164 results match your criteria: "The Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine[Affiliation]"

Article Synopsis
  • The MMPOWER-3 clinical trial initially found no overall benefit of elamipretide for adults with primary mitochondrial myopathy, but a subgroup with nuclear DNA variants showed improvement in a six-minute walk test.
  • A large portion of the trial subjects had mitochondrial DNA variants, with particular improvements noted among those possessing pathogenic variants related to mtDNA maintenance.
  • Further analyses aim to identify trends among responders to assist in designing a more targeted Phase 3 trial for those likely to benefit from elamipretide.
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Necrotizing enterocolitis (NEC) is a known cause of morbidity and mortality in infants with congenital heart disease (CHD), but reports about the burden of cardiogenic NEC frequently conflict. To synthesize the extant literature on the incidence, risk factors, and prognosis of NEC in patients with CHD. Medline, Cochrane, and EMBASE were searched from 1946 through 2023 for studies of NEC in infants 0-12 months of age with CHD.

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Background: Outcomes for children with heart disease improved over the past decades. Quality improvement (QI) research in paediatric cardiac critical care is a key driver of improvement. The availability and variability of QI research across the field is unknown.

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Liver Lymphatic Anatomy and Its Role in Systemic Health and Disease.

Lymphology

October 2024

Jill and Mark Fishman Center for Lymphatic Disorders, Children's Hospital of Philadelphia, Philadelphia, USA.

Lymphatic anatomy of the abdomen is reviewed with focus on hepatic and mesenteric vessels in normal and pathologic conditions. Anatomy and pathophysiology is highlight using both specialized fluoroscopy and T2 Dynamic Contrast MR lymphangiography (DCMRL). Plastic bronchitis, chylothorax, protein losing enteropathy, and both cirrhosis and ascites due to hepatic lymphatics are highlighted.

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Lymphatic flow disorders are reviewed, and a classification based on magnetic resonance lymphography findings outlined. Examples of successful interventional management based on this classification are provided. Presented at the 2023 ISL International Congress of Lymphology, Genoa, Italy in a special symposium on central and regional lymphatic system in health and disease.

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Advances in lymphatic imaging for both diagnosis and intervention are reviewed, and specific examples given for protein-losing enteropathy, multi compartment lymphatic failure, congestive heart failure. Presented at the 2023 ISL International Congress of Lymphology, Genoa, Italy in a special symposium on central and regional lymphatic system in health and disease.

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Serratia marcescens outbreak in a neonatal intensive care unit associated with contaminated handwashing sinks.

Indian J Med Microbiol

December 2024

Hospital Infection Control Department, Guangzhou Women and Children's Medical Center, Guangzhou Medical University, Guangdong Provincial Clinical Research Center for Child Health, Guangzhou, 510623, China. Electronic address:

Article Synopsis
  • The article investigates a S. marcescens outbreak in a neonatal intensive care unit (NICU) and identifies the origin through a case-control study of affected and unaffected neonates.
  • Whole-genome sequencing and various software were used to analyze the outbreak and determine significant risk factors, such as proximity to contaminated sinks and staff rotation patterns.
  • The study concludes that contaminated handwashing sinks were key in spreading S. marcescens, emphasizing the need for better hygiene practices and monitoring of sink contamination to prevent future outbreaks.
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Education Research: Quality and Validity Evidence for a National In-Training Examination for Epilepsy Fellows.

Neurol Educ

December 2023

From the Department of Neurology (J. Moeller), Yale School of Medicine, New Haven, CT; Departments of Neurology and Pediatrics (E.G.-G.), University of California, San Francisco; Departments of Neurology and Pediatrics (F.W.F., S.K.K.), Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia; Department of Neurology (E.L.J.), Johns Hopkins School of Medicine, Baltimore, MD; Department of Neurology and Pediatric Institute (A. Kheder), Emory University School of Medicine, Atlanta, GA; Department of Pediatric Neurology (J. MacLean), Sutter Medical Foundation, Mountain View, CA; Neurology Department (E.L.M.), Kaiser Permanente Los Angeles Medical Center, CA; University of Washington Regional Epilepsy Center (W.G.M.), University of Washington, Seattle; Department of Neurology (J.M.O.), Tufts University, Boston, MA; Department of Neurology (S.S.), Medical University of South Carolina, Charleston; Division of Epilepsy (P.E.V.), Department of Neurology, Brigham and Womens Hospital, Harvard Medical School, Boston, MA; Department of Neurology (L.C.W.-K.), Mayo Clinic College of Medicine, Rochester, MN; American Epilepsy Society (K.J.K., A. Kephart), Chicago, IL; and Department of Neurology (F.A.L.), Zucker School of Medicine at Hofstra-Northwell, Hempstead, NY.

Background And Objectives: Epilepsy education has been transformed over the past 2 decades, leading to a need for structured formative assessment tools. The American Epilepsy Society developed the Epilepsy Fellowship In-Training Examination (EpiFITE) to provide high-quality formative assessment for fellows, to stimulate program improvement, and to guide future learning and teaching. The aim of this study was to explore validity evidence for the EpiFITE in meeting these goals.

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Objective: To identify delivery room (DR) characteristics of patients with transposition of the great arteries (TGA) who underwent preoperative balloon atrial septostomy (BAS).

Study Design: Retrospective cohort study of all patients with prenatally diagnosed TGA delivered at our center between 2013 and 2023 who underwent arterial switch operation during the newborn admission.

Results: A total of 168 patients were included (median gestational age 39.

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Objective: Evaluate the association between results of the room air (RA) challenge and death, respiratory morbidity, and neurodevelopmental impairment (NDI) at 2 years' corrected age.

Study Design: Cohort study of infants born <27 weeks' gestational age who underwent a RA challenge to determine BPD diagnosis at 36 weeks postmenstrual age.

Results: Of 1022 infants eligible for the RA challenge, 554 underwent testing and 223 passed.

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Chylopericardium is a rare complication after cardiac transplantation. We report a case of a 69-year-old woman with persistent chylopericardium after a heart transplantation due to Chagas disease. Failure of conservative treatment led to dynamic contrast-enhanced magnetic resonance lymphangiography and percutaneous radiologic intervention of the lymphatic leakage and symptoms resolution.

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Background: The rates of major neonatal morbidities, such as bronchopulmonary dysplasia, necrotizing enterocolitis, preterm white matter disease, and retinopathy of prematurity, remain high among surviving preterm infants. Exposure to inflammatory stimuli and the subsequent host innate immune response contribute to the risk of developing these complications of prematurity. Notably, the burden of inflammation and associated neonatal morbidity is inversely related to gestational age - leaving primarily but not exclusively the tiniest babies at highest risk.

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Immune cells experience large cell shape changes during environmental patrolling because of the physical constraints that they encounter while migrating through tissues. These cells can adapt to such deformation events using dedicated shape-sensing pathways. However, how shape sensing affects immune cell function is mostly unknown.

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Background: Parent/caregiver-completed developmental testing (PCDT) is integral to developmental care in children; however, there is limited information on its accuracy. In this systematic review, we compared the diagnostic accuracy of PCDT with concurrently administered Bayley Scales of Infant Development for detection of developmental delay (DD) in children below 4 years of age.

Methods: We searched databases PubMed, Embase, CINAHL, PsycINFO and Google Scholar until November 2023.

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Article Synopsis
  • The immune system has the potential to control cancer, but the role of certain immune sensors in cancer aggressiveness, specifically NLRC4, remains largely unexplored in humans.
  • This study found that decreased levels of NLRC4 in colorectal cancer (CRC) cells are linked to poorer immune cell infiltration and worse patient outcomes, indicating its importance in tumor progression.
  • Enhancing NLRC4 expression in CRC cells led to immune reprogramming that improved the function of immune cells, suggesting that targeting NLRC4 could provide a new approach to boosting antitumor responses in various types of carcinoma.
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Objective: To understand factors associated with missed academic time after concussion to improve support for patients. Our goal was to assess patient-specific predictors of total school time lost after pediatric/adolescent concussion.

Study Design: We performed a prospective cohort study of children and adolescents (8-18 years of age) seen within 14 days of concussion from seven pediatric medical centers across the United States.

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Background: The association between chest compression (CC) pause duration and pediatric in-hospital cardiac arrest survival outcomes is unknown. The American Heart Association has recommended minimizing pauses in CC in children to <10 seconds, without supportive evidence. We hypothesized that longer maximum CC pause durations are associated with worse survival and neurological outcomes.

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Emerging Adults With Type 2 Diabetes: Understanding Illness Experience and Transition to Adult Care.

J Adolesc Health

July 2024

Department of Pediatrics, Children's Hospital of Philadelphia and University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania; Policy Lab and Clinical Futures, Children's Hospital of Philadelphia, Roberts Center for Pediatric Research, Philadelphia, Pennsylvania; Leonard Davis Institute of Health Economics, University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania.

Article Synopsis
  • Increasing cases of youth-onset type 2 diabetes (T2D) highlight a need to understand how emerging adults navigate the transition from pediatric to adult care.
  • This study utilized interviews, the Transition Readiness Assessment Questionnaire (TRAQ), and medical records to explore the experiences and challenges faced by these individuals during their transition.
  • Key findings indicate that factors like coping with chronic illness and mental health, along with the need for support, are crucial during this period, with no significant difference in experiences based on TRAQ scores.
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Background: Bronchopulmonary dysplasia (BPD) has become a major cause of morbidity and mortality in preterm infants worldwide, yet its pathogenesis and underlying mechanisms remain poorly understood. The present study sought to explore microRNA-mRNA regulatory networks and immune cells involvement in BPD through a combination of bioinformatic analysis and experimental validation.

Methods: MicroRNA and mRNA microarray datasets were obtained from the Gene Expression Omnibus (GEO) database.

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Introduction: Influenza infections contribute to excess healthcare utilization, morbidity, and mortality in individuals with glomerular disease (GD); however, influenza vaccination may not yield protective immune responses in this high-risk patient population. The objective of the present study was to describe influenza vaccine administration from 2010 to 2019 and explore the effectiveness of influenza vaccination in patients with GD.

Methods: We conducted an observational cohort study using healthcare claims for seasonal influenza vaccination (exposure) as well as influenza and influenza-like illness (outcomes) from commercially insured children and adults <65 years of age with primary GD in the Merative MarketScan Research Databases.

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Objective: To evaluate changes in prevalence and severity of cerebral palsy (CP) among surviving children born at <27 weeks of gestation over time and to determine associations between CP and other developmental domains, functional impairment, medical morbidities, and resource use among 2-year-old children who were born extremely preterm.

Study Design: Retrospective cohort study using prospective registry data, conducted at 25 centers of the Eunice Kennedy Shriver National Institute of Child Health and Human Development Neonatal Research Network. Participants were children born at <27 weeks of gestation and followed at 18 through 26 months of corrected age from 2008 through 2019.

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Objective: To determine the rate of and factors associated with suboptimal discharge antimicrobial prescribing at a tertiary referral children's hospital.

Design: Retrospective cohort.

Setting: Tertiary referral children's hospital.

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Immunotherapy targeting B cells and long-lived plasma cells effectively eliminates pre-existing donor-specific allo-antibodies.

Cell Rep Med

December 2023

Department of Pathology & Laboratory Medicine, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA 19104, USA; Center for Cellular Immunotherapies, University of Pennsylvania, Perelman School of Medicine, Philadelphia, PA 19104, USA. Electronic address:

Pre-existing anti-human leukocyte antigen (HLA) allo-antibodies constitute a major barrier to transplantation. Current desensitization approaches fail due to ineffective depletion of allo-specific memory B cells (Bmems) and long-lived plasma cells (LLPCs). We evaluate the efficacy of chimeric antigen receptor (CAR) T cells targeting CD19 and B cell maturation antigen (BCMA) to eliminate allo-antibodies in a skin pre-sensitized murine model of islet allo-transplantation.

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Objectives: To assess the presence and timing of furosemide diuretic tolerance in infants with bronchopulmonary dysplasia (BPD), and to determine if tolerance is modified by thiazide co-administration.

Study Design: We performed a retrospective cohort study among infants born very preterm with BPD exposed to repeated-dose furosemide for 72 hours, measuring net fluid balance (total intake minus total output) as a surrogate of diuresis in the 3 days before and after exposure. The primary comparison was the difference in fluid balance between the first and third 24 hours of furosemide exposure.

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