinson, Lilian Golzarri Arroyo, Anna L M Macagno, Michael J Ricciardi, Jon Macy, Stanley Perlman, Christina Ludema, Kevin C Maki, Molly Rosenberg

Faculty, Staff and Students Publications

Psychological stress can cause diminished immune response to infectious challenges. The extent this holds true during severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection and the modifying effect of vaccination status is untested. We explored these relationships in a non-random sample standardized to the US adult population. Across 2178 participants in the Aegis study (2021-2022), we identified 507 eligible laboratory-confirmed SARS-CoV-2 infections. We estimated the natural log of nucleocapsid-binding antibody absorbance (N-antibody) at study visits up to 225 days following a positive SARS-CoV-2 test. Linear mixed effects models were fit to estimate the association of baseline perceived stress and, separately, …

A Multi-Country Community Of Practice To Strengthen Quality Improvement In Low- And Middle-Income Countries: A Quality Improvement Program Description, Samhita Bhargava, Heather A Haq, Brodus A Franklin, Elizabeth Davis, Florence Anabwani-Richter, Thobile Bhembe, Lindokuhle P Dlamini, Makhosazana Dlamini, Andy Chapola, Nomsa Kafumba, Chisomo Mzandu Zinyemba, Menard Bvumbwe, Kyakuwa Richard Jjuuko, Jacqueline Balungi Kanywa, Dithan Kiragga, Andreas Boy Isaac, Esther Makhalanyane, Lwamba Nyembo, Retselisitoe Mahlaha, John T Farirai, Eunice W Ketang'enyi, Andrea E M Imsen, Iuliana Costas, Susan B Torrey

Faculty, Staff and Students Publications

Background/objectives: Quality improvement (QI) is widely used in global health to improve patient outcomes, reduce costs, and strengthen service delivery. The Texas Children's Global Health Network (TCGHN) includes nine independent non-governmental organizations supporting healthcare in low- and middle-income countries (LMICs), with pediatric HIV clinical centers of excellence in six countries in sub-Saharan Africa (SSA), supported technically by Baylor College of Medicine. We describe the development of a virtual QI Community of Practice (QICoP) to connect geographically dispersed teams and strengthen local QI capacity.

Methods: In 2022, QI and global health experts convened to design the QICoP and assess site readiness. …

New Applications Of Cardiopulmonary Exercise Testing Parameters Characterize Age-Related Changes In Exercise Performance During Adolescence, Katerina Kourpas, Takeshi Tsuda

Department of Pediatrics Faculty Papers

BACKGROUND: Age-related changes in exercise performance during adolescence are complex because of progressive physical and functional changes, in which multiple factors are involved.

METHODS: Peak and submaximal cardiopulmonary exercise testing (CPET) parameters were studied in healthy children and adolescents.

RESULTS: TTotal 303 subjects were divided into six groups by ages and sex: ≤11 years old (yo) (28/22 for males/females), 12–15 yo (61/98), and ≥16 yo (52/42). Rest and peak heart rate (HR) were comparable in all groups. Absolute peak values of oxygen consumption (˙ VO2), work rate (WR), and oxygen pulse (OP) were higher in older age groups in both …

Hydroxyurea Interferes With Point-Of-Care Creatinine Testing In Children With Sickle Cell Anemia, Ruth Namazzi, Robert Opoka, Charles Ssuuna, Michael Goings, Kathryn Mcelhinney, Dibyadyuti Datta, Russell Ware, Chandy John, Andrea Conroy

Paediatrics and Child Health, East Africa

Objective To evaluate the performance of the point-of-care i-STAT device for creatinine measurement in Ugandan children with sickle cell anemia (SCA), and to determine the clinical impact of hydroxyurea-associated assay interference.

Results description This secondary analysis was nested within a randomized clinical trial involving 248 Ugandan children with SCA. The mean age at enrollment was 32 months, and 115/248 (46.2%) initiated hydroxyurea during 12 months of follow-up as part of routine clinical care. At enrollment, creatinine values measured by i-STAT and reference laboratory testing were clinically comparable. At follow-up, children receiving hydroxyurea had significantly higher creatinine values measured by iSTAT …

Enhancing Lactation Support In The Icu Via Interdisciplinary Collaboration, Lexxi Ellis, Aretha Miller

All Poster Presentations

No abstract provided.

Nurses' Perspectives On Caring For Critically Ill Children During A Measles Outbreak: A Case Report, Nicole Roehrig

All Podium Presentations

No abstract provided.

Corrigendum To Diagnosis Of Post-Hematopoietic Stem Cell Transplant Bronchiolitis Obliterans Syndrome In Children: Time For A Rethink? Transplantation And Cellular Therapy. 2024 Aug; 30(8):760–769, Shivanthan Shanthikumar, William A Gower, Kenneth R Cooke, Anne Bergeron, Kirk R Schultz, Amisha Barochia, Maximiliano Tamae-Kakazu, Edward Charbek, Erin E Reardon, Charlotte Calvo, Alicia Casey, Pi Chun Cheng, Theresa S Cole, Stella M Davies, Shailendra Das, Aliva De, Robin R Deterding, Deborah R Liptzin, Francoise Mechinaud, Jonathan H Rayment, Paul D Robinson, Roopa Siddiah, Anne Stone, Saumini Srinivasin, Christopher T Towe, Gregory A Yanik, Narayan P Iyer, Samuel B Goldfarb

Faculty, Staff and Students Publications

No abstract provided.

Prognostic Value Of Stress Perfusion Cardiac Magnetic Resonance Imaging In Kawasaki Disease With Coronary Artery Aneurysms: A 10-Year Retrospective Cohort Study, Alexander J Kiener, S Kristen Sexson Tejtel, Aimee Liou, Carolyn A Altman, Tam T Doan

Faculty, Staff and Students Publications

Background: Kawasaki disease (KD) with coronary artery aneurysms (CAA) places patients at risk for myocardial ischemia and major cardiac events (MCE). While stress perfusion cardiac magnetic resonance imaging (spCMR) is used for surveillance, its prognostic value has not been established.

Methods: We conducted a retrospective cohort study of KD patients (AHA risk levels 3-5) who underwent clinically indicated vasodilator spCMR at a tertiary pediatric center between October 2014 and December 2024. We evaluated inducible perfusion defects, late gadolinium enhancement (LGE), ventricular function, and MCE (myocardial infarction, ventricular dysfunction, arrhythmia, revascularization, transplant, or death). We performed Cox regression and Kaplan-Meier analyses …

Myocardial T1 And T2 Values Are Associated With Patient Age In Healthy Pediatric Heart Transplant Recipients, Andrew A Lawson, Robyn G Lottes, Defne Magnetta, Andrada Popescu, Kae Watanabe, Cynthia K Rigsby, Michael Markl, Nazia Husain

Faculty, Staff and Students Publications

Background: In pediatric heart transplant recipients (PHTR), myocardial T1 and T2 values are elevated in the setting of acute rejection and with cardiac allograft vasculopathy. In normal, healthy children, T1 and T2 values vary with patient age. Our goal was to identify associations between T1, extracellular volume fraction (ECV), and T2 values and patient- and donor-characteristics in PHTR without history of significant graft pathology.

Methods: We performed a single-center, retrospective chart review of consecutive cardiac magnetic resonance (CMR) studies in PHTR from 2017 to 2023. Exclusion criteria were a prior CMR during the study period, history of any prior antibody-mediated …

How To … Coach Problem Representation To Strengthen Diagnostic Reasoning In Trainees, Cody Clary, Adam Cohen, Satid Thammasitboon

Faculty, Staff and Students Publications

Problem representation (PR) is a concise synthesis of clinical information that captures the core diagnostic problem in a single statement, often referred to as a 'one-liner'. Although central to diagnostic reasoning, this skill is frequently underdeveloped in medical training. Many trainees struggle to transform clinical data into meaningful representations, which can hinder communication, diagnostic reasoning and patient safety. The Assessment of Reasoning Tool-Reconstructed (ART-R) provides a structured framework that educators can adapt as a coaching rubric to strengthen PR during clinical teaching. Using the ART-R, faculty can guide trainees to refine their PR by focusing on three elements: (1) clarity …

Classification Of Gck-Mody In A Child With Mild Hyperglycemia And Islet Autoantibody Positivity: A Diagnostic Challenge, Aman N Yarlagadda, Maria J Redondo, Toni I Pollin, Mustafa Tosur, Colleen O Jodarski, Jennifer J Bell

Faculty, Staff and Students Publications

We report a 9-year-old female who presented with mild, persistent hyperglycemia, with hemoglobin A1c and oral glucose tolerance test results in the prediabetes range, relatively preserved C-peptide, and mildly positive glutamate decarboxylase 65 autoantibody levels. A strong family history of lean, noninsulin-dependent diabetes suggested a possible monogenic cause. Genetic testing with a 28-gene maturity-onset diabetes of the young (MODY) panel identified a heterozygous variant of uncertain significance in GCK (NM_000162.5:c.179C>T [p.Thr60Ile]). Following review by the ClinGen Monogenic Diabetes Variant Curation Expert Panel, the GCK variant was reclassified as pathogenic, confirming a diagnosis of glucokinase (GCK)-MODY/GCK-hyperglycemia, a stable …

Extracorporeal Membrane Oxygenation, Acute Kidney Injury, Fluid Balance, And Continuous Renal Replacement Therapy: Acute Disease Quality Initiative (Adqi) And Extracorporeal Life Support Organization (Elso) Joint Consensus Conference, Katja M Gist, David T Selewski, Kianoush B Kashani, Ayse Akcan-Arikan, Gail Annich, Marta V Antonini, David Askenazi, Linda Awdishu, Brian C Bridges, Michael J Connor, Lui G Forni, Dana Y Fuhrman, Stuart L Goldstein, Stephen M Gorga, Pramod K Guru, Ahmad Kaddourah, Alex Lepage-Farrell, D Michael Mcmullan, Ravindra L Mehta, Shina Menon, Theresa Mottes, Thomas Müeller, Marlies Ostermann, Matthew L Paden, Natalie E Rintoul, Thiago Reis, Claudio Ronco, Emily See, Gurmeet Singh, Sean M Bagshaw, Graeme Maclaren

Faculty, Staff and Students Publications

Extracorporeal membrane oxygenation (ECMO) is increasingly being utilized for life-threatening cardiac and/or respiratory failure refractory to conventional treatment. Acute kidney injury (AKI) and fluid balance disorders commonly occur both before and during ECMO, with approximately half of cases receiving treatment with continuous renal replacement therapy (CRRT). Acute kidney injury, fluid balance disorders, and CRRT influence both short- and long-term outcomes in this population. The 36th Acute Disease Quality Initiative (ADQI) meeting was held in June 2025 to develop multidisciplinary international expert recommendations for AKI, fluid balance, and CRRT during ECMO across the age spectrum. This work encompassed five working groups: …

Plasma Biomarkers Of Brain Injury In Critically Ill Children Receiving Extracorporeal Membrane Oxygenation, Matthew L Friedman, Michael J Bell, Bonnie A Brooks, Adam S Himebauch, Asavari Kamerkar, Kerri Larovere, Laura L Loftis, Jose A Pineda, Ahmed Said, Hitesh S Sandhu, Martin Stengelin, Catherine Demos, Mark S Wainwright, Alina N West, Allan Barnes, Allen D Everett, Ryan J Felling, Sue J Hong, Jennifer L Roem, Cynthia F Salorio, Derek K Ng, Melania M Bembea

Faculty, Staff and Students Publications

Importance: Timely identification of acute brain injury (ABI) in children receiving extracorporeal membrane oxygenation (ECMO) support is critical for early neuroprotective interventions.

Objectives: To determine if elevations in plasma glial fibrillary acidic protein (GFAP), neurofilament light chain (NfL), and tau levels in children receiving ECMO precede new ABI confirmed by neuroimaging, and if they are associated with mortality and functional outcomes.

Design, setting, and participants: This was a prospective observational cohort study conducted from 2019 to 2023, with 18-month follow-up completed in 2025. Children aged 2 days to younger than 18 years at ECMO cannulation were recruited from 11 US …

Polygenic Risk Scores And Hla Class Ii Variants Are Biomarkers Of Corticosteroid Response In Childhood Nephrotic Syndrome., Tiffany Tu, Alejandro Ochoa, Amika Sood, Ashley Dabrik, Megan Chryst-Stangl, Brandon Lane, Guanghong Wu, Frank Donovan, Ursula Harper, Settara Chandrasekharappa, Christopher Esezobor, Adaobi Solarin, David Hooper, Christine Sethna, Sandra Amaral, Mahmoud Kallash, Michelle Rheault, Priya Verghese, Vikas Dharnidharka, Eloise Salmon, Patricia Weng, Tarak Srivastava, Michael E Seifert, Cozumel Pruette, David Selewski, Keisha Gibson, Tracy Hunley, Asiri Abeyagunawardena, Shenal Thalgahagoda, Arvind Bagga, Aditi Sinha, Nicholas Webb, Larry Greenbaum, Ali Gharavi, Krzysztof Kiryluk, Matthias Kretzler, Lisa Guay-Woodford, Simone Sanna-Cherchi, Agnieszka Bierzynska, Ania Koziell, Gavin Welsh, Moin Saleem, Charles Rotimi, Eileen Chambers, Cliburn Chan, Curegn Consortium, Pnrc Glomerular Disease Group, Cibmtr/Nmdp Consortium, Annette Jackson, Adebowale Adeyemo, Rasheed Gbadegesin

Manuscripts, Articles, Book Chapters and Other Papers

INTRODUCTION: Nephrotic syndrome (NS), a common glomerular disease in children, is classified based on response to corticosteroid therapy as either steroid-sensitive nephrotic syndrome (SSNS), or steroid-resistant nephrotic syndrome (SRNS). However, there are no current reliable predictors of therapy response at initial clinical presentation.

METHODS: To evaluate predictors, we conducted genome-wide association studies, developed polygenic risk scores (PRS) for therapy response and analyzed classical HLA alleles in 1,997 children (994 discovery and 1,003 replication/validation cohorts) previously unstudied children with NS and 3,558 ancestry-matched control individuals.

RESULTS: A significant association with HLA loci defined by variants in HLA-DQB1, HLA-DRB1, and HLA-DQA1 were …

Retrospective Chart Review Of Immobilized Lipase Cartridge Use In Children With Short Bowel Syndrome., Elizaveta Khenner, Jennifer Morton, Laura Green, Ashlee Yoder, Kayla Paul, Ann E. Remmers, William San Pablo

Manuscripts, Articles, Book Chapters and Other Papers

This retrospective, single-center chart review studied the use of an in-line, immobilized lipase cartridge (ILC) in children with short bowel syndrome (SBS) at Children's Mercy Hospital. For parenteral nutrition (PN)-dependent patients, the primary endpoint was enteral feeding progression, evaluated by comparing monthly change from baseline in PN and enteral nutrition (EN) after the start of ILC use. For patients with enteral autonomy, changes from baseline in growth measure z-scores were evaluated. In 9 PN-dependent patients (with Type II or III SBS), mean PN use decreased 10.6 kcal/kg/day (15.5%) and mean EN use increased by 6.0 kcal/kg/day (39.9%) after 3.8 to …

Birth-Size Reference Charts For Newborns Admitted To The Neonatal Intensive Care Units., Fu-Sheng Chou, Hung-Wen Yeh, Crystal Hsueh, Irene E. Olsen, Veeral N. Tolia, Reese H. Clark

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Our original birth-size reference charts (the Olsen charts), published in 2010 (weight, length, head circumference) and 2015 (body mass index), had limitations, including manually defined exclusion criteria and the use of completed gestational weeks. This project aimed to update the charts and address those limitations.

METHODS: We analyzed de-identified data from infants admitted to Pediatrix NICUs between 2010 and 2022. In contrast to prior work, newborns with mortality before discharge, multiple births, and congenital anomalies were included; only statistical outliers (defined as two times the interquartile range below the first quartile or above the third quartile) were excluded. Gestational …

A Phase 4, Open-Label, Non-Randomized, Multicenter Study To Evaluate Efficacy (And Safety) Of Intravenous Administration Of Optison For Contrast-Enhanced Echocardiography In Pediatric Patients, Arash Sabati, Shuo Wang, Pushpa Shivaram, Joshua Hawley, David Thompson, Francine Erenberg, Shelby Kutty, Aparna Kulkarni, Tam T Doan, Joshua D Robinson

Faculty, Staff and Students Publications

Contrast-enhanced echocardiography (CE-echo) is commonly used in adult patients to optimize endocardial border delineation (EBD) but there is limited experience in children. A prospective, open-label, non-randomized, multicenter study was performed to evaluate the efficacy of intravenous Optison for CE-echo in pediatric patients and determine the optimal dose of Optison. Subjects ≥ 9 and <  18 years with suboptimal echocardiograms (defined as ≥ 2 contiguous segments that could not be adequately visualized) were enrolled. After a baseline non-contrast echocardiogram (NC-echo), CE-echo was performed with 2 dose levels of Optison based on patient weight. The primary endpoint was visualization of the LV wall segments (LVWS) in apical 2- and 4-chamber views. An EBD score was calculated by assigning a value from 0 (no visualization) to 3 (optimal visualization) for 12 LVWS. 37 subjects across 8 sites completed the study. 30 had complete echocardiograms at both dose levels. 2 patients had usable images at dose 2 only. Mean age was 13.4 years (± 2.6) with 23 males (62%). Mean weight was 73.9 kg (± 36.8) with wide distribution of weight (interquartile range of 42-102 kg). There were 8 patients under 40 kg. Most subjects had a prior medical/surgical history (n = 33, 89%). Both dose levels of Optison CE-echo improved the number of LVWS visualized from 3.7 ± 1.1 on NC-echo to 10.0 ± 0.1 with dose 1 (p <  0.001) and 10.5 ± 0.1 with dose 2 (p <  0.001). EBD score also improved at both dose levels from 11.9 ± 2.5 for NC-echo to 28.5 ± 1.7 for dose 1 (p <  0.001) and 30.2 ± 8.4 for dose 2 (p <  0.001). The number of suboptimal echocardiograms improved from 92.3% ± 10.7 in NC-echo to 26.2% ± 9 with dose 1 (p <  0.001) and 30.8% ± 4.5 with dose 2 (p <  0.001). CE-echo with intravenous Optison in pediatric patients improved the number of LVWS visualized, improved EBD score, and reduced the number of suboptimal echocardiograms at both dosages used in this study. Intravenous Optison can improve LV visualization in pediatric patients with suboptimal echocardiograms.

Practice Patterns In Pediatric Cardiothoracic Presurgical Conferences: A Multicenter Survey Study., Britney Reed, Poonam Puranik, Andrew Rodenbarger, Mira Trivedi, Dilachew A. Adebo, Gary Beasley, Louis Bezold, M Jay Campbell, Michael R. Carr, Joshua Daily, Mark Debrunner, Erica Del Grippo, Carlen G. Fifer, Timothy M. Hoffman, Susan R. Hupp, Joshua D. Kurtz, Gira Morchi, Carl Owada, Renuka Peterson, Michael D. Puchalski, Ryan Romans, Arwa Saidi, Rajesh U. Shenoy, Anoop K. Singh, Robert D. Tunks, Thomas M. Yohannan, Carolyn M. Wilhelm, Jyoti K. Patel

Manuscripts, Articles, Book Chapters and Other Papers

Pediatric cardiothoracic surgeries are high-stakes, complex procedures, typically undergoing prior review at multidisciplinary conferences. This study evaluates practice patterns of conferences throughout the United States (US). Surveys were distributed to fellowship program directors or division directors in 124 US pediatric cardiology centers seeking information on conference logistics, fellow roles, quality improvement (QI), and satisfaction. All 47 responding centers (response rate 38%) conduct presurgical conferences, mostly on a weekly basis (92%) lasting 60-120 min (79%). The conferences are solely virtual (19%) or hybrid (81%). High-volume centers (> 300 surgical cases/year) are more likely to hold multiple conferences (13/20 vs 7/27, p <  0.01) and less likely to designate a moderator (11/20 vs 22/26, p = 0.027). Categorical pediatric cardiology fellows at 33 centers present clinical data (97%), echocardiograms (85%), catheterizations (82%), and cross-sectional imaging (39%), typically beginning in their first year. Most centers report that minor (98%) or major changes (51%) are made to patient management at least "sometimes." Responders rate conferences as very important (median 10/10 on a 10-point Likert scale, IQR 9-10), but satisfaction is more modest (median 7/10, IQR 7-9). Only 17% of centers have a formal QI process. Comments from 42 centers reveal positive themes of collaboration (68%) but also concerns about lengthy (30%) or inefficient (36%) discussion. Conclusions: This survey highlights common practices for pediatric cardiothoracic presurgical conferences. Conferences are collaborative and seen as highly impactful. However, satisfaction varies, and QI efforts are infrequent. These findings highlight opportunities for process improvement and standardization.

Incidental Colorectal Adenomas In Adolescents: Clinical Management, Genetic Evaluation, And Surveillance., Brett J. Hoskins, Shlomi Cohen, Colleen B. Flahive, Isabel Rojas, Aparajita Singh, Thomas M. Attard

Manuscripts, Articles, Book Chapters and Other Papers

The rising incidence of early-onset colorectal cancer has renewed attention to colorectal adenomas in adolescents and young adults (AYA), although these lesions remain rare and poorly characterized in adolescents. Lack of age-specific guidance creates uncertainty regarding genetic evaluation, surveillance intervals, and long-term management when these lesions are identified in adolescents. This narrative review synthesizes current evidence and expert perspective on epidemiology, evaluation, endoscopic management, surveillance, genetic considerations, and psychosocial implications of incidentally identified colorectal adenomas across the AYA spectrum, with emphasis on adolescents as a distinct clinical population. Available data largely derive from adults aged 20-49 years but suggest that …

A 2-Year Randomized Trial Of Pitavastatin Calcium Vs Placebo To Treat Combined Dyslipidemia In Adolescents With Overweight And Obesity., Brian W. Mccrindle, Silva Arslanian, Michele Mietus-Snyder, Elaine M. Urbina, Adam L. Ware, Jessica E. Teng, Felicia Trachtenberg, Mark W. Russell, Amy S. Shah, Mark Cartoski, Julie A. Brothers, Christine San Giovanni, Justin P. Zachariah, Sandra Pena, William T. Mahle, Amy L. Peterson, Sheela N. Magge, Geetha Raghuveer, Binu Sharma, Valiantsina Kazlova, Craig A. Sponseller, D'Andrea Freemon, Mario Stylianou, Sarah D. De Ferranti, Pediatric Heart Network Investigators

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Combined dyslipidemia of overweight/obesity (CDO) is prevalent in youth and is associated with an increased risk of early cardiovascular disease.

OBJECTIVE: We sought to determine the impact and safety of statin therapy for CDO in adolescents.

METHODS: A double-blind, randomized trial was performed across 18 North American sites. Participants aged 10 to 19 years with body mass index ≥85th percentile and CDO defined as non-high-density lipoprotein cholesterol (non-HDL-C) ≥120 mg/dL (3.10 mmol/L) and either low HDL-C or high triglyceride:HDL-C ratio were randomized centrally to receive either pitavastatin calcium 4 mg/d or placebo for 2 years. The primary outcome was …

Early Neurodevelopmental Outcomes In Children With Congenital Heart Disease., Mike Seed, Dawn Ilardi, Valerie Rofeberg, Cynthia Ortinau, Caren Goldberg, Garrett Reichle, Lauren Bush, Justin Elhoff, Amy Jo Lisanti, Jennifer Butcher, Caitlin Rollins, Andrew Van Bergen, Shabnam Peyvandi, Emily Bucholz, Stephanie Cox, Lyla Hampton, Jacqueline Sanz, Sonia Monteiro, Shruti Tewar, Kiona Allen, Caroline Lee, Kristi Glotzbach, Nneka Alexander, Laurel Bear, Corinne Anton, Renee Sananes, Linh Ly, Gina Boucher, Kelly Wolfe, Lindsay Edwards, Elizabeth J. Willen, Alexander Tan, Christina Ortega, Erica Sood, Anjali Sadhwani, Kari Crawford Plant, Lauren Quigley, Jessica Pliego, Elizabeth Valles, Abbey Hines, David Wypij, Thomas Miller

Manuscripts, Articles, Book Chapters and Other Papers

IMPORTANCE: Neurodevelopmental impairments are common in children with congenital heart disease.

OBJECTIVES: To compare neurodevelopmental outcomes by cardiac diagnosis and identify associations between medical and social factors and early neurodevelopmental outcomes among children with congenital heart disease.

DESIGN, SETTING, AND PARTICIPANTS: This cross-sectional analysis used neurodevelopmental follow-up data obtained between May 1, 2019, and June 30, 2022, and entered into the Cardiac Neurodevelopmental Outcome Collaborative (CNOC) registry, with linkage to the Pediatric Cardiac Critical Care Consortium registry. Children younger than 30 months from 25 CNOC sites were studied. Data analysis was performed between January 2023 and December 2024.

EXPOSURES: Patients …

Malignant Transformation Of Sacrococcygeal Teratoma Versus Presacral Teratoma In Currarino Syndrome: Results Of 'The Sct-Study'., Lieke J. Van Heurn, Joep P M Derikx, Nigel J. Hall, Amr Abdelhamid Abouzeid, Simone De Campos Vieira Abib, Lohfa B. Chirdan, Shigehisa Fumino, Seong C. Kim, Oliver Muensterer, Maricarmen Olivos, Shawn D. St Peter, Jos Twisk, Nicolas Vinit, Tianyou Yang, Augusto Zani, L W Ernst Van Heurn, Sct-Study Consortium

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: The risk of malignant transformation and recurrence of Sacrococcygeal Teratoma (SCT) is relatively high, while it is possibly lower in cases associated with Currarino Syndrome (CS). However, the existing literature gives contradictory results. We aimed to examine the risk of malignant transformation in a large cohort of SCT and CS patients.

METHODS: In a global retrospective cohort study, data of consecutive SCT patients and CS patients with presacral teratoma was obtained from 132 institutes in 62 countries. Malignant transformation, defined as malignancy at initial resection, malignant recurrence, or death due to malignancy was analysed for SCT and CS patients. …

Prevalence And Aetiology Of Cerebral Palsy Among Nigerian Children: A Systematic Review And Meta-Analysis., Udochukwu Michael Diala, Aderonke O. Uhunmwangho-Courage, Fatima Abdullahi, Paul Ikhurionan, Caitlin Bakker, Duke Appiah, David Danjuma Shwe, Rose N. Gelineau-Morel, Kabiru Gurama, Olugbenga Akinyemi Ofakunrin, Angela Mcgillivray, Gabriel E. Ofovwe, Tina Slusher

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Nigeria is among the leading countries contributing to the burden of cerebral palsy. Cerebral palsy (CP) is the commonest physical disability in childhood, accounting for 16.2% of child neurology referrals. Many cases of CP are preventable with simple interventions such as neonatal resuscitation and effective phototherapy. Lack of reliable data on the burden of disease limits comprehensive national policy directed to the prevention, holistic care and rehabilitation of patients with cerebral palsy in Nigeria. Therefore, to effectively address this problem, this study aimed to describe the pooled prevalence of CP in Nigeria, its potential causes and types.

METHODS: This …

Infection Prevention And Control Of Candida Auris In Pediatric Settings, Thomas S. Murray, Hana Hakim, Christelle Ilboudo, Lynn Ramírez-Ávila, Jana Shaw, Terri Stillwell, Patricia Budo, Bernard Ebruke, Noa Fleiss, Amanda Green, Matthew Linam, Roshni Mathew, April Mcdougal, Natalie Neu, Zoi Dorothea Pana, Karen Ravin, Ayelet Rosenthal, Jane D. Siegel, Katlyn Steimel, Amy Valencia, Carol Vance, Lars F. Westblade, Lisa Saiman

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Candida auris (also referred to as Candidozyma auris) is an emerging multidrug-resistant fungal pathogen associated with high morbidity and mortality. Existing infection prevention and control (IPC) guidance has largely focused on adult populations, with limited recommendations for pediatric healthcare and non-healthcare settings.

METHODS: The Society for Healthcare Epidemiology of America (SHEA) convened a multidisciplinary expert panel to develop IPC recommendations for C. auris. The panel developed recommendations using a structured, iterative Delphi consensus process with rounds of discussion, refinement, and anonymous electronic voting with predefined consensus thresholds. Panelists reviewed relevant peer-reviewed and gray literature integrated with expert …

Parent Perspectives On Neonatal Research Without Prior Consent: A Mixed-Methods Interview Study., Stephanie A. Kraft, Abigail Brickler, Abril Beretta, Rishabh Jain, Andrea Kelsh, Kaashif A. Ahmad, Justin T. Clapp, Neal W. Dickert, Rebecca A. Dorner, Katherine F. Guttmann, Anup C. Katheria, Stephanie Kukora, Benjamin A. Mooso, Elliott Mark Weiss, Fam-Connect Collaborative

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Recruitment for clinical research in the neonatal intensive care unit (NICU) is difficult, which may lead to low enrollment rates, systematically skewed samples, and nongeneralizable results. Research without prior consent (RWPC), defined as obtaining consent only after infants have been randomized to a treatment arm, may improve enrollment but raises ethical challenges. There is a need to better understand how parents with lived experience of having an infant in the NICU consider RWPC’s impact on critical ethical values related to informed consent, including decision-making, respect, and trust. This mixed-methods study aimed to describe parental perceptions of the acceptability and …

Optimizing Lactation Support: Enhancing Patient-Centered Care And Breastfeeding Self Efficacy Through Data-Driven Survey Analysis, Jessica L. Doshay

Doctor of Nursing Practice Final Manuscripts

Objective: The objective of this project was to implement evaluation of an integrated lactation support model within a multi‑site pediatric primary care organization to assess patient experience and breastfeeding confidence associated with a lactation consultation.

Background: Optimizing the patient experience is an indicator of healthcare quality. Improving breastfeeding outcomes remains a national priority. Structured lactation support delivered by International Board-Certified Lactation Consultants (IBCLCs) enhances breastfeeding outcomes, the impact of this is often undocumented in primary care settings.

Methods: This quality improvement project was conducted within the lactation department of a multicenter pediatric clinic in Southern California. Pediatric clinicians who were …

Coxfa4l2 Upregulation Preserves Residual Cytochrome C Oxidase Activity In Coxfa4-Related Leigh-Like Encephalopathy, Micol Falabella, Sandra Lopez Calcerrada, Jana Aref, Jiaze Gao, William L Macken, Chiara Pizzamiglio, Renata Kabiljo, Anna Lucia Francavilla, Pauline Gaignard, Antoine Pouzet, Jonathan Levy, Giulia Barcia, Jamie K Leighton, Efstathia Chronopoulou, Germaine Pierre, Riza Köksal Özgül, Ali Dursun, Rebecca Halligan, Helen Mundy, Javeria Raza Alvi, Tipu Sultan, William James Craigen, Lisa Emrick, Jill A Rosenfeld, Gehad Elmakkawy, Jihye Kim, Joseph J Gleeson, Aboulfazl Rad, Gabriela Oprea, Maqbool Hussain, Khalil Ur Rehman, Sadia Riaz, Robert W Taylor, Vincent Procaccio, Maha S Zaki, Erika Fernandez-Vizarra, Ciro Leonardo Pierri, Michael G Hanna, Henry Houlden, Reza Maroofian, Cristina Ugalde, Jan-Willem Taanman, Robert D S Pitceathly

Faculty, Staff and Students Publications

Primary mitochondrial diseases (PMDs) affect approximately 1 in 4300 individuals and cause early-onset neuromuscular and multisystem dysfunction with reduced lifespan. They result from pathogenic variants in mitochondrial or nuclear DNA that impair oxidative phosphorylation. Cytochrome c oxidase (COX; complex IV) deficiency is a well-established cause of PMD, leading to a broad spectrum of phenotypes. COXFA4 (cytochrome c oxidase subunit FA4), formerly NDUFA4, is a nuclear-encoded COX subunit, but its role in disease remains poorly defined. We report the largest genetically confirmed cohort of COXFA4-related PMD to date, comprising 13 individuals from 12 families with biallelic pathogenic COXFA4 variants. All present …

Can Behavioral Interventions Promote Health Equity In Pediatric Type 1 Diabetes? A Narrative Review Of Promising Treatments, Paul T. Enlow, Jaquelin Flores Garcia, Julia Price, Kristen A. Torres, David V. Wagner

Department of Pediatrics Faculty Papers

Purpose of review: Synthesize data on whether existing behavioral interventions can improve health equity among youth with type 1 diabetes.

Recent findings: While existing behavioral interventions demonstrated efficacy in improving health and/or psychosocial outcomes, evidence that these same interventions may improve health equity were lacking. Most interventions were evaluated using predominantly White and affluent samples and some studies did not report on racial, ethnic, or sociodemographic characteristics of their sample. Only a few interventions have been adapted for youth from minoritized backgrounds. Recent multisystemic and technology/mHealth interventions recruited samples that were sociodemographically representative of youth experiencing disparities, suggesting that these …