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Full-Text Articles in Medicine and Health Sciences

Coronary Microvascular Dysfunction In Childhood: An Emerging Pathological Entity And Its Clinical Implications, Takeshi Tsuda, Gina Patel Apr 2024

Coronary Microvascular Dysfunction In Childhood: An Emerging Pathological Entity And Its Clinical Implications, Takeshi Tsuda, Gina Patel

Department of Pediatrics Faculty Papers

Coronary microvascular dysfunction (CMD) encompasses a spectrum of structural and functional alterations in coronary microvasculature resulting in impaired coronary blood flow and consequent myocardial ischemia without obstruction in epicardial coronary artery. The pathogenesis of CMD is complex involving both functional and structural alteration in the coronary microcirculation. In adults, CMD is predominantly discussed in context with anginal chest pain or existing ischemic heart disease and its risk factors. The presence of CMD suggests increased risk of adverse cardiovascular events independent of coronary atherosclerosis. Coronary microvascular dysfunction is also known in children but is rarely recognized due to paucity of concommitent …


Delayed Onset Of Neonatal Compartment Syndrome Associated With Compound Fetal Presentation, Nicholas Manini, Hayato Unno Apr 2024

Delayed Onset Of Neonatal Compartment Syndrome Associated With Compound Fetal Presentation, Nicholas Manini, Hayato Unno

Department of Medicine Faculty Papers

Neonatal compartment syndrome, although rare, has a classic presentation with sentinel skin findings and development of swelling, erythema, and tenderness of the affected extremity. Neonatal compartment syndrome requires prompt surgical intervention to preserve the affected limb and ensure its normal growth and development. Our patient was born at term via vaginal delivery complicated by a compound presentation involving the left upper extremity. No physical exam abnormalities were noted at birth, but she developed signs of neonatal compartment syndrome by 15 h of life. She was surgically treated at 22 h of life and recovered well. At one year of age, …


Nf-Κb As An Inducible Regulator Of Inflammation In The Central Nervous System, Sudha Anilkumar, Elizabeth Wright-Jin Mar 2024

Nf-Κb As An Inducible Regulator Of Inflammation In The Central Nervous System, Sudha Anilkumar, Elizabeth Wright-Jin

Department of Medicine Faculty Papers

The NF-κB (nuclear factor K-light-chain-enhancer of activated B cells) transcription factor family is critical for modulating the immune proinflammatory response throughout the body. During the resting state, inactive NF-κB is sequestered by IκB in the cytoplasm. The proteasomal degradation of IκB activates NF-κB, mediating its translocation into the nucleus to act as a nuclear transcription factor in the upregulation of proinflammatory genes. Stimuli that initiate NF-κB activation are diverse but are canonically attributed to proinflammatory cytokines and chemokines. Downstream effects of NF-κB are cell type-specific and, in the majority of cases, result in the activation of pro-inflammatory cascades. Acting as …


Current Strategies For Increasing Knock-In Efficiency In Crispr/Cas9-Based Approaches, Andrés Felipe Leal, Angelica María Herreno-Pachón, Eliana Benincore-Flórez, Amali Karunathilaka, Shunji Tomatsu Feb 2024

Current Strategies For Increasing Knock-In Efficiency In Crispr/Cas9-Based Approaches, Andrés Felipe Leal, Angelica María Herreno-Pachón, Eliana Benincore-Flórez, Amali Karunathilaka, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Since its discovery in 2012, the clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated protein 9 (Cas9) system has supposed a promising panorama for developing novel and highly precise genome editing-based gene therapy (GT) alternatives, leading to overcoming the challenges associated with classical GT. Classical GT aims to deliver transgenes to the cells via their random integration in the genome or episomal persistence into the nucleus through lentivirus (LV) or adeno-associated virus (AAV), respectively. Although high transgene expression efficiency is achieved by using either LV or AAV, their nature can result in severe side effects in humans. For instance, …


Virtual Facilitation Best Practices And Research Priorities: A Scoping Review, Asya Agulnik, Derrecka Boykin, Denalee M O'Malley, Julia Price, Mia Yang, Mark Mckone, Geoffrey Curran, Mona J Ritchie Feb 2024

Virtual Facilitation Best Practices And Research Priorities: A Scoping Review, Asya Agulnik, Derrecka Boykin, Denalee M O'Malley, Julia Price, Mia Yang, Mark Mckone, Geoffrey Curran, Mona J Ritchie

Department of Pediatrics Faculty Papers

BACKGROUND: Facilitation is an implementation strategy that supports the uptake of evidence-based practices. Recently, use of virtual facilitation (VF), or the application of facilitation using primarily video-based conferencing technologies, has become more common, especially since the COVID-19 pandemic. Thorough assessment of the literature on VF, however, is lacking. This scoping review aimed to identify and describe conceptual definitions of VF, evaluate the consistency of terminology, and recommend "best" practices for its use as an implementation strategy.

METHODS: We conducted a scoping review to identify literature on VF following the PRISMA-ScR guidance. A search of PubMed, Embase, Web of Science, and …


Exploring Referral And Service Utilization Patterns Within An Outpatient Interdisciplinary Pediatric Chronic Pain Program, Aimee Hildenbrand, Christina M Amaro, Benjamin Bear, Catherine Soprano, Katherine S Salamon Feb 2024

Exploring Referral And Service Utilization Patterns Within An Outpatient Interdisciplinary Pediatric Chronic Pain Program, Aimee Hildenbrand, Christina M Amaro, Benjamin Bear, Catherine Soprano, Katherine S Salamon

Department of Medicine Faculty Papers

PURPOSE: We examine referral sources and clinical characteristics for youth presenting to an outpatient interdisciplinary pediatric chronic pain program.

PATIENTS AND METHODS: Referral data were extracted from the electronic health record. PROMIS Pediatric Anxiety and Pain Interference Scales were administered at an initial evaluation visit.

RESULTS: The program received 1488 referrals between 2016 and 2019, representing 1338 patients, with increasing volume of referrals over time. Referrals were primarily from orthopedics (19.6%), physical medicine and rehabilitation (18.8%), neurology (14.4%), and rheumatology (12.6%). Patients referred were primarily female (75.4%), White (80.1%), English-speaking (98.4%) adolescents (median=15.0 years). Of those referred, 732 (54.7%) attended …


Molecular Mechanisms In Pathophysiology Of Mucopolysaccharidosis And Prospects For Innovative Therapy, Yasuhiko Ago, Estera Rintz, Krishna Sai Musini, Zhengyu Ma, Shunji Tomatsu Jan 2024

Molecular Mechanisms In Pathophysiology Of Mucopolysaccharidosis And Prospects For Innovative Therapy, Yasuhiko Ago, Estera Rintz, Krishna Sai Musini, Zhengyu Ma, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidoses (MPSs) are a group of inborn errors of the metabolism caused by a deficiency in the lysosomal enzymes required to break down molecules called glycosaminoglycans (GAGs). These GAGs accumulate over time in various tissues and disrupt multiple biological systems, including catabolism of other substances, autophagy, and mitochondrial function. These pathological changes ultimately increase oxidative stress and activate innate immunity and inflammation. We have described the pathophysiology of MPS and activated inflammation in this paper, starting with accumulating the primary storage materials, GAGs. At the initial stage of GAG accumulation, affected tissues/cells are reversibly affected but progress irreversibly to: (1) …


Sepsis Epidemiology In Australian And New Zealand Children (Sentinel): Protocol For A Multicountry Prospective Observational Study, Elliot Long, Meredith L. Borland, Shane George, Shefali Jani, Eunicia Tan, Jocelyn Neutze, Natalie Phillips, Amit Kochar, Simon Craig, Anna Lithgow, Arjun Rao, Stuart Dalziel, Ed Oakley, Stephen Hearps, Sonia Singh, Ben Gelbart, Sarah Mcnab, Fran Balamuth, Scott Weiss, Nathan Kuppermann, Amanda Williams, Franz E. Babl Jan 2024

Sepsis Epidemiology In Australian And New Zealand Children (Sentinel): Protocol For A Multicountry Prospective Observational Study, Elliot Long, Meredith L. Borland, Shane George, Shefali Jani, Eunicia Tan, Jocelyn Neutze, Natalie Phillips, Amit Kochar, Simon Craig, Anna Lithgow, Arjun Rao, Stuart Dalziel, Ed Oakley, Stephen Hearps, Sonia Singh, Ben Gelbart, Sarah Mcnab, Fran Balamuth, Scott Weiss, Nathan Kuppermann, Amanda Williams, Franz E. Babl

Department of Pediatrics Faculty Papers

INTRODUCTION: Sepsis affects 25.2 million children per year globally and causes 3.4 million deaths, with an annual cost of hospitalisation in the USA of US$7.3 billion. Despite being common, severe and expensive, therapies and outcomes from sepsis have not substantially changed in decades. Variable case definitions, lack of a reference standard for diagnosis and broad spectrum of disease hamper efforts to evaluate therapies that may improve sepsis outcomes. This landscape analysis of community-acquired childhood sepsis in Australia and New Zealand will characterise the burden of disease, including incidence, severity, outcomes and cost. Sepsis diagnostic criteria and risk stratification tools will …


Iron Oxide-Coupled Crispr-Ncas9-Based Genome Editing Assessment In Mucopolysaccharidosis Iva Mice, Andrés Leal, Betul Celik, Nidhi Fnu, Shaukat Khan, Shunji Tomatsu Dec 2023

Iron Oxide-Coupled Crispr-Ncas9-Based Genome Editing Assessment In Mucopolysaccharidosis Iva Mice, Andrés Leal, Betul Celik, Nidhi Fnu, Shaukat Khan, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidosis (MPS) IVA is a lysosomal storage disorder caused by mutations in the GALNS gene that leads to the lysosomal accumulation of keratan sulfate (KS) and chondroitin 6-sulfate, causing skeletal dysplasia and cardiopulmonary complications. Current enzyme replacement therapy does not impact the bone manifestation of the disease, supporting that new therapeutic alternatives are required. We previously demonstrated the suitability of the CRISPR-nCas9 system to rescue the phenotype of human MPS IVA fibroblasts using iron oxide nanoparticles (IONPs) as non-viral vectors. Here, we have extended this strategy to an MPS IVA mouse model by inserting the human GALNS cDNA into the …


Feasibility Of Electronic Medication Monitoring Among Adolescents And Emerging Adults With Sickle Cell Disease, Aimee Hildenbrand, Katherine Kidwell, Meghan Mcgrady, Constance Mara, Charles Quinn, Lori Crosby Dec 2023

Feasibility Of Electronic Medication Monitoring Among Adolescents And Emerging Adults With Sickle Cell Disease, Aimee Hildenbrand, Katherine Kidwell, Meghan Mcgrady, Constance Mara, Charles Quinn, Lori Crosby

Department of Pediatrics Faculty Papers

Purpose: To examine the feasibility of using MEMS® bottles to assess adherence among adolescents and emerging adults with sickle cell disease.

Patients and Methods: Eighteen non-Hispanic Black participants with HbSS (M = 17.8 years; 61% male) were given a MEMS® bottle to store hydroxyurea (n = 14) or deferasirox (n = 4).

Results: One hundred percent initiated MEMS® use and 61% sustained use through the 18-week study; at follow-up, only 11% returned their bottle on time. Barriers to MEMS® use included medication changes and transition to adult care; facilitators included tip sheets and reminders.

Conclusion: While MEMS® is acceptable to …


Human Parechovirus Central Nervous System Infection In A Young Infant Cohort, Aspasia Katragkou, Avni Sheth, Christina Gagliardo, Jessica Aquino, Niva Shah, Eberechi Nwaobasi-Iwuh, Christina Melchionne, Paige Black, Stephanie Chiu, Cecilia Di Pentima Dec 2023

Human Parechovirus Central Nervous System Infection In A Young Infant Cohort, Aspasia Katragkou, Avni Sheth, Christina Gagliardo, Jessica Aquino, Niva Shah, Eberechi Nwaobasi-Iwuh, Christina Melchionne, Paige Black, Stephanie Chiu, Cecilia Di Pentima

Department of Pediatrics Faculty Papers

In 2022, a surge in cases of pediatric human parechovirus (HPeV) central nervous system infections in young infants was seen at our institution. Despite the dramatic increase in the number of cases seen that year, the clinical features of the illness were similar to prior years. The recent pediatric HPeV surge highlights the need to evaluate treatment options and standardize follow-up to better understand the long-term prognosis of infants with HPeV infection.


Racial Disparities Among Predicted Bronchopulmonary Dysplasia Risk Outcomes In Premature Infants Born <30 Weeks Gestation, Priyanka Patel, Andrew Ellefson, David A. Paul Nov 2023

Racial Disparities Among Predicted Bronchopulmonary Dysplasia Risk Outcomes In Premature Infants Born <30 Weeks Gestation, Priyanka Patel, Andrew Ellefson, David A. Paul

Department of Pediatrics Faculty Papers

Background and Objective: There is extensive literature to support eliminating race-based risk stratification. The National Institute of Child Health and Human Development (NICHD) calculator, used to predict risk of bronchopulmonary dysplasia (BPD), includes race as a variable. We sought to investigate how utilizing race in determination of risk for BPD may lead to inequitable care.

Methods: The study included a retrospective cohort of infants born <30 weeks gestation between January 2016 and February 2022. The primary outcome was the difference in predictive risk of BPD for non-Hispanic Black compared to non-Hispanic White infants. The secondary outcome was the disparity in theoretical administration of post-natal corticosteroids when the calculator was applied to the cohort. Analysis included paired T-tests and Chi-Square.

Results: Of the 273 infants studied, 154 were non-Hispanic Black (56%). There was no difference between the groups in gestation or respiratory support on day of life (DOL) 14 or 28. …


Surveillance Cardiopulmonary Exercise Testing Can Risk-Stratify Childhood Cancer Survivors: Underlying Pathophysiology Of Poor Exercise Performance And Possible Room For Improvement, Takeshi Tsuda, Kimberly Davidow, Gina D'Aloisio, Joanne Quillen Nov 2023

Surveillance Cardiopulmonary Exercise Testing Can Risk-Stratify Childhood Cancer Survivors: Underlying Pathophysiology Of Poor Exercise Performance And Possible Room For Improvement, Takeshi Tsuda, Kimberly Davidow, Gina D'Aloisio, Joanne Quillen

Department of Pediatrics Faculty Papers

BACKGROUND: Asymptomatic childhood cancer survivors (CCS) frequently show decreased exercise performance. Poor exercise performance may indicate impaired future cardiovascular health.

METHODS: Cardiopulmonary exercise testing (CPET) was performed in asymptomatic off-treatment CCS (age ≥ 10 years). Patients were divided into Normal and Poor performance groups by %predicted maximum VO2 at 80%. Both peak and submaximal CPET values were analyzed.

RESULTS: Thirty-eight males (19 Normal, 19 Poor) and 40 females (18 Normal, 22 Poor) were studied. Total anthracycline dosage was comparable among 4 groups. The body mass index (BMI), although normal, and weight were significantly higher in Poor groups. Peak heart rate …


Post-Acute Sequelae Of Covid-19 (Pasc) In Pediatrics: Factors That Impact Symptom Severity And Referral To Treatment., Catherine Soprano, Ryan Ngo, Casey A Konys, Ashley Bazier, Katherine Salamon Nov 2023

Post-Acute Sequelae Of Covid-19 (Pasc) In Pediatrics: Factors That Impact Symptom Severity And Referral To Treatment., Catherine Soprano, Ryan Ngo, Casey A Konys, Ashley Bazier, Katherine Salamon

Department of Medicine Faculty Papers

The post-acute sequelae of COVID-19 (PASC) is a complex condition. While there are emerging studies on its effects in adults, there is scarce research regarding the long-term effects of COVID-19 infection among youth. Several researchers have likened long-haul COVID-19 to chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME) and postural orthostatic tachycardia syndrome (POTS). In adults, the prognosis for these diagnoses is less promising than that in youth; however, there is currently very little information available on the presentation of youth with PASC. A better understanding of the specific symptom presentation for youth diagnosed with PASC is necessary. Retrospective chart reviews were conducted …


Mucopolysaccharidosis Iva: Current Disease Models And Drawbacks, Andrés Felipe Leal, Carlos Javier Alméciga-Díaz, Shunji Tomatsu Nov 2023

Mucopolysaccharidosis Iva: Current Disease Models And Drawbacks, Andrés Felipe Leal, Carlos Javier Alméciga-Díaz, Shunji Tomatsu

Department of Pediatrics Faculty Papers


Mucopolysaccharidosis IVA (MPS IVA) is a rare disorder caused by mutations in the N-acetylgalactosamine-6-sulfate-sulfatase (GALNS) encoding gene. GALNS leads to the lysosomal degradation of the glycosaminoglyccreasans keratan sulfate and chondroitin 6-sulfate. Impaired GALNS enzymes result in skeletal and non-skeletal complications in patients. For years, the MPS IVA pathogenesis and the assessment of promising drugs have been evaluated using in vitro (primarily fibroblasts) and in vivo (mainly mouse) models. Even though value information has been raised from those studies, these models have several limitations. For instance, chondrocytes have been well recognized as primary cells affected in MPS IVA and responsible for …


Clinical Factors Associated With Need For Neurosurgical Care In Young Children With Imaging For Macrocephaly: A Case Control Study, Jessica F. Rohde, Jeffrey Campbell, Julie Barbera, Elena Taylor, Ashok Ramachandra, Christopher Gegg, Andrea Scherer, Joseph Piatt Nov 2023

Clinical Factors Associated With Need For Neurosurgical Care In Young Children With Imaging For Macrocephaly: A Case Control Study, Jessica F. Rohde, Jeffrey Campbell, Julie Barbera, Elena Taylor, Ashok Ramachandra, Christopher Gegg, Andrea Scherer, Joseph Piatt

Department of Pediatrics Faculty Papers

BACKGROUND: Macrocephaly is present in 2.3% of children with important neurosurgical conditions in the differential diagnosis. The objective of this study was to identify clinical associations with actionable imaging findings among children with head imaging for macrocephaly.

METHODS: We conducted a case-control study of head imaging studies ordered for macrocephaly among children 24 months and younger in a multistate children's health system. Four neurosurgeons reviewed the images, determining cases to be a 'concern' if neurosurgical follow-up or intervention was indicated. Electronic health records were reviewed to collect patient-level data and to determine if surgery was performed. Controls were matched 3:1 …


Noninvasive Point Of Care Device For Assessing Cardiac Response To Acute Volume Changes, Harry A. Silber, Nisha A. Gilotra, Thomas L. Miller Oct 2023

Noninvasive Point Of Care Device For Assessing Cardiac Response To Acute Volume Changes, Harry A. Silber, Nisha A. Gilotra, Thomas L. Miller

Department of Pediatrics Faculty Papers

Purpose: The change in the amplitude of a peripheral pulse in response to a Valsalva maneuver has diagnostic utility for assessing volume status at the bedside. We have developed a device to automatically quantify the Valsalva pulse response (VPR) to a standardized Valsalva maneuver that the device guides a user to perform. In this study, we sought to determine whether VPR by the device, Indicor, is sensitive enough to detect the acute increase in central pressure and volume load that occurs with a passive leg raise (PLR) in healthy volunteers.

Methods: Healthy volunteers were tested semirecumbently at 45 degrees, then …


Normal Values Of Nerve Conduction Studies In Children Aged 7 Days To 14 Years Referred To Electrodiagnosis Clinic Of Iranian Children’S Medical Center, Masood Ghahvechi Akbari, Fazel Mahmoodpoor, Mahmoodreza Ashrafi, Elahe Rezaee, Sahar Ghorbanpour, Seyede Zahra Emami Razavi, Mohaddeseh Azadvari, Reza Shervin Badv, Gholamreza Zamani, Ali Reza Tavasoli, Morteza Heidari, Zahra Rezaei, Setareh Rohani, Mahmoud Mohammadi Oct 2023

Normal Values Of Nerve Conduction Studies In Children Aged 7 Days To 14 Years Referred To Electrodiagnosis Clinic Of Iranian Children’S Medical Center, Masood Ghahvechi Akbari, Fazel Mahmoodpoor, Mahmoodreza Ashrafi, Elahe Rezaee, Sahar Ghorbanpour, Seyede Zahra Emami Razavi, Mohaddeseh Azadvari, Reza Shervin Badv, Gholamreza Zamani, Ali Reza Tavasoli, Morteza Heidari, Zahra Rezaei, Setareh Rohani, Mahmoud Mohammadi

Jefferson Institute of Molecular Medicine Papers and Presentations

Background: The normal values of nerve conduction studies (NCS) are different in children compared to adults. Moreover, racial and geographical factors can affect these values.

Objectives: The present study aimed to investigate the normal NCS values in children of different ages.

Methods: The present cross-sectional study included children referred to the Electrodiagnosis Clinic of the Children’s Medical Center in Iran, who had normal NCS results based on the references and had no exclusion criteria. The patients were divided into 8 age groups (7 days to one month, 1 - 3 months, 3 - 6 months, 6 - 12 months, 1 …


Comparison Of Bacterial Culture With Biofire® Filmarray® Multiplex Pcr Screening Of Archived Cerebrospinal Fluid Specimens From Children With Suspected Bacterial Meningitis In Nigeria, S Obaro, F Hassan-Hanga, N Medugu, Rasaq Olaosebikan, G Olanipekun, B Jibir, S Gambo, Theresa Ajose, Carissa Duru, B Ebruke, H D Davies Oct 2023

Comparison Of Bacterial Culture With Biofire® Filmarray® Multiplex Pcr Screening Of Archived Cerebrospinal Fluid Specimens From Children With Suspected Bacterial Meningitis In Nigeria, S Obaro, F Hassan-Hanga, N Medugu, Rasaq Olaosebikan, G Olanipekun, B Jibir, S Gambo, Theresa Ajose, Carissa Duru, B Ebruke, H D Davies

Department of Pharmacology and Experimental Therapeutics Faculty Papers

BACKGROUND: Diagnosis of bacterial meningitis remains a challenge in most developing countries due to low yield from bacterial culture, widespread use of non-prescription antibiotics, and weak microbiology laboratories. The objective of this study was to compare the yield from standard bacterial culture with the multiplex nested PCR platform, the BioFire® FilmArray® Meningitis/Encephalitis Panel (BioFire ME Panel), for cases with suspected acute bacterial meningitis.

METHODS: Following Gram stain and bacterial culture on cerebrospinal fluid (CSF) collected from children aged less than 5 years with a clinical suspicion of acute bacterial meningitis (ABM) as defined by the WHO guidelines, residual CSF specimens …


Sickle Cell Disease Treatment With Arginine Therapy (Start): Study Protocol For A Phase 3 Randomized Controlled Trial., Chris A Rees, David C. Brousseau, Daniel M Cohen, Anthony Villella, Carlton Dampier, Kathleen Brown, Andrew Campbell, Corrie E Chumpitazi, Gladstone Airewele, Todd Chang, Christopher Denton, Angela Ellison, Alexis Thompson, Fahd Ahmad, Nitya Bakshi, Keli D Coleman, Sara Leibovich, Deborah Leake, Dunia Hatabah, Hagar Wilkinson, Michelle Robinson, T Charles Casper, Elliott Vichinsky, Claudia R Morris Aug 2023

Sickle Cell Disease Treatment With Arginine Therapy (Start): Study Protocol For A Phase 3 Randomized Controlled Trial., Chris A Rees, David C. Brousseau, Daniel M Cohen, Anthony Villella, Carlton Dampier, Kathleen Brown, Andrew Campbell, Corrie E Chumpitazi, Gladstone Airewele, Todd Chang, Christopher Denton, Angela Ellison, Alexis Thompson, Fahd Ahmad, Nitya Bakshi, Keli D Coleman, Sara Leibovich, Deborah Leake, Dunia Hatabah, Hagar Wilkinson, Michelle Robinson, T Charles Casper, Elliott Vichinsky, Claudia R Morris

Department of Pediatrics Faculty Papers

BACKGROUND: Despite substantial illness burden and healthcare utilization conferred by pain from vaso-occlusive episodes (VOE) in children with sickle cell disease (SCD), disease-modifying therapies to effectively treat SCD-VOE are lacking. The aim of the Sickle Cell Disease Treatment with Arginine Therapy (STArT) Trial is to provide definitive evidence regarding the efficacy of intravenous arginine as a treatment for acute SCD-VOE among children, adolescents, and young adults.

METHODS: STArT is a double-blind, placebo-controlled, randomized, phase 3, multicenter trial of intravenous arginine therapy in 360 children, adolescents, and young adults who present with SCD-VOE. The STArT Trial is being conducted at 10 …


Mobile Health As A Potential Tool To Treat Children In Obesity, Olivia Carey Aug 2023

Mobile Health As A Potential Tool To Treat Children In Obesity, Olivia Carey

Master of Science in Physician Assistant Studies Capstone Presentations (Center City)

Introduction

  • Pediatric obesity prevalence is estimated to be 22% in 2020 of all children in the US3
  • Obesity is defined as greater or equal to the 95th percentile for age and sex4
  • Childhood obesity leads to adulthood hypertension, hyperlipidemia, respiratory complications, and reduced quality of life44
  • The AAP released new 2023 guidelines that define the standard of care as a multi-modal, chronic-care treatment plan that prescribes healthy eating choices, an increase in physical activity, and mental health support.5
  • Mobile health is the use of mobile phones and wireless technology in healthcare3
  • A meta analysis of …


Role Of Suction-Based Airway Clearance Devices In Pediatric Foreign Body Airway Obstruction, Rebecca Braver Aug 2023

Role Of Suction-Based Airway Clearance Devices In Pediatric Foreign Body Airway Obstruction, Rebecca Braver

Master of Science in Physician Assistant Studies Capstone Presentations (Center City)

Introduction

  • Foreign body airway obstruction (FBAO) represents a significant cause of morbidity and mortality in the pediatric population. Factors including pediatric anatomy, chewing and swallowing difficulties, developmental stages, habits and behaviors, and children’s toys and foods increase risk of FBAO in children.1,2
  • BLS guidelines for FBAO management in responsive children ages 1-8 call for a series of subdiaphragmatic abdominal thrusts, which increase intrathoracic pressure to force air and the foreign body out of the airway.3,4 Abdominal thrusts have higher reports of injury compared to all other FBAO interventions, with risk of thoracic, vascular, and gastro-esophageal injury.5-7
  • Novel …


Evaluation Of The Orally Bioavailable 4-Phenylbutyrate-Tethered Trichostatin A Analogue Ar42 In Models Of Spinal Muscular Atrophy, Casey J. Lumpkin, Ashlee W. Harris, Andrew J. Connell, Ryan W. Kirk, Joshua A. Whiting, Luciano Saieva, Livio Pellizzoni, Arthur H.M. Burghes, Matthew E.R. Butchbach Jun 2023

Evaluation Of The Orally Bioavailable 4-Phenylbutyrate-Tethered Trichostatin A Analogue Ar42 In Models Of Spinal Muscular Atrophy, Casey J. Lumpkin, Ashlee W. Harris, Andrew J. Connell, Ryan W. Kirk, Joshua A. Whiting, Luciano Saieva, Livio Pellizzoni, Arthur H.M. Burghes, Matthew E.R. Butchbach

Department of Pediatrics Faculty Papers

Proximal spinal muscular atrophy (SMA) is a leading genetic cause for infant death in the world and results from the selective loss of motor neurons in the spinal cord. SMA is a consequence of low levels of SMN protein and small molecules that can increase SMN expression are of considerable interest as potential therapeutics. Previous studies have shown that both 4-phenylbutyrate (4PBA) and trichostatin A (TSA) increase SMN expression in dermal fibroblasts derived from SMA patients. AR42 is a 4PBA-tethered TSA derivative that is a very potent histone deacetylase inhibitor. SMA patient fibroblasts were treated with either AR42, AR19 (a …


Bone Growth Induction In Mucopolysaccharidosis Iva Mouse, Estera Rintz, Angélica María Herreño-Pachón, Betul Celik, Fnu Nidhi, Shaukat Khan, Eliana Benincore-Flórez, Shunji Tomatsu Jun 2023

Bone Growth Induction In Mucopolysaccharidosis Iva Mouse, Estera Rintz, Angélica María Herreño-Pachón, Betul Celik, Fnu Nidhi, Shaukat Khan, Eliana Benincore-Flórez, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidosis IVA (MPS IVA; Morquio A syndrome) is caused by a deficiency of the N-acetylgalactosamine-6-sulfate-sulfatase (GALNS) enzyme, leading to the accumulation of glycosaminoglycans (GAG), keratan sulfate (KS) and chondroitin-6-sulfate (C6S), mainly in cartilage and bone. This lysosomal storage disorder (LSD) is characterized by severe systemic skeletal dysplasia. To this date, none of the treatment options for the MPS IVA patients correct bone pathology. Enzyme replacement therapy with elosulfase alpha provides a limited impact on bone growth and skeletal lesions in MPS IVA patients. To improve bone pathology, we propose a novel gene therapy with a small peptide as a growth-promoting …


Usability And Acceptability Testing Of A Plan Of Safe Care In A Mobile Health Platform, Krystyna R. Isaacs, Elina Bajracharya, Shantae Taylor, Katie Chung, Yukiko Washio, Trenee Parker, David A. Paul, Tony X. Ma May 2023

Usability And Acceptability Testing Of A Plan Of Safe Care In A Mobile Health Platform, Krystyna R. Isaacs, Elina Bajracharya, Shantae Taylor, Katie Chung, Yukiko Washio, Trenee Parker, David A. Paul, Tony X. Ma

Department of Pediatrics Faculty Papers

Purpose: Women who are pregnant or parenting while recovering from substance use disorder (SUD) are at risk for insufficient recovery support. With the federal mandate, implementation has been left to each state for the Plan of Safe Care (POSC), leading to challenges in providing comprehensive care coordination and meeting federal reporting requirements.

Methods: This research tests the usability and acceptability of a POSC platform, called SAFE4BOTH, which combines a mobile health (mHealth) app for use by mothers with substance use disorder (MSUD) with a web-based case management system for use by stakeholders to reduce the issue of fragmented postnatal maternal …


The Child Healthcare At Mater Pediatric Study (Champs): A 2-Arm Cluster Randomized Control Trial Of Group Well Child Care For Mothers In Treatment For Opioid Use Disorder And Their Children, Vanessa L. Short, Diane J. Abatemarco, Erica Sood, Dennis J. Hand, Meghan Gannon, Jobayer Hossain, Neera K. Goyal May 2023

The Child Healthcare At Mater Pediatric Study (Champs): A 2-Arm Cluster Randomized Control Trial Of Group Well Child Care For Mothers In Treatment For Opioid Use Disorder And Their Children, Vanessa L. Short, Diane J. Abatemarco, Erica Sood, Dennis J. Hand, Meghan Gannon, Jobayer Hossain, Neera K. Goyal

Department of Pediatrics Faculty Papers

BACKGROUND: Studies suggest that group-based well child care-a shared medical appointment where families come together as a group to receive pediatric primary care-increases patient-reported satisfaction and adherence to recommended care. Evidence supporting the use of group well child care for mothers with opioid use disorder, however, is lacking. The overall objective of the Child Healthcare at MATER Pediatric Study (CHAMPS) trial is to evaluate a group model of well child care for mothers with opioid use disorder and their children.

METHODS: CHAMPS is a single-site 2-arm cluster randomized controlled trial. A total of 108 mother-child dyads will be enrolled into …


Contributions To Auditory System Conduction Velocity: Insights With Multi-Modal Neuroimaging And Machine Learning In Children With Asd And Xyy Syndrome, Jeffrey I. Berman, Luke Bloy, Lisa Blaskey, Carissa R. Jackel, Judith S. Miller, Judith Ross, J. Christopher Edgar, Thimothy P.L. Roberts May 2023

Contributions To Auditory System Conduction Velocity: Insights With Multi-Modal Neuroimaging And Machine Learning In Children With Asd And Xyy Syndrome, Jeffrey I. Berman, Luke Bloy, Lisa Blaskey, Carissa R. Jackel, Judith S. Miller, Judith Ross, J. Christopher Edgar, Thimothy P.L. Roberts

Department of Pediatrics Faculty Papers

Introduction: The M50 electrophysiological auditory evoked response time can be measured at the superior temporal gyrus with magnetoencephalography (MEG) and its latency is related to the conduction velocity of auditory input passing from ear to auditory cortex. In children with autism spectrum disorder (ASD) and certain genetic disorders such as XYY syndrome, the auditory M50 latency has been observed to be elongated (slowed).

Methods: The goal of this study is to use neuroimaging (diffusion MR and GABA MRS) measures to predict auditory conduction velocity in typically developing (TD) children and children with autism ASD and XYY syndrome.

Results: Non-linear TD …


Use Of Integrative, Complementary, And Alternative Medicine In Children With Epilepsy: A Global Scoping Review, Zahra Zhu, Daniela Dluzynski, Nouran Hammad, Deepika Pugalenthi, Sarah A. Walser, Rea Mittal, Debopam Samanta, Melanie L. Brown, Ali A. Asadi-Pooya, Angelina Kakooza-Mwesige, Alberto Spalice, Martina Capponi, Alain Lekoubou, Ashutosh Kumar, Sita Paudel, Paul R. Carney, Gayatra Mainali, Sunil Naik Apr 2023

Use Of Integrative, Complementary, And Alternative Medicine In Children With Epilepsy: A Global Scoping Review, Zahra Zhu, Daniela Dluzynski, Nouran Hammad, Deepika Pugalenthi, Sarah A. Walser, Rea Mittal, Debopam Samanta, Melanie L. Brown, Ali A. Asadi-Pooya, Angelina Kakooza-Mwesige, Alberto Spalice, Martina Capponi, Alain Lekoubou, Ashutosh Kumar, Sita Paudel, Paul R. Carney, Gayatra Mainali, Sunil Naik

Department of Neurology Faculty Papers

(1) Background: Epilepsy is one of the most common chronic neurological disorders in childhood. Complementary and alternative medicine (CAM) use is highly prevalent in patients with epilepsy. Despite CAM’s widespread and increasing popularity, its prevalence, forms, perceived benefits, and potential risks in pediatric epilepsy are rarely explored. (2) Methods: We performed a scoping review of the available literature on the use of CAM in pediatric epilepsy. (3) Results: Overall, global cross-sectional studies showed a variable degree of CAM usage among children with epilepsy, ranging from 13 to 44% in prevalence. Popular types of CAMs reported were supplements, cannabis products, aromatherapy, …


Impact Of Intravenous Fluid Administration On Cardiac Output And Oxygenation During Cardiopulmonary Resuscitation, Jennifer Lutz, Yosef Levenbrown, Md Jobayer Hossain, Anne Hesek, Kelly E. Massa, James P. Keith, Thomas H. Shaffer Mar 2023

Impact Of Intravenous Fluid Administration On Cardiac Output And Oxygenation During Cardiopulmonary Resuscitation, Jennifer Lutz, Yosef Levenbrown, Md Jobayer Hossain, Anne Hesek, Kelly E. Massa, James P. Keith, Thomas H. Shaffer

Department of Pediatrics Faculty Papers

Background: The efect of intravenous fuid (IVF) administration during cardiopulmonary resuscitation (CPR) is an unexplored factor that may improve cardiac output (CO) during CPR. The aim of this study was to determine the efect of IVF administration on CO and oxygenation during CPR.

Methods: This experimental animal study was performed in a critical care animal laboratory. Twenty-two Landrace-Yorkshire female piglets weighing 27–37 kg were anesthetized, intubated, and placed on positive pressure ventilation. Irreversible cardiac arrest was induced with bupivacaine. CPR was performed with a LUCAS 3 mechanical compression device. Pigs were randomized into IVF or no-IVF groups. Pigs in the …


Trauma-Informed And Family-Centered Paediatric Resuscitation: Defining Domains And Practices, Nancy Kassam-Adams, Lucas Butler, Julia Price, Marcie Gawel, Leila Graham, Sage Myers, Marc Auerbach Mar 2023

Trauma-Informed And Family-Centered Paediatric Resuscitation: Defining Domains And Practices, Nancy Kassam-Adams, Lucas Butler, Julia Price, Marcie Gawel, Leila Graham, Sage Myers, Marc Auerbach

Department of Pediatrics Faculty Papers

Aim

For paediatric patients and families, resuscitation can be an extremely stressful experience with significant medical and psychological consequences. Psychological sequelae may be reduced when healthcare teams apply patient- and family-centered care and trauma-informed care, yet there are few specific instructions for effective family-centered or trauma-informed behaviours that are observable and teachable. We aimed to develop a framework and tools to address this gap.

Methods

We reviewed relevant policy statements, guidelines, and research to define core domains of family-centered and trauma-informed care, and identified observable evidence-based practices in each domain. We refined this list of practices via review of provider/team …