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Articles 1 - 30 of 33
Full-Text Articles in Medicine and Health Sciences
Eliminating Contamination In Umbilical Cord Blood Culture Sampling For Early-Onset Neonatal Sepsis., Vilmaris Quinones Cardona, Vanessa Lowery, David Cooperberg, Endla K Anday, Alison J Carey
Eliminating Contamination In Umbilical Cord Blood Culture Sampling For Early-Onset Neonatal Sepsis., Vilmaris Quinones Cardona, Vanessa Lowery, David Cooperberg, Endla K Anday, Alison J Carey
Department of Pediatrics Faculty Papers
Introduction: Despite the advantages of umbilical cord blood culture (UCBC) use for diagnosis of early onset sepsis (EOS), contamination rates have deterred neonatologists from its widespread use. We aimed to implement UCBC collection in a level III neonatal intensive care unit (NICU) and apply quality improvement (QI) methods to reduce contamination in the diagnosis of early onset sepsis. Methods: Single-center implementation study utilizing quality improvement methodology to achieve 0% contamination rate in UCBC samples using the Plan-Do-Study-Act (PDSA) model for improvement. UCBC was obtained in conjunction with peripheral blood cultures (PBC) in neonates admitted to the NICU due to maternal …
Coping In Pediatric Burn Survivors And Its Relation To Social Functioning And Self-Concept, Mira D H Snider, Sarah Young, Paul T Enlow, Corrine Ahrabi-Nejad, Ariel M Aballay, Christina L Duncan
Coping In Pediatric Burn Survivors And Its Relation To Social Functioning And Self-Concept, Mira D H Snider, Sarah Young, Paul T Enlow, Corrine Ahrabi-Nejad, Ariel M Aballay, Christina L Duncan
Department of Pediatrics Faculty Papers
Pediatric burn survivors experience increased risk for bullying, stigmatization, body image concerns, and problematic social functioning. Although coping behaviors are associated with engagement in social supports and positive self-concept in multiple pediatric illness populations, their relation has not been examined in pediatric burns. This study examined coping in relation to social functioning and self-concept in 51 pediatric burn survivors aged 7–17years (M=12.54; SD=2.65). Survivors and their caregivers completed the Child Coping Strategies Checklist (CCSC; youth report); the Burn Injury Social Questionnaire (BISQ; parent and youth report); and the Piers-Harris Children’s Self-Concept Scale-2 (PH-2; youth report). Associations between coping, social functioning, …
Evaluation Of Pediatric Rheumatology Telehealth Satisfaction During The Covid-19 Pandemic, Lindsay N Waqar-Cowles, John Chuo, Pamela F Weiss, Sabrina Gmuca, Marianna Lanoue, Jon M Burnham
Evaluation Of Pediatric Rheumatology Telehealth Satisfaction During The Covid-19 Pandemic, Lindsay N Waqar-Cowles, John Chuo, Pamela F Weiss, Sabrina Gmuca, Marianna Lanoue, Jon M Burnham
College of Population Health Faculty Papers
Background: During the Coronavirus disease 2019 pandemic, ambulatory pediatric rheumatology healthcare rapidly transformed to a mainly telehealth model. However, pediatric patient and caregiver satisfaction with broadly deployed telehealth programs remains largely unknown. This study aimed to evaluate patient/caregiver satisfaction with telehealth and identify the factors associated with satisfaction in a generalizable sample of pediatric rheumatology patients.
Methods: Patients with an initial telehealth video visit with a rheumatology provider between April and June 2020 were eligible. All patients/caregivers were sent a post-visit survey to assess a modified version of the Telehealth Usability Questionnaire (TUQ) and demographic and clinical characteristics. TUQ total …
Disparities In Delaware Caregiver Beliefs About The Covid-19 Vaccine For Their Children, Thao-Ly Phan, Paul T Enlow, Michael K. Wong, Amanda M. Lewis, Anne E Kazak, Jonathan M. Miller
Disparities In Delaware Caregiver Beliefs About The Covid-19 Vaccine For Their Children, Thao-Ly Phan, Paul T Enlow, Michael K. Wong, Amanda M. Lewis, Anne E Kazak, Jonathan M. Miller
Department of Pediatrics Faculty Papers
Objective: To describe sociodemographic disparities in caregiver beliefs about the COVID-19 vaccine for their children. Methods: This was a cross-sectional study, linking caregiver-reported data to geocoded sociodemographic data from child EHRs. Caregivers of children receiving care in a Delaware pediatric healthcare system were invited to complete a survey about COVID-19 vaccine beliefs from March 19 to April 16, 2021. Results: 1499 caregivers participated (18% Black, 11% Hispanic, 32% public insurance, 12% rural). 54% of caregivers intended to vaccinate their children, while 34% were unsure and 12% would not. Caregivers of younger children (aOR 3.70, CI 2.36-5.79), Black children (aOR 2.11, …
Assessment Of Temporal Variations In Adherence To Nrp Using Video Recording In The Delivery Room, Amy J Sloane, Kaitlin M Kenaley, Michael T Favara
Assessment Of Temporal Variations In Adherence To Nrp Using Video Recording In The Delivery Room, Amy J Sloane, Kaitlin M Kenaley, Michael T Favara
Department of Medicine Faculty Papers
INTRODUCTION: Video recording and video evaluation tools have been successfully used to evaluate neonatal resuscitation performance. The objective of our study was to evaluate differences in Neonatal Resuscitation Program (NRP) adherence at time of birth between three temporal resuscitative periods using scored video recordings.
METHODS: This is a retrospective review of in-situ resuscitation video recordings from a level 3 perinatal center between 2017 and 2018. The modified Neonatal Resuscitation Assessment (mNRA) scoring tool was used as a surrogate marker to assess NRP adherence during daytime, evening, and nighttime hours.
RESULTS: A total of 260 resuscitations, of which 258 were births …
Promoter Considerations In The Design Of Lentiviral Vectors For Use In Treating Lysosomal Storage Diseases, Estera Rintz, Takashi Higuchi, Hiroshi Kobayashi, Deni S Galileo, Grzegorz Wegrzyn, Shunji Tomatsu
Promoter Considerations In The Design Of Lentiviral Vectors For Use In Treating Lysosomal Storage Diseases, Estera Rintz, Takashi Higuchi, Hiroshi Kobayashi, Deni S Galileo, Grzegorz Wegrzyn, Shunji Tomatsu
Department of Pediatrics Faculty Papers
More than 50 lysosomal storage diseases (LSDs) are associated with lysosomal dysfunctions with the frequency of 1:5,000 live births. As a result of missing enzyme activity, the lysosome dysfunction accumulates undegraded or partially degraded molecules, affecting the entire body. Most of them are life-threatening diseases where patients could die within the first or second decade of life. Approximately 20 LSDs have the approved treatments, which do not provide the cure for the disorder. Therefore, the delivery of missing genes through gene therapy is a promising approach for LSDs. Over the years, ex vivo lentiviral-mediated gene therapy for LSDs has been …
Health Care Policy And Congenital Heart Disease: 2020 Focus On Our 2030 Future, Devyani Chowdhury, Jonathan N Johnson, Carissa M Baker-Smith, Robert D B Jaquiss, Arjun K Mahendran, Valerie Curren, Aarti Bhat, Angira Patel, Audrey C Marshall, Stephanie Fuller, Bradley S Marino, Christina M Fink, Keila N Lopez, Lowell H Frank, Mishaal Ather, Natalie Torentinos, Olivia Kranz, Vivian Thorne, Ryan R Davies, Stuart Berger, Christopher Snyder, Arwa Saidi, Kenneth Shaffer
Health Care Policy And Congenital Heart Disease: 2020 Focus On Our 2030 Future, Devyani Chowdhury, Jonathan N Johnson, Carissa M Baker-Smith, Robert D B Jaquiss, Arjun K Mahendran, Valerie Curren, Aarti Bhat, Angira Patel, Audrey C Marshall, Stephanie Fuller, Bradley S Marino, Christina M Fink, Keila N Lopez, Lowell H Frank, Mishaal Ather, Natalie Torentinos, Olivia Kranz, Vivian Thorne, Ryan R Davies, Stuart Berger, Christopher Snyder, Arwa Saidi, Kenneth Shaffer
Division of Cardiology Faculty Papers
The congenital heart care community faces a myriad of public health issues that act as barriers toward optimum patient outcomes. In this article, we attempt to define advocacy and policy initiatives meant to spotlight and potentially address these challenges. Issues are organized into the following 3 key facets of our community: patient population, health care delivery, and workforce. We discuss the social determinants of health and health care disparities that affect patients in the community that require the attention of policy makers. Furthermore, we highlight the many needs of the growing adults with congenital heart disease and those with comorbidities, …
Clinical Assessment Of Ventricular Wall Stress In Understanding Compensatory Hypertrophic Response And Maladaptive Ventricular Remodeling, Takeshi Tsuda
Department of Pediatrics Faculty Papers
Ventricular wall stress (WS) is an important hemodynamic parameter to represent myocardial oxygen demand and ventricular workload. The normalization of WS is regarded as a physiological feedback signal that regulates the rate and extent of ventricular hypertrophy to maintain myocardial homeostasis. Although hypertrophy is an adaptive response to increased biomechanical stress, persistent hypertrophic stimulation forces the stressed myocardium into a progressive maladaptive process called ventricular remodeling, consisting of ventricular dilatation and dysfunction in conjunction with the development of myocyte hypertrophy, apoptosis, and fibrosis. The critical determinant of this pathological transition is not fully understood, but an energetic mismatch due to …
The Pnpla3 Rs738409 Variant But Not Mboat7 Rs641738 Is A Risk Factor For Nonalcoholic Fatty Liver Disease In Obese U.S. Children Of Hispanic Ethnicity, Sana Mansoor, Anshu Maheshwari, Matthew D. Di Guglielmo, Katryn Furuya, Makala Wang, Erin Crowgey, Zarela Molle-Rios, Zhaoping He
The Pnpla3 Rs738409 Variant But Not Mboat7 Rs641738 Is A Risk Factor For Nonalcoholic Fatty Liver Disease In Obese U.S. Children Of Hispanic Ethnicity, Sana Mansoor, Anshu Maheshwari, Matthew D. Di Guglielmo, Katryn Furuya, Makala Wang, Erin Crowgey, Zarela Molle-Rios, Zhaoping He
Department of Pediatrics Faculty Papers
Purpose: The rs641738 C>T in membrane-bound O-acyltransferase domain-containing protein 7 (MBOAT7) is implicated, along with the rs738409 C>G polymorphism in patatin-like phospholipase domain-containing protein 3 (PNPLA3), in nonalcoholic fatty liver disease (NAFLD). The association of these polymorphisms and NAFLD are investigated in Hispanic children with obesity.
Methods: Obese children with and without NAFLD were enrolled at a pediatric tertiary care health system and genotyped for MBOAT7 rs641738 C>T and PNPLA3 rs738409 C>G. NAFLD was characterized by the ultrasonographic presence of hepatic steatosis along with persistently elevated liver enzymes. Genetic variants and demographic and biochemical data were …
Restrictive Versus Liberal Transfusion Thresholds In Very Low Birth Weight Infants: A Systematic Review With Meta-Analysis, Peng Wang, Xing Wang, Haidong Deng, Linjie Li, Weelic Chong, Yang Hai, Yu Zhang
Restrictive Versus Liberal Transfusion Thresholds In Very Low Birth Weight Infants: A Systematic Review With Meta-Analysis, Peng Wang, Xing Wang, Haidong Deng, Linjie Li, Weelic Chong, Yang Hai, Yu Zhang
Department of Medicine Faculty Papers
BACKGROUND: To assess the efficacy and safety of restrictive versus liberal red blood cell transfusion thresholds in very low birth weight infants.
METHODS: We searched MEDLINE, EMBASE, and Cochrane database without any language restrictions. The last search was conducted in August 15, 2020. All randomized controlled trials comparing the use of restrictive versus liberal red blood cell transfusion thresholds in very low birth weight (VLBW) infants were selected. Pooled risk ratio (RR) for dichotomous variable with 95% confidence intervals were assessed by a random-effects model. The primary outcome was all-cause mortality.
RESULTS: Overall, this meta-analysis included 6 randomized controlled trials …
Glycosaminoglycans As Biomarkers For Mucopolysaccharidoses And Other Disorders, Paige Amendum, Shaukat Khan, Seiji Yamaguchi, Hironori Kobayashi, Yasuhiko Ago, Yasuyuki Suzuki, Betul Celik, Estera Rintz, Jobayer Hossain, Wendi Xiao, Shunji Tomatsu
Glycosaminoglycans As Biomarkers For Mucopolysaccharidoses And Other Disorders, Paige Amendum, Shaukat Khan, Seiji Yamaguchi, Hironori Kobayashi, Yasuhiko Ago, Yasuyuki Suzuki, Betul Celik, Estera Rintz, Jobayer Hossain, Wendi Xiao, Shunji Tomatsu
Department of Pediatrics Faculty Papers
Glycosaminoglycans (GAGs) are present in proteoglycans, which play critical physiological roles in various tissues. They are known to be elevated in mucopolysaccharidoses (MPS), a group of rare inherited metabolic diseases in which the lysosomal enzyme required to break down one or more GAG is deficient. In a previous study, we found elevation of GAGs in a subset of patients without MPS. In the current study, we aim to investigate serum GAG levels in patients with conditions beyond MPS. In our investigated samples, the largest group of patients had a clinical diagnosis of viral or non-viral encephalopathy. Clinical diagnoses and conditions …
Targeted Provider Education And Pre-Visit Planning Increase Rates Of Formal Depression Screening In Childhood-Onset Sle., Evan Mulvihill, Rebecca Furru, Alana Goldstein-Leever, Kyla Driest, Stephanie Lemle, Darby Macdonald, Emily Frost, Vidya Sivaraman
Targeted Provider Education And Pre-Visit Planning Increase Rates Of Formal Depression Screening In Childhood-Onset Sle., Evan Mulvihill, Rebecca Furru, Alana Goldstein-Leever, Kyla Driest, Stephanie Lemle, Darby Macdonald, Emily Frost, Vidya Sivaraman
Department of Pediatrics Faculty Papers
BACKGROUND: Despite being at high risk for depression, patients with childhood-onset systemic lupus erythematosus (c-SLE) are infrequently and inconsistently screened for depression by their pediatric rheumatologists. We aimed to systematically increase rates of formal depression screening for c-SLE patients in an academic Pediatric Rheumatology clinic.
METHODS: Our multi-disciplinary quality improvement (QI) team used electronic health record (EHR) documentation to retroactively calculate baseline rates of documented depression screening using the Patient Health Questionnaire-9 (PHQ-9). We then engaged key stakeholders to develop a clinical workflow for formal depression screening in the clinic. We also provided education to providers regarding mental health disorders …
Outcomes Of Intravenous Chemotherapy (Chemoreduction) For Retinoblastoma Based On Patient Age In 964 Eyes Of 554 Patients., Zeynep Bas, Lauren A. Dalvin, Sameeksha Tadepalli, Raksha Rao, Amish Shah, Ann M. Leahey, Carol L. Shields
Outcomes Of Intravenous Chemotherapy (Chemoreduction) For Retinoblastoma Based On Patient Age In 964 Eyes Of 554 Patients., Zeynep Bas, Lauren A. Dalvin, Sameeksha Tadepalli, Raksha Rao, Amish Shah, Ann M. Leahey, Carol L. Shields
Wills Eye Hospital Papers
PURPOSE: To evaluate retinoblastoma control after intravenous chemotherapy (chemoreduction) by patient age at presentation.
DESIGN: Retrospective case series.
METHODS: This study included 964 eyes of 554 patients treated with chemoreduction at Ocular Oncology Service at Wills Eye Hospital. Patients received 6 monthly cycles of standard chemoreduction. Additional therapies for tumor control were performed as needed.
RESULTS: Of 964 eyes, a comparison by age group (24 months) revealed more advanced age group with higher frequency of group E tumor (15% vs. 25% vs. 32% vs. 39%, P < 0.001). By treatment outcomes, complete tumor control was achieved with chemoreduction alone more often in less advanced age group (46% vs. 30% vs. 17% vs. 8%, P < 0.001). Additional treatment after chemoreduction was needed more often in more advanced age group with external beam radiotherapy (EBRT; 9% vs. 16% vs. 20% vs. 15%, P = 0.006) or enucleation (12% vs. 18% vs. 26% vs. 37%, P < 0.001). Over time (1994-1998 vs. 1999-2003 vs. 2004-2008 vs. 2009-2013 vs. 2014-2019), the paradigm for additional required treatment after chemoreduction shifted toward less EBRT (27% vs. 24% vs. 14% vs. 7% vs. 2%, P < 0.001) and more intra-arterial (0% vs. 0% vs. 1% vs. 25% vs. 48%, P < 0.001) and intravitreal (0% vs. 0% vs. 3% vs. 10% vs. 20%, P < 0.001) chemotherapy.
CONCLUSIONS: Chemoreduction is a safe and effective treatment method for patients with retinoblastoma, demonstrating …
Genomic Variability In The Survival Motor Neuron Genes (Smn1 And Smn2): Implications For Spinal Muscular Atrophy Phenotype And Therapeutics Development, Matthew E R Butchbach
Genomic Variability In The Survival Motor Neuron Genes (Smn1 And Smn2): Implications For Spinal Muscular Atrophy Phenotype And Therapeutics Development, Matthew E R Butchbach
Department of Pediatrics Faculty Papers
Spinal muscular atrophy (SMA) is a leading genetic cause of infant death worldwide that is characterized by loss of spinal motor neurons leading to muscle weakness and atrophy. SMA results from the loss of survival motor neuron 1 (SMN1) gene but retention of its paralog SMN2. The copy numbers of SMN1 and SMN2 are variable within the human population with SMN2 copy number inversely correlating with SMA severity. Current therapeutic options for SMA focus on increasing SMN2 expression and alternative splicing so as to increase the amount of SMN protein. Recent work has demonstrated that not all …
What Children Learn About Death From Film, Elissa Miller, Md
What Children Learn About Death From Film, Elissa Miller, Md
Department of Family & Community Medicine Presentations and Grand Rounds
Objectives
- Discuss popular films and how they affect what children know about death
- Discuss child development and development-related concepts of death and dying
- Discuss using films to help discuss death and dying with children
Capacity Building For A New Multicenter Network Within The Echo Idea States Pediatric Clinical Trials Network., Robert D Annett, Scott Bickel, John C Carlson, Kelly Cowan, Sara Cox, Mark J Fisher, J Dean Jarvis, Alberta S Kong, Jessica S Kosut, Kurtis R Kulbeth, Abbot Laptook, Pearl A Mcelfish, Mary M Mcnally, Lee Pachter, Do, Barbara A Pahud, Lee A Pyles, Jennifer Shaw, Kari Simonsen, Jessica Snowden, Christine B Turley, Andrew M Atz
Capacity Building For A New Multicenter Network Within The Echo Idea States Pediatric Clinical Trials Network., Robert D Annett, Scott Bickel, John C Carlson, Kelly Cowan, Sara Cox, Mark J Fisher, J Dean Jarvis, Alberta S Kong, Jessica S Kosut, Kurtis R Kulbeth, Abbot Laptook, Pearl A Mcelfish, Mary M Mcnally, Lee Pachter, Do, Barbara A Pahud, Lee A Pyles, Jennifer Shaw, Kari Simonsen, Jessica Snowden, Christine B Turley, Andrew M Atz
Department of Medicine Faculty Papers
Introduction: Research capacity building is a critical component of professional development for pediatrician scientists, yet this process has been elusive in the literature. The ECHO IDeA States Pediatric Clinical Trials Network (ISPCTN) seeks to implement pediatric trials across medically underserved and rural populations. A key component of achieving this objective is building pediatric research capacity, including enhancement of infrastructure and faculty development. This article presents findings from a site assessment inventory completed during the initial year of the ISPCTN.
Methods: An assessment inventory was developed for surveying ISPCTN sites. The inventory captured site-level activities designed to increase clinical trial research …
Amplified Pain Syndrome In Children, Catherine Soprano, Md, Katherine S. Salamon, Phd
Amplified Pain Syndrome In Children, Catherine Soprano, Md, Katherine S. Salamon, Phd
Department of Family & Community Medicine Presentations and Grand Rounds
Objectives
- Describe chronic pain conditions
- Define chronic pain from biopsychosocial perspective
- Learn ways to describe treatment philosophy of pediatric chronic pain to families
Seeking Similarities Rather Than Differences With Adults To Aid In Therapeutic Advancement For Children., Mara L. Becker, Walter K. Kraft
Seeking Similarities Rather Than Differences With Adults To Aid In Therapeutic Advancement For Children., Mara L. Becker, Walter K. Kraft
Department of Pharmacology and Experimental Therapeutics Faculty Papers
No abstract provided.
Adolescent And Young Adult Oncology Patient And Provider Perspectives In Philadelphia (Aya-4p), Christoher Terry, Md, Miriam Pomerantz, Lsw, Jodi Sandos, Lcsw, Lindsay Wilde, Md
Adolescent And Young Adult Oncology Patient And Provider Perspectives In Philadelphia (Aya-4p), Christoher Terry, Md, Miriam Pomerantz, Lsw, Jodi Sandos, Lcsw, Lindsay Wilde, Md
House Staff Quality Improvement and Patient Safety Conference (2020-)
Primary Objectives
- To assess the current perspective of AYA patients regarding medical services and psychosocial support delivered to AYAs in Philadelphia
- To assess the current knowledge and perspective of healthcare providers at the SKCC about the care and services needed by AYA patients
Plasma Adenosine Deaminase (Ada)-1 And -2 Demonstrate Robust Ontogeny Across The First Four Months Of Human Life., Oludare A. Odumade, Alec L. Plotkin, Jensen Pak, Olubukola T. Idoko, Matthew A. Pettengill, Tobias R. Kollmann, Al Ozonoff, Beate Kampmann, Ofer Levy, Kinga K. Smolen
Plasma Adenosine Deaminase (Ada)-1 And -2 Demonstrate Robust Ontogeny Across The First Four Months Of Human Life., Oludare A. Odumade, Alec L. Plotkin, Jensen Pak, Olubukola T. Idoko, Matthew A. Pettengill, Tobias R. Kollmann, Al Ozonoff, Beate Kampmann, Ofer Levy, Kinga K. Smolen
Department of Pathology, Anatomy, and Cell Biology Faculty Papers
Background: Human adenosine deaminases (ADAs) modulate the immune response: ADA1
Methods: In a subgroup of the EPIC002-study, clinical data and plasma samples were collected from 540 Gambian infants at four time-points: day of birth; first week of life; one month of age; and four months of age. Concentrations of total extracellular ADA, ADA1, and ADA2 were measured by chromogenic assay and evaluated in relation to clinical data. Plasma cytokines/chemokine were measured across the first week of life and correlated to ADA concentrations.
Results: ADA2 demonstrated a steady rise across the first months of life, while ADA1 concentration significantly decreased 0.79-fold …
Automated Assessment Of Thoracic-Abdominal Asynchrony In Patients With Morquio Syndrome., Madhavi V Ratnagiri, Yan Zhu, Tariq Rahman, Mary Theroux, Shunji Tomatsu, Thomas H Shaffer
Automated Assessment Of Thoracic-Abdominal Asynchrony In Patients With Morquio Syndrome., Madhavi V Ratnagiri, Yan Zhu, Tariq Rahman, Mary Theroux, Shunji Tomatsu, Thomas H Shaffer
Department of Pediatrics Faculty Papers
Morquio syndrome is a rare disease caused by a disorder in the storage of mucopolysaccharides that affects multiple organs, including musculoskeletal, respiratory, cardiovascular, and digestive systems. Respiratory failure is one of the leading causes of mortality in Morquio patients; thus, respiratory function testing is vital to the management of the disease. An automated respiratory assessment methodology using the pneuRIP device and a machine-learning algorithm was developed. pneuRIP is a noninvasive approach that uses differences between thoracic and abdominal movements (thoracic-abdominal asynchrony) during respiration to assess respiratory status. The technique was evaluated on 17 patients with Morquio (9 females and 8 …
Long-Term Safety Of Growth Hormone Treatment In Childhood: Two Large Observational Studies: Nordinet Ios And Answer., Lars Sävendahl, Michel Polak, Philippe Backeljauw, Joanne C Blair, Bradley S Miller, Tilman R Rohrer, Anita Hokken-Koelega, Alberto Pietropoli, Nicky Kelepouris, Judith L. Ross
Long-Term Safety Of Growth Hormone Treatment In Childhood: Two Large Observational Studies: Nordinet Ios And Answer., Lars Sävendahl, Michel Polak, Philippe Backeljauw, Joanne C Blair, Bradley S Miller, Tilman R Rohrer, Anita Hokken-Koelega, Alberto Pietropoli, Nicky Kelepouris, Judith L. Ross
Department of Pediatrics Faculty Papers
CONTEXT: Growth hormone (GH) treatment has a generally good safety profile; however, concerns about increased mortality risk in adulthood have been raised.
OBJECTIVE: This work aims to assess the long-term safety of GH treatment in clinical practice.
METHODS: Data were collected from 676 clinics participating in 2 multicenter longitudinal observational studies: the NordiNet International Outcome Study (2006-2016, Europe) and ANSWER Program (2002-2016, USA). Pediatric patients treated with GH were classified into 3 risk groups based on diagnosis. Intervention consisted of daily GH treatment, and main outcome measures included incidence rates (events/1000 patient-years) of adverse drug reactions (ADRs), serious adverse events …
Safety And Improved Efficacy Signals Following Gene Therapy In Childhood Blindness Caused By Gucy2d Mutations, Samuel G. Jacobson, Artur V. Cideciyan, Allen Ho, Igor V. Peshenko, Alexandra V. Garafalo, Alejandro J. Roman, Alexander Sumaroka, Vivian Wu, Arun Krishnan, Rebecca Sheplock, Sanford Boye, Alexander Dizhoor, Shannon Boye
Safety And Improved Efficacy Signals Following Gene Therapy In Childhood Blindness Caused By Gucy2d Mutations, Samuel G. Jacobson, Artur V. Cideciyan, Allen Ho, Igor V. Peshenko, Alexandra V. Garafalo, Alejandro J. Roman, Alexander Sumaroka, Vivian Wu, Arun Krishnan, Rebecca Sheplock, Sanford Boye, Alexander Dizhoor, Shannon Boye
Wills Eye Hospital Papers
A first-in-human clinical trial of gene therapy in Leber congenital amaurosis due to mutations in the GUCY2D gene is underway, and early results are summarized. A recombinant adeno-associated virus serotype 5 (rAAV5) vector carrying the human GUCY2D gene was delivered by subretinal injection to one eye in three adult patients with severe visual loss, nystagmus, but preserved retinal structure. Safety and efficacy parameters were monitored for 9 months post-operatively. No systemic toxicity was detected; there were no serious adverse events, and ocular adverse events resolved. P1 and P2 showed statistically significant rod photoreceptor vision improvement by full-field stimulus testing in …
A Career Of Honor And Humility: Interview With A Pioneer In Ophthalmology, Eli Cehelyk
A Career Of Honor And Humility: Interview With A Pioneer In Ophthalmology, Eli Cehelyk
inSIGHT
Imagine being a first-time parent in your early 30s, and you have just learned that your three-month old infant has retinoblastoma. Thanksgiving is right around the corner and you have no idea what the future holds. How would you feel? What thoughts would be racing through your head? With both hope and anxiety, you make an appointment at Wills Eye to see Dr. Carol Shields, a world-renowned expert on ocular oncology. With confidence and compassion, she walks into the room with her entire team and says, “Don’t worry, we will get everything under control for you. We will take your …
The Limping Child, Bob Wiley, Md
The Limping Child, Bob Wiley, Md
Department of Family & Community Medicine Presentations and Grand Rounds
Learning Objectives
- Review the definition of limp
- Describe the approach to initial evaluation of a limping child, including key items in the history and physical exam
- Appreciate the broad differential that one must keep when evaluating a limping child
- Understand the different types of limp and how they can be used to formulate a differential
- Recognize red flag signs and symptoms which require more urgent evaluation
- Use cases to demonstrate some of the common presentations that are seen by the GP
A Diagnostic Dilemma Of Antiglutamic Acid Decarboxylase 65 (Anti-Gad 65) And Mycoplasma Pneumoniae Antibodies In A Girl Presenting With Acute-Onset Obsessive-Compulsive Disorder., Cecilia Freeman, Antanoid J Langeveldt, Robyn R Miller
A Diagnostic Dilemma Of Antiglutamic Acid Decarboxylase 65 (Anti-Gad 65) And Mycoplasma Pneumoniae Antibodies In A Girl Presenting With Acute-Onset Obsessive-Compulsive Disorder., Cecilia Freeman, Antanoid J Langeveldt, Robyn R Miller
Department of Pediatrics Faculty Papers
Acute-onset obsessive-compulsive disorder can be challenging, especially when triggered by an underlying disease process. Clinicians often turn to Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS), but it is important to consider a broad differential in these patients. We present a case of a 9-year-old girl with acute-onset obsessive-compulsive behavior likely triggered by a post-infectious phenomenon that ultimately resolved following treatment with plasmapheresis.
Phenotype Expression Variability In Children With Gabrb3 Heterozygous Mutations., Abdulhafeez M. Khair, Alana E. Salvucci
Phenotype Expression Variability In Children With Gabrb3 Heterozygous Mutations., Abdulhafeez M. Khair, Alana E. Salvucci
Department of Pediatrics Faculty Papers
GABRB3 gene is a recently identified gene located in 15q12 chromosome and encodes for gamma-aminobutyric acid (GABA) receptor subunit beta-3 protein, which is linked to the GABAA receptor. The gene is believed to share a role in inhibitory GABAergic synapses, GABA iron-gated channel function, and possible cellular response to histamine. The β3 subunit is expressed in cerebral grey matter, thalami, hippocampi, and cerebellum, among other structures. Faulty GABRB3 function is linked to several neurological disorders and clinical syndromes. However, the spectrum of such disorders is not yet well known. We present three case reports highlighting the potentially expanding clinical phenotype …
Correspondence Between Perceived Pubertal Development And Hormone Levels In 9-10 Year-Olds From The Adolescent Brain Cognitive Development Study., Megan M Herting, Kristina A Uban, Marybel Robledo Gonzalez, Fiona C Baker, Eric C Kan, Wesley K Thompson, Douglas A Granger, Matthew D Albaugh, Andrey P Anokhin, Kara S Bagot, Marie T Banich, Deanna M Barch, Arielle Baskin-Sommers, Florence J Breslin, B J Casey, Bader Chaarani, Linda Chang, Duncan B Clark, Christine C Cloak, R Todd Constable, Linda B Cottler, Rada K Dagher, Mirella Dapretto, Anthony S Dick, Nico Dosenbach, Gayathri J Dowling, Julie A Dumas, Sarah Edwards, Thomas Ernst, Damien A Fair, Sarah W Feldstein-Ewing, Edward G Freedman, Bernard F Fuemmeler, Hugh Garavan, Dylan G Gee, Jay N Giedd, Paul E A Glaser, Aimee Goldstone, Kevin M Gray, Samuel W Hawes, Andrew C Heath, Mary M Heitzeg, John K Hewitt, Charles J Heyser, Elizabeth A Hoffman, Rebekah S Huber, Marilyn A. Huestis, Luke W Hyde, M Alejandra Infante, Masha Y Ivanova, Joanna Jacobus, Terry L Jernigan, Nicole R Karcher, Angela R Laird, Kimberly H Leblanc, Krista Lisdahl, Monica Luciana, Beatriz Luna, Hermine H Maes, Andrew T Marshall, Michael J Mason, Erin C Mcglade, Amanda S Morris, Bonnie J Nagel, Gretchen N Neigh, Clare E Palmer, Martin P Paulus, Alexandra S Potter, Leon I Puttler, Nishadi Rajapakse, Kristina Rapuano, Gloria Reeves, Perry F Renshaw, Claudiu Schirda, Kenneth J Sher, Chandni Sheth, Paul D Shilling, Lindsay M Squeglia, Matthew T Sutherland, Susan F Tapert, Rachel L Tomko, Deborah Yurgelun-Todd, Natasha E Wade, Susan R B Weiss, Robert A Zucker, Elizabeth R Sowell
Correspondence Between Perceived Pubertal Development And Hormone Levels In 9-10 Year-Olds From The Adolescent Brain Cognitive Development Study., Megan M Herting, Kristina A Uban, Marybel Robledo Gonzalez, Fiona C Baker, Eric C Kan, Wesley K Thompson, Douglas A Granger, Matthew D Albaugh, Andrey P Anokhin, Kara S Bagot, Marie T Banich, Deanna M Barch, Arielle Baskin-Sommers, Florence J Breslin, B J Casey, Bader Chaarani, Linda Chang, Duncan B Clark, Christine C Cloak, R Todd Constable, Linda B Cottler, Rada K Dagher, Mirella Dapretto, Anthony S Dick, Nico Dosenbach, Gayathri J Dowling, Julie A Dumas, Sarah Edwards, Thomas Ernst, Damien A Fair, Sarah W Feldstein-Ewing, Edward G Freedman, Bernard F Fuemmeler, Hugh Garavan, Dylan G Gee, Jay N Giedd, Paul E A Glaser, Aimee Goldstone, Kevin M Gray, Samuel W Hawes, Andrew C Heath, Mary M Heitzeg, John K Hewitt, Charles J Heyser, Elizabeth A Hoffman, Rebekah S Huber, Marilyn A. Huestis, Luke W Hyde, M Alejandra Infante, Masha Y Ivanova, Joanna Jacobus, Terry L Jernigan, Nicole R Karcher, Angela R Laird, Kimberly H Leblanc, Krista Lisdahl, Monica Luciana, Beatriz Luna, Hermine H Maes, Andrew T Marshall, Michael J Mason, Erin C Mcglade, Amanda S Morris, Bonnie J Nagel, Gretchen N Neigh, Clare E Palmer, Martin P Paulus, Alexandra S Potter, Leon I Puttler, Nishadi Rajapakse, Kristina Rapuano, Gloria Reeves, Perry F Renshaw, Claudiu Schirda, Kenneth J Sher, Chandni Sheth, Paul D Shilling, Lindsay M Squeglia, Matthew T Sutherland, Susan F Tapert, Rachel L Tomko, Deborah Yurgelun-Todd, Natasha E Wade, Susan R B Weiss, Robert A Zucker, Elizabeth R Sowell
Institute of Emerging Health Professions Faculty Papers
Aim: To examine individual variability between perceived physical features and hormones of pubertal maturation in 9-10-year-old children as a function of sociodemographic characteristics.
Methods: Cross-sectional metrics of puberty were utilized from the baseline assessment of the Adolescent Brain Cognitive Development (ABCD) Study—a multi-site sample of 9–10 year-olds (n = 11,875)—and included perceived physical features via the pubertal development scale (PDS) and child salivary hormone levels (dehydroepiandrosterone and testosterone in all, and estradiol in females). Multi-level models examined the relationships among sociodemographic measures, physical features, and hormone levels. A group factor analysis (GFA) was implemented to extract latent variables of pubertal …
Identifying The Roadblocks To Successful Pediatric Imaging Without Sedation Or Anesthesia, Iman Soliman, Ammie M. White, Md, J. Christopher Edgar, Phd, Laura Cadge, Ccls, Margaret Hamel-Daymon, Crnp, Sandra Saade-Lemus, Md, Rochelle Bagatell, Md, Elizabeth T. Drum, Md, Lisa States, Md
Identifying The Roadblocks To Successful Pediatric Imaging Without Sedation Or Anesthesia, Iman Soliman, Ammie M. White, Md, J. Christopher Edgar, Phd, Laura Cadge, Ccls, Margaret Hamel-Daymon, Crnp, Sandra Saade-Lemus, Md, Rochelle Bagatell, Md, Elizabeth T. Drum, Md, Lisa States, Md
Phase 1
Background: Modern imaging modalities allow for assessment of a wide array of medical conditions. Because it is difficult for young children to remain still during lengthy imaging studies, there is a need for sedation and general anesthesia (GA). A growing literature, however, suggests that there may be deleterious neurocognitive effects of sedation/GA in young children. As such, it is in our patients’ best interest to critically evaluate the use of sedation/GA.
Objective: To identify the roadblocks to obtaining clinical pediatric magnetic resonance imaging (MRI) exams without sedation/GA.
Materials and Methods: Participants included 63 parents/guardians of 5- to 9-year-old patients who …
Callicore, Cole Miller, Galen Ogg, Jasmine Phun, Yajnesh Vedanaparti, Morgan Hutchinson, Md
Callicore, Cole Miller, Galen Ogg, Jasmine Phun, Yajnesh Vedanaparti, Morgan Hutchinson, Md
Phase 1
Transporting children in SPICA casts in a safe, comfortable, and convenient manner so that the parents can feel more at ease.