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Full-Text Articles in Medical Specialties

Influence Of Novel Cyp2c-Haplotype On Proton Pump Inhibitor Pharmacokinetics In Children., Kathyrn Kyler, Andrea Gaedigk, Susan Abdel-Rahman, Vincent S. Staggs, Robin E. Pearce, Paul C. Toren, J Steven Leeder, Valentina Shakhnovich Apr 2024

Influence Of Novel Cyp2c-Haplotype On Proton Pump Inhibitor Pharmacokinetics In Children., Kathyrn Kyler, Andrea Gaedigk, Susan Abdel-Rahman, Vincent S. Staggs, Robin E. Pearce, Paul C. Toren, J Steven Leeder, Valentina Shakhnovich

Manuscripts, Articles, Book Chapters and Other Papers

In this brief report, we provide an analysis of the influence of a novel CYP2C haplotype (CYP2C:TG) on proton pump inhibitor (PPI) pharmacokinetics (PK) in children. The CYP2C:TG haplotype has been proposed to be associated with increased CYP2C19 activity. We sought to determine if this CYP2C:TG haplotype resulted in similar alterations in metabolism for proton pump inhibitors, which are primarily metabolized by CYP2C19. In a cohort of 41 children aged 6-21 participating in a PPI pharmacokinetic study, effects of the CYP2C:TG allele were assessed by fitting two linear regression models for each of the six PK outcomes assessed, the second …


Investigating The Use Of Finerenone In Children With Chronic Kidney Disease And Proteinuria: Design Of The Fiona And Open-Label Extension Studies., Franz Schaefer, Giovanni Montini, Hee Gyung Kang, Johan Vande Walle, Joshua Zaritsky, Michiel F. Schreuder, Mieczyslaw Litwin, Andrea Scalise, Helen Scott, James Potts, Pablo Iveli, Stefanie Breitenstein, Bradley A. Warady Mar 2024

Investigating The Use Of Finerenone In Children With Chronic Kidney Disease And Proteinuria: Design Of The Fiona And Open-Label Extension Studies., Franz Schaefer, Giovanni Montini, Hee Gyung Kang, Johan Vande Walle, Joshua Zaritsky, Michiel F. Schreuder, Mieczyslaw Litwin, Andrea Scalise, Helen Scott, James Potts, Pablo Iveli, Stefanie Breitenstein, Bradley A. Warady

Manuscripts, Articles, Book Chapters and Other Papers

INTRODUCTION: Proteinuria is a modifiable risk factor for chronic kidney disease (CKD) progression in children. Finerenone, a selective, non-steroidal, mineralocorticoid receptor antagonist (MRA) has been approved to treat adults with CKD associated with type 2 diabetes mellitus (T2DM) following results from the phase III clinical trials FIDELIO-DKD (NCT02540993) and FIGARO-DKD (NCT02545049). In a pre-specified pooled analysis of both studies (N = 13,026), finerenone was shown to have an acceptable safety profile and was efficacious in decreasing the risk of adverse kidney and cardiovascular outcomes and of proteinuria.

OBJECTIVE: FIONA and the associated open-label extension (OLE) study aim to demonstrate that …


Pediatric Beta Blocker Therapy: A Comprehensive Review Of Development And Genetic Variation To Guide Precision-Based Therapy In Children, Adolescents, And Young Adults., Mollie Walton, Jonathan B. Wagner Mar 2024

Pediatric Beta Blocker Therapy: A Comprehensive Review Of Development And Genetic Variation To Guide Precision-Based Therapy In Children, Adolescents, And Young Adults., Mollie Walton, Jonathan B. Wagner

Manuscripts, Articles, Book Chapters and Other Papers

Beta adrenergic receptor antagonists, known as beta blockers, are one of the most prescribed medications in both pediatric and adult cardiology. Unfortunately, most of these agents utilized in the pediatric clinical setting are prescribed off-label. Despite regulatory efforts aimed at increasing pediatric drug labeling, a majority of pediatric cardiovascular drug agents continue to lack pediatric-specific data to inform precision dosing for children, adolescents, and young adults. Adding to this complexity is the contribution of development (ontogeny) and genetic variation towards the variability in drug disposition and response. In the absence of current prospective trials, the purpose of this comprehensive review …


Infant Appendicitis: A Case Presentation Of Appendicitis In A Nine-Month-Old Infant With Respiratory Syncytial Virus (Rsv) And Otitis Media And Review Of Literature., Leslie Hueschen, April Mcneill-Johnson Mar 2024

Infant Appendicitis: A Case Presentation Of Appendicitis In A Nine-Month-Old Infant With Respiratory Syncytial Virus (Rsv) And Otitis Media And Review Of Literature., Leslie Hueschen, April Mcneill-Johnson

Manuscripts, Articles, Book Chapters and Other Papers

Appendicitis in children < 5 years old is uncommon and even less common in children < 1 year old. Symptoms of appendicitis can be non-specific and mimic other common pediatric diseases, causing delays in diagnosis. Without a timely diagnosis, young children with appendicitis are at risk of developing worsening disease, intra-abdominal abscess, perforation, and bacteremia. We submit a case of a nine-month-old male infant presenting with fever, vomiting, and irritability seen the day prior and treated for otitis media, who was ultimately diagnosed with appendicitis with concomitant viral infection (respiratory syncytial virus and human rhinovirus/enterovirus) and treated with intravenous antibiotics and laparoscopic appendectomy. This case illustrates how easy it is to misdiagnose infant appendicitis due to factors including normal developmental speech barriers, nonspecific presentations, and overlap of symptoms with many other common childhood illnesses, along with a challenging abdominal examination. Delay in diagnosis leads to increased rates of perforation and complications. Providers should trust abnormal physical examination findings, especially abdominal guarding against palpation, and keep a wide differential diagnosis in order to diagnose appendicitis in young children.


Real-World Evidence On The Dosing And Safety Of C.E.R.A. In Pediatric Dialysis Patients: Findings From The International Pediatric Dialysis Network Registries., Laura Kohlhas, Milena Studer, Loes Rutten-Jacobs, Sylvie Meyer Reigner, Anja Sander, Hui-Kim Yap, Karel Vondrak, Paula A. Coccia, Francisco Cano, Claus Peter Schmitt, Bradley A. Warady, Franz Schaefer, Ipdn Collaborators Mar 2024

Real-World Evidence On The Dosing And Safety Of C.E.R.A. In Pediatric Dialysis Patients: Findings From The International Pediatric Dialysis Network Registries., Laura Kohlhas, Milena Studer, Loes Rutten-Jacobs, Sylvie Meyer Reigner, Anja Sander, Hui-Kim Yap, Karel Vondrak, Paula A. Coccia, Francisco Cano, Claus Peter Schmitt, Bradley A. Warady, Franz Schaefer, Ipdn Collaborators

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: This retrospective real-world study used data from two registries, International Pediatric Peritoneal Dialysis Network (IPPN) and International Pediatric Hemodialysis Network (IPHN), to characterize the efficacy and safety of continuous erythropoietin receptor activator (C.E.R.A.) in pediatric patients with chronic kidney disease (CKD) on peritoneal dialysis (PD) or hemodialysis (HD).

METHODS: IPPN and IPHN collect prospective data (baseline and every 6 months) from pediatric PD and HD centers worldwide. Demographics, clinical characteristics, dialysis information, treatment, laboratory parameters, number and causes of hospitalization events, and deaths were extracted for patients on C.E.R.A. treatment (IPPN: 2007-2021; IPHN: 2013-2021).

RESULTS: We analyzed 177 patients …


Associations Between Anemia And Fgf23 In The Ckid Study., Elizabeth Thomas, Alexandra M. Klomhaus, Marciana L. Laster, Susan L. Furth, Bradley A. Warady, Isidro B. Salusky, Mark R. Hanudel Mar 2024

Associations Between Anemia And Fgf23 In The Ckid Study., Elizabeth Thomas, Alexandra M. Klomhaus, Marciana L. Laster, Susan L. Furth, Bradley A. Warady, Isidro B. Salusky, Mark R. Hanudel

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Fibroblast growth factor 23 (FGF23) is a bone-derived hormone that plays a central role in chronic kidney disease-mineral bone disorder and is associated with CKD progression and cardiovascular morbidity. Factors related to CKD-associated anemia, including iron deficiency, can increase FGF23 production. This study aimed to assess whether anemia and/or iron deficiency are associated with increased circulating concentrations of FGF23 in the large, well-characterized Chronic Kidney Disease in Children (CKiD) study cohort.

METHODS: Hemoglobin concentrations, iron parameters, C-terminal (total) FGF23, intact FGF23, and relevant covariables were measured in cross-sectional analysis of CKiD study subjects.

RESULTS: In 493 pediatric patients with …


Remote Delivery Of Allied And Behavioral Healthcare During Covid-19 For Children With Developmental Disabilities., Lisa D. Wiggins, Lin H. Tian, Sarah C. Tinker, Marshalyn Yeargin-Allsopp, Carolyn G. Diguiseppi, Cy Nadler, Patrick S. Powell, Eric J. Moody, Maureen S. Durkin, M Danielle Fallin, A Blythe Ryerson, Joann M. Thierry, Britney Robinson, Karen Pazol Mar 2024

Remote Delivery Of Allied And Behavioral Healthcare During Covid-19 For Children With Developmental Disabilities., Lisa D. Wiggins, Lin H. Tian, Sarah C. Tinker, Marshalyn Yeargin-Allsopp, Carolyn G. Diguiseppi, Cy Nadler, Patrick S. Powell, Eric J. Moody, Maureen S. Durkin, M Danielle Fallin, A Blythe Ryerson, Joann M. Thierry, Britney Robinson, Karen Pazol

Manuscripts, Articles, Book Chapters and Other Papers

OBJECTIVE: Many children with autism spectrum disorder (ASD) and other developmental disabilities (DD) transitioned to telehealth services due to the COVID-19 pandemic. Our objectives were to describe reductions in allied and behavioral healthcare services and receipt of caregiver training to deliver services at home because of COVID-19 for children with ASD and other DD, and factors associated with worse response to remote delivery of services for children with ASD.

METHOD: Prior to the pandemic, children 2 to 5 years of age were enrolled in a multi-site case-control study and completed a developmental assessment. Caregivers completed questionnaires on child behavior problems …


The Association Between Diet Quality And Glycemic Outcomes Among People With Type 1 Diabetes., Melanie B. Gillingham, Martin Chase Marak, Michael C. Riddell, Peter Calhoun, Robin L. Gal, Susana R. Patton, Peter G. Jacobs, Jessica R. Castle, Mark A. Clements, Francis J. Doyle, Michael R. Rickels, Corby K. Martin Mar 2024

The Association Between Diet Quality And Glycemic Outcomes Among People With Type 1 Diabetes., Melanie B. Gillingham, Martin Chase Marak, Michael C. Riddell, Peter Calhoun, Robin L. Gal, Susana R. Patton, Peter G. Jacobs, Jessica R. Castle, Mark A. Clements, Francis J. Doyle, Michael R. Rickels, Corby K. Martin

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: The amount and type of food consumed impacts the glycemic response and insulin needs of people with type 1 diabetes mellitus (T1DM). Daily variability in consumption, reflected in diet quality, may acutely impact glycemic levels and insulin needs.

OBJECTIVE: Type 1 Diabetes Exercise Initiative (T1DEXI) data were examined to evaluate the impact of daily diet quality on near-term glycemic control and interaction with exercise.

METHODS: Using the Remote Food Photography Method, ≤8 d of dietary intake data were analyzed per participant. Diet quality was quantified with the Healthy Eating Index-2015 (HEI), where a score of 100 indicates the highest-quality …


Racial And Ethnic Disparities In The Co-Occurrence Of Intellectual Disability And Autism: Impact Of Incorporating Measures Of Adaptive Functioning., Sarah M. Furnier, Ronald Gangnon, Julie L. Daniels, Susan Ellis Weismer, Cy Nadler, Karen Pazol, Nuri M. Reyes, Steven Rosenberg, Eric Rubenstein, Lisa D. Wiggins, Marshalyn Yeargin-Allsopp, Maureen S. Durkin Mar 2024

Racial And Ethnic Disparities In The Co-Occurrence Of Intellectual Disability And Autism: Impact Of Incorporating Measures Of Adaptive Functioning., Sarah M. Furnier, Ronald Gangnon, Julie L. Daniels, Susan Ellis Weismer, Cy Nadler, Karen Pazol, Nuri M. Reyes, Steven Rosenberg, Eric Rubenstein, Lisa D. Wiggins, Marshalyn Yeargin-Allsopp, Maureen S. Durkin

Manuscripts, Articles, Book Chapters and Other Papers

Intellectual disability (ID) commonly co-occurs in children with autism. Although diagnostic criteria for ID require impairments in both cognitive and adaptive functioning, most population-based estimates of the frequency of co-occurring ID in children with autism-including studies of racial and ethnic disparities in co-occurring autism and ID-base the definition of ID solely on cognitive scores. The goal of this analysis was to examine the effect of including both cognitive and adaptive behavior criteria on estimates of co-occurring ID in a well-characterized sample of 2- to 5-year-old children with autism. Participants included 3264 children with research or community diagnoses of autism enrolled …


Special Considerations For The Adolescent With Obesity: An Obesity Medicine Association (Oma) Clinical Practice Statement (Cps) 2024., Suzanne Cuda, Valerie O'Hara, Marisa Censani, Rushika Conroy, Brooke Sweeney, Jennifer Paisley, Cristina Fernandez Md, Meredith Dreyer, Allen Browne, Nancy T. Browne Mar 2024

Special Considerations For The Adolescent With Obesity: An Obesity Medicine Association (Oma) Clinical Practice Statement (Cps) 2024., Suzanne Cuda, Valerie O'Hara, Marisa Censani, Rushika Conroy, Brooke Sweeney, Jennifer Paisley, Cristina Fernandez Md, Meredith Dreyer, Allen Browne, Nancy T. Browne

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: This Obesity Medicine Association (OMA) Clinical Practice Statement (CPS) details special considerations for the management of the adolescent with obesity. The information in this CPS is based on scientific evidence, supported by medical literature, and derived from the clinical experiences of members of the OMA.

METHODS: The scientific information and clinical guidance in this CPS are based on scientific evidence, supported by the medical literature, and derived from the clinical perspectives of the authors.

RESULTS: This OMA Clinical Practice Statement addresses special considerations in the management and treatment of adolescents with overweight and obesity.

CONCLUSIONS: This OMA Clinical Practice …


Epigenome-Wide Association Study Identifies Neonatal Dna Methylation Associated With Two-Year Attention Problems In Children Born Very Preterm., Marie Camerota, Barry M. Lester, Francisco Xavier Castellanos, Brian S. Carter, Jennifer Check, Jennifer Helderman, Julie A. Hofheimer, Elisabeth C. Mcgowan, Charles R. Neal, Steven L. Pastyrnak, Lynne M. Smith, Thomas Michael O'Shea, Carmen J. Marsit, Todd M. Everson Feb 2024

Epigenome-Wide Association Study Identifies Neonatal Dna Methylation Associated With Two-Year Attention Problems In Children Born Very Preterm., Marie Camerota, Barry M. Lester, Francisco Xavier Castellanos, Brian S. Carter, Jennifer Check, Jennifer Helderman, Julie A. Hofheimer, Elisabeth C. Mcgowan, Charles R. Neal, Steven L. Pastyrnak, Lynne M. Smith, Thomas Michael O'Shea, Carmen J. Marsit, Todd M. Everson

Manuscripts, Articles, Book Chapters and Other Papers

Prior research has identified epigenetic predictors of attention problems in school-aged children but has not yet investigated these in young children, or children at elevated risk of attention problems due to preterm birth. The current study evaluated epigenome-wide associations between neonatal DNA methylation and attention problems at age 2 years in children born very preterm. Participants included 441 children from the Neonatal Neurobehavior and Outcomes in Very Preterm Infants (NOVI) Study, a multi-site study of infants born < 30 weeks gestational age. DNA methylation was measured from buccal swabs collected at NICU discharge using the Illumina MethylationEPIC Bead Array. Attention problems were assessed at 2 years of adjusted age using the attention problems subscale of the Child Behavior Checklist (CBCL). After adjustment for multiple testing, DNA methylation at 33 CpG sites was associated with child attention problems. Differentially methylated CpG sites were located in genes previously linked to physical and mental health, including several genes associated with ADHD in prior epigenome-wide and genome-wide association studies. Several CpG sites were located in genes previously linked to exposure to prenatal risk factors in the NOVI sample. Neonatal epigenetics measured at NICU discharge could be useful in identifying preterm children at risk for long-term attention problems and related psychiatric disorders, who could benefit from early prevention and intervention efforts.


Notes From The Field: Reemergence Of Mycoplasma Pneumoniae Infections In Children And Adolescents After The Covid-19 Pandemic, United States, 2018-2024., Chris Edens, Benjamin R. Clopper, Jourdan Devies, Alvaro Benitez, Erin R. Mckeever, Dylan Johns, Bernard Wolff, Rangaraj Selvarangan, Jennifer E. Schuster, Geoffrey A. Weinberg, Peter G. Szilagyi, Fatimah S. Dawood, Lakshmi Radhakrishnan, Christina Quigley, Leila C. Sahni, Natasha Halasa, Laura S. Stewart, Meredith L. Mcmorrow, Brett Whitaker, Danielle M. Zerr, Vasanthi Avadhanula, John V. Williams, Marian G. Michaels, Aaron Kite-Powell, Janet A. Englund, Mary Allen Staat, Kathleen Hartnett, Heidi L. Moline, Adam L. Cohen, Maureen Diaz Feb 2024

Notes From The Field: Reemergence Of Mycoplasma Pneumoniae Infections In Children And Adolescents After The Covid-19 Pandemic, United States, 2018-2024., Chris Edens, Benjamin R. Clopper, Jourdan Devies, Alvaro Benitez, Erin R. Mckeever, Dylan Johns, Bernard Wolff, Rangaraj Selvarangan, Jennifer E. Schuster, Geoffrey A. Weinberg, Peter G. Szilagyi, Fatimah S. Dawood, Lakshmi Radhakrishnan, Christina Quigley, Leila C. Sahni, Natasha Halasa, Laura S. Stewart, Meredith L. Mcmorrow, Brett Whitaker, Danielle M. Zerr, Vasanthi Avadhanula, John V. Williams, Marian G. Michaels, Aaron Kite-Powell, Janet A. Englund, Mary Allen Staat, Kathleen Hartnett, Heidi L. Moline, Adam L. Cohen, Maureen Diaz

Manuscripts, Articles, Book Chapters and Other Papers

No abstract provided.


Degree Of Uncertainty In Reporting Imaging Findings For Necrotizing Enterocolitis: A Secondary Analysis From A Pilot Randomized Diagnostic Trial., Alain Cuna, Disa Rathore, Kira Bourret, Erin K. Opfer, Sherwin S. Chan Feb 2024

Degree Of Uncertainty In Reporting Imaging Findings For Necrotizing Enterocolitis: A Secondary Analysis From A Pilot Randomized Diagnostic Trial., Alain Cuna, Disa Rathore, Kira Bourret, Erin K. Opfer, Sherwin S. Chan

Manuscripts, Articles, Book Chapters and Other Papers

Diagnosis of necrotizing enterocolitis (NEC) relies heavily on imaging, but uncertainty in the language used in imaging reports can result in ambiguity, miscommunication, and potential diagnostic errors. To determine the degree of uncertainty in reporting imaging findings for NEC, we conducted a secondary analysis of the data from a previously completed pilot diagnostic randomized controlled trial (2019-2020). The study population comprised sixteen preterm infants with suspected NEC randomized to abdominal radiographs (AXRs) or AXR + bowel ultrasound (BUS). The level of uncertainty was determined using a four-point Likert scale. Overall, we reviewed radiology reports of 113 AXR and 24 BUS …


Economic Impacts Of The Covid-19 Pandemic On Families Of Children With Autism And Other Developmental Disabilities., Olivia M. Pokoski, Hayley Crain, Carolyn Diguiseppi, Sarah M. Furnier, Eric J. Moody, Cy Nadler, Karen Pazol, Jessica Sanders, Lisa D. Wiggins, Maureen S. Durkin Feb 2024

Economic Impacts Of The Covid-19 Pandemic On Families Of Children With Autism And Other Developmental Disabilities., Olivia M. Pokoski, Hayley Crain, Carolyn Diguiseppi, Sarah M. Furnier, Eric J. Moody, Cy Nadler, Karen Pazol, Jessica Sanders, Lisa D. Wiggins, Maureen S. Durkin

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: To control the spread of the coronavirus disease (COVID-19), many jurisdictions throughout the world enacted public health measures that had vast socio-economic implications. In emergency situations, families of children with developmental disabilities (DDs), including autism, may experience increased difficulty accessing therapies, economic hardship, and caregiver stress, with the potential to exacerbate autism symptoms. Yet, limited research exists on the economic impacts of the COVID-19 pandemic on families of children with autism or another DD compared to families of children from the general population.

OBJECTIVES: To assess impact of the COVID-19 pandemic related to parental employment and economic difficulties in …


Pediatric Spinal Ependymoma With Chromothripsis Of Chromosome 6: A Case Report And Review Of The Literature., Keela R. Scott, Melissa Gener, Elena Repnikova Feb 2024

Pediatric Spinal Ependymoma With Chromothripsis Of Chromosome 6: A Case Report And Review Of The Literature., Keela R. Scott, Melissa Gener, Elena Repnikova

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Ependymomas are the third most common central nervous system tumor in the pediatric population; however, spinal ependymomas in children are rare. Ependymomas affecting the spinal cord most frequently occur in adults of 20-40 years of age. The current World Health Organization classification system for ependymomas is now composed of ten different entities based on histopathology, location, and molecular studies, with evidence that the new classification system more accurately predicts clinical outcomes.

CASE PRESENTATION: We present the case of a 16-year-old Caucasian female patient with a history of type 2 neurofibromatosis with multiple schwannomas, meningioma, and spinal ependymoma. Chromosome analysis …


Molecular-Guided Therapy For The Treatment Of Patients With Relapsed And Refractory Childhood Cancers: A Beat Childhood Cancer Research Consortium Trial., Giselle L Saulnier Sholler, Genevieve Bergendahl, Elizabeth C. Lewis, Jacqueline Kraveka, William Ferguson, Abhinav B. Nagulapally, Karl Dykema, Valerie I Brown, Michael S. Isakoff, Joseph Junewick, Deanna Mitchell, Jawhar Rawwas, William Roberts, Don Eslin, Javier Oesterheld, Randal K. Wada, Devang Pastakia, Virginia Harrod, Kevin Ginn, Raya Saab, Kevin Bielamowicz, Jason Glover, Eugenia Chang, Gina K. Hanna, Daniel Enriquez, Tyler Izatt, Rebecca F Halperin, Abigail Moore, Sara A. Byron, William P D Hendricks, Jeffrey M. Trent Feb 2024

Molecular-Guided Therapy For The Treatment Of Patients With Relapsed And Refractory Childhood Cancers: A Beat Childhood Cancer Research Consortium Trial., Giselle L Saulnier Sholler, Genevieve Bergendahl, Elizabeth C. Lewis, Jacqueline Kraveka, William Ferguson, Abhinav B. Nagulapally, Karl Dykema, Valerie I Brown, Michael S. Isakoff, Joseph Junewick, Deanna Mitchell, Jawhar Rawwas, William Roberts, Don Eslin, Javier Oesterheld, Randal K. Wada, Devang Pastakia, Virginia Harrod, Kevin Ginn, Raya Saab, Kevin Bielamowicz, Jason Glover, Eugenia Chang, Gina K. Hanna, Daniel Enriquez, Tyler Izatt, Rebecca F Halperin, Abigail Moore, Sara A. Byron, William P D Hendricks, Jeffrey M. Trent

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Children with relapsed central nervous system (CNS tumors), neuroblastoma, sarcomas, and other rare solid tumors face poor outcomes. This prospective clinical trial examined the feasibility of combining genomic and transcriptomic profiling of tumor samples with a molecular tumor board (MTB) approach to make real‑time treatment decisions for children with relapsed/refractory solid tumors.

METHODS: Subjects were divided into three strata: stratum 1-relapsed/refractory neuroblastoma; stratum 2-relapsed/refractory CNS tumors; and stratum 3-relapsed/refractory rare solid tumors. Tumor samples were sent for tumor/normal whole-exome (WES) and tumor whole-transcriptome (WTS) sequencing, and the genomic data were used in a multi-institutional MTB to make real‑time treatment …


Relationship Between Obesity And Global Longitudinal Strain In The Pediatric Single Ventricle Fontan Population Across Ventricular Morphologies., Nitin Madan, Doaa Aly, Melanie Kathol, Amulya Buddhavarapu, Thomas Rieth, Ashley K. Sherman, Daniel Forsha Feb 2024

Relationship Between Obesity And Global Longitudinal Strain In The Pediatric Single Ventricle Fontan Population Across Ventricular Morphologies., Nitin Madan, Doaa Aly, Melanie Kathol, Amulya Buddhavarapu, Thomas Rieth, Ashley K. Sherman, Daniel Forsha

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Obesity is associated with diminished myocardial function as measured by strain echocardiography in children and young adults with normal cardiac anatomy. Data are lacking about the effect of obesity on myocardial strain in patients with a single ventricle. In this study, the relationship between body mass index (BMI) and single ventricle myocardial strain in the Fontan population was assessed.

METHODS AND RESULTS: Thirty-eight abnormal BMI Fontan cases (21 overweight and 17 obese) and 30 normal BMI Fontan controls matched based on single ventricular morphology, age, and sex were included in the study. Ventricular morphology was categorized as single right …


Opportunities For Pharmacogenetic Testing To Guide Dosing Of Medications In Youths With Medicaid., Sonya Tang Girdwood, Matthew Hall, James W. Antoon, Kathryn Kyler, Derek J. Williams, Samir S. Shah, Lucas E. Orth, Jennifer Goldman, James A. Feinstein, Laura B. Ramsey Feb 2024

Opportunities For Pharmacogenetic Testing To Guide Dosing Of Medications In Youths With Medicaid., Sonya Tang Girdwood, Matthew Hall, James W. Antoon, Kathryn Kyler, Derek J. Williams, Samir S. Shah, Lucas E. Orth, Jennifer Goldman, James A. Feinstein, Laura B. Ramsey

Manuscripts, Articles, Book Chapters and Other Papers

IMPORTANCE: There are an increasing number of medications with a high level of evidence for pharmacogenetic-guided dosing (PGx drugs). Knowledge of the prevalence of dispensings of PGx drugs and their associated genes may allow hospitals and clinical laboratories to determine which pharmacogenetic tests to implement.

OBJECTIVES: To investigate the prevalence of outpatient dispensings of PGx drugs among Medicaid-insured youths, determine genes most frequently associated with PGx drug dispenses, and describe characteristics of youths who were dispensed at least 1 PGx drug.

DESIGN, SETTING, AND PARTICIPANTS: This serial cross-sectional study includes data from 2011 to 2019 among youths aged 0 to …


Prenatal Diagnosis Of A Ductal-Dependent Branch Pulmonary Artery: Extra Vessels In The 3-Vessel And Trachea View., Anmol Goyal, Maria Kiaffas, Tara Swanson, Melanie Kathol, Sanket Shah, Nitin Madan Feb 2024

Prenatal Diagnosis Of A Ductal-Dependent Branch Pulmonary Artery: Extra Vessels In The 3-Vessel And Trachea View., Anmol Goyal, Maria Kiaffas, Tara Swanson, Melanie Kathol, Sanket Shah, Nitin Madan

Manuscripts, Articles, Book Chapters and Other Papers

Obtaining a three-vessel (3V) and three-vessel and trachea (3VT) view from the fetal upper mediastinum is now considered to be part of standard imaging protocol for routine obstetric cardiac screening examinations. We report two fetal cases of an anomalous pulmonary artery origin, utilizing the standard 3V and 3VT views. Further imaging led to a rare diagnosis of bilateral ductus arteriosus with discontinuous branch pulmonary arteries in the absence of any other congenital heart defect. We briefly discuss the imaging features, differential diagnoses, and management of this rare entity.


Vitamin D Oral Replacement In Children With Obesity Related Asthma: Vdora1 Randomized Clinical Trial., Brian O'Sullivan, Song Ounpraseuth, Laura James, Marc Majure, Jason Lang, Zhuopei Hu, Alan Simon, Scott Bickel, Brian Ely, L E Faricy, Maryam Garza, Melody Greer, Daniel Hsia, Akilah Jefferson, Lisa Knight, Jeannette Lee, Deborah Liptzin, Mehtap Haktanir Abul, Tamara T. Perry, Fred Prior, Christine Sangiovanni, Jade B. Tam-Williams, Brian Wu, Jessica Snowden, Echo Idea States Pediatric Clinical Trial Network Feb 2024

Vitamin D Oral Replacement In Children With Obesity Related Asthma: Vdora1 Randomized Clinical Trial., Brian O'Sullivan, Song Ounpraseuth, Laura James, Marc Majure, Jason Lang, Zhuopei Hu, Alan Simon, Scott Bickel, Brian Ely, L E Faricy, Maryam Garza, Melody Greer, Daniel Hsia, Akilah Jefferson, Lisa Knight, Jeannette Lee, Deborah Liptzin, Mehtap Haktanir Abul, Tamara T. Perry, Fred Prior, Christine Sangiovanni, Jade B. Tam-Williams, Brian Wu, Jessica Snowden, Echo Idea States Pediatric Clinical Trial Network

Manuscripts, Articles, Book Chapters and Other Papers

Children with asthma and obesity are more likely to have lower vitamin D levels, but the optimal replacement dose is unknown in this population. The objective of this study is identifying a vitamin D dose in children with obesity-related asthma that safely achieves serum vitamin D levels of ≥ 40 ng/mL. This prospective multisite randomized controlled trial recruited children/adolescents with asthma and body mass index ≥ 85% for age/sex. Part 1 (dose finding), evaluated 4 oral vitamin D regimens for 16 weeks to identify a replacement dose that achieved serum vitamin D levels ≥ 40 ng/mL. Part 2 compared the …


Health Equity And Policy Considerations For Pediatric And Adult Congenital Heart Disease Care Among Minoritized Populations In The United States., Keila N. Lopez, Kiona Y. Allen, Carissa M. Baker-Smith, Katia Bravo-Jaimes, Joseph Burns, Bianca Cherestal, Jason F. Deen, Brittany K. Hills, Jennifer H. Huang, Ramiro W. Lizano Santamaria, Carlos A. Lodeiro, Valentina Melo, Jasmine S. Moreno, Flora Nuñez Gallegos, Harris Onugha, Tony A. Pastor, Michelle C. Wallace, Deidra A. Ansah Jan 2024

Health Equity And Policy Considerations For Pediatric And Adult Congenital Heart Disease Care Among Minoritized Populations In The United States., Keila N. Lopez, Kiona Y. Allen, Carissa M. Baker-Smith, Katia Bravo-Jaimes, Joseph Burns, Bianca Cherestal, Jason F. Deen, Brittany K. Hills, Jennifer H. Huang, Ramiro W. Lizano Santamaria, Carlos A. Lodeiro, Valentina Melo, Jasmine S. Moreno, Flora Nuñez Gallegos, Harris Onugha, Tony A. Pastor, Michelle C. Wallace, Deidra A. Ansah

Manuscripts, Articles, Book Chapters and Other Papers

Achieving health equity in populations with congenital heart disease (CHD) requires recognizing existing disparities throughout the lifespan that negatively and disproportionately impact specific groups of individuals. These disparities occur at individual, institutional, or system levels and often result in increased morbidity and mortality for marginalized or racially minoritized populations (population subgroups (e.g., ethnic, racial, social, religious) with differential power compared to those deemed to hold the majority power in the population). Creating actionable strategies and solutions to address these health disparities in patients with CHD requires critically examining multilevel factors and health policies that continue to drive health inequities, including …


The Durability Of Antibody Responses Of Two Doses Of High-Dose Relative To Two Doses Of Standard-Dose Inactivated Influenza Vaccine In Pediatric Hematopoietic Cell Transplant Recipients: A Multi-Center Randomized Controlled Trial., Jennifer E. Schuster, Lubna Hamdan, Daniel E. Dulek, Carrie L. Kitko, Einas Batarseh, Zaid Haddadin, Laura S. Stewart, Anna Stahl, Molly Potter, Herdi Rahman, Spyros A. Kalams, Claire E. Bocchini, Elizabeth A. Moulton, Susan E. Coffin, Monica I. Ardura, Rachel L. Wattier, Gabriela Maron, Michael Grimley, Grant Paulsen, Christopher J. Harrison, Jason L. Freedman, Paul A. Carpenter, Janet A. Englund, Flor M. Munoz, Lara Danziger-Isakov, Andrew J. Spieker, Natasha B. Halasa, Pediatric Hct Flu Study Jan 2024

The Durability Of Antibody Responses Of Two Doses Of High-Dose Relative To Two Doses Of Standard-Dose Inactivated Influenza Vaccine In Pediatric Hematopoietic Cell Transplant Recipients: A Multi-Center Randomized Controlled Trial., Jennifer E. Schuster, Lubna Hamdan, Daniel E. Dulek, Carrie L. Kitko, Einas Batarseh, Zaid Haddadin, Laura S. Stewart, Anna Stahl, Molly Potter, Herdi Rahman, Spyros A. Kalams, Claire E. Bocchini, Elizabeth A. Moulton, Susan E. Coffin, Monica I. Ardura, Rachel L. Wattier, Gabriela Maron, Michael Grimley, Grant Paulsen, Christopher J. Harrison, Jason L. Freedman, Paul A. Carpenter, Janet A. Englund, Flor M. Munoz, Lara Danziger-Isakov, Andrew J. Spieker, Natasha B. Halasa, Pediatric Hct Flu Study

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Our previous study established a 2-dose regimen of high-dose trivalent influenza vaccine (HD-TIV) to be immunogenically superior compared to a 2-dose regimen of standard-dose quadrivalent influenza vaccine (SD-QIV) in pediatric allogeneic hematopoietic cell transplant (HCT) recipients. However, the durability of immunogenicity and the role of time post-HCT at immunization as an effect modifier are unknown.

METHODS: This phase II, multi-center, double-blinded, randomized controlled trial compared HD-TIV to SD-QIV in children 3-17 years old who were 3-35 months post-allogeneic HCT, with each formulation administered twice, 28-42 days apart. Hemagglutination inhibition (HAI) titers were measured at baseline, 28-42 days following each …


Clarification Of Adverse Drug Reactions By A Pharmacovigilance Team Results In Increased Antibiotic Re-Prescribing At A Freestanding United States Children's Hospital., Keith Feldman, Sarah Suppes, Jennifer Goldman Jan 2024

Clarification Of Adverse Drug Reactions By A Pharmacovigilance Team Results In Increased Antibiotic Re-Prescribing At A Freestanding United States Children's Hospital., Keith Feldman, Sarah Suppes, Jennifer Goldman

Manuscripts, Articles, Book Chapters and Other Papers

Documentation of adverse drug reactions (ADRs) is a key factor in guiding future prescribing. However, incomplete documentation is common and often fails to distinguish implicated drugs as true allergies. This in turn leads to unnecessary avoidance of implicated drug classes and may result in sub-optimal prescribing. Pharmacovigilance (PV) programs utilize a systematic approach to clarify ADR documentation and are known to improve patient safety. Yet it remains unclear if PV alters prescribing. Or, if the existence of the ADR documentation itself continues to prompt avoidance of implicated drugs. To address this, our work presents a retrospective cohort study assessing if …


Development Of Right Ventricular Electromechanical Dyssynchrony Following Surgical Repair Of Tetralogy Of Fallot In Infants., Andrew W. Mccrary, Sydney D. Collins, Zebulon Z. Spector, P Andrea Kropf, Piers C A Barker, Joseph Kisslo, Daniel Forsha Jan 2024

Development Of Right Ventricular Electromechanical Dyssynchrony Following Surgical Repair Of Tetralogy Of Fallot In Infants., Andrew W. Mccrary, Sydney D. Collins, Zebulon Z. Spector, P Andrea Kropf, Piers C A Barker, Joseph Kisslo, Daniel Forsha

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: In adolescents and adults with tetralogy of Fallot (TOF), right ventricle (RV) electromechanical dyssynchrony (EMD) due to right bundle branch block (RBBB) is associated with reduced exercise capacity and RV dysfunction. While the development of RBBB following surgical repair of tetralogy of Fallot (rTOF) is a frequent sequela, it is not known whether EMD is present in every patient immediately following rTOF. The specific timing of the onset of RBBB following rTOF therefore provides an opportunity to assess whether acute RBBB is associated with the simultaneous acquisition of EMD.

METHODS: Transthoracic echocardiography with speckle tracking analysis for RV global …


Achieving Reliable Patient Reported Outcomes Collection To Measure Health Care Improvement In A Learning Health Network: Lessons From Pediatric Rheumatology Care And Outcomes Improvement Network., Nancy Pan, Esi M. Morgan, Meghan Ryan, Beth Gottlieb, Julia G. Harris, Tzielan Lee, Y Ingrid Goh Jan 2024

Achieving Reliable Patient Reported Outcomes Collection To Measure Health Care Improvement In A Learning Health Network: Lessons From Pediatric Rheumatology Care And Outcomes Improvement Network., Nancy Pan, Esi M. Morgan, Meghan Ryan, Beth Gottlieb, Julia G. Harris, Tzielan Lee, Y Ingrid Goh

Manuscripts, Articles, Book Chapters and Other Papers

INTRODUCTION: Data from the Pediatric Rheumatology Care and Outcomes Improvement Network (PR-COIN) registry suggests that reliable collection of patient-reported outcomes (PROs) varies across sites. The objective of this study was to better understand the practices of collecting PROs at PR-COIN sites.

METHODS: A REDCap survey was sent to the lead representative for each PR-COIN site. Registry data were analyzed to better understand the completion rates of PROs. Interviews of physician leaders of high performing sites were conducted by videoconference, audiotranscribed and themes were summarized. Quantitative data were analyzed using descriptive statistics and qualitative data were thematically analyzed.

RESULTS: All 23 …


Postprandial Glucose Variability Following Typical Meals In Youth Living With Type 1 Diabetes., Susana R. Patton, Simon Bergford, Jennifer L. Sherr, Robin L. Gal, Peter Calhoun, Mark A. Clements, Michael C. Riddell, Corby K. Martin Jan 2024

Postprandial Glucose Variability Following Typical Meals In Youth Living With Type 1 Diabetes., Susana R. Patton, Simon Bergford, Jennifer L. Sherr, Robin L. Gal, Peter Calhoun, Mark A. Clements, Michael C. Riddell, Corby K. Martin

Manuscripts, Articles, Book Chapters and Other Papers

We explored the association between macronutrient intake and postprandial glucose variability in a large sample of youth living with T1D and consuming free-living meals. In the Type 1 Diabetes Exercise Initiative Pediatric (T1DEXIP) Study, youth took photographs before and after their meals on 3 days during a 10 day observation period. We used the remote food photograph method to obtain the macronutrient content of youth's meals. We also collected physical activity, continuous glucose monitoring, and insulin use data. We measured glycemic variability using standard deviation (SD) and coefficient of variation (CV) of glucose for up to 3 h after meals. …


Distinct Reproductive Phenotypes Segregate With Differences In Body Weight In Adolescent Polycystic Ovary Syndrome., Angie Chen-Patterson, Angelina Bernier, Tania S. Burgert, Vanessa Davis, Tazeena Khan, David Geller, Emily Paprocki, Rachana Shah, Selma F. Witchel, Camila Pereira-Eshraghi, Aviva B. Sopher, Melanie G. Cree, Laura C. Torchen Jan 2024

Distinct Reproductive Phenotypes Segregate With Differences In Body Weight In Adolescent Polycystic Ovary Syndrome., Angie Chen-Patterson, Angelina Bernier, Tania S. Burgert, Vanessa Davis, Tazeena Khan, David Geller, Emily Paprocki, Rachana Shah, Selma F. Witchel, Camila Pereira-Eshraghi, Aviva B. Sopher, Melanie G. Cree, Laura C. Torchen

Manuscripts, Articles, Book Chapters and Other Papers

INTRODUCTION: Polycystic ovary syndrome (PCOS) is a heterogenous clinical syndrome defined by hyperandrogenism and irregular menses. In adult women with PCOS, discrete metabolic and reproductive subgroups have been identified. We hypothesize that distinct phenotypes can be distinguished between adolescent girls who are lean (LN-G) and girls with obesity (OB-G) at the time of PCOS diagnosis.

METHODS: Data were extracted from the CALICO multisite PCOS database. Clinical data collected at the time of diagnosis were available in 354 patients (81% with obesity) from 7 academic centers. Patients with body mass index (BMI) < 85th percentile for age and sex were characterized as lean (LN-G) and those with BMI percentile ≥ 95th percentile as obese (OB-G). We compared metabolic and reproductive phenotypes in LN-G and OB-G.

RESULTS: Reproductive phenotypes differed between the groups, with LN-G …


Lessons Learnt In The First Year Of An Australian Pediatric Cardio Oncology Clinic., Claudia Toro, Ben Felmingham, Mangesh Jhadav, David S. Celermajer, Andre La Gerche, John O'Sullivan, Sanjeev Kumar, Marion K. Mateos, Joy M. Fulbright, Dinisha Govender, Lane Collier, Michael Cheung, David D. Eisenstat, Peter W. Lange, Julian Ayer, David A. Elliott, Rachel Conyers Dec 2023

Lessons Learnt In The First Year Of An Australian Pediatric Cardio Oncology Clinic., Claudia Toro, Ben Felmingham, Mangesh Jhadav, David S. Celermajer, Andre La Gerche, John O'Sullivan, Sanjeev Kumar, Marion K. Mateos, Joy M. Fulbright, Dinisha Govender, Lane Collier, Michael Cheung, David D. Eisenstat, Peter W. Lange, Julian Ayer, David A. Elliott, Rachel Conyers

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Modern oncological therapies together with chemotherapy and radiotherapy have broadened the agents that can cause cardiac sequelae, which can manifest for pediatric oncology patients while on active treatment. Recommendations for high-risk patients who should be monitored in a pediatric cardio-oncology clinic have previously been developed by expert Delphi consensus by our group. In 2022 we opened our first multidisciplinary pediatric cardio-oncology clinic adhering to these recommendations in surveillance and management.

OBJECTIVES: Our pediatric cardio-oncology clinic aimed to: (i) Document cardiovascular toxicities observed within a pediatric cardio-oncology clinic and. (ii) Evaluate the applicability of the Australian and New Zealand Pediatric …


Effects Of A Preventive Mental Health Curriculum Embedded Into A Scholarly Gaming Course On Adolescent Self-Esteem: Prospective Matched Pairs Experiment., Christopher Jenson, Sharon Fitzgerald Wolff, Libby Milkovich Dec 2023

Effects Of A Preventive Mental Health Curriculum Embedded Into A Scholarly Gaming Course On Adolescent Self-Esteem: Prospective Matched Pairs Experiment., Christopher Jenson, Sharon Fitzgerald Wolff, Libby Milkovich

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Positive self-esteem predicts happiness and well-being and serves as a protective factor for favorable mental health. Scholarly gaming within the school setting may serve as a channel to deliver a mental health curriculum designed to improve self-esteem.

OBJECTIVE: This study aims to evaluate the impact of a scholarly gaming curriculum with and without an embedded preventive mental health curriculum, Mental Health Moments (MHM), on adolescents' self-esteem.

METHODS: The scholarly gaming curriculum and MHM were developed by 3 educators and a school-based health intervention expert. The scholarly gaming curriculum aligned with academic guidelines from the International Society for Technology Education, …


Association Between Remote Monitoring And Interstage Morbidity And Death In Patients With Single-Ventricle Heart Disease Across Socioeconomic Groups., Bianca Cherestal, Lori A. Erickson, Janelle R. Noel-Macdonnell Phd, Girish S. Shirali, Hayley S. Hancock, Doaa Aly, Matthew Files, Sarah Clauss, Natalie Jayaram Dec 2023

Association Between Remote Monitoring And Interstage Morbidity And Death In Patients With Single-Ventricle Heart Disease Across Socioeconomic Groups., Bianca Cherestal, Lori A. Erickson, Janelle R. Noel-Macdonnell Phd, Girish S. Shirali, Hayley S. Hancock, Doaa Aly, Matthew Files, Sarah Clauss, Natalie Jayaram

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Despite improvements in survival over time, the mortality rate for infants with single-ventricle heart disease remains high. Infants of low socioeconomic status (SES) are particularly vulnerable. We sought to determine whether use of a novel remote monitoring program, the Cardiac High Acuity Monitoring Program, mitigates differences in outcomes by SES.

METHODS AND RESULTS: Within the Cardiac High Acuity Monitoring Program, we identified 610 infants across 11 centers from 2014 to 2021. All enrolled families had access to a mobile application allowing for near-instantaneous transfer of patient information to the care team. Patients were divided into SES tertiles on the …