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Articles 331 - 360 of 1417
Full-Text Articles in Pediatrics
Understanding Guardian Perceptions Of Whole-Foods Plant-Based Diets As Interventions In Pediatric Medicine, Vijay R. Luthra
Understanding Guardian Perceptions Of Whole-Foods Plant-Based Diets As Interventions In Pediatric Medicine, Vijay R. Luthra
Honors Undergraduate Theses
The obesity epidemic is widespread in American society, often affecting children who bear its consequences innocently. Due to a lack of nutrition knowledge, children rely on caregivers to meet their nutritional needs and instill healthy habits. This study examines parents' attitudes, knowledge, and behaviors, as well as perceived barriers to adopting a whole-foods plant-based diet (WFPD) for their children. Using a modified survey instrument by Morton et al., this study focused on parents and children regarding dietary choices. Participants were recruited from the waiting area of a pediatric clinic at the Medical College of Georgia between April and May 2023. …
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
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
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 …
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
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 …
Balancing Honest Assessment And Compassion For Learners Experiencing Burnout: A Workshop And Feedback Tool For Clinical Teachers, Gal Barak, Dana Foradori, H Barrett Fromme, Linessa Zuniga, Andrea Dean
Balancing Honest Assessment And Compassion For Learners Experiencing Burnout: A Workshop And Feedback Tool For Clinical Teachers, Gal Barak, Dana Foradori, H Barrett Fromme, Linessa Zuniga, Andrea Dean
Faculty, Staff and Students Publications
INTRODUCTION: Burnout in medical trainees challenges their work effectiveness and impedes their ability to learn. Teachers in the clinical learning environment (CLE) are well situated to identify burnout and are often responsible for learner assessment.
METHODS: We developed a workshop to improve clinical teachers' identification and understanding of learner burnout while empowering them to provide constructive feedback and support. Building on best-practice feedback techniques and utilizing the Maslach Burnout Inventory (MBI) as a framework, we designed the GetINBurnOUT method to provide feedback and support for learners experiencing burnout. Applying Kern's six-step approach to curriculum development, we created and implemented a …
International Society For Pediatric And Adolescent Diabetes Clinical Practice Consensus Guidelines 2024: Diabetes Technologies - Insulin Delivery, Torben Biester, Cari Berget, Charlotte Boughton, Laura Cudizio, Laya Ekhlaspour, Marisa E Hilliard, Leenatha Reddy, Suzanne Sap Ngo Um, Melissa Schoelwer, Jennifer L Sherr, Klemen Dovc
International Society For Pediatric And Adolescent Diabetes Clinical Practice Consensus Guidelines 2024: Diabetes Technologies - Insulin Delivery, Torben Biester, Cari Berget, Charlotte Boughton, Laura Cudizio, Laya Ekhlaspour, Marisa E Hilliard, Leenatha Reddy, Suzanne Sap Ngo Um, Melissa Schoelwer, Jennifer L Sherr, Klemen Dovc
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 chapter builds on the 2022 ISPAD guidelines, and summarizes recent advances in the technology behind insulin administration, with special emphasis on insulin pump therapy, especially on glucose-responsive integrated technology that is feasible with the use of automated insulin delivery (AID) systems in children and adolescents. The International Society for Pediatric and Adolescent Diabetes (ISPAD) guidelines represent a rich repository that serves as the only comprehensive …
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
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 …
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
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 …
Should Targeted Temperature Management Be Used In Cardiogenic Shock Patients? Systematic Review And Meta-Analysis, Adam Nieborek, Jerzy R Ladny, Milosz J Jaguszewski, Andrea Denegri, Aleksandra Sikora, Szymon Pucylo, Aleksandra Gasecka, Michal Pruc, Ivan Savytsky, Lukasz Szarpak
Should Targeted Temperature Management Be Used In Cardiogenic Shock Patients? Systematic Review And Meta-Analysis, Adam Nieborek, Jerzy R Ladny, Milosz J Jaguszewski, Andrea Denegri, Aleksandra Sikora, Szymon Pucylo, Aleksandra Gasecka, Michal Pruc, Ivan Savytsky, Lukasz Szarpak
Faculty, Staff and Students Publications
BACKGROUND: Therapeutic hypothermia, or targeted temperature management (TTM), is a strategy of reducing the core body temperature of survivors of sudden cardiac arrest, cardiogenic shock (CS) or stroke. Therefore, a systematic literature review and meta-analysis were performed to tackle the question about whether the implementation of TTM is actually beneficial for patients with CS.
METHODS: Study was designed as a systematic review and meta-analysis. PubMed, Cochrane Library, Web of Science and Scopus were searched from these databases inception to July 17, 2022. Eligible studies were those comparing TTM and non-TTM treatment in CS patients. Data were pooled with the Mantel-Haenszel …
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
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 …
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
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 …
Colonic Malakoplakia In A Pediatric Renal Transplant Recipient Case Report, Kristen G Valencia Deray, Richard Kellermayer, Alexis C Gomez, Kalyani R Patel, Peace Imani, Seiji Kitagawa, Claire E Bocchini, Alvaro Orjuela
Colonic Malakoplakia In A Pediatric Renal Transplant Recipient Case Report, Kristen G Valencia Deray, Richard Kellermayer, Alexis C Gomez, Kalyani R Patel, Peace Imani, Seiji Kitagawa, Claire E Bocchini, Alvaro Orjuela
Faculty, Staff and Students Publications
Malakoplakia is a rare, chronic granulomatous disease that mainly affects the genitourinary system of immunocompromised adults. It is caused by a bactericidal deficit in macrophages and, therefore, the treatment includes antimicrobials that reach high concentrations in macrophages. To our knowledge, we present the first case of malakoplakia in a pediatric solid organ transplant recipient. Our patient is a 15-year-old male renal transplant recipient who presented with recurrent diarrhea. Blood, urine, and gastrointestinal pathogen panel testing were positive for enteroaggregative Escherichia coli. A colonoscopy revealed diffuse malakoplakia. He had a complete resolution of symptoms with trimethoprim-sulfamethoxazole therapy. Unfortunately, his malakoplakia recurred …
Recent Trends In The Incidence Of Gastric Cancer In The United States, Kyle S Liu, Syed Ahsan Raza, Hashem B El-Serag, Aaron P Thrift
Recent Trends In The Incidence Of Gastric Cancer In The United States, Kyle S Liu, Syed Ahsan Raza, Hashem B El-Serag, Aaron P Thrift
Faculty, Staff and Students Publications
BACKGROUND: Gastric cancer (GC) incidence rates overall in the United States have declined over recent decades and are predicted to continue declining. However, there have been mixed recent findings regarding the potential stabilization of rates and potential divergent trends by age group. We used the most recent cancer data for the United States and examined trends in GC between 1992 and 2019, overall and in important subgroups of the population.
METHODS: Age-adjusted GC incidence rates and trends in adults 20 years or older were calculated using data from the Surveillance, Epidemiology, and End Results (SEER) 12 program. Secular trends were …
To Compare The Efficacy Of Oral Deflazacort, Oral Prednisolone And Inhaled Budesonide In The Treatment Of Salbutamol Refractory Acute Asthma In Children, Santosh Kondekar, Unmesh Dev M, Surbhi Rathi, Gulrej N. Shaikh
To Compare The Efficacy Of Oral Deflazacort, Oral Prednisolone And Inhaled Budesonide In The Treatment Of Salbutamol Refractory Acute Asthma In Children, Santosh Kondekar, Unmesh Dev M, Surbhi Rathi, Gulrej N. Shaikh
Digital Journal of Clinical Medicine
Abstract
Background: A 22-month open-label randomized comparative study was conducted at tertiary care centre attached to medical college in metropolitan city in Maharashtra from Dec 2011 to Oct 2013, aiming to compare the effectiveness of three corticosteroid treatments - oral deflazacort, oral prednisolone, and inhaled budesonide in managing salbutamol-refractory asthma in children aged 1-14 years.
Methods: 90 afebrile children were equally allocated to the different treatment arms. Treatment arm A received oral Deflazacort (1mg/kg/day alternate day), treatment arm B received oral prednisolone (1mg/kg/day), and treatment arm C received budesonide inhaler (100 mcg/puff twice a day) for seven days, along with …
– Foreign Body Ingestion In Children(<12 Years) At A Tertiary Care Hospital: A Retrospective Cohort Study Studying Role Of Socioeconomic Status And Parental Supervision., Khyati Gupta, Gargi Rekhawar, Anisha Jain, Vishal D. Sawant, Sushma Save
– Foreign Body Ingestion In Children(<12 Years) At A Tertiary Care Hospital: A Retrospective Cohort Study Studying Role Of Socioeconomic Status And Parental Supervision., Khyati Gupta, Gargi Rekhawar, Anisha Jain, Vishal D. Sawant, Sushma Save
Digital Journal of Clinical Medicine
Background- Foreign body ingestion is a long-standing problem concerning pediatric population. We aim to evaluate the correlation between the median time interval between ingestion of foreign body and arrival to the hospital with socioeconomic factors, effect of parental supervision and number of children in the family. We also aim to find out the deciding factors for proper selection of method of removal of ingested foreign bodies.
Methods- The data was collected from patient’s admission files, medical and endoscopic records. Chi-square test was used to calculate the corelation of time since ingestion with the socioeconomic status of family, method of removal …
Assessing Discharge Knowledge For Caregivers Of Newborn (Using Video Discharge Instructions As An Adjunct To Standard Written Instructions) In Covid Period – A Randomized Controlled Trial, Jayesh Yadav, Khyati Gupta, Vishal D. Sawant
Assessing Discharge Knowledge For Caregivers Of Newborn (Using Video Discharge Instructions As An Adjunct To Standard Written Instructions) In Covid Period – A Randomized Controlled Trial, Jayesh Yadav, Khyati Gupta, Vishal D. Sawant
Digital Journal of Clinical Medicine
Background: Studies have demonstrated that caregivers of newborn frequently face challenges in comprehending the instructions provided to them upon discharge. Video discharge instructions provide a clear and concise message and can potentially alleviate factors which have an impact on comprehension of caregivers like illiteracy, communication barriers and limited physician time. We aim to assess the impact of incorporating video-based discharge instructions on caregivers' comprehension of newborn care and post-discharge follow-up, specifically in the context of the COVID-19 pandemic.
Methods: Caregivers of newborn in the COVID times were randomized into written only and written with video discharge instruction groups; …
Predictive Analytics In Bronchopulmonary Dysplasia: Past, Present, And Future, Bryan G Mcomber, Alvaro G Moreira, Kelsey Kirkman, Sebastian Acosta, Craig Rusin, Binoy Shivanna
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
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 …
Improving Health Outcomes In Children And Adolescents With Asthma, Elizabeth Kate Coffey Moore
Improving Health Outcomes In Children And Adolescents With Asthma, Elizabeth Kate Coffey Moore
Theses and Dissertations (ETD)
Asthma is among the most prevalent chronic illnesses found in the United States, affecting millions of people under the age of 18 each year. The disease burden for affected children, adolescents, their families, and the healthcare system is immense. Finding ways to mitigate its impact is critical to improving the lives of affected children, adolescents, and their families. The three chapters included in this dissertation aimed to document the asthma triggers that children and adolescents are exposed to and strategies for reducing them and to demonstrate using Imogene King’s conceptual framework to guide nursing practice to care for and nursing …
Smooth Muscle Contractile Responses To Bile Acids In Mouse Ileum Require Tgr5 But Not Asbt, Diana S Chang, Krishnakant G Soni, Geoffrey A Preidis
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 …
Hepatic Failure And Hyperbilirubinemia Secondary To Hemolytic Disease Of The Newborn, Matthew Reinhardt, Marya L Strand, Dajana Sabic
Hepatic Failure And Hyperbilirubinemia Secondary To Hemolytic Disease Of The Newborn, Matthew Reinhardt, Marya L Strand, Dajana Sabic
Faculty, Staff and Students Publications
Anti-D antibody is the most common cause of hemolytic disease of the newborn (HDN). Other antigens have emerged, causing significant damage to the newborn. We report a case of a 31-week dichorionic diamniotic twin born emergently to a mother with anti-C and anti-D antibodies who required multiple percutaneous umbilical cord blood sampling (PUBS) and transfusions. Extensive resuscitation at birth was required due to severe anemia, hypovolemia, and prematurity. Ensuing liver damage occurred with subsequent profound coagulopathy, transaminitis, and direct hyperbilirubinemia that required significant support. This patient developed several unique findings including the inability to monitor oxygen saturations due to dermal …
Efficacy And Safety Of Lumasiran For Infants And Young Children With Primary Hyperoxaluria Type 1: 30-Month Analysis Of The Phase 3 Illuminate-B Trial, Yaacov Frishberg, Wesley Hayes, Hadas Shasha-Lavsky, David J Sas, Mini Michael, Anne-Laure Sellier-Leclerc, Julien Hogan, Richard Willey, John M Gansner, Daniella Magen
Efficacy And Safety Of Lumasiran For Infants And Young Children With Primary Hyperoxaluria Type 1: 30-Month Analysis Of The Phase 3 Illuminate-B Trial, Yaacov Frishberg, Wesley Hayes, Hadas Shasha-Lavsky, David J Sas, Mini Michael, Anne-Laure Sellier-Leclerc, Julien Hogan, Richard Willey, John M Gansner, Daniella Magen
Faculty, Staff and Students Publications
BACKGROUND: Primary hyperoxaluria type 1 (PH1) is a genetic disorder resulting in overproduction of hepatic oxalate, potentially leading to recurrent kidney stones, nephrocalcinosis, chronic kidney disease, and kidney failure. Lumasiran, the first RNA interference therapeutic approved for infants and young children, is a liver-directed treatment that reduces hepatic oxalate production. Lumasiran demonstrated sustained efficacy with an acceptable safety profile over 12 months in infants and young children (age <6 >years) with PH1 in ILLUMINATE-B (clinicaltrials.gov: NCT03905694), an ongoing, Phase 3, multinational, open-label, single-arm study.
METHODS: Here, we report interim efficacy and safety findings from ILLUMINATE-B following 30 months of lumasiran treatment. …
Understanding The Role Of Ampa Receptors In Autism: Insights From Circuit And Synapse Dysfunction, Andres Jimenez-Gomez, Megan X Nguyen, Jason S Gill
Understanding The Role Of Ampa Receptors In Autism: Insights From Circuit And Synapse Dysfunction, Andres Jimenez-Gomez, Megan X Nguyen, Jason S Gill
Faculty, Staff and Students Publications
Autism spectrum disorders represent a diverse etiological spectrum that converge on a syndrome characterized by discrepant deficits in developmental domains often highlighted by concerns in socialization, sensory integration, and autonomic functioning. Importantly, the incidence and prevalence of autism spectrum disorders have seen sharp increases since the syndrome was first described in the 1940s. The wide etiological spectrum and rising number of individuals being diagnosed with the condition lend urgency to capturing a more nuanced understanding of the pathogenic mechanisms underlying the autism spectrum disorders. The current review seeks to understand how the disruption of AMPA receptor (AMPAr)-mediated neurotransmission in the …
Mecp2-Related Disorders While Gene-Based Therapies Are On The Horizon, Katherine Allison, Mirjana Maletic-Savatic, Davut Pehlivan
Mecp2-Related Disorders While Gene-Based Therapies Are On The Horizon, Katherine Allison, Mirjana Maletic-Savatic, Davut Pehlivan
Faculty, Staff and Students Publications
The emergence of new genetic tools has led to the discovery of the genetic bases of many intellectual and developmental disabilities. This creates exciting opportunities for research and treatment development, and a few genetic disorders (e.g., spinal muscular atrophy) have recently been treated with gene-based therapies. MECP2 is found on the X chromosome and regulates the transcription of thousands of genes. Loss of MECP2 gene product leads to Rett Syndrome, a disease found primarily in females, and is characterized by developmental regression, motor dysfunction, midline hand stereotypies, autonomic nervous system dysfunction, epilepsy, scoliosis, and autistic-like behavior. Duplication of MECP2 causes …
Metabolic Bioactivation Of Antidepressants: Advance And Underlying Hepatotoxicity, Saleh M Khalil, Kevin R Mackenzie, Mirjana Maletic-Savatic, Feng Li
Metabolic Bioactivation Of Antidepressants: Advance And Underlying Hepatotoxicity, Saleh M Khalil, Kevin R Mackenzie, Mirjana Maletic-Savatic, Feng Li
Faculty, Staff and Students Publications
Many drugs that serve as first-line medications for the treatment of depression are associated with severe side effects, including liver injury. Of the 34 antidepressants discussed in this review, four have been withdrawn from the market due to severe hepatotoxicity, and others carry boxed warnings for idiosyncratic liver toxicity. The clinical and economic implications of antidepressant-induced liver injury are substantial, but the underlying mechanisms remain elusive. Drug-induced liver injury may involve the host immune system, the parent drug, or its metabolites, and reactive drug metabolites are one of the most commonly referenced risk factors. Although the precise mechanism by which …
Optimizing Immunogenicity And Product Presentation Of A Sars-Cov-2 Subunit Vaccine Composition: Effects Of Delivery Route, Heterologous Regimens With Self-Amplifying Rna Vaccines, And Lyophilization, William R Lykins, Jeroen Pollet, Jessica A White, Brian Keegan, Leroy Versteeg, Ulrich Strych, Wen-Hsiang Chen, Raodoh Mohamath, Gabi Ramer-Denisoff, Sierra Reed, Christina Renshaw, 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
Optimizing Immunogenicity And Product Presentation Of A Sars-Cov-2 Subunit Vaccine Composition: Effects Of Delivery Route, Heterologous Regimens With Self-Amplifying Rna Vaccines, And Lyophilization, William R Lykins, Jeroen Pollet, Jessica A White, Brian Keegan, Leroy Versteeg, Ulrich Strych, Wen-Hsiang Chen, Raodoh Mohamath, Gabi Ramer-Denisoff, Sierra Reed, Christina Renshaw, 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: Dozens of vaccines have been approved or authorized internationally in response to the ongoing SARS-CoV-2 pandemic, covering a range of modalities and routes of delivery. For example, mucosal delivery of vaccines via the intranasal (i.n.) route has been shown to improve protective mucosal responses in comparison to intramuscular (i.m.) delivery. As we gain knowledge of the limitations of existing vaccines, it is of interest to understand if changes in product presentation or combinations of multiple vaccine modalities can further improve immunological outcomes.
METHODS: We investigated a commercial-stage SARS-CoV-2 receptor binding domain (RBD) antigen adjuvanted with a clinical-stage TLR-7/8 agonist …
Crystal Structure Of Trichinella Spiralis Calreticulin And The Structural Basis Of Its Complement Evasion Mechanism Involving C1q, Zhihui Jia, Wen Yu, Jingmo Li, Mingming Zhang, Bin Zhan, Liming Yan, Zhenhua Ming, Yuli Cheng, Xiaolin Tian, Shuai Shao, Jingjing Huang, Xinping Zhu
Crystal Structure Of Trichinella Spiralis Calreticulin And The Structural Basis Of Its Complement Evasion Mechanism Involving C1q, Zhihui Jia, Wen Yu, Jingmo Li, Mingming Zhang, Bin Zhan, Liming Yan, Zhenhua Ming, Yuli Cheng, Xiaolin Tian, Shuai Shao, Jingjing Huang, Xinping Zhu
Faculty, Staff and Students Publications
Helminths produce calreticulin (CRT) to immunomodulate the host immune system as a survival strategy. However, the structure of helminth-derived CRT and the structural basis of the immune evasion process remains unclarified. Previous study found that the tissue-dwelling helminth Trichinella spiralis produces calreticulin (TsCRT), which binds C1q to inhibit activation of the complement classical pathway. Here, we used x-ray crystallography to resolve the structure of truncated TsCRT (TsCRTΔ), the first structure of helminth-derived CRT. TsCRTΔ was observed to share the same binding region on C1q with IgG based on the structure and molecular docking, which explains the inhibitory effect of TsCRT …
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
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 …
Researching Covid To Enhance Recovery (Recover) Pediatric Study Protocol: Rationale, Objectives And Design, Rachel S Gross, Tanayott Thaweethai, Erika B Rosenzweig, James Chan, Lori B Chibnik, Mine S Cicek, Amy J Elliott, Valerie J Flaherman, Andrea S Foulkes, Margot Gage Witvliet, Richard Gallagher, Maria Laura Gennaro, Terry L Jernigan, Elizabeth W Karlson, Stuart D Katz, Patricia A Kinser, Lawrence C Kleinman, Michelle F Lamendola-Essel, Joshua D Milner, Sindhu Mohandas, Praveen C Mudumbi, Jane W Newburger, Kyung E Rhee, Amy L Salisbury, Jessica N Snowden, Cheryl R Stein, Melissa S Stockwell, Kelan G Tantisira, Moriah E Thomason, Dongngan T Truong, David Warburton, John C Wood, Shifa Ahmed, Almary Akerlundh, Akram N Alshawabkeh, Brett R Anderson, Judy L Aschner, Andrew M Atz, Robin L Aupperle, Fiona C Baker, Venkataraman Balaraman, Dithi Banerjee, Deanna M Barch, Arielle Baskin-Sommers, Sultana Bhuiyan, Marie-Abele C Bind, Amanda L Bogie, Tamara Bradford, Natalie C Buchbinder, Elliott Bueler, Hülya Bükülmez, B J Casey, Linda Chang, Maryanne Chrisant, Duncan B Clark, Rebecca G Clifton, Katharine N Clouser, Lesley Cottrell, Kelly Cowan, Viren D'Sa, Mirella Dapretto, Soham Dasgupta, Walter Dehority, Audrey Dionne, Kirsten B Dummer, Matthew D Elias, Shari Esquenazi-Karonika, Danielle N Evans, E Vincent S Faustino, Alexander G Fiks, Daniel Forsha, John J Foxe, Naomi P Friedman, Greta Fry, Sunanda Gaur, Dylan G Gee, Kevin M Gray, Stephanie Handler, Ashraf S Harahsheh, Keren Hasbani, Andrew C Heath, Camden Hebson, Mary M Heitzeg, Christina M Hester, Sophia Hill, Laura Hobart-Porter, Travis K F Hong, Carol R Horowitz, Daniel S Hsia, Matthew Huentelman, Kathy D Hummel, Katherine Irby, Joanna Jacobus, Vanessa L Jacoby, Pei-Ni Jone, David C Kaelber, Tyler J Kasmarcak, Matthew J Kluko, Jessica S Kosut, Angela R Laird, Jeremy Landeo-Gutierrez, Sean M Lang, Christine L Larson, Peter Paul C Lim, Krista M Lisdahl, Brian W Mccrindle, Russell J Mcculloh, Kimberly Mchugh, Alan L Mendelsohn, Torri D Metz, Julie Miller, Elizabeth C Mitchell, Lerraughn M Morgan, Eva M Müller-Oehring, Erica R Nahin, Michael C Neale, Manette Ness-Cochinwala, Sheila M Nolan, Carlos R Oliveira, Onyekachukwu Osakwe, Matthew E Oster, R Mark Payne, Michael A Portman, Hengameh Raissy, Isabelle G Randall, Suchitra Rao, Harrison T Reeder, Johana M Rosas, Mark W Russell, Arash A Sabati, Yamuna Sanil, Alice I Sato, Michael S Schechter, Rangaraj Selvarangan, S Kristen Sexson Tejtel, Divya Shakti, Kavita Sharma, Lindsay M Squeglia, Shubika Srivastava, Michelle D Stevenson, Jacqueline Szmuszkovicz, Maria M Talavera-Barber, Ronald J Teufel, Deepika Thacker, Felicia Trachtenberg, Mmekom M Udosen, Megan R Warner, Sara E Watson, Alan Werzberger, Jordan C Weyer, Marion J Wood, H Shonna Yin, William T Zempsky, Emily Zimmerman, Benard P Dreyer, Recover-Pediatric Consortium
Researching Covid To Enhance Recovery (Recover) Pediatric Study Protocol: Rationale, Objectives And Design, Rachel S Gross, Tanayott Thaweethai, Erika B Rosenzweig, James Chan, Lori B Chibnik, Mine S Cicek, Amy J Elliott, Valerie J Flaherman, Andrea S Foulkes, Margot Gage Witvliet, Richard Gallagher, Maria Laura Gennaro, Terry L Jernigan, Elizabeth W Karlson, Stuart D Katz, Patricia A Kinser, Lawrence C Kleinman, Michelle F Lamendola-Essel, Joshua D Milner, Sindhu Mohandas, Praveen C Mudumbi, Jane W Newburger, Kyung E Rhee, Amy L Salisbury, Jessica N Snowden, Cheryl R Stein, Melissa S Stockwell, Kelan G Tantisira, Moriah E Thomason, Dongngan T Truong, David Warburton, John C Wood, Shifa Ahmed, Almary Akerlundh, Akram N Alshawabkeh, Brett R Anderson, Judy L Aschner, Andrew M Atz, Robin L Aupperle, Fiona C Baker, Venkataraman Balaraman, Dithi Banerjee, Deanna M Barch, Arielle Baskin-Sommers, Sultana Bhuiyan, Marie-Abele C Bind, Amanda L Bogie, Tamara Bradford, Natalie C Buchbinder, Elliott Bueler, Hülya Bükülmez, B J Casey, Linda Chang, Maryanne Chrisant, Duncan B Clark, Rebecca G Clifton, Katharine N Clouser, Lesley Cottrell, Kelly Cowan, Viren D'Sa, Mirella Dapretto, Soham Dasgupta, Walter Dehority, Audrey Dionne, Kirsten B Dummer, Matthew D Elias, Shari Esquenazi-Karonika, Danielle N Evans, E Vincent S Faustino, Alexander G Fiks, Daniel Forsha, John J Foxe, Naomi P Friedman, Greta Fry, Sunanda Gaur, Dylan G Gee, Kevin M Gray, Stephanie Handler, Ashraf S Harahsheh, Keren Hasbani, Andrew C Heath, Camden Hebson, Mary M Heitzeg, Christina M Hester, Sophia Hill, Laura Hobart-Porter, Travis K F Hong, Carol R Horowitz, Daniel S Hsia, Matthew Huentelman, Kathy D Hummel, Katherine Irby, Joanna Jacobus, Vanessa L Jacoby, Pei-Ni Jone, David C Kaelber, Tyler J Kasmarcak, Matthew J Kluko, Jessica S Kosut, Angela R Laird, Jeremy Landeo-Gutierrez, Sean M Lang, Christine L Larson, Peter Paul C Lim, Krista M Lisdahl, Brian W Mccrindle, Russell J Mcculloh, Kimberly Mchugh, Alan L Mendelsohn, Torri D Metz, Julie Miller, Elizabeth C Mitchell, Lerraughn M Morgan, Eva M Müller-Oehring, Erica R Nahin, Michael C Neale, Manette Ness-Cochinwala, Sheila M Nolan, Carlos R Oliveira, Onyekachukwu Osakwe, Matthew E Oster, R Mark Payne, Michael A Portman, Hengameh Raissy, Isabelle G Randall, Suchitra Rao, Harrison T Reeder, Johana M Rosas, Mark W Russell, Arash A Sabati, Yamuna Sanil, Alice I Sato, Michael S Schechter, Rangaraj Selvarangan, S Kristen Sexson Tejtel, Divya Shakti, Kavita Sharma, Lindsay M Squeglia, Shubika Srivastava, Michelle D Stevenson, Jacqueline Szmuszkovicz, Maria M Talavera-Barber, Ronald J Teufel, Deepika Thacker, Felicia Trachtenberg, Mmekom M Udosen, Megan R Warner, Sara E Watson, Alan Werzberger, Jordan C Weyer, Marion J Wood, H Shonna Yin, William T Zempsky, Emily Zimmerman, Benard P Dreyer, Recover-Pediatric Consortium
Faculty, Staff and Students Publications
IMPORTANCE: The prevalence, pathophysiology, and long-term outcomes of COVID-19 (post-acute sequelae of SARS-CoV-2 [PASC] or "Long COVID") in children and young adults remain unknown. Studies must address the urgent need to define PASC, its mechanisms, and potential treatment targets in children and young adults.
OBSERVATIONS: We describe the protocol for the Pediatric Observational Cohort Study of the NIH's REsearching COVID to Enhance Recovery (RECOVER) Initiative. RECOVER-Pediatrics is an observational meta-cohort study of caregiver-child pairs (birth through 17 years) and young adults (18 through 25 years), recruited from more than 100 sites across the US. This report focuses on two of …
Anomalous Aortic Origin Of A Coronary Artery In Pediatric Patients, Tam T Doan, Charles Puelz, Craig Rusin, Silvana Molossi
Anomalous Aortic Origin Of A Coronary Artery In Pediatric Patients, Tam T Doan, Charles Puelz, Craig Rusin, Silvana Molossi
Faculty, Staff and Students Publications
PURPOSE OF REVIEW: We present a contemporary approach to risk assessment and management of patients with anomalous aortic origin of a coronary artery (AAOCA).
RECENT FINDINGS: Anomalous left coronary artery from the right aortic sinus (L-AAOCA) with interarterial course carries a high risk of sudden cardiac death (SCD); therefore, current guidelines recommend exercise restriction and surgical intervention. Recent data in intraseptal and juxtacommissural L-AAOCA showed inducible perfusion abnormalities, leading to consideration of surgical intervention. Anomalous right coronary artery from the left aortic sinus (R-AAOCA) carries a much lower risk and stress perfusion imaging is helpful in identifying patients with inducible …