Pediatrics Commons^™

Ispad Clinical Practice Consensus Guidelines 2024: Screening, Staging, And Strategies To Preserve Beta-Cell Function In Children And Adolescents With Type 1 Diabetes, Michael J Haller, Kirstine J Bell, Rachel E J Besser, Kristina Casteels, Jenny J Couper, Maria E Craig, Helena Elding Larsson, Laura Jacobsen, Karin Lange, Tal Oron, Emily K Sims, Cate Speake, Mustafa Tosur, Francesca Ulivi, Anette-G Ziegler, Diane K Wherrett, M Loredana Marcovecchio

Faculty, Staff and Students Publications

The International Society for Pediatric and Adolescent Diabetes (ISPAD) guidelines represent a rich repository that serves as the only comprehensive set of clinical recommendations for children, adolescents, and young adults living with diabetes worldwide. This guideline serves as an update to the 2022 ISPAD consensus guideline on staging for type 1 diabetes (T1D). Key additions include an evidence-based summary of recommendations for screening for risk of T1D and monitoring those with early-stage T1D. In addition, a review of clinical trials designed to delay progression to Stage 3 T1D and efforts seeking to preserve beta-cell function in those with Stage 3 …

Wall Motion Assessment By Feature Tracking In Pediatric Patients With Coronary Anomalies Undergoing Dobutamine Stress Cmr, Shagun Sachdeva, Silvana Molossi, Dana Reaves-O'Neal, Prakash Masand, Tam T Doan

Faculty, Staff and Students Publications

BACKGROUND: Left ventricular (LV) wall motion assessment is an important adjunct in addition to perfusion defects in assessing ischemic changes. This study aims to investigate the feasibility and utility of performing feature tracking (FT) in pediatric patients with coronary anomalies undergoing dobutamine stress CMR to assess wall motion abnormalities (WMA) and perfusion defects.

METHOD: This is a retrospective study where 10 patients with an inducible first-pass perfusion (FPP) defect and 10 without were selected. Global LV circumferential strain/strain rate (GCS/GCSR) was measured at rest and at peak stress (systole and diastole) using a commercially available feature tracking software. Peak GCS …

Treatment Of High- And Intermediate-High-Risk Pulmonary Embolism By The Pulmonary Embolism Response Team: Focus On Catheter-Directed Therapies, Arkadiusz Pietrasik, Paweł Kurzyna, Piotr Szwed, Karolina Jasińska-Gniadzik, Aleksandra Gąsecka, Szymona Darocha, Dariusz Zieliński, Łukasz Szarpak, Janusz Kochman, Marcin Grabowski, Grzegorz Opolski, Adam Torbicki, Marcin Kurzyna

Faculty, Staff and Students Publications

BACKGROUND: Multidisciplinary Pulmonary Embolism Response Teams (PERTs) were established to individualize the treatment of high-risk (HR) and intermediate-high-risk (IHR) pulmonary embolism (PE) patients, which pose a challenge in clinical practice.

METHODS: We retrospectively collected the data of all HR and IHR acute PE patients consulted by PERT CELZAT between September 2017 and October 2022. The patient population was divided into four different treatment methods: anticoagulation alone (AC), systemic thrombolysis (ST), surgical embolectomy (SE), and catheter-directed therapies (CDTx). Baseline clinical characteristics, risk stratification, PE severity parameters, and treatment outcomes were compared between the four groups.

RESULTS: Of the 110 patients with …

Meta-Analysis Of Postoperative Myocardial Injury As A Predictor Of Mortality After Living Donor Liver Transplantation, Krzysztof Jankowski, Frank W Peacock, Michal Pruc, Teresa Malecka-Massalska, Lukasz Szarpak

Faculty, Staff and Students Publications

BACKGROUND: The purpose of this study was to perform a systematic review and meta-analysis to investigate postoperative myocardial injury, as expressed by the postoperative concentration of high-sensitivity cardiac troponin I (hs-cTnI) as a predictor of mortality among living donor liver transplantation (LDLT) patients.

METHODS: PubMed, Scopus, Embase and the Cochrane Library were searched through to September 1st 2022. The primary endpoint included in-hospital mortality. Secondary endpoints were 1-year mortality and re-transplantation occurrence. Estimates are expressed as risk ratios (RRs) and 95% confidence intervals (95% CIs). Heterogeneity was assessed with the I² test.

RESULTS: During the search, 2 studies were found …

Early Administration Of Steroids In The Ambulance Setting: An Observational Design Trial (Easi-As-Odt), Jennifer N Fishe, Gerard Garvan, Andrew Bertrand, Shannon Burcham, Phyllis Hendry, Manish Shah, Kathryn Kothari, David W Ashby, Daniel Ostermeyer, Lauren Riney, Olga Semenova, Benjamin Abo, Benjamin Abes, Nichole Shimko, Emily Myers, Marshall Frank, Tim Turner, Mac Kemp, Kim Landry, Greg Roland, Kathryn V Blake

Faculty, Staff and Students Publications

BACKGROUND: In the emergency department (ED), prompt administration of systemic corticosteroids for pediatric asthma exacerbations decreases hospital admission rates. However, there is sparse evidence for whether earlier administration of systemic corticosteroids by emergency medical services (EMS) clinicians, prior to ED arrival, further improves pediatric asthma outcomes.

METHODS: Early Administration of Steroids in the Ambulance Setting: An Observational Design Trial is a multicenter, observational, nonrandomized stepped-wedge design study with seven participating EMS agencies who adopted an oral systemic corticosteroid (OCS) into their protocols for pediatric asthma treatment. Using univariate analyses and multivariable mixed-effects models, we compared hospital admission rates for pediatric …

Race, Ethnicity, Language, And The Treatment Of Low-Risk Febrile Infants, Colleen K Gutman, Paul L Aronson, Nidhi V Singh, Michelle L Pickett, Kamali Bouvay, Rebecca S Green, Britta Roach, Hannah Kotler, Jessica L Chow, Emily A Hartford, Mark Hincapie, Ryan St Pierre-Hetz, Jessica Kelly, Laura Sartori, Jennifer A Hoffmann, Jacqueline B Corboy, Kelly R Bergmann, Bolanle Akinsola, Vanessa Ford, Natalie J Tedford, Theresa T Tran, Sasha Gifford, Amy D Thompson, Andrew Krack, Mary Jane Piroutek, Samantha Lucrezia, Sunhee Chung, Nabila Chowdhury, Kathleen Jackson, Tabitha Cheng, Christian D Pulcini, Nirupama Kannikeswaran, Larissa L Truschel, Karen Lin, Jamie Chu, Neh D Molyneaux, Myto Duong, Leslie Dingeldein, Jerri A Rose, Carly Theiler, Sonali Bhalodkar, Emily Powers, Muhammad Waseem, Ahmed Lababidi, Xinyu Yan, Xiang-Yang Lou, Rosemarie Fernandez, K Casey Lion

Faculty, Staff and Students Publications

IMPORTANCE: Febrile infants at low risk of invasive bacterial infections are unlikely to benefit from lumbar puncture, antibiotics, or hospitalization, yet these are commonly performed. It is not known if there are differences in management by race, ethnicity, or language.

OBJECTIVE: To investigate associations between race, ethnicity, and language and additional interventions (lumbar puncture, empirical antibiotics, and hospitalization) in well-appearing febrile infants at low risk of invasive bacterial infection.

DESIGN, SETTING, AND PARTICIPANTS: This was a multicenter retrospective cross-sectional analysis of infants receiving emergency department care between January 1, 2018, and December 31, 2019. Data were analyzed from December 2022 …

Expert Review Of Child And Caregiver Critiques Of A Therapeutic Guided Imagery Therapy Mobile Application Targeting Disorders Of Gut-Brain Interaction In Children, John M Hollier, Tiantá A Strickland, C Michael Fordis, Robert J Shulman, Debbe Thompson

Faculty, Staff and Students Publications

BACKGROUND: A guided imagery therapy mobile application (GIT App) is a novel platform for treating children with disorders of gut-brain interaction (DGBI). Previous feedback from child/caregiver dyads suggested modifications for our App prototype. However, their feedback had the potential to affect the intervention's efficacy. Thus, we aimed to have their critiques vetted by relevant experts prior to further App refinement.

OBJECTIVE: Compare expert reviews of the GIT App with end-users' (i.e., child/caregiver dyads') feedback.

METHODS: This mixed-methods study with experts included a hands-on App evaluation, a survey assessing usability, and focus groups comparing their perspectives with those previously provided by …

Protein Biomarkers Gdf15 And Fgf21 To Differentiate Mitochondrial Hepatopathies From Other Pediatric Liver Diseases, Johan L K Van Hove, Marisa W Friederich, Dana K Strode, Roxanne A Van Hove, Kristen R Miller, Rohit Sharma, Hardik Shah, Jane Estrella, Linda Gabel, Simon Horslen, Rohit Kohli, Mark A Lovell, Alexander G Miethke, Jean P Molleston, Rene Romero, James E Squires, Estella M Alonso, Stephen L Guthery, Binita M Kamath, Kathleen M Loomes, Philip Rosenthal, Krupa R Mysore, Laurel A Cavallo, Pamela L Valentino, John C Magee, Shikha S Sundaram, Ronald J Sokol, Childhood Liver Disease Research Network (Children)

Faculty, Staff and Students Publications

BACKGROUND: Mitochondrial hepatopathies (MHs) are primary mitochondrial genetic disorders that can present as childhood liver disease. No recognized biomarkers discriminate MH from other childhood liver diseases. The protein biomarkers growth differentiation factor 15 (GDF15) and fibroblast growth factor 21 (FGF21) differentiate mitochondrial myopathies from other myopathies. We evaluated these biomarkers to determine if they discriminate MH from other liver diseases in children.

METHODS: Serum biomarkers were measured in 36 children with MH (17 had a genetic diagnosis); 38 each with biliary atresia, α1-antitrypsin deficiency, and Alagille syndrome; 20 with NASH; and 186 controls.

RESULTS: GDF15 levels compared to controls were …

Smooth Muscle Contractile Responses To Bile Acids In Mouse Ileum Require Tgr5 But Not Asbt, Diana S Chang, Krishnakant G Soni, Geoffrey A Preidis

Faculty, Staff and Students Publications

BACKGROUND: Many disorders of gut-brain interaction (DGBIs) are more prevalent in women than men and feature alterations in gastrointestinal motility and bile acid homeostasis. Mechanisms by which bile acids regulate gastrointestinal motility are poorly characterized. We recently validated an adapted tissue bath technique using everted mouse ileum, which revealed differential contractile responses to ursodeoxycholic acid (UDCA) and deoxycholic acid (DCA). Here, we aimed to determine whether these responses are dependent on host sex, the plasma membrane bile acid receptor TGR5, or the apical sodium-dependent bile acid transporter ASBT.

METHODS: Ileal segments from male and female mice were everted and suspended …

The Impact Of Obesity-Associated Glycine Deficiency On The Elimination Of Endogenous And Exogenous Metabolites Via The Glycine Conjugation Pathway, Hong Chang Tan, Jean W Hsu, E Shyong Tai, Shaji Chacko, Jean-Paul Kovalik, Farook Jahoor

Faculty, Staff and Students Publications

BACKGROUND: Glycine is an integral component of the human detoxification system as it reacts with potentially toxic exogenous and endogenously produced compounds and metabolites via the glycine conjugation pathway for urinary excretion. Because individuals with obesity have reduced glycine availability, this detoxification pathway may be compromised. However, it should be restored after bariatric surgery because of increased glycine production.

OBJECTIVE: To examine the impact of obesity-associated glycine deficiency on the glycine conjugation pathway. We hypothesize that the synthesis rates of acylglycines from endogenous and exogenous sources are significantly reduced in individuals with obesity but increase after bariatric surgery.

METHODS: We …

Diagnosis And Management Of Shrimp Allergy, Bin Brenda Su, Warren Blackmon, Chun Xu, Christopher Holt, Nathaniel Boateng, Darren Wang, Vibha Szafron, Aikaterini Anagnostou, Sara Anvari, Carla M Davis

Faculty, Staff and Students Publications

Shrimp allergy, the most common food allergy in the United States, affects up to 2% of the population. Its etiology is multi-factorial with the combination of genetic predisposition and environmental exposures. This review summarizes the latest diagnosis and management strategies for shrimp allergy. Currently, the double-blind, placebo-controlled food challenge is the gold standard for diagnosis. Moreover, mainstream and experimental management strategies include food allergen avoidance, the FDA-approved omalizumab, and oral immunotherapy. Herein, we emphasize the urgent need to develop more effective diagnostic tools and therapies for shrimp allergy.

Applying Market Basket Analysis To Determine Complex Coassociations Among Food Allergens In Children With Food Protein-Induced Enterocolitis Syndrome (Fpies), Ankona Banerjee, Kenneth Nobleza, Cynthia Haddad, Joshua Eubanks, Ruchit Rana, Nicholas L Rider, Lisa Pompeii, Duc Nguyen, Sara Anvari

Faculty, Staff and Students Publications

BACKGROUND: Food protein-induced enterocolitis syndrome (FPIES) is a non-IgE-mediated food allergy, characterized by delayed onset of repetitive vomiting occurring 1 to 4 h following ingestion of a food allergen. Managing FPIES requires strict avoidance of the food trigger. The concern with FPIES is determining the risk of another FPIES food trigger reaction due to potential coassociations with other foods or food groups. An effective statistical approach for analyzing FPIES-related data is essential to identify common coallergens and their associations.

METHODS: This study employed Market Basket Analysis, a data-mining technique, to examine correlations and patterns among allergens in FPIES patients at …

Levofloxacin Prophylaxis For Pediatric Leukemia Patients: Monitoring Of Outcomes For Sustained Benefit And Consequences, Andrea L Davis, Alexandra M Stevens, Julienne Brackett, Lucila Marquez, Catherine E Foster, Adriana Sarmiento Clemente, Hannah E Sauer, Grant T Stimes, Judith R Campbell

Faculty, Staff and Students Publications

Levofloxacin prophylaxis reduces bloodstream infections in neutropenic patients with acute myeloid leukemia or relapsed acute lymphoblastic leukemia. A retrospective, longitudinal cohort study compares incidence of bacteremia, multidrug-resistant organisms (MDRO), and Clostridioides difficile (CDI) between time periods of levofloxacin prophylaxis implementation. Benefits were sustained without increasing MDRO or CDI.

Posttransplantation Late Complications Increase Over Time For Patients With Scid: A Primary Immune Deficiency Treatment Consortium (Pidtc) Landmark Study, Hesham Eissa, Monica S Thakar, Ami J Shah, Brent R Logan, Linda M Griffith, Huaying Dong, Roberta E Parrott, Richard J O'Reilly, Jasmeen Dara, Neena Kapoor, Lisa Forbes Satter, Sharat Chandra, Malika Kapadia, Shanmuganathan Chandrakasan, Alan Knutsen, Soma C Jyonouchi, Lyndsay Molinari, Ahmad Rayes, Christen L Ebens, Pierre Teira, Blachy J Dávila Saldaña, Lauri M Burroughs, Sonali Chaudhury, Deepak Chellapandian, Alfred P Gillio, Fredrick Goldman, Harry L Malech, Kenneth Desantes, Geoff D E Cuvelier, Jacob Rozmus, Ralph Quinones, Lolie C Yu, Larisa Broglie, Victor Aquino, Evan Shereck, Theodore B Moore, Mark T Vander Lugt, Talal I Mousallem, Joeseph H Oved, Morna Dorsey, Hisham Abdel-Azim, Caridad Martinez, Jacob H Bleesing, Susan Prockop, Donald B Kohn, Jeffrey J Bednarski, Jennifer Leiding, Rebecca A Marsh, Troy Torgerson, Luigi D Notarangelo, Sung-Yun Pai, Michael A Pulsipher, Jennifer M Puck, Christopher C Dvorak, Elie Haddad, Rebecca H Buckley, Morton J Cowan, Jennifer Heimall

Faculty, Staff and Students Publications

Background: The Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children in the United States and Canada onto a retrospective multicenter natural history study of hematopoietic cell transplantation (HCT).

Objective: We investigated outcomes of HCT for severe combined immunodeficiency (SCID).

Methods: We evaluated the chronic and late effects (CLE) after HCT for SCID in 399 patients transplanted from 1982 to 2012 at 32 PIDTC centers. Eligibility criteria included survival to at least 2 years after HCT without need for subsequent cellular therapy. CLE were defined as either conditions present at any time before 2 years from HCT that remained unresolved (chronic), …

Safety And Efficacy Of Intravenous Sotalol Following Congenital Heart Surgery, Ellis Rochelson, Maria Gutierrez, Santiago O Valdés, Katherine Lemming, Taylor S Howard, Tam Dan N Pham, Christina Y Miyake, Vicken Asadourian, Raajen Patel, Jeffrey J Kim

Faculty, Staff and Students Publications

No abstract provided.

Choice Of Adjuvant And Antigen Composition Alters The Immunogenic Profile Of A Sars-Cov-2 Subunit Vaccine, William R Lykins, Jeroen Pollet, Jessica A White, Brian Keegan, Leroy Versteeg, Ulrich Strych, Wen-Hsiang Chen, Raodoh Mohamath, Gabi Ramer-Denisoff, Sierra Reed, Samuel Beaver, Alana Gerhardt, Emily A Voigt, Mark A Tomai, Robert Sitrin, Robert K M Choy, Frederick J Cassels, Peter J Hotez, Maria Elena Bottazzi, Christopher B Fox

Faculty, Staff and Students Publications

Introduction: Since their introduction, adjuvanted recombinant subunit vaccines against COVID-19 have played a pivotal role in protecting global populations. Optimizing the immune response's quality, amplitude, and durability to these vaccines depends on the appropriate adjuvant choice and dose in combination with the selected antigen.

Methods: Here, we employed a preclinical mouse model to study the adaptive humoral and cellular immune responses to a SARS-CoV-2 receptor binding domain (RBD) antigen formulated with one of four different immune agonists [GLA, 3M-052, CpG-1826 (CpG), and dmLT], in combination with one of two different immune-stimulating formulations, a stabilized squalene emulsion (SE) or aluminum hydroxide …

Diagnostic Accuracy Of Urinary N Terminal Pro-B Type Natriuretic Peptides For Hemodynamically Significant-Patent Ductus Arteriosus In Preterm Infants, Srirupa Hari Gopal, Ganga Gokulakrishnan, Madhulika Kulkarni, Caraciolo J Fernandes, Mohan Pammi

Faculty, Staff and Students Publications

Background: Echocardiography is the gold standard for the diagnosis hemodynamically significant-patent ductus arteriosus (hs-PDA). It requires trained personnel and is not readily available. Urinary biomarkers can be used as an adjunct.

Objective: The objective of this study was to systematically review the diagnostic accuracy of urinary N terminal pro-B type natriuretic peptides (NT-proBNP) for hs-PDA in preterm neonates.

Methods: We included studies that evaluated urinary NT-proBNP and urinary NT-proBNP/creatinine ratio (index tests) in preterm neonates with hs-PDA (participants) in comparison with echocardiogram (reference standard). Methodological quality and certainty of evidence were assessed using Quality Assessment of Diagnostic-Accuracy Studies (QUADAS-2) and …

Predictive Analytics In Bronchopulmonary Dysplasia: Past, Present, And Future, Bryan G Mcomber, Alvaro G Moreira, Kelsey Kirkman, Sebastian Acosta, Craig Rusin, Binoy Shivanna

Faculty, Staff and Students Publications

Bronchopulmonary dysplasia (BPD) remains a significant complication of prematurity, impacting approximately 18,000 infants annually in the United States. Advances in neonatal care have not reduced BPD, and its management is challenged by the rising survival of extremely premature infants and the variability in clinical practices. Leveraging statistical and machine learning techniques, predictive analytics can enhance BPD management by utilizing large clinical datasets to predict individual patient outcomes. This review explores the foundations and applications of predictive analytics in the context of BPD, examining commonly used data sources, modeling techniques, and metrics for model evaluation. We also highlight bioinformatics' potential role …

Long-Term Efficacy And Safety Of Cardiac Genome Editing For Catecholaminergic Polymorphic Ventricular Tachycardia, Oliver M Moore, Yuriana Aguilar-Sanchez, Satadru K Lahiri, Mohit M Hulsurkar, J Alberto Navarro-Garcia, Tarah A Word, Joshua A Keefe, Dean Barazi, Elda M Munivez, Charles T Moore, Vaidya Parthasarathy, Jaysón Davidson, William R Lagor, So Hyun Park, Gang Bao, Christina Y Miyake, Xander H T Wehrens

Faculty, Staff and Students Publications

INTRODUCTION: Heterozygous autosomal-dominant single nucleotide variants in RYR2 account for 60% of cases of catecholaminergic polymorphic ventricular tachycardia (CPVT), an inherited arrhythmia disorder associated with high mortality rates. CRISPR/Cas9-mediated genome editing is a promising therapeutic approach that can permanently cure the disease by removing the mutant RYR2 allele. However, the safety and long-term efficacy of this strategy have not been established in a relevant disease model.

AIM: The purpose of this study was to assess whether adeno-associated virus type-9 (AAV9)-mediated somatic genome editing could prevent ventricular arrhythmias by removal of the mutant allele in mice that are heterozygous for

METHODS …

Case Report: P40phox Deficiency Underlying Pediatric-Onset Systemic Lupus Erythematosus, Alejandro Nieto-Patlán, Natalia S Fernández Dávila, Yuqing Wang, Michelle Zelnick, Eyal Muscal, Martha Curry, James R Lupski, Steven M Holland, Bo Yuan, Douglas B Kuhns, Tiphanie P Vogel, Ivan K Chinn

Faculty, Staff and Students Publications

Introduction

Systemic lupus erythematosus is a multi-faceted autoimmune disorder of complex etiology. Pre-pubertal onset of pediatric systemic lupus erythematosus (pSLE) is uncommon and should raise suspicion for a genetic driver of disease. Autosomal recessive p40^phox deficiency is a rare immunologic disorder characterized by defective but not abolished NADPH oxidase activity with residual production of reactive oxygen species (ROS) by phagocytic cells.

Case presentation

We report the case of a now 18-year-old female with pSLE onset at 7 years of age. She presented with recurrent fever and malar rash. Aspects of her immune dysregulation over time have included typical pSLE …

Development Of A Mini Pig Model Of Peanut Allergy, Akhilesh Kumar Shakya, Brittany Backus, Lazar D Nesovic, Malini Mallick, Olivia Banister, Carla M Davis, Sara Anvari, Harvinder Singh Gill

Faculty, Staff and Students Publications

INTRODUCTION: The prevalence of peanut allergies is increasing, emphasizing the need for an animal model to enhance our understanding of peanut allergy pathogenesis and to advance diagnostic tools and therapeutic interventions. While mice are frequently used as model organisms, their allergic responses do not fully mirror those observed in humans, warranting the exploration of a higher animal model. The porcine gastrointestinal system closely resembles that of humans, and exhibits allergy symptoms akin to human responses, making pigs a promising model for peanut allergy research.

METHODS: In this study we compared two allergen sensitization protocols involving either topical allergen application after …

Citrulline And Adi-Peg20 Reduce Inflammation In A Juvenile Porcine Model Of Acute Endotoxemia, Caitlin Vonderohe, Barbara Stoll, Inka Didelija, Trung Nguyen, Mahmoud Mohammad, Yava Jones-Hall, Miguel A Cruz, Juan Marini, Douglas Burrin

Faculty, Staff and Students Publications

BACKGROUND: Arginine is a conditionally essential amino acid that is depleted in critically ill or surgical patients. In pediatric and adult patients, sepsis results in an arginine-deficient state, and the depletion of plasma arginine is associated with greater mortality. However, direct supplementation of arginine can result in the excessive production of nitric oxide (NO), which can contribute to the hypotension and macrovascular hypo-reactivity observed in septic shock. Pegylated arginine deiminase (ADI-PEG20, pegargiminase) reduces plasma arginine and generates citrulline that can be transported intracellularly to generate local arginine and NO, without resulting in hypotension, while maintaining microvascular patency. The objective of …

Safety And Efficacy Of Intravenous Sotalol Following Congenital Heart Surgery, Ellis Rochelson, Maria Gutierrez, Santiago O Valdés, Katherine Lemming, Taylor S Howard, Tam Dan N Pham, Christina Y Miyake, Vicken Asadourian, Raajen Patel, Jeffrey J Kim

Faculty, Staff and Students Publications

No abstract provided.

Changes In Patterns Of Referral For Inpatient Rehabilitation Cancer Patients Due To Covid-19: A Retrospective Study, Ekta Gupta, Amy H Ng, Aline Rozman De Moraes, Jack B Fu, Jegy M Tennison, Maaheen Ahmed, Bryan M Fellman, Eduardo Bruera

Faculty, Staff and Students Publications

There is a paucity of literature on the effect of COVID-19 on hospital processes. We hypothesized that COVID-19 was associated with decreased cancer physiatry referrals in 2020. This is a retrospective cohort study of consecutive patients from April to July 2019 and 2020 admitted at an academic quaternary cancer center. The main outcomes were number of hospital admissions, rate, and characteristics of inpatient rehabilitation admissions and change in percentage of physiatry referrals as the primary endpoint. Results showed that in 2019, there were 387 referrals from 10,274 inpatient admissions (3.8%; 95% confidence interval, 2.4-4.2), compared with 337 referrals from 7051 …

A Case Report Navigating Cvid And Sarcoidosis Overlaps In Pediatric Nephritis, Amanda Salih, Amanda Brown, Amanda Grimes, Sana Hasan, Manuel Silva-Carmona, Leyat Tal, Joud Hajjar

Faculty, Staff and Students Publications

Common variable immunodeficiency (CVID) can be complicated by granulomatous disease, often granulomatous lymphocytic interstitial lung disease (GLILD). Granulomatous interstitial nephritis represents an atypical presentation in pediatrics. Our patient is a previously healthy 13-year-old white male with a recent diagnosis of CVID. He presented with a rash and laboratory findings included pancytopenia (white blood cells 2.6 cells × 103/μl, hemoglobin 11.8 g/dl, platelets 60 × 103/μl), hypercalcemia (14.9 mg/dl), elevated Vit D 1,25 OH level (>200 pg/ml), hyperuricemia (8.8 mg/dl), and acute kidney injury (AKI) (serum creatinine 1.1 mg/dl; baseline 0.64 mg/dl). A broad infectious workup was unremarkable. The rash …

Co-Transmitting Interneurons In The Mouse Olfactory Bulb Regulate Olfactory Detection And Discrimination, Ariel M Lyons-Warren, Evelyne K Tantry, Elizabeth H Moss, Mikhail Y Kochukov, Benjamin D W Belfort, Joshua Ortiz-Guzman, Zachary Freyberg, Benjamin R Arenkiel

Faculty, Staff and Students Publications

Co-transmission of multiple neurotransmitters from a single neuron increases the complexity of signaling information within defined neuronal circuits. Superficial short-axon cells in the olfactory bulb release both dopamine and γ-aminobutyric acid (GABA), yet the specific targets of these neurotransmitters and their respective roles in olfaction have remained unknown. Here, we implement intersectional genetics in mice to selectively block GABA or dopamine release from superficial short-axon cells to identify their distinct cellular targets, impact on circuit function, and behavioral contribution of each neurotransmitter toward olfactory behaviors. We provide functional and anatomical evidence for divergent superficial short-axon cell signaling onto downstream neurons …

How To Peer Review A Neurology Education Manuscript, Daniel G Di Luca, Steven M Lazar, Preeta Gupta, Marie Charmaine S Lukban, Cole Crowson, Dara V F Albert, Jeremy J Moeller, Andres Fernandez, Andrew M Southerland, Zachary London

Faculty, Staff and Students Publications

Peer review is an essential process in scientific research, ensuring the comprehensiveness, accuracy, and suitability of manuscripts for publication. Neurology education research differs from biomedical clinical research in several ways. These differences encompass specific paradigms, the use of theoretical frameworks, and different methodological approaches. Despite the high number of studies and journal publications on neurology education, there is a dearth of resources and guidance on how to perform a formal review on this specific literature. This article aims to review the distinctive features of neurology education from clinical research while proposing an organizational framework and model for performing peer reviews …

Pediatric Endocrinology Milestones 20-Guide To Their Implementation, Cara V Tillotson, Imen Becetti, Katherine Hwu, Laura Page, Sowmya Krishnan, Dianne Stafford, Takara Stanley, Patricia Vuguin, Jennifer M Barker

Faculty, Staff and Students Publications

The Milestones were initiated by the Accreditation Council for Graduate Medical Education (ACGME) to provide a framework for monitoring a trainee's progression throughout residency/fellowship. The Milestones describe stepwise skill progression through six core domains of clinical competency: Patient Care, Medical Knowledge, Interpersonal and Communication Skills, Practice-based Learning and Improvement, Professionalism, and Systems-based Practice. Since their introduction in 2013, several barriers to implementation have emerged. Thus, the ACGME launched the Milestones 2.0 project to develop updated specialty-specific milestones. The Pediatric Endocrinology Milestones 2.0 project aimed to improve upon Milestones 1.0 by addressing common limitations, providing resources for faculty to easily incorporate …

Early Cellular And Molecular Signatures Correlate With Severity Of West Nile Virus Infection, Ho-Joon Lee, Yujiao Zhao, Ira Fleming, Sameet Mehta, Xiaomei Wang, Brent Vander Wyk, Shannon E Ronca, Heather Kang, Chih-Hung Chou, Benoit Fatou, Kinga K Smolen, Ofer Levy, Clary B Clish, Ramnik J Xavier, Hanno Steen, David A Hafler, J Christopher Love, Alex K Shalek, Leying Guan, Kristy O Murray, Steven H Kleinstein, Ruth R Montgomery

Faculty, Staff and Students Publications

Infection with West Nile virus (WNV) drives a wide range of responses, from asymptomatic to flu-like symptoms/fever or severe cases of encephalitis and death. To identify cellular and molecular signatures distinguishing WNV severity, we employed systems profiling of peripheral blood from asymptomatic and severely ill individuals infected with WNV. We interrogated immune responses longitudinally from acute infection through convalescence employing single-cell protein and transcriptional profiling complemented with matched serum proteomics and metabolomics as well as multi-omics analysis. At the acute time point, we detected both elevation of pro-inflammatory markers in innate immune cell types and reduction of regulatory T cell …

Genotype, Oxidase Status, And Preceding Infection Or Autoinflammation Do Not Affect Allogeneic Hct Outcomes For Cgd, Jennifer W Leiding, Danielle E Arnold, Suhag Parikh, Brent Logan, Rebecca A Marsh, Linda M Griffith, Ruizhe Wu, Sharon Kidd, Kanwaldeep Mallhi, Deepak Chellapandian, Stephanie J Si Lim, Eyal Grunebaum, E Liana Falcone, Luis Murguia-Favela, Debbi Grossman, Vinod K Prasad, Jennifer R Heimall, Fabien Touzot, Lauri M Burroughs, Jack Bleesing, Neena Kapoor, Jasmeen Dara, Olatundun Williams, Malika Kapadia, Benjamin R Oshrine, Jeffrey J Bednarski, Ahmad Rayes, Hey Chong, Geoffrey D E Cuvelier, Lisa R Forbes Satter, Caridad Martinez, Mark T Vander Lugt, Lolie C Yu, Shanmuganathan Chandrakasan, Avni Joshi, Susan E Prockop, Blachy J Dávila Saldaña, Victor Aquino, Larisa A Broglie, Christen L Ebens, Lisa M Madden, Kenneth Desantes, Jordan Milner, Hemalatha G Rangarajan, Ami J Shah, Alfred P Gillio, Alan P Knutsen, Holly K Miller, Theodore B Moore, Pamela Graham, Andrea Bauchat, Nancy J Bunin, Pierre Teira, Aleksandra Petrovic, Sharat Chandra, Hisham Abdel-Azim, Morna J Dorsey, Olga Birbrayer, Morton J Cowan, Christopher C Dvorak, Elie Haddad, Donald B Kohn, Luigi D Notarangelo, Sung-Yun Pai, Jennifer M Puck, Michael A Pulsipher, Troy R Torgerson, Harry L Malech, Elizabeth M Kang

Faculty, Staff and Students Publications

Chronic granulomatous disease (CGD) is a primary immunodeficiency characterized by life-threatening infections and inflammatory conditions. Hematopoietic cell transplantation (HCT) is the definitive treatment for CGD, but questions remain regarding patient selection and impact of active disease on transplant outcomes. We performed a multi-institutional retrospective and prospective study of 391 patients with CGD treated either conventionally (non-HCT) enrolled from 2004 to 2018 or with HCT from 1996 to 2018. Median follow-up after HCT was 3.7 years with a 3-year overall survival of 82% and event-free survival of 69%. In a multivariate analysis, a Lansky/Karnofsky score < 90 and use of HLA-mismatched donors negatively affected survival. Age, genotype, and oxidase status did not affect outcomes. Before HCT, patients had higher infection density, higher frequency of noninfectious lung and liver diseases, and more steroid use than conventionally treated patients; however, these issues did not adversely affect HCT survival. Presence of pre-HCT inflammatory conditions was associated with chronic graft-versus-host disease. Graft failure or receipt of a second HCT occurred in 17.6% of the patients and was associated with melphalan-based conditioning and/or early mixed chimerism. At 3 to 5 years after HCT, patients had improved growth and nutrition, resolved infections and inflammatory disease, and lower rates of antimicrobial prophylaxis or corticosteroid use compared with both their baseline and those of conventionally treated patients. HCT leads to durable resolution of CGD symptoms and lowers the burden of the disease. Patients with active infection or inflammation are candidates for transplants; HCT should be considered before the development of comorbidities that could affect performance status. This trial was registered at www.clinicaltrials.gov as #NCT02082353.