Pediatrics Commons^™

Identifying And Improving System Barriers To Promote Successful Breastfeeding In Resident Physicians, Brigette Schuman, Michelle Drobny, Elizabeth Savage, Colleen Zimmermann

Articles, Abstracts, and Reports

No abstract provided.

Phlebotomy-Free Days In Common Conditions Among Hospitalized Children And The Association With Clinical Outcomes, Megan Collins, M Hall, Ss Shah, Mj Molloy, Pl Aronson, Jm Cotter, Mj Steiner, Mj Tchou, Jr Stephens, Jessica L. Markham

Posters

Background: Phlebotomy is an invasive procedure associated with pain and iatrogenic anemia. Minimizing phlebotomy for hospitalized children could improve their experience and avoid unnecessary tests. Objective: To describe: 1) the prevalence of phlebotomy-free days among children hospitalized with common conditions and 2) the association of phlebotomy-free days with clinical outcomes. Design/Methods: We performed a multicenter, cross-sectional study of children hospitalized 1/1/2018 to 12/31/2019 with an All Patient Refined Diagnosis Related Group (APR-DRG) for common infections across 38 hospitals in the Pediatric Health Information System (PHIS) database. We excluded patients with length of stay (LOS) < 2 days, medical complexity, interhospital transfers, and those receiving intensive care. We defined phlebotomy-free days (PFDs) as hospital days with no laboratory blood testing and measured the proportion of PFDs per hospital day (PFD ratio) for each condition and hospital. Hospitals were grouped into low, moderate and high average PFD ratios. Adjusted outcomes were compared across groups and included LOS, costs, and all-cause 14- and 30-day readmission rates. Results: We identified 126,135 patient encounters (Table 1). Bronchiolitis (N=31,302), non-bacterial gastroenteritis (N=20,430), and pneumonia (N=16,031) accounted for the greatest number of hospital days. Bronchiolitis (0.78) and pneumonia (0.54) had the highest overall PFD ratios, while bone and joint infections (0.28) and non-bacterial gastroenteritis (0.30) had the lowest overall PFD ratios. There was wide variation across hospitals and conditions in PFD ratios (Figure 1). We identified 8 hospitals with low, 21 with moderate, and 9 with high PFD ratios (Figure 1 and Table 1). There were statistically significant but small differences in the distributions of age, payer, and H-RISK among patients in the low, moderate, and high PFD hospital groups (Table 1). There were no differences in adjusted outcomes across low, moderate, and high PFD hospital groups (Table 2). Conclusion(s): Among children hospitalized with common infectious conditions, there was variation across conditions and hospitals in the proportion of PFDs per hospital day. Hospitals with low, moderate and high ratios of PFDs had no differences in outcomes. Our data suggest at least some laboratory overuse and opportunities to improve the experience and care of children hospitalized with infections.

Sepsis-Induced Acute Lung Injury And The Development Of Bronchopulmonary Dysplasia In Premature Infants, Jacob S. Ward, Hung-Wen Yeh, Megan Tucker, Venkatesh Sampath

Posters

Background: Bronchopulmonary dysplasia (BPD) is a significant cause of morbidity and mortality in premature infants with several known risk factors. Recent literature described the harmful effect of inflammation on the developing lung. Recently, we showed that late-onset sepsis (LOS) and necrotizing enterocolitis (NEC) causes acute lung injury (ALI) in preterm infants as measured by increases in pulmonary severity scores (PSS). The aim of this study was to investigate the association between LOS-induced ALI and BPD outcomes. We hypothesized that LOS would increase the likelihood of developing BPD, and the associated LOS-induced ALI will add to the ability to predict development …

Implementing Team Birth To Improve Patient Satisfaction On An Ldrp (Team Birthsingleroom ) Unit, Deanna Higgins, Alyson Willard, Teresa Rangel

Articles, Abstracts, and Reports

No abstract provided.

Treatment Of Post-Operative Pain In Children With Severe Neurological Impairment, Jordan Keys

Presentations

Background: The assessment of pain in children with severe neurologic impairment (SNI) can be more challenging than in neurotypical children.

Objective: To describe the type, number of classes, and duration of post-operative pain medications for procedures common among children with SNI. Secondarily, to describe the variability in pain management strategies across children’s hospitals, specifically for opioid administration.

Design/Methods: This retrospective cohort study of the Pediatric Health Information System included children hospitalized with SNI aged 0-21 years old who underwent common procedures during 2019. We defined SNI using previously described high-intensity neurologic impairment (HINI) diagnosis codes and identified six common ( …

Pediatric Malaria With Respiratory Distress: Prognostic Significance Of Point-Of-Care Lactate, Catherine Mitran, Robert Opoka, Andrea Conroy, Sophie Namasopo, Kevin Kain, Michael Hawkes

Paediatrics and Child Health, East Africa

Respiratory distress (RD) in pediatric malaria portends a grave prognosis. Lactic acidosis is a biomarker of severe disease. We investigated whether lactate, measured at admission using a handheld device among children hospitalized with malaria and RD, was predictive of subsequent mortality. We performed a pooled analysis of Ugandan children under five years of age hospitalized with malaria and RD from three past studies. In total, 1324 children with malaria and RD (median age 1.4 years, 46% female) from 21 health facilities were included. Median lactate level at admission was 4.6 mmol/L (IQR 2.6–8.5) and 586 patients (44%) had hyperlactatemia (lactate …

Examining The Feasibility Of Telehealth Trauma-Focused Cognitive Behavioural Therapy (Tf-Cbt) With Young People In Foster Care, Jill Mcleigh, Adrianna Martin, Laura Lamminen

Behavioral Health/Developmental Delay

Despite the high rate of trauma exposure among young people with child welfare involvement, various systematic and patient barriers exist that inhibit utilization of evidence-based trauma treatments. One strategy for alleviating barriers to such treatments is using telehealth. A few studies have found that the clinical outcomes of telehealth TF-CBT are comparable to those found from clinic-based, in-person treatment administration. Studies have yet to examine the feasibility of telehealth TF-CBT with young people in care. The current study sought to address this gap by examining outcomes for patients who received telehealth TF-CBT, along with factors that may have impacted successful …

Using Multiple Imputation Of Real-World Data To Estimate Clinical Remission In Pediatric Inflammatory Bowel Disease, Nanhua Zhang, Chunyan Liu, Steven J. Steiner, Richard B. Colletti, Robert Baldassano, Shiran Chen, Stanley Cohen, Michael D. Kappelman, Shehzad Saeed, Laurie S. Conklin, Richard Strauss, Sheri Volger, Eileen King, Kim Hung Lo

Pediatrics Faculty Publications

Aim: To evaluate the performance of the multiple imputation (MI) method for estimating clinical effectiveness in pediatric Crohn's disease in the ImproveCareNow registry; to address the analytical challenge of missing data. Materials & methods: Simulation studies were performed by creating missing datasets based on fully observed data from patients with moderate-to-severe Crohn's disease treated with non-ustekinumab biologics. MI was used to impute sPCDAI remission statuses in each simulated dataset. Results: The true remission rate (75.1% [95% CI: 72.6%, 77.5%]) was underestimated without imputation (72.6% [71.8%, 73.3%]). With MI, the estimate was 74.8% (74.4%, 75.2%). Conclusion: MI reduced nonresponse bias and …

Acute Necrotizing Encephalopathy In An Infant: A Case Report, Elisamia Ngowi, Zeenat Juneja, Jacqueline Gabone, Maria Bulimba, Mohamedraza Ebrahim, Yaser Abdallah

Paediatrics and Child Health, East Africa

Background: Acute necrotizing encephalopathy is a rare, severe neurological condition, characterized by symmetrical necrotic brain lesions affecting the thalamus, cerebral white matter, brain stem, and cerebellum. Diagnosis relies on characteristic clinical and radiological features.

Methodology: We describe a 10-month-old female infant presenting with multiple seizures following a brief febrile upper respiratory illness.

Findings: MRI of the brain showed bilateral symmetrical abnormal signal intensities involving both thalami and caudate nucleus, consistent with ANE. Management of ANE is symptom-related but high-dose steroids and IVIG have been used.

Conclusion: This case highlights the importance of recognizing ANE beyond its …

Exploring Modern Trends And Prevention Strategies For Childhood Obesity: A Comprehensive Literature Review, Anya E. Bogen

Honors Program: Senior Projects (Public)

This literature review seeks to provide an overview of the current research on childhood obesity. The study examines the prevalence and causes of childhood obesity, including genetic, environmental, and behavioral factors. The review also discusses the health consequences associated with childhood obesity, such as the increased risk for chronic diseases and psychological and social problems. Additionally, the review explores the effectiveness of interventions designed to prevent childhood obesity, including individual lifestyle modifications, education from health practitioners and school programs, and global, national, and state programs to promote wellness. The literature review concludes that childhood obesity is a complex and multifactorial …

Innovative Management Of Posterior Upper Thigh Pain In Two Adolescent Athletes, Thomas Munro, Brian Harvey

Posters

Case history: Two 16-year-old males with posterior upper leg pain. Athlete A reports he had been exploding up to dunk a basketball when he felt a pop with immediate pain in his posterior LLE. He was treated conservatively with activity modification and PT resulting in improvement of symptoms and return to full activity. He returned 15-months after the initial injury due to recurrence of pain. Athlete B stated he had tweaked his hamstring multiple times over the summer and had been working with a physical therapist for rehabilitation. He had attempted full rest for a couple weeks with graded return …

A Metabolic, Mechanical, Multi-Organ Masterpiece: Dural Device Support Bridge To En-Bloc Heart-Liver Transplantation In Propionic Acidemia, Rebecca Juhl, Brian Birnbaum, Aliessa P. Barnes, William Gibson, Bhargava Mullapudi, Beth Lang, Megan Faseler, Daniel E. Heble, Victoria Urban, Ryan T. Fischer, Jennifer L. Gannon, David Sutcliffe

Posters

Introduction: Propionic Acidemia (PA) is a disorder related to abnormal protein and lipid metabolism resulting in progressive neurological injury and dilated cardiomyopathy (DCM). Interventions for PA and secondary disease manifestations can require multi-organ transplantation. Herein we report the case of a child with PA and end-stage DCM requiring left ventricular assist device (LVAD) support with eventual heart-liver transplant. Case Report: A 17 year old male diagnosed in childhood with PA developed chronically progressive DCM culminating in end stage heart failure with acute decompensations. In a recurrent admission, he progressed to require dual inotropic support and systemic anticoagulation for new LV …

Consensus-Based Evaluation Of Outcome Measures In Pediatric Stroke Care: A Toolkit, Samantha J Feldman, Lauren A Beslow, Ryan J Felling, Laura A Malone, Michaela Waak, Stuart Fraser, Nihal Bakeer, Jo Ellen M Lee, Victoria Sherman, Melissa M Howard, Beth Anne Cavanaugh, Robyn Westmacott, Lori C Jordan

Faculty, Staff and Student Publications

Following a pediatric stroke, outcome measures selected for monitoring functional recovery and development vary widely. We sought to develop a toolkit of outcome measures that are currently available to clinicians, possess strong psychometric properties, and are feasible for use within clinical settings. A multidisciplinary group of clinicians and scientists from the International Pediatric Stroke Organization comprehensively reviewed the quality of measures in multiple domains described in pediatric stroke populations including global performance, motor and cognitive function, language, quality of life, and behavior and adaptive functioning. The quality of each measure was evaluated using guidelines focused on responsiveness and sensitivity, reliability, …

Monogenic Early-Onset Lymphoproliferation And Autoimmunity: Natural History Of Stat3 Gain-Of-Function Syndrome, Jennifer W Leiding, Tiphanie P Vogel, Valentine G J Santarlas, Rahul Mhaskar, Madison R Smith, Alexandre Carisey, Alexander Vargas-Hernández, Manuel Silva-Carmona, Maximilian Heeg, Anne Rensing-Ehl, Bénédicte Neven, Jérôme Hadjadj, Sophie Hambleton, Timothy Ronan Leahy, Kornvalee Meesilpavikai, Charlotte Cunningham-Rundles, Cullen M Dutmer, Svetlana O Sharapova, Mervi Taskinen, Ignatius Chua, Rosie Hague, Christian Klemann, Larysa Kostyuchenko, Tomohiro Morio, Akaluck Thatayatikom, Ahmet Ozen, Anna Scherbina, Cindy S Bauer, Sarah E Flanagan, Eleonora Gambineri, Lisa Giovannini-Chami, Jennifer Heimall, Kathleen E Sullivan, Eric Allenspach, Neil Romberg, Sean G Deane, Benjamin T Prince, Melissa J Rose, John Bohnsack, Talal Mousallem, Rohith Jesudas, Maria Marluce Dos Santos Vilela, Michael O'Sullivan, Jana Pachlopnik Schmid, Štěpánka Průhová, Adam Klocperk, Matthew Rees, Helen Su, Sami Bahna, Safa Baris, Lisa M Bartnikas, Amy Chang Berger, Tracy A Briggs, Shannon Brothers, Vanessa Bundy, Alice Y Chan, Shanmuganathan Chandrakasan, Mette Christiansen, Theresa Cole, Matthew C Cook, Mukesh M Desai, Ute Fischer, David A Fulcher, Silvanna Gallo, Amelie Gauthier, Andrew R Gennery, José Gonçalo Marques, Frédéric Gottrand, Bodo Grimbacher, Eyal Grunebaum, Emma Haapaniemi, Sari Hämäläinen, Kaarina Heiskanen, Tarja Heiskanen-Kosma, Hal M Hoffman, Luis Ignacio Gonzalez-Granado, Anthony L Guerrerio, Leena Kainulainen, Ashish Kumar, Monica G Lawrence, Carina Levin, Timi Martelius, Olaf Neth, Peter Olbrich, Alejandro Palma, Niraj C Patel, Tamara Pozos, Kahn Preece, Saúl Oswaldo Lugo Reyes, Mark A Russell, Yael Schejter, Christine Seroogy, Jan Sinclair, Effie Skevofilax, Daniel Suan, Daniel Suez, Paul Szabolcs, Helena Velasco, Klaus Warnatz, Kelly Walkovich, Austen Worth, Stat3 Gof Working Group Members, Mikko R J Seppänen, Troy R Torgerson, Georgios Sogkas, Stephan Ehl, Stuart G Tangye, Megan A Cooper, Joshua D Milner, Lisa R Forbes Satter

Faculty, Staff and Students Publications

BACKGROUND: In 2014, germline signal transducer and activator of transcription (STAT) 3 gain-of-function (GOF) mutations were first described to cause a novel multisystem disease of early-onset lymphoproliferation and autoimmunity.

OBJECTIVE: This pivotal cohort study defines the scope, natural history, treatment, and overall survival of a large global cohort of patients with pathogenic STAT3 GOF variants.

METHODS: We identified 191 patients from 33 countries with 72 unique mutations. Inclusion criteria included symptoms of immune dysregulation and a biochemically confirmed germline heterozygous GOF variant in STAT3.

RESULTS: Overall survival was 88%, median age at onset of symptoms was 2.3 years, and median …

Genomic Analysis Of Two Phlebotomine Sand Fly Vectors Of Leishmania From The New And Old World, Frédéric Labbé, Maha Abdeladhim, Jenica Abrudan, Alejandra Saori Araki, Ricardo N Araujo, Peter Arensburger, Joshua B Benoit, Reginaldo Pecanha Brazil, Rafaela V Bruno, Gustavo Bueno Da Silva Rivas, Vinicius Carvalho De Abreu, Jason Charamis, Iliano V Coutinho-Abreu, Samara G Da Costa-Latgé, Alistair Darby, Viv M Dillon, Scott J Emrich, Daniela Fernandez-Medina, Nelder Figueiredo Gontijo, Catherine M Flanley, Derek Gatherer, Fernando A Genta, Sandra Gesing, Gloria I Giraldo-Calderón, Bruno Gomes, Eric Roberto Guimaraes Rocha Aguiar, James G C Hamilton, Omar Hamarsheh, Mallory Hawksworth, Jacob M Hendershot, Paul V Hickner, Jean-Luc Imler, Panagiotis Ioannidis, Emily C Jennings, Shaden Kamhawi, Charikleia Karageorgiou, Ryan C Kennedy, Andreas Krueger, José M Latorre-Estivalis, Petros Ligoxygakis, Antonio Carlos A Meireles-Filho, Patrick Minx, Jose Carlos Miranda, Michael J Montague, Ronald J Nowling, Fabiano Oliveira, João Ortigão-Farias, Marcio G Pavan, Marcos Horacio Pereira, Andre Nobrega Pitaluga, Roenick Proveti Olmo, Marcelo Ramalho-Ortigao, José M C Ribeiro, Andrew J Rosendale, Mauricio R V Sant'anna, Steven E Scherer, Nágila F C Secundino, Douglas A Shoue, Caroline Da Silva Moraes, João Silveira Moledo Gesto, Nataly Araujo Souza, Zainulabueddin Syed, Samuel Tadros, Rayane Teles-De-Freitas, Erich L Telleria, Chad Tomlinson, Yara M Traub-Csekö, João Trindade Marques, Zhijian Tu, Maria F Unger, Jesus Valenzuela, Flávia V Ferreira, Karla P V De Oliveira, Felipe M Vigoder, John Vontas, Lihui Wang, Gareth D Weedall, Elyes Zhioua, Stephen Richards, Wesley C Warren, Robert M Waterhouse, Rod J Dillon, Mary Ann Mcdowell

Faculty, Staff and Students Publications

Phlebotomine sand flies are of global significance as important vectors of human disease, transmitting bacterial, viral, and protozoan pathogens, including the kinetoplastid parasites of the genus Leishmania, the causative agents of devastating diseases collectively termed leishmaniasis. More than 40 pathogenic Leishmania species are transmitted to humans by approximately 35 sand fly species in 98 countries with hundreds of millions of people at risk around the world. No approved efficacious vaccine exists for leishmaniasis and available therapeutic drugs are either toxic and/or expensive, or the parasites are becoming resistant to the more recently developed drugs. Therefore, sand fly and/or reservoir control …

Discrepancies In Age-Based Medication Dosing, Chad Ruger, Anne Keeling, Sera Jacob, Cassidy Macarthur

Articles, Abstracts, and Reports

No abstract provided.

Leadership Across The Career Continuum: Using Self-Reflection Effective Leadership Practices For Optimal Team Functioning, Emily Bonanni, Grant M. Latta, Michael Platt, Christine Irvin, Angela Etzenhouser, Megan Collins, Sian Best, Jonathan Ermer, Joy L. Solano, Jacqueline M. Walker

Presentations

Have you ever found yourself in a formal or informal leadership role and felt underprepared because of a lack of formal leadership training? Have you noticed unused potential in those around you and wondered how you maximize it? As academic pediatricians, we are identified as leaders by patients and their families, trainees of all levels, members of our multi-disciplinary care teams, and our peers. We are a group of passionate clinician educators who have found ourselves in both formal and informal leadership roles and have found success in these roles with leadership training. Our interactive workshop will give you the …

Insidious Ankle Swelling, Catharine Kral, Brian Harvey

Posters

10-year-old female presented to clinic for left heel pain and swelling. She participates in dance for an estimated 8-9 hours a week. Symptoms started four weeks prior to presentation. She does not remember any inciting event, fall, trauma, or other mechanism of injury prior to onset. She says the pain is localized to the lateral aspect of her calcaneus and extending up to her Achilles. She has never seen any bruising, erythema, or skin changes overlaying the swollen region. Family reports she intermittently has a limp. It is most tender to touch or when she bumps her foot on something. …

Infusion Reaction In Hem/Onc Patients, Children's Mercy Kansas City

Clinical Pathways (Formally Known As Care Process Models)

No abstract provided.

Use Of High-Flow Cannula In Pediatric Patients With Respiratory Failure: A Prospective Cohort Study In Three High-Altitude Hospitals, Natalia Ante-Ardila, Camilo Novoa Garnica, Paola Mora Umaña, Olga Lucía Baquero Castañeda, Alexandra Jiménez Cháves, Melisa Sofía Naranjo, Juan G Piñeros, Carolina Bonilla, Luz M Mejía, María L Mesa-Rubio, Sonia Restrepo-Gualteros, Pedro Barrera, Sergio Moreno-Lopez, Paola Rueda-Guevara, Andrea Ramírez Varela

Faculty, Staff and Student Publications

BACKGROUND AND AIMS: Acute respiratory failure (ARF) is a common cause of morbimortality, and a frequent reason for admission to the pediatric intensive care unit (PICU). It requires a high-flow oxygen device as treatment. Our aim is to determine the frequency and main indications for the use of high-flow nasal cannula (HFNC), and the prevalence of HFNC failure and its main causes, in three hospitals.

METHODS: It is a multicenter prospective cohort study, developed in three hospitals in Bogota. Eligible patients were children older than 1 month and younger than 18 years who presented ARF and required management with an …

Sports Medicine Eras, Children's Mercy Kansas City

Clinical Pathways (Formally Known As Care Process Models)

No abstract provided.

Use Of High-Flow Cannula In Pediatric Patients With Respiratory Failure: A Prospective Cohort Study In Three High-Altitude Hospitals, Natalia Ante-Ardila, Camilo Novoa Garnica, Paola Mora Umaña, Olga Lucía Baquero Castañeda, Alexandra Jiménez Cháves, Melisa Sofía Naranjo, Juan G Piñeros, Carolina Bonilla, Luz M Mejía, María L Mesa-Rubio, Sonia Restrepo-Gualteros, Pedro Barrera, Sergio Moreno-Lopez, Paola Rueda-Guevara, Andrea Ramírez Varela, Fara Group

Faculty, Staff and Student Publications

BACKGROUND AND AIMS: Acute respiratory failure (ARF) is a common cause of morbimortality, and a frequent reason for admission to the pediatric intensive care unit (PICU). It requires a high-flow oxygen device as treatment. Our aim is to determine the frequency and main indications for the use of high-flow nasal cannula (HFNC), and the prevalence of HFNC failure and its main causes, in three hospitals.

METHODS: It is a multicenter prospective cohort study, developed in three hospitals in Bogota. Eligible patients were children older than 1 month and younger than 18 years who presented ARF and required management with an …

Sexual Health And Relationship Abuse Interventions In Pediatric Primary Care: A Systematic Review, Susheel Kant Khetarpal, Sarah Tiffany-Appleton, Erin E Mickievicz, Romina L Barral, Kimberly A Randell, Jeff R Temple, Elizabeth Miller, Maya I Ragavan

Faculty, Staff and Student Publications

PURPOSE: Supporting adolescents in developing healthy relationships and promoting sexual and reproductive health (SRH) is an important responsibility of pediatric primary care providers. Less is known about evidence-based interventions in pediatric settings focused on healthy relationships and SRH.

METHODS: We conducted a systematic review to describe SRH and healthy relationship/adolescent relationship abuse (ARA) interventions for pediatric primary care over the past 20 years. Eligible articles were original research on an SRH-focused or ARA-focused intervention, conducted in-person within pediatric primary care or school-based health centers specifically for middle or high school-aged adolescents. Data abstracted from included articles included intervention description, content, …

Recruitment And Retention Of Primary Care Practices In The Southeastern Collaboration To Improve Blood Pressure Control, James M Shikany, Monika M Safford, Andrea L Cherrington, Jacqueline R Halladay, Muna Anabtawi, Erica L Richman, Alyssa D Adams, Charlotte Holt, Suzanne Oparil, Orysya Soroka, Doyle M Cummings

Faculty, Staff and Student Publications

BACKGROUND: Racial disparities related to hypertension prevalence and control persist, with Black persons continuing to have both high prevalence and suboptimal control. The Black Belt region of the US Southeast is characterized by multiple critical priority populations: rural, low-income, and minority (Black).

METHODS: In a cluster-randomized, controlled, pragmatic implementation trial, the Southeastern Collaboration to Improve Blood Pressure Control evaluated two multi-component, multi-level functional interventions - peer coaching (PC) and practice facilitation (PF) (separately and combined) - as adjuncts to usual care to improve blood pressure control in the Black Belt. The overall goal was to randomize 80 primary care practices …

Bronchiolitis: Hypertonic Saline, Children's Mercy Kansas City

Clinical Critically Appraised Topics

No abstract provided.

Febrile Young Infants Age <=60d With Covid-19 Infection: Review Of Clinical Presentation, Inflammatory Markers, Hospitalization Rate And Clinical Outcomes, Megumi T Mori, Tibisay Villalobos Md, Susan K. Yaeger Md, Claudia F. Busse Md

Department of Pediatrics

No abstract provided.

Nfkb2 Haploinsufficiency Identified Via Screening For Ifn-Α2 Autoantibodies In Children And Adolescents Hospitalized With Sars-Cov-2-Related Complications, Aaron Bodansky, Sara E Vazquez, Janet Chou, Tanya Novak, Amer Al-Musa, Cameron Young, Margaret Newhams, Suden Kucukak, Laura D Zambrano, Anthea Mitchell, Chung-Yu Wang, Kristin Moffitt, Natasha B Halasa, Laura L Loftis, Stephanie P Schwartz, Tracie C Walker, Elizabeth H Mack, Julie C Fitzgerald, Shira J Gertz, Courtney M Rowan, Katherine Irby, Ronald C Sanders, Michele Kong, Jennifer E Schuster, Mary A Staat, Matt S Zinter, Natalie Z Cvijanovich, Keiko M Tarquinio, Bria M Coates, Heidi R Flori, Mary K Dahmer, Hillary Crandall, Melissa L Cullimore, Emily R Levy, Brandon Chatani, Ryan Nofziger, Overcoming Covid-19 Network Study Group Investigators, Raif S Geha, Joseph Derisi, Angela P Campbell, Mark Anderson, Adrienne G Randolph

Faculty, Staff and Students Publications

BACKGROUND: Autoantibodies against type I IFNs occur in approximately 10% of adults with life-threatening coronavirus disease 2019 (COVID-19). The frequency of anti-IFN autoantibodies in children with severe sequelae of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection is unknown.

OBJECTIVE: We quantified anti-type I IFN autoantibodies in a multicenter cohort of children with severe COVID-19, multisystem inflammatory syndrome in children (MIS-C), and mild SARS-CoV-2 infections.

METHODS: Circulating anti-IFN-α2 antibodies were measured by a radioligand binding assay. Whole-exome sequencing, RNA sequencing, and functional studies of peripheral blood mononuclear cells were used to study any patients with levels of anti-IFN-α2 autoantibodies exceeding …

Incidence Rates Of Medically Attended Covid-19 In Infants Less Than 6 Months Of Age, Isabel Griffin, Stephanie A Irving, Carmen Sofia Arriola, Angela P Campbell, De-Kun Li, Fatimah S Dawood, Caroline Doughty-Skierski, Jeannette R Ferber, Nickolas Ferguson, Louise Hadden, Jillian T Henderson, Mary Juergens, Venkatesh Kancharla, Allison L Naleway, Gabriella Newes-Adeyi, Erin Nicholson, Roxana Odouli, Lawrence Reichle, Mo Sanyang, Kate Woodworth, Flor M Munoz

Faculty, Staff and Students Publications

BACKGROUND: Studies suggest infants may be at increased risk of severe coronavirus disease 2019 (COVID-19) relative to older children, but few data exist regarding the incidence of COVID-19 episodes and associated risk factors. We estimate incidence rates and describe characteristics associated with medically attended COVID-19 episodes among infants younger than 6 months of age.

METHODS: We analyzed electronic medical record data from a cohort of infants born March 1, 2020-February 28, 2021. Data from 3 health care delivery systems included demographic characteristics, maternal and infant outpatient visit and hospitalization diagnoses and severe acute respiratory syndrome coronavirus syndrome 2 (SARS-CoV-2) test …

Efficacy And Safety Of Lumasiran For Infants And Young Children With Primary Hyperoxaluria Type 1: 12-Month Analysis Of The Phase 3 Illuminate-B Trial, Wesley Hayes, David J Sas, Daniella Magen, Hadas Shasha-Lavsky, Mini Michael, Anne-Laure Sellier-Leclerc, Julien Hogan, Taylor Ngo, Marianne T Sweetser, John M Gansner, Tracy L Mcgregor, Yaacov Frishberg

Faculty, Staff and Students Publications

BACKGROUND: Primary hyperoxaluria type 1 (PH1) is a rare genetic disease that causes progressive kidney damage and systemic oxalosis due to hepatic overproduction of oxalate. Lumasiran demonstrated efficacy and safety in the 6-month primary analysis period of the phase 3, multinational, open-label, single-arm ILLUMINATE-B study of infants and children < 6 years old with PH1 (ClinicalTrials.gov: NCT03905694 (4/1/2019); EudraCT: 2018-004,014-17 (10/12/2018)). Outcomes in the ILLUMINATE-B extension period (EP) for patients who completed ≥ 12 months on study are reported here.

METHODS: Of the 18 patients enrolled in the 6-month primary analysis period, all entered the EP and completed ≥ 6 additional months of lumasiran treatment (median (range) duration of total exposure, 17.8 (12.7-20.5) months).

RESULTS: Lumasiran treatment was previously reported to reduce spot urinary oxalate:creatinine ratio by 72% at month 6, …

Insulin-Like Growth Factor-1 Supplementation Promotes Brain Maturation In Preterm Pigs, Line I Christiansen, Bo Holmqvist, Xiaoyu Pan, Kristine Holgersen, Sandy E H Lindholm, Nicole L Henriksen, Douglas G Burrin, David Ley, Thomas Thymann, Per Torp Sangild, Stanislava Pankratova

Faculty, Staff and Students Publications

Very preterm infants show low levels of insulin-like growth factor-1 (IGF-1), which is associated with postnatal growth restriction and poor neurologic outcomes. It remains unknown whether supplemental IGF-1 may stimulate neurodevelopment in preterm neonates. Using cesarean-delivered preterm pigs as a model of preterm infants, we investigated the effects of supplemental IGF-1 on motor function and on regional and cellular brain development. Pigs were treated with 2.25 mg/kg/d recombinant human IGF-1/IGF binding protein-3 complex from birth until day 5 or 9 before the collection of brain samples for quantitative immunohistochemistry (IHC), RNA sequencing, and quantitative PCR analyses. Brain protein synthesis was …