Medicine and Health Sciences Commons^™

Covid-19 Pandemic Impacts On Behavioral And Emotional Health Of Young Children With Autism., Olivia M. Pokoski, Hayley M. Crain, Sarah M. Furnier, Ronald E. Gangnon, Cy Nadler, Eric J. Moody, Karen Pazol, Maria A. Stanley, Lisa D. Wiggins, Maureen S. Durkin

Manuscripts, Articles, Book Chapters and Other Papers

OBJECTIVE: To test initial hypotheses that the coronavirus disease 2019 (COVID-19) pandemic was associated with decreases in adaptive behavior and increases in behavioral and emotional problems of children with autism; greater impacts for children who lost specialty services; and greater behavioral and emotional problems for children with autism vs control participants.

METHOD: Eligible participants (N = 1,158) enrolled in phase 3 of the multisite, case-control Study to Explore Early Development (SEED) before March 31, 2020, were between 2 and 5 years old, and completed follow-up assessments between January and July 2021. Caregivers completed a COVID-19 Impact Assessment Questionnaire, Vineland Adaptive …

Cardiovascular And Thromboembolic Events In Children And Adults With Glomerular Disease: Findings From The Cure Glomerulonephropathy (Curegn) Network., Shikha Wadhwani, Sarah A. Mansfield, Abigail R. Smith, Bruce M. Robinson, Eman Abdelghani, Amira Al-Uzri, Isa F. Ashoor, Sharon M. Bartosh, Aftab S. Chishti, Salim S. Hayek, Michelle A. Hladunewich, Bryce A. Kerlin, Siddharth S. Madapoosi, Laura H. Mariani, Amy K. Mottl, Michelle N. Rheault, Michelle M. O'Shaughnessy, Christopher John Sperati, Tarak Srivastava, David T. Selewski, Chia-Shi Wang, Craig S. Wong, Donald J. Weaver, Myda Khalid, Glomerulonephropathy (Curegn) Study Consortium

Manuscripts, Articles, Book Chapters and Other Papers

RATIONALE & OBJECTIVE: Cardiovascular (CV) and thromboembolic (TE) events are known complications of glomerular disease (GD), but their incidence and risk factors are poorly characterized. This analysis describes CV and TE outcomes in the Cure GlomeruloNephropathy (CureGN) Network.

STUDY DESIGN: Prospective cohort study.

SETTING & PARTICIPANTS: CureGN is a prospective cohort study of children and adults with biopsy-proven minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), membranous nephropathy (MN), or IgA nephropathy (IgAN)/vasculitis (IgAV). Data from 2,545 children and adults (23% MCD, 23% MN, 25% FSGS, 29% IgAN/IgAV) was analyzed.

EXPOSURE: Estimated glomerular filtration rate (eGFR), proteinuria, serum albumin, tobacco …

Postoperative Caregiver Decision Regret Following Velopharyngeal Dysfunction Surgery., Rohit Nallani, Nathan Farrokhian, Meghan Tracy, Kevin J. Sykes, Jill M. Arganbright

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Decision regret (DR) is a powerful factor in medical decision-making, particularly for surgical decisions which carry multiple risks, impact quality of life (QOL), and have uncertain outcomes. While prior studies have investigated caregiver DR after tonsillectomy and otologic surgery, there is a paucity of data assessing DR following pediatric otolaryngology procedures. The aim of this study is to measure DR following velopharyngeal dysfunction (VPD) surgery.

METHODS: A cross-sectional survey study with chart review was performed of caregivers of children who had VPD surgery performed by a single surgeon at a tertiary children's hospital. Demographic, medical, and surgical history were …

New Frontiers: The Intern Experience In A Newly Established Integrated Plastic Surgery Program, Hendrik Lintel, Imran Rizvi, Michael Haydon, Erik S Marques, Matthew R Greives, Mohin A Bhadkamkar

Faculty, Staff and Student Publications

The plastic surgery residency pathway has undergone many changes since its inception. The advent of integrated training has largely taken over as the predominant residency model with the addition of a few new programs every year. There is currently a paucity of literature describing the experiences of first-year interns in a new integrated residency program transitioning from the independent model. In this article, we seek to elucidate some of the advantages and difficulties encountered by the initial residents of a newly established integrated program and the steps taken to pave the way for future matched integrated trainees.

Measuring And Interpreting Individual Differences In Fetal, Infant, And Toddler Neurodevelopment, Halie A Olson, M Catalina Camacho, Gavkhar Abdurokhmonova, Sahar Ahmad, Emily M Chen, Haerin Chung, Renata Di Lorenzo, Áine T Dineen, Melanie Ganz, Roxane Licandro, Caroline Magnain, Natasha Marrus, Sarah A Mccormick, Tara M Rutter, Lauren Wagner, Kali Woodruff Carr, Lilla Zöllei, Kelly A Vaughn, Kathrine Skak Madsen

Faculty, Staff and Student Publications

As scientists interested in fetal, infant, and toddler (FIT) neurodevelopment, our research questions often focus on how individual children differ in their neurodevelopment and the predictive value of those individual differences for long-term neural and behavioral outcomes. Measuring and interpreting individual differences in neurodevelopment can present challenges: Is there a "standard" way for the human brain to develop? How do the semantic, practical, or theoretical constraints that we place on studying "development" influence how we measure and interpret individual differences? While it is important to consider these questions across the lifespan, they are particularly relevant for conducting and interpreting research …

Enhancing Seizure Control In Ultra-Refractory Postencephalitic Epilepsies Using Multinodal Network Neuromodulation, Subhiksha Srinivasan, Surya Suresh, Ganne Chaitanya, Manoj Saranathan, Nitin Tandon, Sandipan Pati

Faculty, Staff and Student Publications

This case series reports the formidable challenge posed by postencephalitic epilepsies, characterized by frequent drug-resistant seizures and neuropsychiatric and cognitive comorbidities. Polypharmacy is frequently required, and surgical resection may not be feasible due to multifocality. Neuromodulation therapies, including Deep Brain Stimulation (DBS) and Responsive Neurostimulation (RNS), offer a potential lifeline. In this case series, we shed light on the intricate landscape of seizure management and neuropsychiatric comorbidities in five individuals with frequent seizures (often weekly) and ultra-refractory epilepsy (defined as resistance to more than six different antiseizure medications, including failed epilepsy surgery) following catastrophic encephalitis. Four out of five patients …

Understanding And Addressing Mental Health Challenges Of Families Admitted To The Neonatal Intensive Care Unit., Ashley D. Osborne, Daphna Yasova Barbeau, Tiffany Gladdis, Kara Hansen, Tonia Branche, Emily R. Miller, Christine C. Pazandak, Margaret K. Hoge, Michelle Spencer, Diana Montoya-Williams, Ryan Barbeau, Heather Padratzik, Stephen Lassen

Manuscripts, Articles, Book Chapters and Other Papers

This article reviews the psychological distress experienced by NICU families, including anxiety, postpartum depression (PPD), and post-traumatic stress disorder (PTSD), in addition to providing recommendations for clinicians at the individual, institutional, and national level. Currently, mental health screenings, specialized evaluations, and treatment options are not routinely offered to NICU families and are frequently under-utilized when offered. Here we provide expert opinion recommendations to address challenges in supporting universal screening, offering bedside interventions, including trained mental health professionals in care plans, updating neonatology training competencies, and advocating for policies that support the mental health of NICU families. We advocate that mental …

Epidemiology Of Shigella Species And Serotypes In Children: A Retrospective Substudy Of The Mal-Ed Observational Birth Cohort Study, Elizabeth T. Rogawski Mcquade, Jie Liu, Mustafa Mahfuz, Alexandre Havt, Furqan Kabir, Adil Kalam, Fatima Aziz, Sahrish Muneer, Najeeha Talat Iqbal, Zulfiqar Ahmed Bhutta

Department of Paediatrics and Child Health

Background: Shigellosis is a leading cause of diarrhoea in children globally. We aimed to assess the burden of specific Shigella species and Shigella flexneri serotypes, characterise their clinical syndromes and natural immunity, and evaluate their relevance as causes of diarrhoea and linear growth faltering.
Methods: In the MAL-ED birth cohort study, children younger than 17 days were enrolled from Nov 3, 2009, to Feb 29, 2012, in Bangladesh, Brazil, India, Nepal, Pakistan, Peru, Tanzania, and South Africa and were followed up for 2 years. In this retrospective substudy, we retested Shigella quantitative PCR-positive diarrhoeal and non-diarrhoeal stool samples with molecular …

Clinical Manifestations, Disease Penetrance, And Treatment In Individuals With Socs1 Insufficiency: A Registry-Based And Population-Based Study, Jerome Hadjadj, Anna Wolfers, Oleg Borisov, Derek Hazard, Ronan Leahy, Marie Jeanpierre, Alexandre Belot, Shahrzad Bakhtiar, Fabian Hauck, Pui Y Lee, Stefano Volpi, Serena Palmeri, Vincent Barlogis, Nathalie Aladjidi, Georg Ebetsberger-Dachs, Jerome Avouac, Fabienne Charbit-Henrion, Morgane Cheminant, Jean Donadieu, Sujal Ghosh, David P Hoytema Van Konijnenburg, Julia Körholz, Jacinta Bustamante, Jeremie Rosain, Lisa Forbes Satter, Ingrid Selmeryd, Georgios Sogkas, Benedicte Neven, Frederic Rieux-Laucat, Stephan Ehl

Faculty, Staff and Students Publications

Background: Suppressor of cytokine signalling 1 (SOCS1) insufficiency is an inborn error of immunity affecting the negative regulation of cytokine and growth factor signalling. We aimed to enhance the understanding of clinical manifestations, disease trajectories, disease penetrance, and the effect of Janus kinase (JAK) inhibition in individuals with SOCS1 insufficiency.

Methods: This study used data from two independent cohorts: the European Society for Immunodeficiencies (ESID) registry and the UK Biobank. Participants from the ESID registry were from nine European countries (Austria, Belgium, France, Germany, Ireland, Italy, Portugal, Sweden, and Ukraine), China, Taiwan, and the USA. Participants from the ESID registry …

Plasma Microbial Cell-Free Dna Sequencing For The Detection Of Kingella Kingae Pediatric Spinal Infections, Asim A Ahmed, Sarah Y Park, Matthew J Smollin, Martin S Lindner, Adriana Sarmiento Clemente, Ana Del Valle Penella, P Marcelo Laufer, Carolina Sanchez-Vegas, Manuel R Cotilla, Marian E Melish, Connie Trieu, Nazia A Kabani, Joshua Cooper, Audrey R Lloyd, David W Kimberlin, John C Arnold, Alejandro Jordan-Villegas, Lori E Patterson, Catherine E Foster, Pablo Yagupsky

Faculty, Staff and Students Publications

Background: Diagnosis of Kingella kingae skeletal system infections is made challenging by the microbe's fastidious nature. Detection and quantification of circulating microbial cell-free DNA (mcfDNA) in plasma by the Karius Test, a commercial metagenomic sequencing test, may offer promise in diagnosing pediatric spinal infections caused by difficult-to-culture organisms such as K. kingae .

Methods: Plasma mcfDNA sequencing detections of K. kingae from April 2018 to December 2020 were reviewed to identify pediatric (age < 18 years) patients. Medical charts of those with spinal infections were reviewed, and mcfDNA sequencing diagnostic performance was compared with usual care tests (ie, cultures, polymerase chain reaction).

Results: Ten children with K. kingae spinal infections were identified across 7 institutions. The median age was 16.5 months (range 11-23 months). All case-patients had vertebral osteomyelitis …

Ophthalmology Examinations In Children With Skull Fractures And Underlying Focal Hemorrhage, Karli Breeden, Cindy W Christian, Joanne N Wood, Gil Binenbaum, Daniel M Lindberg, Angela Bachim, Colleen J Bressler, Lori Frasier, Terra Frazier, Nicole R Johnson, Natalie Laub, Megan M Letson, Tagrid Ruiz-Maldonado, Matthew Valente, Jenna Kiely, Jan Leonard, M Katherine Henry

Faculty, Staff and Students Publications

Objectives: To assess the frequency and yield of retinal examination in children below 2 years old undergoing abuse evaluations in the setting of skull fracture(s) and small underlying intracranial hemorrhage.

Methods: This cross-sectional study used CAPNET, a multicenter child physical abuse network, to identify children below 2 years with a skull fracture(s) and intracranial injury limited to an underlying small focal intracranial hemorrhage undergoing subspeciality child abuse evaluations. Our outcomes of interest were (1) the performance of a retinal examination, (2) the identification of retinal hemorrhages, and (3) associations of clinical factors and CAPNET site with the performance of retinal …

Mitral Annular Disjunction Is Associated With Ventricular Arrhythmias In Children With Marfan Syndrome, Carlos A Sanchez Parra, Ruthie M Vaughan, Alejandra Iturralde Chavez, Christina Y Miyake, Santiago O Valdes, Ricardo H Pignatelli, Anitha Parthiban, Shaine A Morris, Tam T Doan

Faculty, Staff and Students Publications

No abstract provided.

An Electronic, Preclinic, Social Determinants Survey Shows Pediatric Atherosclerotic Risk Factor Referral Identifies At-Risk Families, Justin R Rahman, Bethany S Zachariah, Christina Y Miyake, Deidra Ansah, Justin P Zachariah

Faculty, Staff and Students Publications

Objective: Atherosclerotic risk factors are prevalent in childhood. Risk factors are associated with social determinants of health (SDoH), but have never been demonstrated in a clinical pediatric cardiology cohort. This report details the first use of a remotely deployed, previsit, self-administered electronic survey for the ascertainment of SDoH in youth referred to pediatric cardiology. We hypothesized that patients referred for atherosclerotic risk factors would have higher risk of SDoH factors.

Study design: SDoH were assessed using validated instruments from the PRAPARE toolkit and the Accountable Health Communities Health-Related Social Needs Screening Tool automatically deployed days before a scheduled visit in …

Twenty-Five Years Of Palivizumab: A Global Historic Review Of Its Impact On The Burden Of Respiratory Syncytial Virus Disease In Children, Xavier Carbonell-Estrany, Eric A F Simões, Louis Bont, Paolo Manzoni, Heather J Zar, Anne Greenough, Octavio Ramilo, Renato Stein, Barbara Law, Asuncion Mejias, Manuel Sanchez Luna, Paul A Checchia, Leonard Krilov, Marcello Lanari, Ron Dagan, Brigitte Fauroux, Bernhard Resch, Terho Heikkinen, Joseph B Domachowske, Joanne G Wildenbeest, Federico Martinon-Torres, Richard Thwaites, Merih Cetinkaya, Adel S Alharbi, Carlos E Rodriguez-Martinez, Daniel E Noyola, Asiah Kassim, Satoshi Kusuda, Ji-Man Kang, Barry Rodgers-Gray, Anna Platonova, Fungwe Jah, Bosco Paes

Faculty, Staff and Students Publications

Introduction: Respiratory syncytial virus (RSV) causes significant morbidity and mortality in young children. For 25 years, palivizumab has been the only effective pharmaceutical RSV preventive.

Areas covered: We summarize the development and a quarter-century of real-world evidence with palivizumab. We highlight its positive impact on the burden of RSV in high-risk children. Based on lessons learnt from its implementation, we suggest strategies for effective and equitable deployment of newer RSV preventives.

Expert opinion: Following failure of the formalin-inactivated RSV vaccine in 1967, RSV intravenous immunoglobulin was approved in 1996 after three decades' research. Subsequently, palivizumab emerged as the most effective …

Point-Of-Care Ultrasound For Guiding Pulmonary Artery Catheter Placement, Erik Su, Saul Flores

Faculty, Staff and Students Publications

No abstract provided.

Neuroanatomical Features Of Naa10 And Naa15-Related Neurodevelopmental Syndromes, Rahi Patel, Rikhil Makwana, Carolina Christ, Elaine Marchi, Christina Y Miyake, Fabricio Guimaraes Goncalves, Gholson J Lyon, Matthew T Whitehead

Faculty, Staff and Students Publications

Background: NAA10-related and NAA15-related neurodevelopmental (ND) syndromes present with intellectual disability, hypotonia, cardiac abnormalities, and delayed development. While data exists on the clinical manifestations of these conditions, there are few reports describing the neuroanatomical abnormalities present on imaging.

Objective: We aim to provide neuroimaging analyses for a subset of probands with NAA10- and NAA15-related neurodevelopmental symptoms and assess the severity and number of neuroanatomical anomalies and their associated functional impairments to better understand the pathophysiology of these disease processes.

Materials and methods: Neuroimaging studies were obtained from 26 probands (18 with pathogenic variants in NAA10, 8 with pathogenic variants in …

Functional Mobility And Pain Are Improved For 6 Years After Adolescent Bariatric Surgery, Neil Thivalapill, Todd M Jenkins, Thomas H Inge, Changchun Xie, Anita P Courcoulas, Carroll M Harmon, Michael A Helmrath, Stephanie Sisley, Marc P Michalsky, Justin R Ryder, Teen‐Longitudinal Assessment Of Bariatric Surgery (Teen‐Labs) Consortium

Faculty, Staff and Students Publications

Objective: The long-term durability of improvements in functional mobility and musculoskeletal pain for adolescents after metabolic and bariatric surgery (MBS) is unknown.

Methods: We used the Teen-Longitudinal Assessment of Bariatric Surgery (Teen-LABS) study to determine the change in mobility and pain among adolescents who underwent MBS. From standardized 400-m walk tests, we analyzed walk time, heart rate (HR) parameters, and musculoskeletal pain.

Results: The mean walk time improved from 383 s (95% CI: 368-399) prior to surgery to 351 s (95% CI: 330-372) by 6 years. The mean resting HR was 90 beats per minute (bpm; 95% CI: 87-93) preoperatively …

Are Education, Socioeconomic Status, And Household Size Dependent Variables Influencing The Developmental Origins Of Inflammatory Bowel Disease?, Richard Kellermayer

Faculty, Staff and Students Publications

No abstract provided.

Severe Gastrointestinal Manifestations In Childhood-Onset Lupus: A Single-Center Cohort And Review Of The Literature, James Orozco, Jessica Nguyen, Sara Mm Yasrebi, Marnie Blalock, Sara L Grisales, Craig L Jensen, Marietta M Deguzman

Faculty, Staff and Students Publications

Objective

Systemic lupus erythematosus (SLE) is a complex autoimmune condition with diverse manifestations. Childhood-onset SLE (cSLE) presents more severely than adult-onset disease with a higher incidence of severe neurologic or renal manifestations and lower estimated survival rates. There is limited literature on the range of severe gastrointestinal manifestations at presentation in cSLE, and this lack of information results in underappreciation of these potentially life-threatening complications and little guidance regarding management of these patients.

Methods

We reviewed cases of patients with cSLE and severe GI manifestations at presentation diagnosed at our institution and additionally provide a comprehensive review of existing cases …

Design Of The Type 1 Diabetes And Life (T1dal) Pilot And Feasibility Study: A Brief Telehealth Intervention Targeting Health-Related Quality Of Life Across Clinical Settings, Marisa E Hilliard, Wendy Levy, Ruth S Weinstock, Korey K Hood, Paula M Trief, Daniel J Desalvo, Yuliana Rojas, Kyrah Holland, Aika K Schneider-Utaka, Selma A Alamarie, Lynn Agostini, Se-Kang Kim, Maartje De Wit, Meghan E Mcgrady, Laurel H Messer, Barbara J Anderson

Faculty, Staff and Students Publications

Background: Type 1 diabetes (T1D) management is demanding and can impact quality of life among persons with diabetes (PWDs) and their family members. Behavioral intervention research has largely focused on adolescents, and previous interventions that have benefitted quality of life have limited potential for implementation in routine care. This trial is piloting a brief behavioral intervention that targets health-related quality of life (HRQOL), which can be implemented with PWDs of all ages in a range of clinical care settings. The aims are to (1) evaluate intervention feasibility and acceptability, (2) explore pre-post change in psychosocial and clinical outcomes, and (3) …

Sci – U: Validation Of An Updated Self-Care Inventory For Contemporary Diabetes Management In Adolescents With Type 1 Diabetes, Sarah S Jaser, Tamim A Hamdan, Marisa E Hilliard, Joyce P Yi-Frazier, Randi Streisand, Faisal S Malik, Daniel J Desalvo, Maeve B O'Donnell, Annette M La Greca

Faculty, Staff and Students Publications

Aims: The Self-Care Inventory is a widely used measure to assess diabetes self-management behaviors. We sought to adapt and streamline the measure to reflect advances in diabetes management, including increased use of continuous glucose monitor and automated insulin delivery systems.

Methods: Through an expert-driven, iterative process, the updated Self-Care Inventory (SCI-U) was created by modifying items to reflect modern diabetes management and reducing the measure to 8 items. The measure was administered to 369 adolescents with type 1 diabetes in 4 regions of the United States, at baseline of two separate intervention trials (mean age = 15.5 ± 1.5, 56 …

Measles, Mumps, Rubella Immunization And The Resurgence Of Measles In America, Peter J Hotez

Faculty, Staff and Students Publications

No abstract provided.

Surveying Management Strategies For Pediatric Autoimmune Hepatitis, Lillie Baca

University Honors Theses

Pediatric autoimmune hepatitis (PAIH) is a long-term condition that requires ongoing treatment and regular monitoring. It is characterized by its elevation of liver enzymes, interface hepatitis, and the specific presence of circulating autoantibodies. There is a general lack of consensus on appropriate measures of diagnostic criteria, treatment medication, and beneficial practices for monitoring the disease. The existing literature contains several subtle discrepancies that challenge a practitioner's judgement for care management of children with autoimmune hepatitis. This study is a retrospective chart review aimed at understanding the current diagnostics, treatment, and long-term management strategies of PAIH at a single center. Also, …

Protocol Optimization For Out-Of-Hospital Cardiac Arrest: A Quality Initiative To Decrease Epinephrine Administration, Jessica Aird Do, Aaron Singer Md, David Rayburn Md, Mph, Meg Marino Md

Quality Improvement & Patient Safety Forum

No abstract provided.

American Society Of Hematology/International Society On Thrombosis And Haemostasis 2024 Updated Guidelines For Treatment Of Venous Thromboembolism In Pediatric Patients, Paul Monagle, Muayad Azzam, Rachel Bercovitz, Marisol Betensky, Rukhmi Bhat, Tina Biss, Brian Branchford, Leonardo R. Brandão, Anthony K.C. Chan, Vincent E.S. Faustino, Julie Jaffray, Sophie Jones, Hassan Kawtharany, Bryce A. Kerlin, Nicole Kucine, Riten Kumar, Christoph Male, Marie Claude Pelland-Marcotte, Leslie Raffini, Chittalsinh Raulji, Sarah E. Sartain, Clifford M. Takemoto, Cristina Tarango, C. Heleen Van Ommen, Maria C. Velez, Sara K. Vesely, John Wiernikowski, Suzan Williams, Hope P. Wilson, Et Al

School of Medicine Faculty Publications

Background: The American Society of Hematology (ASH) guidelines on treatment of pediatric venous thromboembolism (VTE) were published in 2018. In the last 6 years, there has been a 10-fold increase in the number of children involved in VTE treatment trials. Objective: The ASH Committee on Quality and Guidelines agreed to update the pediatric guidelines in conjunction with the International Society on Thrombosis and Haemostasis (ISTH). These ASH/ISTH evidence-based guidelines are intended to support patients, clinicians, and other health care professionals in the management of pediatric patients with VTE. Methods: ASH/ISTH formed a multidisciplinary guideline panel to minimize potential bias from …

Portal Vein Obstruction In Pediatric Liver Transplant Patients: An Evaluation Of Self-Reported Management Practices., Lydia Sieben, Bader A. Alfares, Ruben H. De Kleine, Barbara E. Wildhaber, Thomas Casswall, Greg Nowak, Martin Delle, Denise Aldrian, Valeria Berchtold, Georg F. Vogel, Piotr Kaliciński, Malgorzata Markiewicz-Kijewska, Adam Kolesnik, Jesús Quintero, María Mercadal Hally, Mauricio Larrarte King, Paolo Marra, Michela Bravi, Domenico Pinelli, Mureo Kasahara, Seisuke Sakamoto, Hajime Uchida, Vidyadhar Mali, Marion Aw, Stéphanie Franchi-Abella, Emmanuel Gonzales, Florent Guérin, Guillermo Cervio, Julia Minetto, Sergio Sierre, Martín De Santibañes, Victoria Ardiles, Jimmy Walker Uño, Helen Evans, David Duncan, John Mccall, Steffen Hartleif, Ekkehard Sturm, Jai V. Patel, Marumbo Mtegha, Raj Prasad, Cristina T. Ferreira, Luiza S. Nader, Marco Farina, Catalina Jaramillo, Manuel I. Rodriguez-Davalos, Peter Feola, Amit A. Shah, Phoebe M. Wood, Michael R. Acord, Ryan T. Fischer, Bhargava Mullapudi, Richard J. Hendrickson, Rajeev Khanna, Viniyendra Pamecha, Amar Mukund, Khalid Sharif, Girish Gupte, Simon Mcguirk, Gilda Porta, Winita Hardikar, Marco Spada, Tommaso Alterio, Giuseppe Maggiore, Marisa Beretta, Rudi A J Dierckx, Reinoud P H Bokkers, Hubert P J Van Der Doef, Portal Registry Investigators

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND AND AIMS: Portal vein obstruction (PVO) is a known complication after pediatric liver transplantation (pLT). Effective management strategies are crucial in improving patient outcomes. This study investigated the various practice patterns related to PVO management to clarify the degree of consensus on the diverse facets of care involved in addressing PVO after pLT.

METHODS: A self-reported evaluation was conducted using a scanned, paper-based survey among specialized pLT centers participating in the Portal vein Obstruction Revascularization Therapy After Liver transplantation (PORTAL) registry. The survey consisted of 30 questions covering the current practices regarding PVO, including experience, team composition, follow-up and …

Extracorporeal Membrane Oxygenation For Systemic Lupus Erythematosus: An Elso Registry Analysis, Maria Pereira, Danielle Guffey, Katherine Doane, Eyal Muscal, Carla Levin, Peter Rycus, Marc Anders, Andrea Ontaneda

Faculty, Staff and Students Publications

The success of extracorporeal membrane oxygenation (ECMO) in treating Systemic Lupus Erythematosus (SLE) and the risk factors associated with mortality remain uncertain. Methods: We describe the survival outcomes at discharge of the largest SLE cohort on ECMO support. We performed a retrospective cohort study of the Extracorporeal Life Support Organization registry database from 2012 to 2022. Pediatric and adult survivor groups were analyzed using descriptive statistics for the primary study outcome of survival to hospital discharge. Risk predictors for survival were determined by logistic regression.Results: We included 48 children and 368 adults with SLE. Overall, 198 patients (54%) survived …

Implementation Of A Health Care Transition Pathway For Children And Adults With Inborn Errors Of Metabolism: A Pilot Project, Brittany M. Murray

Doctor of Nursing Practice Scholarly Projects

Background: Children with medically complex diseases require highly specialized care by a highly specialized team. Those with rare disease, including inborn errors of metabolism (IEMs), are a medically complex population and face significant challenges undergoing the health care transition from a child/family-centered model to an adult/patient-centered model of care. The project site follows patients with IEMs in the Metabolism Program, but previously did not have a coordinated process and had insufficient resources for patients/families for the health care transition.

Methods: The Plan-Do-Study-Act (PDSA) method was utilized as the model for improvement. Due to the complexity of the population and time …

Reducing Surgery For Pediatric Posttonsillectomy Hemorrhage Using Tranexamic Acid: A Quality Improvement Initiative, Laura A. Petrauskas, Janavi Sethurathnam, Ansley J. Kunnath, Rahul K. Sharma, John Ceremsak, Ryan H. Belcher, James D. Phillips, Jay A. Werkhaven, Amy S. Whigham, Lyndy J. Wilcox, Christopher T. Wootten, Frank W. Virgin, Jason S. Park

School of Medicine Faculty Publications

Objective: Evaluate the use of tranexamic acid (TXA) and observation as a management option for pediatric patients presenting with posttonsillectomy hemorrhage (PTH). Study Design: Retrospective analysis of a prospectively implemented quality improvement initiative with a historical control comparison group. Setting: Tertiary children's hospital. Methods: Patients < 18 years of age who underwent adenotonsillectomy (AT) and returned to the Emergency Department for PTH were included. Patients who were stable without large volume or active bleeding were given intravenous TXA and admitted for overnight observation. Data were compared in a before-and-after analysis: preprotocol (April 2022 to March 2023) versus postprotocol (April 2023 to March 2024). For cost-effectiveness analysis, we analyzed aggregated claims data from a commercial claims database. Results: Preprotocol 1800 adenotonsillectomies were performed, and 40 procedures were performed for control of hemorrhage (2.2 per 100 AT). Postprotocol 2356 adenotonsillectomies were performed, and 30 procedures were performed to control hemorrhage (1.3 per 100 AT) showing a significant reduction in return to the operating room (relative risk [RR] = 0.59, 95% confidence interval [CI] [0.358, 0.916], P-value.020). There were no reported adverse events attributable to TXA. An estimated 21 surgeries were avoided, and 26 additional patients were observed in the hospital during the postprotocol period, for an estimated net cost savings of $174,970. Conclusion: The implementation of a standardized TXA protocol significantly reduced the need for return to the operating room for PTH in pediatric patients, without complications and with net cost savings to the health care system.

Identification Of Surrogate Biomarkers For Mucopolysaccharidosis Type Iva, Yasuhiko Ago, Shaukat Khan, Kimberly Klipner, Allison Bradford, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidosis type IVA (MPS IVA, Morquio A syndrome) is a rare inherited disorder characterized by skeletal dysplasia due to deficient N-acetylgalactosamine-6-sulfate sulfatase activity, resulting in glycosaminoglycan (GAG) accumulation. Identifying accurate biomarkers reflecting clinical severity and therapeutic response remains challenging. This study evaluated potential surrogate biomarkers, including N-terminal pro-C-type natriuretic peptide (NT-proCNP), collagen types I and II, mono-sulfated keratan sulfate (KS), di-sulfated KS, and chondroitin-6-sulfate (C6S), in blood and urine samples from 60 patients ranging from 1 to 62 years of age. NT-proCNP levels were significantly elevated in patients of all ages and negatively correlated with growth impairment, especially after 8 …