Medicine and Health Sciences Commons^™

Approach For Defining Human Adenovirus Infection And Disease For Central Review Adjudication In Clinical Studies., Brian T. Fisher, Jesse Blumenstock, Craig L K Boge, Sydney Shuster, Alix E. Seif, Michael Green, Marian G. Michaels, Jessie L. Alexander, Monica I. Ardura, Tamara P. Miller, Diego R. Hijano, William J. Muller, Jennifer E. Schuster, Abby M. Green, Daniel E. Dulek, Adriana E. Kajon, Lara Danziger-Isakov

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BACKGROUND: Pediatric allogeneic hematopoietic cell transplant (allo-HCT) recipients are at risk for morbidity and mortality from human adenovirus (HAdV). HAdV can be detected in an asymptomatic state, referred to as infection or with signs or symptoms of illness, referred to as disease. Standardized case definitions are needed to distinguish infection from disease and allow for consistent reporting in both observational cohort studies and therapeutic clinical trials.

METHODS: A working group of experts in virology, transplant infectious disease, and HCT was assembled to develop HAdV infection and disease definitions with the degree of certainty (i.e., possible, probable, and proven). Definitions were …

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

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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 …

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

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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 …

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

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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 …

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

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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 …

Barriers To The Diagnosis, Care, And Management Of Pediatric Patients With Ehlers-Danlos Syndrome In The United States: A Qualitative Analysis., William R. Black, Lora L. Black, Jordan T. Jones

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Objectives: Ehlers-Danlos Syndromes (EDS) are a family of heritable connective tissue diseases. Primary practitioners are capable of diagnosing and managing EDS; however, few are knowledgeable and comfortable enough to see patients with EDS, resulting in delays in diagnosis and care. This study explores the barriers physicians experience with diagnosing, managing, and caring for patients with EDS, and potential resolutions to those barriers. Methods: As part of a larger online study, providers (n = 107) in the United States were asked to specify "What information would improve (their) comfort" in diagnosing, caring for, and managing EDS via open-ended questions. Results …

Risk Factors For Health Impairments In Children After Hospitalization For Acute Covid-19 Or Mis-C., Aline B. Maddux, Cameron C. Young, Suden Kucukak, Laura D. Zambrano, Margaret M. Newhams, Caitlin K. Rollins, Natasha B. Halasa, Shira J. Gertz, Elizabeth H. Mack, Stephanie Schwartz, Michele Kong, Laura L. Loftis, Katherine Irby, Courtney M. Rowan, Keiko M. Tarquinio, Matt S. Zinter, Hillary Crandall, Natalie Z. Cvijanovich, Jennifer E. Schuster, Julie C. Fitzgerald, Mary A. Staat, Charlotte V. Hobbs, Ryan A. Nofziger, Steven Shein, Heidi Flori, Melissa L. Cullimore, Brandon M. Chatani, Emily R. Levy, Katri V. Typpo, Janet R. Hume, Angela P. Campbell, Adrienne G. Randolph, Overcoming Covid-19 Investigators

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OBJECTIVE: To identify risk factors for persistent impairments after pediatric hospitalization for acute coronavirus disease 2019 (COVID-19) or multisystem inflammatory syndrome in children (MIS-C) during the SARS-CoV-2 pandemic.

METHODS: Across 25 U.S. Overcoming COVID-19Network hospitals, we conducted a prospective cohort study of patients

RESULTS: Of 232 children with acute COVID-19, 71 (30.6%) had persistent symptoms and 50 (21.6%) had activity impairments at follow-up; for MIS-C (n = 241), 56 (23.2%) had persistent symptoms and 58 (24.1%) had activity impairments. In adjusted analyses of patients with acute COVID-19, receipt of mechanical ventilation was associated with persistent symptoms [aRR 1.83 …

Barriers And Facilitators Of Family Rules And Routines During Pediatric Cancer Treatment., Carolyn R. Bates, Isabella K. Pallotto, Rachel Moore, Lynne Covitz, Meredith Dreyer

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OBJECTIVE: Pediatric nurses work closely with families of children with new cancer diagnoses and can provide essential supports to promote coping and adjustment. This cross-sectional qualitative study aimed to gather caregiver perspectives on barriers and facilitators to adaptive family functioning during the early phases of cancer treatment, with a focus on family rules and routines.

METHODS: Caregivers (N = 44) of a child diagnosed with cancer and receiving active treatment completed a semi-structured interview about their engagement in family rules and routines. Time since diagnosis was abstracted from the medical record. A multi-pass inductive coding strategy was utilized to extract …

Sars-Cov-2 Infections In Patients Enrolled On The Children's Oncology Group Standard-Risk B-Cell Acute Lymphoblastic Leukemia Trial, Aall1731., Caitlin W. Elgarten, John A. Kairalla, Joel Thompson, Tamara P. Miller, Cindy Wang, Susan Conway, Mignon L. Loh, Elizabeth A. Raetz, Sumit Gupta, Rachel E. Rau, Anne Angiolillo, Karen R. Rabin, Sarah Alexander

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Hematologic malignancy is a risk factor for severe coronavirus disease 2019 (COVID-19) in adults; however, data specific to children with leukemia are limited. High-quality infectious adverse event data from the ongoing Children's Oncology Group (COG) standard-risk B acute lymphoblastic leukemia/lymphoma (ALL/LLy) trial, AALL1731, were analyzed to provide a disease-specific estimate of SARS-CoV-2 infection outcomes in pediatric ALL. Of 253 patients with reported infections, the majority (77.1%) were asymptomatic or mildly symptomatic (CTCAE grade 1/2) and there was a single COVID-19-related death. These data suggest SARS-CoV-2 infection does not confer substantial morbidity among young patients with B-lymphoblastic leukemia/lymphoma (B-ALL/LLy).

Climate Change Imperils Pediatric Health: Child Advocacy Through Fossil Fuel Divestment., Sandra H. Jee, Elizabeth Friedman, Ruth A. Etzel, Vi T. Nguyen, Todd L. Sack, Kathi J. Kemper

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Climate change poses an existential threat to children's health. Divestment of ownership stakes in fossil fuel companies is one tool available to pediatricians to address climate change. Pediatricians are trusted messengers regarding children's health and therefore bear a unique responsibility to advocate for climate and health policies that affect children. Among the impacts of climate change on pediatric patients are allergic rhinitis and asthma; heat-related illnesses; premature birth; injuries from severe storms and fires; vector-borne diseases; and mental illnesses. Children are disproportionately affected as well by climate-related displacement of populations, drought, water shortages, and famine. The human-generated burning of fossil …

Trends In The Representation Of Women In The American Society Of Pediatric Nephrology Program At The Pediatric Academic Society Annual Meetings 2012-2021., Bahar Barani Najafabadi, David T. Selewski, Danielle E. Soranno, Darcy K. Weidemann

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INTRODUCTION: Women are under-represented in virtually all fields of academic medicine. Even in pediatrics, a field that historically attracts a workforce with a majority of women physicians, substantial gender disparities persist in leadership positions. However, previous studies of gender representation in various academic settings are limited to small studies or aggregate pediatric subspecialties, thereby omitting important granularity within each subspecialty. No prior studies have investigated potential gender disparities in pediatric nephrology. The aim of this study is to determine the representation of women physicians in leadership and speaking roles in the annual American Society of Pediatric Nephrology (ASPN) meeting.

METHODS: …

Risk And Resilient Functioning Of Families Of Children With Cancer During The Covid-19 Pandemic., Renee Gilbert, Carolyn R Bates, Devanshi Khetawat, Meredith Dreyer Gillette, Rachel Moore

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Previous literature highlights the impact of COVID-19 on family functioning. Less is known about the impact of the pandemic on families of pediatric cancer patients. In order to determine universal and unique risk and resilience factors of these families during the pandemic, a qualitative analysis was conducted on families currently receiving cancer treatment at a Midwestern hospital. Results of the data analysis depict ways in which these families have been impacted by and have adapted to COVID-19. These findings suggest that families of pediatric cancer patients have unique experiences in the context of COVID-19, in addition to universal experiences outlined …

Impact Of Duodenal Pathology On Oral Drug Bioavailability And Disease Outcomes In Pediatric Crohn's Disease., Rebecca Casini, Carrie A. Vyhlidal, Julia M. Bracken, Ashley K. Sherman, Atif Ahmed, Vivekanand Singh, Veronica Williams, Valentina Shakhnovich

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Background: Crohn's disease with upper gastrointestinal tract involvement occurs more often in children than adults and has the potential to interfere with oral drug absorption. We aimed to compare disease outcomes in children receiving oral azathioprine for the treatment of Crohn's disease with (DP) and without (NDP) duodenal pathology at diagnosis.

Methods: Duodenal villous length, body mass index (BMI), and laboratory studies were compared in DP vs. NDP during the first year post-diagnosis, using parametric/nonparametric tests and regression analysis (SAS v9.4); the data are reported as the median (interquartile range) or the mean ± standard deviation. Thiopurine metabolite concentration (pmol/8 …

Gender Dysphoria In Adolescents With Ehlers-Danlos Syndrome., Jordan T. Jones, William R. Black, Christine Moser, Eric T. Rush, Lindsey Malloy-Walton

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OBJECTIVES: Ehlers-Danlos Syndrome represents a family of heritable connective tissue disorders that include joint hypermobility, tissue fragility, and skin hyperextensibility. Ehlers-Danlos Syndrome presents with clinical sequela across multiple body systems that require multidisciplinary care. Little is known about adolescents with Ehlers-Danlos Syndrome who are transgender and gender diverse. To date, there have been no reports of transgender and gender diverse youth in pediatric patients with Ehlers-Danlos Syndrome. The objective of this study was to characterize transgender and gender diverse adolescents with Ehlers-Danlos Syndrome seen in a pediatric multidisciplinary specialty clinic.

METHODS: A retrospective chart review was performed and it was …

A Review Of Ferric Citrate Clinical Studies, And The Rationale And Design Of The Ferric Citrate And Chronic Kidney Disease In Children (Fit4kid) Trial., Mark R. Hanudel, Marciana L. Laster, Anthony A. Portale, Aditi Dokras, Raymond P. Quigley, German A Lozano Guzman, Joshua J. Zaritsky, Nicole A. Hayde, Frederick J. Kaskel, Mark M. Mitsnefes, Jorge A. Ramirez, Peace D. Imani, Poyyapakkam R. Srivaths, Amy J. Kogon, Michelle R. Denburg, Tom D. Blydt-Hansen, Loretta Z. Reyes, Larry A. Greenbaum, Darcy K. Weidemann, Bradley A. Warady, David A. Elashoff, Susan R. Mendley, Tamara Isakova, Isidro B. Salusky

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Pediatric chronic kidney disease (CKD) is characterized by many co-morbidities, including impaired growth and development, CKD-mineral and bone disorder, anemia, dysregulated iron metabolism, and cardiovascular disease. In pediatric CKD cohorts, higher circulating concentrations of fibroblast growth factor 23 (FGF23) are associated with some of these adverse clinical outcomes, including CKD progression and left ventricular hypertrophy. It is hypothesized that lowering FGF23 levels will reduce the risk of these events and improve clinical outcomes. Reducing FGF23 levels in CKD may be accomplished by targeting two key stimuli of FGF23 production-dietary phosphate absorption and iron deficiency. Ferric citrate is approved for use …

A Methodological And Clinical Approach To Measured Energy Expenditure In The Critically Ill Pediatric Patient., Jaime Silva-Gburek, Paola Hong Zhu, Marwa Mansour, David Walding, Jorge A. Coss-Bu

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The metabolic response to injury and stress is characterized initially by a decreased energy expenditure (Ebb phase) followed by an increased metabolic expenditure (Flow phase). Indirect calorimetry is a methodology utilized to measure energy expenditure and substrate utilization by measuring gas exchange in exhaled air and urinary nitrogen. The use of indirect calorimetry in critically ill patients requires precise equipment to obtain accurate measurements. The most recent guidelines suggested that measured energy expenditure by indirect calorimetry be used to determine energy requirements. This article reviews the methodological and clinical use of indirect calorimetry in critically ill pediatric patients.

Iv Sotalol Use In Pediatric And Congenital Heart Patients: A Multicenter Registry Study., Lindsey Malloy-Walton, Nicholas H. Von Bergen, Seshadri Balaji, Peter S. Fischbach, Jason M. Garnreiter, S Yukiko Asaki, Jeffrey P. Moak, Luis A. Ochoa, Philip M. Chang, Hoang H. Nguyen, Akash R. Patel, Christa Kirk, Ashley K. Sherman, Jennifer N. Avari Silva, J Philip Saul

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Background There is limited information regarding the clinical use and effectiveness of IV sotalol in pediatric patients and patients with congenital heart disease, including those with severe myocardial dysfunction. A multicenter registry study was designed to evaluate the safety, efficacy, and dosing of IV sotalol. Methods and Results A total of 85 patients (age 1 day-36 years) received IV sotalol, of whom 45 (53%) had additional congenital cardiac diagnoses and 4 (5%) were greater than 18 years of age. In 79 patients (93%), IV sotalol was used to treat supraventricular tachycardia and 4 (5%) received it to treat ventricular arrhythmias. …

Barriers, Access And Management Of Paediatric Epilepsy With Telehealth., Kari Gali, Sucheta Joshi, Sarah Hueneke, Alexis Katzenbach, Linda Radecki, Trisha Calabrese, Linda Fletcher, Cristina Trandafir, Carey Wilson, Monisha Goyal, Courtney J. Wusthoff, Jean-Baptist Lepichon, Rhonda Corvalan, April Golson, Jessica Hardy, Michael Smith, Elizabeth Cook, Joshua L. Bonkowsky

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Access to paediatric neurology care is complex, resulting in significant wait times and negative patient outcomes. The goal of the American Academy of Pediatrics National Coordinating Center for Epilepsy's project, Access Improvement and Management of Epilepsy with Telehealth (AIM-ET), was to identify access and management challenges in the deployment of telehealth technology. AIM-ET organised four paediatric neurology teams to partner with primary-care providers (PCP) and their multidisciplinary teams. Telehealth visits were conducted for paediatric epilepsy patients. A post-visit survey assessed access and satisfaction with the telehealth visit compared to an in-person visit. Pre/post surveys completed by PCPs and neurologists captured …

Data-Driven Clustering Identifies Features Distinguishing Multisystem Inflammatory Syndrome From Acute Covid-19 In Children And Adolescents., Alon Geva, Manish M. Patel, Margaret M. Newhams, Cameron C. Young, Mary Beth F. Son, Michele Kong, Aline B. Maddux, Mark W. Hall, Becky J. Riggs, Aalok R. Singh, John S. Giuliano, Charlotte V. Hobbs, Laura L. Loftis, Gwenn E. Mclaughlin, Stephanie P. Schwartz, Jennifer E. Schuster, Christopher J. Babbitt, Natasha B. Halasa, Shira J. Gertz, Sule Doymaz, Janet R. Hume, Tamara T. Bradford, Katherine Irby, Christopher L. Carroll, John K. Mcguire, Keiko M. Tarquinio, Courtney M. Rowan, Elizabeth H. Mack, Natalie Z. Cvijanovich, Julie C. Fitzgerald, Philip C. Spinella, Mary A. Staat, Katharine N. Clouser, Vijaya L. Soma, Heda Dapul, Mia Maamari, Cindy Bowens, Kevin M. Havlin, Peter M. Mourani, Sabrina M. Heidemann, Steven M. Horwitz, Leora R. Feldstein, Mark W. Tenforde, Jane W. Newburger, Kenneth D. Mandl, Adrienne G. Randolph, Overcoming Covid-19 Investigators

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Background: Multisystem inflammatory syndrome in children (MIS-C) consensus criteria were designed for maximal sensitivity and therefore capture patients with acute COVID-19 pneumonia.

Methods: We performed unsupervised clustering on data from 1,526 patients (684 labeled MIS-C by clinicians)clusters, followed by recursive feature elimination to identify characteristics of potentially misclassified MIS-C-labeled patients.

Findings: Of 94 clinical features tested, 46 were retained for clustering. Cluster 1 patients (N = 498; 92% labeled MIS-C) were mostly previously healthy (71%), with mean age 7·2 ± 0·4 years, predominant cardiovascular (77%) and/or mucocutaneous (82%) involvement, high inflammatory biomarkers, and mostly SARS-CoV-2 PCR negative (60%). Cluster …

Pooled Safety Analysis Of Tisagenlecleucel In Children And Young Adults With B Cell Acute Lymphoblastic Leukemia., John E. Levine, Stephan A. Grupp, Michael A. Pulsipher, Andrew C. Dietz, Susana Rives, Douglas Myers, Keith August, Michael R. Verneris, Jochen Buechner, Theodore W. Laetsch, Henrique Bittencourt, Andre Baruchel, Michael W. Boyer, Barbara De Moerloose, Muna Qayed, Stella M. Davies, Christine L. Phillips, Timothy A. Driscoll, Peter Bader, Krysta Schlis, Patricia A. Wood, Rajen Mody, Lan Yi, Mimi Leung, Lamis K. Eldjerou, Carl H. June, Shannon L. Maude

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Background: Tisagenlecleucel, an anti-CD19 chimeric antigen receptor T cell therapy, has demonstrated efficacy in children and young adults with relapsed/refractory B cell acute lymphoblastic leukemia (B-ALL) in two multicenter phase 2 trials (ClinicalTrials.gov, NCT02435849 (ELIANA) and NCT02228096 (ENSIGN)), leading to commercialization of tisagenlecleucel for the treatment of patients up to age 25 years with B-ALL that is refractory or in second or greater relapse.

Methods: A pooled analysis of 137 patients from these trials (ELIANA: n=79; ENSIGN: n=58) was performed to provide a comprehensive safety profile for tisagenlecleucel.

Results: Grade 3/4 tisagenlecleucel-related adverse events (AEs) were reported in 77% of …

A Multicenter Evaluation Of Viral Bloodstream Detections In Children Presenting To The Emergency Department With Suspected Systemic Infection., Christina A. Rostad, Neena Kanwar, Jumi Yi, Claudia R. Morris, Jennifer Dien Bard, Amy Leber, James Dunn, Kimberle C. Chapin, Anne J. Blaschke, Judy A. Daly, Leslie Hueschen, Matthew Jones, Elizabeth Ott, Jeffrey Bastar, Kevin M. Bourzac, Rangaraj Selvarangan

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BACKGROUND: Fever is a common symptom in children presenting to the Emergency Department (ED). We aimed to describe the epidemiology of systemic viral infections and their predictive values for excluding serious bacterial infections (SBIs), including bacteremia, meningitis and urinary tract infections (UTIs) in children presenting to the ED with suspected systemic infections.

METHODS: We enrolled children who presented to the ED with suspected systemic infections who had blood cultures obtained at seven healthcare facilities. Whole blood specimens were analyzed by an experimental multiplexed PCR test for 7 viruses. Demographic and laboratory results were abstracted.

RESULTS: Of the 1114 subjects enrolled, …

Factors Associated With Reduced Infliximab Exposure In The Treatment Of Pediatric Autoimmune Disorders: A Cross-Sectional Prospective Convenience Sampling Study., Ryan S. Funk, Valentina Shakhnovich, Yu Kyoung Cho, Kishore Polireddy, Taina Hudson, Kyle Gress, Mara L. Becker

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BACKGROUND: Inadequate systemic exposure to infliximab (IFX) is associated with treatment failure. This work evaluated factors associated with reduced IFX exposure in children with autoimmune disorders requiring IFX therapy.

METHODS: In this single-center cross-sectional prospective study IFX trough concentrations and anti-drug antibodies (ADAs) were measured in serum from children diagnosed with inflammatory bowel disease (IBD) (n = 73), juvenile idiopathic arthritis (JIA) (n = 16), or uveitis (n = 8) receiving maintenance IFX infusions at an outpatient infusion clinic in a tertiary academic pediatric hospital. IFX concentrations in combination with population pharmacokinetic modeling were used to estimate IFX clearance. Patient …

Leveraging Institutional Support To Build An Integrated Multidisciplinary Care Model In Pediatric Inflammatory Bowel Disease., Jennifer Verrill Schurman, Craig A. Friesen

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While the biopsychosocial nature of inflammatory bowel disease (IBD) is now well accepted by clinicians, the need for integrated multidisciplinary care is not always clear to institutional administrators who serve as decision makers regarding resources provided to clinical programs. In this commentary, we draw on our own experience in building successful integrated care models within a division of pediatric gastroenterology (GI) to highlight key considerations in garnering initial approval, as well as methods to maintain institutional support over time. Specifically, we discuss the importance of making a strong case for the inclusion of a psychologist in pediatric IBD care, justifying …

Tisagenlecleucel Infusion In Patients With Relapsed/Refractory All And Concurrent Serious Infection., Erin Hall, Dwight E. Yin, Rakesh K. Goyal, Atif Ahmed, Grace S. Mitchell, Shawn D. St Peter, Terrie Flatt, Ibrahim A. Ahmed, Weijie Li, Richard J. Hendrickson, Keith August, Douglas Myers

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BACKGROUND: Tisagenlecleucel, an anti-CD19 chimeric antigen receptor T (CAR-T) cell therapy, has demonstrated durable efficacy and a manageable safety profile in pediatric and young adult patients with relapsed/refractory B-cell acute lymphoblastic leukemia (B-ALL) in the ELIANA pivotal trial and real-world experience. Experience from investigator-led studies prior to ELIANA suggests that infections and inflammatory conditions may exacerbate the severity of cytokine release syndrome (CRS) associated with CAR-T cell therapy, leading to extreme caution and strong restrictions for on-study and commercial infusion of tisagenlecleucel in patients with active infection. CRS intervention with interleukin (IL)-6 blockade and/or steroid therapy was introduced late in …

Severe Acute Respiratory Syndrome Coronavirus 2 Infections In Children: Multicenter Surveillance, United States, January-March 2020., Brian Rha, Joana Y. Lively, Janet A. Englund, Mary A. Staat, Geoffrey A. Weinberg, Rangaraj Selvarangan, Natasha B. Halasa, John V. Williams, Julie A. Boom, Leila C. Sahni, Marian G. Michaels, Laura S. Stewart, Christopher J. Harrison, Peter G. Szilagyi, Monica M. Mcneal, Eileen J. Klein, Bonnie Strelitz, Kirsten Lacombe, Elizabeth Schlaudecker, Mary Moffatt, Jennifer E. Schuster, Barbara A. Pahud, Gina Weddle, Robert W. Hickey, Vasanthi Avadhanula, Mary E. Wikswo, Aron J. Hall, Aaron T. Curns, Susan I. Gerber, Gayle Langley

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Previous reports of coronavirus disease 2019 among children in the United States have been based on health jurisdiction reporting. We performed severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) testing on children enrolled in active, prospective, multicenter surveillance during January-March 2020. Among 3187 children, only 4 (0.1%) SARS-CoV-2-positive cases were identified March 20-31 despite evidence of rising community circulation.

Sleep Patterns And Problems Among Children With 22q11 Deletion Syndrome., Jill M. Arganbright, Meghan Tracy, Susan Starling Hughes, David G. Ingram Md

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BACKGROUND: To delineate sleep habits and problems in children with 22q11.2 deletion syndrome (22q11DS).

METHODS: Thirty children, age 1-15 (mean 6.8) years, participated in the study, which was an internet-based anonymous survey of parents of children with 22q11DS administered via the 22q11.2 Foundation. The main outcome was the Childhood Sleep Habits Questionnaire (CSHQ).

RESULTS: Scores on the CSHQ demonstrated clinically significant sleep problems in 29 of the 30 children. When compared with previously reported normative values for typically developing children of the same age, children with 22q11DS had significantly greater sleep problems. Only 30% of children had previously undergone sleep …

Extracorporeal Membrane Oxygenation As A Bridge To Chimeric Antigen Receptor T-Cell Therapy For Severe Refractory Sepsis In The Setting Of Relapsed Refractory Pediatric Acute Lymphoblastic Leukemia: A Case Report., Alyssa Stoner, Jenna Miller, Terrie Flatt, Jessica S. Wallisch

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Pediatric oncology patients with sepsis are at higher risk of morbidity and mortality compared with pediatric patients without malignancy. Historically, patients with relapsed and/or refractory disease were not considered candidates for aggressive life support strategies including extracorporeal membrane oxygenation support.

Case Summary: We report a 4-year-old female with relapsed refractory pre-B cell acute lymphoblastic leukemia preparing for chimeric antigen receptor T cell therapy with tisagenlecleucel who was admitted with fever and neutropenia. She progressed to refractory septic shock secondary to

Conclusion: The ability to provide chimeric antigen receptor T-cell therapy at designated institutions can augment extracorporeal membrane oxygenation candidacy discussions …

Urinary Epidermal Growth Factor As A Marker Of Disease Progression In Children With Nephrotic Syndrome., Debbie S. Gipson, Howard Trachtman, Anne Waldo, Keisha L. Gibson, Sean Eddy, Katherine M. Dell, Tarak Srivastava, Kevin V. Lemley, Larry A. Greenbaum, Sangeeta Hingorani, Kevin E. Meyers, Frederick J. Kaskel, Kimberly J. Reidy, Christine B. Sethna, Cheryl L. Tran, Chia-Shi Wang, Katherine R. Tuttle, Gia Oh, Alicia M. Neu, Elizabeth Brown, Jen-Jar Lin, Jennifer Lai Yee, Therese M M. Roth, Jonathan P. Troost, Brenda W. Gillespie, Matthew G. Sampson, Matthias Kretzler, Wenjun Ju

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Introduction: Childhood-onset nephrotic syndrome has a variable clinical course. Improved predictive markers of long-term outcomes in children with nephrotic syndrome are needed. This study tests the association between baseline urinary epidermal growth factor (uEGF) excretion and longitudinal kidney function in children with nephrotic syndrome.

Methods: The study evaluated 191 participants younger than 18 years enrolled in the Nephrotic Syndrome Study Network, including 118 with their first clinically indicated kidney biopsy (68 minimal change disease; 50 focal segmental glomerulosclerosis) and 73 with incident nephrotic syndrome without a biopsy. uEGF was measured at baseline for all participants and normalized by the urine …

Musculoskeletal Pain Outcomes Pre- And Post Intrathecal Baclofen Pump Implant In Children With Cerebral Palsy: A Prospective Cohort Study., Chantel C. Barney, Alyssa M. Merbler, Jean Stansbury, Linda E. Krach, Michael D. Partington, Patrick Graupman, Peter D. Kim, Debbie Song, Frank J. Symons

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Objective: To characterize musculoskeletal pain intensity, duration, frequency, and interference with activities of daily living in children with cerebral palsy (CP) before and after intrathecal baclofen pump placement.

Design: Prospective cohort study.

Setting: Children's tertiary hospital.

Participants: Participants were children with CP (N=32; 53% male; mean age, 9.9y; age range, 4-17y). The majority of participants had a CP diagnosis of quadriplegia (76%) and relied on wheeled mobility (91%).

Interventions: Assessments were completed pre- and post intrathecal baclofen pump implant.

Main outcome measures: Because of considerable patient heterogeneity, both pain measures (Brief Pain Inventory, Dalhousie Pain Interview) were completed by proxy …

Incidence Of Initial Renal Replacement Therapy Over The Course Of Kidney Disease In Children., Derek K. Ng, Matthew B. Matheson, Bradley A. Warady, Susan R. Mendley, Susan L. Furth, Alvaro Muñoz

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The Chronic Kidney Disease in Children Study, a prospective cohort study with data collected from 2003 to 2018, provided the first opportunity to characterize the incidence of renal replacement therapy (RRT) initiation over the life course of pediatric kidney diseases. In the current analysis, parametric generalized gamma models were fitted and extrapolated for RRT overall and by specific treatment modality (dialysis or preemptive kidney transplant). Children were stratified by type of diagnosis: nonglomerular (mostly congenital; n = 650), glomerular-hemolytic uremic syndrome (HUS; n = 49), or glomerular-non-HUS (heterogeneous childhood onset; n = 216). Estimated durations of time to RRT after …