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Articles 1 - 30 of 77

Full-Text Articles in Analytical, Diagnostic and Therapeutic Techniques and Equipment

Safety And Efficacy Of Individualised Exercise And Nad+ Precursor Supplementation In Patients With Friedreich’S Ataxia In The Usa: A Single-Centre, 2 × 2 Factorial, Randomised Controlled Trial, Kimberly Lin, Anna Bucha, Kara Mcsweeney, Kristin Wade, Antoneta Karaj, Jaclyn Tamaroff, Shannon O'Malley, Nicole Chung, Nicolette Cilenti, Julianne Wanner, Gabriel Adzika, Clementina Mesaros, Ian Blair, Teerapat Rojsajjakul, Suraj Serai, Jennifer Farmer, Kyle Bryant, Yingying Lu, Michael Harhay, David Weber, Stephen Paridon, Erin Seifert, Mary Putt, Payman Zamani, Joseph Baur, David Lynch, Shana Mccormack May 2026

Safety And Efficacy Of Individualised Exercise And Nad+ Precursor Supplementation In Patients With Friedreich’S Ataxia In The Usa: A Single-Centre, 2 × 2 Factorial, Randomised Controlled Trial, Kimberly Lin, Anna Bucha, Kara Mcsweeney, Kristin Wade, Antoneta Karaj, Jaclyn Tamaroff, Shannon O'Malley, Nicole Chung, Nicolette Cilenti, Julianne Wanner, Gabriel Adzika, Clementina Mesaros, Ian Blair, Teerapat Rojsajjakul, Suraj Serai, Jennifer Farmer, Kyle Bryant, Yingying Lu, Michael Harhay, David Weber, Stephen Paridon, Erin Seifert, Mary Putt, Payman Zamani, Joseph Baur, David Lynch, Shana Mccormack

Department of Pathology, Anatomy, and Cell Biology Faculty Papers

BACKGROUND:Friedreich's ataxia is a rare, chronic, progressive, neurodegenerative condition affecting multiple organ systems, including neurological, musculoskeletal, cardiac, and endocrine systems, and is marked by low cardiopulmonary fitness. We tested the effect of exercise and NAD+ precursor supplementation with nicotinamide riboside, which have each shown benefits in animal and early clinical studies, on cardiopulmonary fitness in individuals with Friedreich's ataxia.

METHODS: This 12-week, outpatient, phase 2, single-site (Children's Hospital of Philadelphia, Philadelphia, PA, USA), randomised, 2 × 2 factorial clinical trial recruited individuals aged 10–40 years with an ejection fraction of 45% or greater who were able to exercise. A …


Virtual Reality For The Management Of Postoperative Pain And Anxiety In Children And Adolescents Undergoing Nuss Repair Of Pectus Excavatum: Randomized Controlled Trial, Charlotte M. Walter, Dillon Froass, Nora Bell, Lauren Haack, Chloe Boehmer, Claudia Bruguera Torres, Rachel Spivak, Max Chou, Kristie Geisler, Keith O'Conor, Sara E. Williams, Lili Ding, Christopher D. King, Vanessa A. Olbrecht Mar 2026

Virtual Reality For The Management Of Postoperative Pain And Anxiety In Children And Adolescents Undergoing Nuss Repair Of Pectus Excavatum: Randomized Controlled Trial, Charlotte M. Walter, Dillon Froass, Nora Bell, Lauren Haack, Chloe Boehmer, Claudia Bruguera Torres, Rachel Spivak, Max Chou, Kristie Geisler, Keith O'Conor, Sara E. Williams, Lili Ding, Christopher D. King, Vanessa A. Olbrecht

Department of Medicine Faculty Papers

BACKGROUND: Virtual reality (VR) is a novel technology with implications for pain and sensory processing. VR may serve as a novel, scalable method to deliver clinically validated therapy for pain management as an alternative or adjunct to opioids for acute pain. Given that psychological factors and pain perception are both components of postoperative pain, it may also be beneficial to incorporate modalities that decrease anxiety, such as active relaxation and guided meditation with VR. Unfortunately, these therapies are not widely available due to multiple barriers. VR has the potential to deliver pain-reducing, psychologically based therapy to children, thereby enhancing multimodal …


Evaluating The American Society Of Hematology Quality Measure On Timeliness Of Analgesics For Sickle Cell Disease Pain Crisis, Ibrahim Gwarzo, Harish Chandra Dega, Paula Tanabe, Robin Miller, David C. Brousseau Feb 2026

Evaluating The American Society Of Hematology Quality Measure On Timeliness Of Analgesics For Sickle Cell Disease Pain Crisis, Ibrahim Gwarzo, Harish Chandra Dega, Paula Tanabe, Robin Miller, David C. Brousseau

Department of Medicine Faculty Papers

The American Society of Hematology (ASH) proposed the median time to first emergency department (ED) administration of pain medication for patients with sickle cell disease (SCD) vaso-occlusive pain episodes (VOE) as a site-level quality measure. Generalizable studies assessing current guideline adherence recommending pain medications within 60 minutes are lacking. We leveraged multisite electronic health record data from Epic's Cosmos research platform to analyze ED encounters for SCD VOE from 1 January 2019 to 31 December 2024, with administration of at least 1 pain medication. We calculated the quality measure (median time to first pain medication) and ranked sites based on …


Epidemiology Of Healthcare-Associated Ventriculitis And Meningitis (Hcavm) And Community-Acquired Meningitis/Encephalitis And Evaluation Of An Off-Label Pcr Panel For Hcavm Diagnosis, Jaime Nassur, Katarina Schnell, Christine Dolon, Laura Walters, Devin Weber, Matthew Pettengill Feb 2026

Epidemiology Of Healthcare-Associated Ventriculitis And Meningitis (Hcavm) And Community-Acquired Meningitis/Encephalitis And Evaluation Of An Off-Label Pcr Panel For Hcavm Diagnosis, Jaime Nassur, Katarina Schnell, Christine Dolon, Laura Walters, Devin Weber, Matthew Pettengill

Department of Medicine Faculty Papers

Healthcare-associated ventriculitis and meningitis (HCAVM) is associated with high morbidity and mortality, but rapid diagnostic tests intended to cover microorganisms that typically cause HCAVM are not commercially available. We sought to evaluate the etiology of HCAVM compared to community-acquired meningitis/encephalitis (CAME) at our healthcare system, and to study the utility of repurposing a multiplex molecular panel intended for positive blood cultures for HCAVM cases. Our epidemiologic review demonstrated that HCAVM cases were primarily caused by bacteria and fungi also associated with bloodstream infections, in contrast with CAME cases, which were primarily caused by herpesviruses and Cryptococcus. Eighty-two cerebrospinal fluid …


An Analysis Of Pediatric Retinoblastoma Medical Malpractice Litigation, Victoria Miller, Alyssa D. Reese, Katherine Foote, Alejandro Navarro, Andrew Galfano, Christian Hietanen Jan 2026

An Analysis Of Pediatric Retinoblastoma Medical Malpractice Litigation, Victoria Miller, Alyssa D. Reese, Katherine Foote, Alejandro Navarro, Andrew Galfano, Christian Hietanen

Department Surgery Faculty Publications

BACKGROUND: Retinoblastoma is a cancer of the retina that is most commonly diagnosed in the pediatric population. Unfortunately, this cancer can spread, leading to poor outcomes such as enucleation. The purpose of this study was to determine the characteristics of medical malpractice litigation associated with a diagnosis of retinoblastoma in the pediatric population.

METHODS: A search for all available cases associated with "retinoblastoma" in the Westlaw Campus Research Legal Database was conducted. This spanned from database inception to August 2023. Cases were included if there was a complaint of medical malpractice related to retinoblastoma present in a pediatric patient. Demographic …


35 Individuals With Huwe1-Related Neurodevelopmental Disorder And Suggested Clinical Evaluations, Mindy H. Li, Deziree L. Coleman, Kelsey Hogan, Danielle Luz, Lindsay Bhandari, Newell Belnap, Tiffany Busa, Charles Coutton, Klaus Dieterich, Svetlana Gorokhova, Clara Hildebrandt, Rachel Logan, Milena Mariani, Manuela Morleo, Vincenzo Nigro, John Pappas, Rachel Rabin, Kelly Schoch, Angelo Selicorni, Vandana Shashi, Rebecca Spillman, Jennifer Sullivan, Charlotte Tardy, Samantha A. Schrier Vergano, Brock Grill, Kristin Baranano Jan 2026

35 Individuals With Huwe1-Related Neurodevelopmental Disorder And Suggested Clinical Evaluations, Mindy H. Li, Deziree L. Coleman, Kelsey Hogan, Danielle Luz, Lindsay Bhandari, Newell Belnap, Tiffany Busa, Charles Coutton, Klaus Dieterich, Svetlana Gorokhova, Clara Hildebrandt, Rachel Logan, Milena Mariani, Manuela Morleo, Vincenzo Nigro, John Pappas, Rachel Rabin, Kelly Schoch, Angelo Selicorni, Vandana Shashi, Rebecca Spillman, Jennifer Sullivan, Charlotte Tardy, Samantha A. Schrier Vergano, Brock Grill, Kristin Baranano

Department of Pediatrics Faculty Publications

HUWE1 (HECT, UBA, and WWE Domain Containing E3 Ubiquitin Protein Ligase1, OMIM 300697), located at Xp11.22, encodes a ubiquitin ligase that is highly conserved across species. Genetic variants in HUWE1 described in multiple independent studies cause X-linked intellectual disability, including in the patients identified by Juberg, Marsidi, and Brooks. This report describes 35 additional cases of individuals with variants in HUWE1 and suggested guidelines for clinical management. Our study includes several female cases, which have not been widely reported previously. Our findings confirm earlier reported clinical features including developmental delay, autism, hypotonia, short stature, and dysmorphic facial features as well …


The Crucial Role Of Machine Learning Models In Predicting Current Childhood Asthma: Model Comparison, Calibration, And Shap-Based Interpretation, Aditya Chakraborty, A. K.M. Raquibul Bashar Jan 2026

The Crucial Role Of Machine Learning Models In Predicting Current Childhood Asthma: Model Comparison, Calibration, And Shap-Based Interpretation, Aditya Chakraborty, A. K.M. Raquibul Bashar

Epidemiology, Biostatistics, & Environmental Health Faculty Publications

Background: Asthma is one of the most prominent chronic diseases in children and one of the most challenging ailments to diagnose in infants and preschoolers in the United States. Predictive models can be instrumental in improving early diagnosis, personalized treatment strategies, and disease progression. By utilizing nationalized data, this study focuses on building and comparing high-performing analytical predictive models based on the relevant risk factors and identifying the most influential predictors.

Methods: We analyzed cross-sectional BRFSS Asthma Call-Back Survey data (2011-2020; N = 9,813) and randomly split participants into training and testing sets. An XGBoost model (hyperparameters tuned via grid …


Vasoactive Drug Use In Children With Community Acquired Septic Shock In Australia And New Zealand, Elliot Long, Stephen Hearps, Amanda Williams, Ben Gelbart, Warwick Butt, Thomas Rozen, Sarah Mcnab, Meredith Borland, Simon Erickson, Shefali Jani, Marino Festa, Eunicia Tan, Natalie Phillips, Sainath Raman, Amit Kochar, Subodh Ganu, Simon Craig, Anna Lithgow, Arjun Rao, Emma Whyte, Stuart Dalziel, Shane George, Fran Balamuth, Scott Weiss, Nathan Kuppermann, Franz Babl Dec 2025

Vasoactive Drug Use In Children With Community Acquired Septic Shock In Australia And New Zealand, Elliot Long, Stephen Hearps, Amanda Williams, Ben Gelbart, Warwick Butt, Thomas Rozen, Sarah Mcnab, Meredith Borland, Simon Erickson, Shefali Jani, Marino Festa, Eunicia Tan, Natalie Phillips, Sainath Raman, Amit Kochar, Subodh Ganu, Simon Craig, Anna Lithgow, Arjun Rao, Emma Whyte, Stuart Dalziel, Shane George, Fran Balamuth, Scott Weiss, Nathan Kuppermann, Franz Babl

Department of Medicine Faculty Papers

BACKGROUND: Vasoactive agents are a critical supportive therapy for children with sepsis. We describe the choice and use patterns of vasoactive agents in children with sepsis.

METHODS: Prospective observational study conducted in 11 hospitals in Australia and New Zealand through the Paediatric Research in Emergency Departments International Collaborative (PREDICT) Network from April 2021 to December 2023. Children aged 0-< 18 years with suspected sepsis were included. Children admitted to hospital and treated with parenteral antibiotics and either 1) a provisional diagnosis of sepsis, and/or 2) treatment for suspected sepsis (fluid bolus to treat poor perfusion). The frequency and sequence of use of vasoactive agents, and contributors to the choice of initial vasoactive were collected.

FINDINGS: 6232 children with suspected sepsis were included; median age of 2.1 years (IQR 0.3-7.1 years), in-hospital mortality of 60 (1.0%), in whom a subset of 306 (4.9%) met Phoenix sepsis criteria. Vasoactive agents were used in 179 (2.9%) children overall …


Parent Needs, Recommendations, And Systemic Challenges Affecting Pediatric Hypertension Detection, Abbas H. Zaidi, Erica D. Sood, Sarah De Ferranti, Samuel Gidding, Varsha Zadokar, Jonathan M. Miller, Anne E Kazak Sep 2025

Parent Needs, Recommendations, And Systemic Challenges Affecting Pediatric Hypertension Detection, Abbas H. Zaidi, Erica D. Sood, Sarah De Ferranti, Samuel Gidding, Varsha Zadokar, Jonathan M. Miller, Anne E Kazak

Department of Pediatrics Faculty Papers

Pediatric hypertension (HTN) affects 3%-5% of children in the United States, yet only 25% are diagnosed and 60% lack recommended follow-up care. Skepticism about elevated blood pressure (BP) readings and reluctance to use antihypertensive medications by parents and clinicians highlight the need for stakeholder-informed strategies to address these challenges. This study examined parents' perceived needs, their recommended strategies to improve HTN detection, and contextual health system challenges. Parents and clinicians from 10 pediatric primary care clinics participated in semi-structured qualitative interviews. Only parents of children with documented stage 2 BP readings and a HTN diagnosis, but with gaps in care …


Predictors Of Final Visual Outcome In Patients With Leber Hereditary Optic Neuropathy Treated With Lenadogene Nolparvovec Gene Therapy, Robert C Sergott, Valerio Carelli, Nancy Newman, Valérie Biousse, Patrick Yu-Wai-Man, Catherine Vignal-Clermont, Constant Josse, Magali Taiel, José-Alain Sahel, Piero Barboni Jul 2025

Predictors Of Final Visual Outcome In Patients With Leber Hereditary Optic Neuropathy Treated With Lenadogene Nolparvovec Gene Therapy, Robert C Sergott, Valerio Carelli, Nancy Newman, Valérie Biousse, Patrick Yu-Wai-Man, Catherine Vignal-Clermont, Constant Josse, Magali Taiel, José-Alain Sahel, Piero Barboni

Wills Eye Hospital Papers

PURPOSE: This exploratory analysis aimed to identify predictive factors of final best-corrected visual acuity (BCVA) in patients with Leber hereditary optic neuropathy (LHON) harboring the m.11778G>A mutation who received lenadogene nolparvovec gene therapy.

METHODS: The following covariates were individually evaluated as possible factors associated with improved final BCVA: age, gender, timing of treatment, baseline BCVA value, and baseline optical coherence tomography (OCT) parameters. Univariate analyses were performed from three phase 3 studies (RESCUE, REVERSE, and REFLECT), using BCVA at 1.5 years post-treatment as the dependent variable.

RESULTS: In 113 eyes treated at least 6 months after disease onset, the …


Validation Of Established Thyroid Ultrasound Volume Norms In A Chernobyl Cohort, Lydia B. Zablotska, Robert J. Mcconnell, Aleksandr V. Rozhko, Patrick O'Kane, Vasilina Yauseyenka, Mark P. Little, Victor Minenko, Vladimir Drozdovitch, Tamara Moskvicheva, Maureen Hatch, Tamara Yeudachkova, Kiyohiko Mabuchi, Elizabeth K. Cahoon Jul 2025

Validation Of Established Thyroid Ultrasound Volume Norms In A Chernobyl Cohort, Lydia B. Zablotska, Robert J. Mcconnell, Aleksandr V. Rozhko, Patrick O'Kane, Vasilina Yauseyenka, Mark P. Little, Victor Minenko, Vladimir Drozdovitch, Tamara Moskvicheva, Maureen Hatch, Tamara Yeudachkova, Kiyohiko Mabuchi, Elizabeth K. Cahoon

Department of Radiology Faculty Papers

OBJECTIVE: To establish thyroid ultrasound volume norms appropriate for studies of diffuse goiter in a cohort of children and adolescents from an iodine-deficient population exposed to 131I by the Chernobyl fallout.

METHODS: A cohort of 11,970 Belarusians aged ≤18 years at the time of the 1986 Chernobyl accident with individual thyroid radiation dose estimates was screened 10-18 years later. From these, a low-dose subset of 2,392 with no thyroid diseases was selected to construct age- and sex-specific normative values for thyroid ultrasound volume, compared to Belarusian Ministry of Health (MOH) norms and existing WHO and European standards.

RESULTS: Cohort-specific values …


Effect Of Testosterone Treatment During Puberty In Boys With Klinefelter Syndrome (The Tipy Study): Protocol For A Nationwide Randomised, Double-Blinded, Placebo-Controlled Study, Ida Dyhr Caspersen, Andrés Felipe Østergaard Fritzbøger, Jørgen Holm Petersen, Niels Birkebæk, Ann-Margrethe Rønholt Christensen, Anders Jørgen Schou, Kurt Kristensen, Judith L. Ross, Shanlee Davis, Gary Butler, Sophie Van Rijn, Anders Juul, Lise Aksglaede Mar 2025

Effect Of Testosterone Treatment During Puberty In Boys With Klinefelter Syndrome (The Tipy Study): Protocol For A Nationwide Randomised, Double-Blinded, Placebo-Controlled Study, Ida Dyhr Caspersen, Andrés Felipe Østergaard Fritzbøger, Jørgen Holm Petersen, Niels Birkebæk, Ann-Margrethe Rønholt Christensen, Anders Jørgen Schou, Kurt Kristensen, Judith L. Ross, Shanlee Davis, Gary Butler, Sophie Van Rijn, Anders Juul, Lise Aksglaede

Department of Pediatrics Faculty Papers

INTRODUCTION: Klinefelter syndrome (KS) is a genetic condition characterised by the presence of an extra X chromosome in males (47,XXY). KS is associated with various phenotypic characteristics in adult life, including infertility, hypogonadism and increased risk of type II diabetes, cardiovascular disease and osteoporosis. Additionally, individuals with KS often experience mental health challenges and functional impairments that significantly impact their quality of life. Currently, testosterone replacement therapy (TRT) in adolescence is considered the first-line treatment by some physicians for patients with KS and biochemical signs of hypogonadism. However, comprehensive evidence on its effectiveness in preventing typical phenotypic traits associated with …


External Validation Of The Phoenix Sepsis Score In Children With Suspected Community-Acquired Sepsis, Elliot Long, Meredith Borland, Shane George, Shefali Jani, Eunicia Tan, Natalie Phillips, Amit Kochar, Simon Craig, Anna Lithgow, Arjun Rao, Stuart Dalziel, Ed Oakley, Stephen Hearps, Ben Gelbart, Sarah Mcnab, Fran Balamuth, Scott Weiss, Nathan Kuppermann, Charlotte Brad, Amanda Williams, Franz Babl Mar 2025

External Validation Of The Phoenix Sepsis Score In Children With Suspected Community-Acquired Sepsis, Elliot Long, Meredith Borland, Shane George, Shefali Jani, Eunicia Tan, Natalie Phillips, Amit Kochar, Simon Craig, Anna Lithgow, Arjun Rao, Stuart Dalziel, Ed Oakley, Stephen Hearps, Ben Gelbart, Sarah Mcnab, Fran Balamuth, Scott Weiss, Nathan Kuppermann, Charlotte Brad, Amanda Williams, Franz Babl

Department of Medicine Faculty Papers

IMPORTANCE: The novel Phoenix Sepsis Score and sepsis criteria were derived and validated using a multicountry dataset and proposed as a new definition for sepsis in children.

OBJECTIVE: To externally validate the Phoenix Sepsis Score and sepsis criteria in a cohort of children hospitalized with suspected community-acquired sepsis.

DESIGN, SETTING, AND PARTICIPANTS: This diagnostic study used data from the multicenter, multicountry Sepsis Epidemiology in Australian and New Zealand Emergency Departments (SENTINEL) study, collected from 2021 to 2023 and including 90-day follow-up. Children admitted to the hospital through 11 emergency departments in Australia and New Zealand and treated with parenteral antibiotics …


Multiparametric Mri Along With Machine Learning Predicts Prognosis And Treatment Response In Pediatric Low-Grade Glioma, Anahita Fathi Kazerooni, Adam Kraya, Komal Rathi, Meen Chul Kim, Arastoo Vossough, Nastaran Khalili, Ariana Familiar, Deep Gandhi, Neda Khalili, Varun Kesherwani, Debanjan Haldar, Hannah Anderson, Run Jin, Aria Mahtabfar, Sina Bagheri, Yiran Guo, Qi Li, Xiaoyan Huang, Yuankun Zhu, Alex Sickler, Matthew R Lueder, Saksham Phul, Mateusz Koptyra, Phillip Storm, Jeffrey Ware, Yuanquan Song, Christos Davatzikos, Jessica Foster, Sabine Mueller, Michael J Fisher, Adam Resnick, Ali Nabavizadeh Jan 2025

Multiparametric Mri Along With Machine Learning Predicts Prognosis And Treatment Response In Pediatric Low-Grade Glioma, Anahita Fathi Kazerooni, Adam Kraya, Komal Rathi, Meen Chul Kim, Arastoo Vossough, Nastaran Khalili, Ariana Familiar, Deep Gandhi, Neda Khalili, Varun Kesherwani, Debanjan Haldar, Hannah Anderson, Run Jin, Aria Mahtabfar, Sina Bagheri, Yiran Guo, Qi Li, Xiaoyan Huang, Yuankun Zhu, Alex Sickler, Matthew R Lueder, Saksham Phul, Mateusz Koptyra, Phillip Storm, Jeffrey Ware, Yuanquan Song, Christos Davatzikos, Jessica Foster, Sabine Mueller, Michael J Fisher, Adam Resnick, Ali Nabavizadeh

Department of Neurosurgery Faculty Papers

Pediatric low-grade gliomas (pLGGs) exhibit heterogeneous prognoses and variable responses to treatment, leading to tumor progression and adverse outcomes in cases where complete resection is unachievable. Early prediction of treatment responsiveness and suitability for immunotherapy has the potential to improve clinical management and outcomes. Here, we present a radiogenomic analysis of pLGGs, integrating MRI and RNA sequencing data. We identify three immunologically distinct clusters, with one group characterized by increased immune activity and poorer prognosis, indicating potential benefit from immunotherapies. We develop a radiomic signature that predicts these immune profiles with over 80% accuracy. Furthermore, our clinicoradiomic model predicts progression-free …


Magnetization Transfer Ratio In The Typically Developing Pediatric Spinal Cord: Normative Data And Age Correlation, Sara Naghizadeh Kashani, Iswarya Vel, Zahra Sadeghi Adl, Shiva Shahrampour, Devon Middleton, Mahdi Alizadeh, Laura Krisa, Scott Faro, Slimane Tounekti, Julien Cohen-Adad, Feroze Mohamed Jan 2025

Magnetization Transfer Ratio In The Typically Developing Pediatric Spinal Cord: Normative Data And Age Correlation, Sara Naghizadeh Kashani, Iswarya Vel, Zahra Sadeghi Adl, Shiva Shahrampour, Devon Middleton, Mahdi Alizadeh, Laura Krisa, Scott Faro, Slimane Tounekti, Julien Cohen-Adad, Feroze Mohamed

Department of Radiology Faculty Papers

BACKGROUND AND PURPOSE: This study presents automated atlas-based magnetization transfer (MT) measurements of the typically developing pediatric cervical spinal cord (SC). We report normative MT ratio (MTR) values from the whole cervical cord white matter (WM) and WM tracts, examining variations with age, sex, height, and weight.

METHODS: MT scans of 33 healthy females (mean age = 12.8) and 22 males (mean age = 13.09) were acquired from the cervical SC (C2-C7) using a 3.0 T MRI. Data were processed using the SC Toolbox, segmented, and registered to the PAM50 template. Affine and non-rigid transformations co-registered the PAM50 WM atlas …


Congenital Posterior Urethroperineal Fistula Presenting As A Cystic Mass, Muhammad Siddiqui, Sang Woo Pak, Preeya Mistry, Sarah Starnes, Stacy White, Anthony Zarka, Lori Gurien Jan 2025

Congenital Posterior Urethroperineal Fistula Presenting As A Cystic Mass, Muhammad Siddiqui, Sang Woo Pak, Preeya Mistry, Sarah Starnes, Stacy White, Anthony Zarka, Lori Gurien

South Atlantic Division GME Research Day 2025

No abstract provided.


Cutaneous Adverse Effects From Diabetes Devices In Pediatric Patients With Type 1 Diabetes Mellitus: Systematic Review, Alicia Podwojniak, Joseph Flemming, Isabella J Tan, Hira Ghani, Zachary Neubauer, Anne Jones Nov 2024

Cutaneous Adverse Effects From Diabetes Devices In Pediatric Patients With Type 1 Diabetes Mellitus: Systematic Review, Alicia Podwojniak, Joseph Flemming, Isabella J Tan, Hira Ghani, Zachary Neubauer, Anne Jones

Rowan-Virtua School of Osteopathic Medicine Departmental Research

BACKGROUND: Continuous glucose monitoring (CGM) and continuous subcutaneous insulin infusions (CSIIs) are the current standard treatment devices for type 1 diabetes (T1D) management. With a high prevalence of T1D beginning in pediatrics and carrying into adulthood, insufficient glycemic control leads to poor patient outcomes. Dermatologic complications such as contact dermatitis, lipodystrophies, and inflammatory lesions are among those associated with CGM and CSII, which reduce glycemic control and patient compliance.

OBJECTIVE: This systematic review aims to explore the current literature surrounding dermatologic complications of CGM and CSII as well as the impact on patient outcomes.

METHODS: A systematic review of the …


Pediatric Cardiac Xenotransplantation And Expanded Access: Ethical Considerations, Daniel J Hurst, Christopher Bobier, Anthony Merlocco, Luz A Padilla, Daniel Rodger, David Cleveland, John D Cleveland Oct 2024

Pediatric Cardiac Xenotransplantation And Expanded Access: Ethical Considerations, Daniel J Hurst, Christopher Bobier, Anthony Merlocco, Luz A Padilla, Daniel Rodger, David Cleveland, John D Cleveland

Rowan-Virtua School of Osteopathic Medicine Departmental Research

Due to the current organ shortage waitlist, alternatives to allotransplantation are necessary. Xenotransplantation is currently being pursued as one such alternative in adults in need of kidney or heart transplantation. Cardiac xenotransplantation of genetically modified pig hearts has been conducted twice in adults under the United States Food and Drug Administration (FDA) expanded access criteria. Because of the shortage of transplantable hearts for children as well as the lack of mechanical circulatory support in this population, pediatric researchers are exploring FDA expanded access in high-risk neonates and infants who lack alternative options for survival. The adult cardiac xenotransplantation experience with …


Early Bolus Epinephrine Administration During Pediatric Cardiopulmonary Resuscitation For Bradycardia With Poor Perfusion: An Icu-Resuscitation Study, Amanda O'Halloran, Ron Reeder, Robert Berg, Tageldin Ahmed, Michael Bell, Robert Bishop, Matthew Bochkoris, Candice Burns, Joseph Carcillo, Todd Carpenter, J. Michael Dean, J. Wesley Diddle, Myke Federman, Richard Fernandez, Ericka Fink, Deborah Franzon, Aisha Frazier, Stuart Friess, Kathryn Graham, Mark Hall, David Hehir, Christopher M Horvat, Leanna Huard, Martha Kienzle, Todd Kilbaugh, Tensing Maa, Arushi Manga, Patrick Mcquillen, Kathleen Meert, Peter Mourani, Vinay Nadkarni, Maryam Naim, Daniel Notterman, Murray Pollack, Anil Sapru, Carleen Schneiter, Matthew Sharron, Neeraj Srivastava, Bradley Tilford, Alexis Topjian, Shirley Viteri, David Wessel, Heather Wolfe, Andrew Yates, Athena Zuppa, Robert Sutton, Ryan Morgan Jul 2024

Early Bolus Epinephrine Administration During Pediatric Cardiopulmonary Resuscitation For Bradycardia With Poor Perfusion: An Icu-Resuscitation Study, Amanda O'Halloran, Ron Reeder, Robert Berg, Tageldin Ahmed, Michael Bell, Robert Bishop, Matthew Bochkoris, Candice Burns, Joseph Carcillo, Todd Carpenter, J. Michael Dean, J. Wesley Diddle, Myke Federman, Richard Fernandez, Ericka Fink, Deborah Franzon, Aisha Frazier, Stuart Friess, Kathryn Graham, Mark Hall, David Hehir, Christopher M Horvat, Leanna Huard, Martha Kienzle, Todd Kilbaugh, Tensing Maa, Arushi Manga, Patrick Mcquillen, Kathleen Meert, Peter Mourani, Vinay Nadkarni, Maryam Naim, Daniel Notterman, Murray Pollack, Anil Sapru, Carleen Schneiter, Matthew Sharron, Neeraj Srivastava, Bradley Tilford, Alexis Topjian, Shirley Viteri, David Wessel, Heather Wolfe, Andrew Yates, Athena Zuppa, Robert Sutton, Ryan Morgan

Department of Pediatrics Faculty Papers

BACKGROUND: Half of pediatric in-hospital cardiopulmonary resuscitation (CPR) events have an initial rhythm of non-pulseless bradycardia with poor perfusion. Our study objectives were to leverage granular data from the ICU-RESUScitation (ICU-RESUS) trial to: (1) determine the association of early epinephrine administration with survival outcomes in children receiving CPR for bradycardia with poor perfusion; and (2) describe the incidence and time course of the development of pulselessness.

METHODS: Prespecified secondary analysis of ICU-RESUS, a multicenter cluster randomized trial of children (< 19 years) receiving CPR in 18 intensive care units in the United States. Index events (October 2016-March 2021) lasting ≥ 2 min with a documented initial rhythm of bradycardia with poor perfusion were included. Associations between early epinephrine (first 2 min of CPR) and outcomes were evaluated with Poisson multivariable regression controlling for a priori pre-arrest characteristics. Among patients with arterial lines, intra-arrest blood pressure waveforms were reviewed to determine presence of a pulse during CPR interruptions. The temporal nature of progression to pulselessness was described and outcomes were compared between patients according to subsequent pulselessness status.

RESULTS: Of 452 eligible subjects, 322 (71%) received early epinephrine. The early epinephrine group had higher pre-arrest severity of illness …


Resection And Reconstruction Of Ewing Sarcoma Of The Cuboid Utilizing Vascularized Fibular Autograft: A Case Report, Brandon Cunha, Alexandre Arkader May 2024

Resection And Reconstruction Of Ewing Sarcoma Of The Cuboid Utilizing Vascularized Fibular Autograft: A Case Report, Brandon Cunha, Alexandre Arkader

Rowan-Virtua Research Day

Ewing Sarcoma (EWS) rarely presents in the bones of the foot, and particularly in the cuboid, making it an exceedingly rare occurrence. We present the case of a 7-year-old female diagnosed with EWS of the right cuboid, detailing the management and outcomes of this rare presentation. The patient, now almost 5 years post-initial local control, exhibits restored foot function, a normalized gait, and no evidence of disease recurrence. This case underscores the potential for preserving lower extremity function and improving postoperative quality of life in rare presentations of EWS through innovative reconstruction strategies.


Cost-Effectiveness And Outcomes Of Utilizing Tisagenlecleucel Therapy (Car T-Cell) In Pediatric Acute Lymphoblastic Leukemia In Comparison To Standard Of Care (Soc) Therapies: A Scoping Review, Andrew Atschinow, Evangeline Attota, Warren Chan, Pooja Kasarapu, Priyal Shah, Karina Vizzoni May 2024

Cost-Effectiveness And Outcomes Of Utilizing Tisagenlecleucel Therapy (Car T-Cell) In Pediatric Acute Lymphoblastic Leukemia In Comparison To Standard Of Care (Soc) Therapies: A Scoping Review, Andrew Atschinow, Evangeline Attota, Warren Chan, Pooja Kasarapu, Priyal Shah, Karina Vizzoni

Rowan-Virtua Research Day

Aims

This review aims to assess the correlations between outcomes and cost of treatment methods for pediatric acute lymphoblastic leukemia patients, specifically comparing CAR T-cell therapy and Standard-of-Care (SoC) therapy. The socioeconomic background of patients will also be taken into consideration to see if there are differences in their outcomes.

Methods

Peer-reviewed publications were collected from PubMed and Web of Science. The keyword strings used were “acute lymphoblastic leukemia,” “pediatric acute lymphoblastic leukemia,” “pediatric,” “CAR T-cell therapy,” and “cost-effectiveness.” 27 citations were obtained. Titles were screened by 6 authors. Articles met the inclusion criteria including potential Quality-Adjusted Life Year (QALY) …


Parents' Perceptions Of Eye-Gaze Technology Use By Children With Complex Communication Needs, Sandra Masayko, Joy Mcgowan, Namrata Grampurohit May 2024

Parents' Perceptions Of Eye-Gaze Technology Use By Children With Complex Communication Needs, Sandra Masayko, Joy Mcgowan, Namrata Grampurohit

Department of Occupational Therapy Faculty Papers

PURPOSE: Some preschool students with complex communication needs explore eye-gaze computer technology (EGCT) and adopt computer-based augmentative and alternative communication (AAC). The objective of this study was to follow preschool explorers of EGCT who are now school aged to describe the children's use of technology and parents' perceptions of its utility for communication, participation, or leisure.

METHOD: Ten parents completed survey questions by Internet and phone and reported their perceptions of nine children's effectiveness in the use and acceptance of AAC and the support they received in implementing technology. The results are reported as a descriptive study.

RESULTS: All children …


Recurrent Intussusception In Child Despite Repeated Surgical Correction, Carlos Cordova, Alexandra Lawlor, Tillie Schumann, Sydney Solomon, Lily Wiedrich Jan 2024

Recurrent Intussusception In Child Despite Repeated Surgical Correction, Carlos Cordova, Alexandra Lawlor, Tillie Schumann, Sydney Solomon, Lily Wiedrich

East Florida Division GME Research Day 2024

No abstract provided.


Evidence-Based Clinical Practice Guideline: Comprehensive Pediatric Eye And Vision Examination, American Optometric Association Oct 2020

Evidence-Based Clinical Practice Guideline: Comprehensive Pediatric Eye And Vision Examination, American Optometric Association

Optometric Clinical Practice

Republished with the written permission granted from the American Optometric Association, October 2, 2020.

Developed by the AOA Evidence-Based Optometry Guideline Development Group

Approved by the AOA Board of Trustees February 12, 2017

Diane T. Adamczyk, O.D., Chair – State University of New York, College of Optometry, New York, New York

John F. Amos, O.D., M.S. – University of Alabama at Birmingham School of Optometry, Birmingham, Alabama, Dean and Professor Emeritus

Felix M. Barker, II, O.D., M.S. – W. G. (Bill) Hefner VAMC, Salisbury, North Carolina

Benjamin P. Casella, OD – Private Practice – Casella Eye Center, Augusta, Georgia

Linda …


What's All The Buzzy About? Using Cryotherapy And Vibration For Pain During Vaccinations In Children, Marta L. Byma May 2020

What's All The Buzzy About? Using Cryotherapy And Vibration For Pain During Vaccinations In Children, Marta L. Byma

Evidence-Based Practice Project Reports

Vaccinations are the most common painful needle procedure, with an estimated 12 billion injections given per year (CDC, 2019). The usual method for administration of vaccinations is through needle puncture, which is often painful. Children often report receiving a shot as one of the most feared and painful experiences (McMurtry et al., 2015). The purpose of this evidence-based practice (EBP) project was to improve patient experiences by decreasing the pain that is associated with vaccinations through the use of a nonpharmacological method for comfort via the Buzzy® device. After thorough analysis and synthesis of the literature, the Buzzy® device that …


Genomic Prediction Of Relapse In Recipients Of Allogeneic Haematopoietic Stem Cell Transplantation., J Ritari, K Hyvärinen, S Koskela, M Itälä-Remes, R Niittyvuopio, A Nihtinen, U Salmenniemi, M Putkonen, L Volin, T Kwan, T Pastinen, J Partanen Jan 2019

Genomic Prediction Of Relapse In Recipients Of Allogeneic Haematopoietic Stem Cell Transplantation., J Ritari, K Hyvärinen, S Koskela, M Itälä-Remes, R Niittyvuopio, A Nihtinen, U Salmenniemi, M Putkonen, L Volin, T Kwan, T Pastinen, J Partanen

Manuscripts, Articles, Book Chapters and Other Papers

Allogeneic haematopoietic stem cell transplantation currently represents the primary potentially curative treatment for cancers of the blood and bone marrow. While relapse occurs in approximately 30% of patients, few risk-modifying genetic variants have been identified. The present study evaluates the predictive potential of patient genetics on relapse risk in a genome-wide manner. We studied 151 graft recipients with HLA-matched sibling donors by sequencing the whole-exome, active immunoregulatory regions, and the full MHC region. To assess the predictive capability and contributions of SNPs and INDELs, we employed machine learning and a feature selection approach in a cross-validation framework to discover the …


Serological Proteomic Screening And Evaluation Of A Recombinant Egg Antigen For The Diagnosis Of Low-Intensity Schistosoma Mansoni Infections In Endemic Area In Brazil, Vanessa Silva-Moraes, Lisa Marie Shollenberger, William Castro-Borges, Ana Lucia Teles Rabello, Donald A. Harn, Lia Carolina Soares Medeiros, Wander De Jesus Jeremias, Liliane Maria Vidal Siqueira, Caroline Stephane Salviano Pereira, Maria Luysa Camargos Pedrosa, Nathalie Bonatti Franco Almeida, Aureo Almeida, Jose Roberto Lambertucci, Nidia Francisca De Figueiredo Carneiro, Paulo Marcos Zech Coelho, Refaella Fortini Queiroz Grenfell Jan 2019

Serological Proteomic Screening And Evaluation Of A Recombinant Egg Antigen For The Diagnosis Of Low-Intensity Schistosoma Mansoni Infections In Endemic Area In Brazil, Vanessa Silva-Moraes, Lisa Marie Shollenberger, William Castro-Borges, Ana Lucia Teles Rabello, Donald A. Harn, Lia Carolina Soares Medeiros, Wander De Jesus Jeremias, Liliane Maria Vidal Siqueira, Caroline Stephane Salviano Pereira, Maria Luysa Camargos Pedrosa, Nathalie Bonatti Franco Almeida, Aureo Almeida, Jose Roberto Lambertucci, Nidia Francisca De Figueiredo Carneiro, Paulo Marcos Zech Coelho, Refaella Fortini Queiroz Grenfell

Biological Sciences Faculty Publications

Background

Despite decades of use of control programs, schistosomiasis remains a global public health problem. To further reduce prevalence and intensity of infection, or to achieve the goal of elimination in low-endemic areas, there needs to be better diagnostic tools to detect low-intensity infections in low-endemic areas in Brazil. The rationale for development of new diagnostic tools is that the current standard test Kato-Katz (KK) is not sensitive enough to detect low-intensity infections in low-endemic areas. In order to develop new diagnostic tools, we employed a proteomics approach to identify biomarkers associated with schistosome-specific immune responses in hopes of developing …


Innocent Heart Murmur., Arpan R. Doshi Dec 2018

Innocent Heart Murmur., Arpan R. Doshi

Manuscripts, Articles, Book Chapters and Other Papers

Heart murmur is the most common reason for a referral to a pediatric cardiologist. Virtually all children have a heart murmur during their childhood. Less than 1% of murmurs are pathological in children. Innocent/functional heart murmur is the most common type of heart murmur. There are multiple theories proposed to identify etiology of innocent heart murmur with varying consensus, but everybody agrees that innocent heart murmur does not carry any morbidity or mortality risk. Even today, heart murmur is associated with high physician uncertainty and parental anxiety. Extensive cardiac evaluation for such a benign finding is also associated with high …


A Multicenter Study To Evaluate Pulmonary Function In Osteogenesis Imperfecta., Allison Tam, Shan Chen, Evan Schauer, Ingo Grafe, Venkata Bandi, Jay R. Shapiro, Robert D. Steiner, Peter A. Smith, Michael B. Bober, Tracy Hart, David Cuthbertson, Jeffrey Krischer, Mary Mullins, Peter H. Byers, Robert A. Sandhaus, Michaela Durigova, Francis H. Glorieux, Frank Rauch, Vernon Reid Sutton, Brendan Lee, Members Of The Brittle Bone Disorders Consortium, Eric T. Rush, Sandesh C S Nagamani Dec 2018

A Multicenter Study To Evaluate Pulmonary Function In Osteogenesis Imperfecta., Allison Tam, Shan Chen, Evan Schauer, Ingo Grafe, Venkata Bandi, Jay R. Shapiro, Robert D. Steiner, Peter A. Smith, Michael B. Bober, Tracy Hart, David Cuthbertson, Jeffrey Krischer, Mary Mullins, Peter H. Byers, Robert A. Sandhaus, Michaela Durigova, Francis H. Glorieux, Frank Rauch, Vernon Reid Sutton, Brendan Lee, Members Of The Brittle Bone Disorders Consortium, Eric T. Rush, Sandesh C S Nagamani

Manuscripts, Articles, Book Chapters and Other Papers

Pulmonary complications are a significant cause for morbidity and mortality in osteogenesis imperfecta (OI). However, to date, there have been few studies that have systematically evaluated pulmonary function in individuals with OI. We analyzed spirometry measurements, including forced vital capacity (FVC) and forced expiratory volume in the first second (FEV1 ), in a large cohort of individuals with OI (n = 217) enrolled in a multicenter, observational study. We show that individuals with the more severe form of the disease, OI type III, have significantly reduced FVC and FEV1 which do not follow the expected trends of the …


Pediatric Nasogastric Tube Placement And Verification: Best Practice Recommendations From The Novel Project., Sharon Y. Irving, Gina Rempel, Beth Lyman, Wednesday Marie A Sevilla, Ladonna Northington, Peggi Guenter, American Society For Parenteral And Enteral Nutrition Dec 2018

Pediatric Nasogastric Tube Placement And Verification: Best Practice Recommendations From The Novel Project., Sharon Y. Irving, Gina Rempel, Beth Lyman, Wednesday Marie A Sevilla, Ladonna Northington, Peggi Guenter, American Society For Parenteral And Enteral Nutrition

Manuscripts, Articles, Book Chapters and Other Papers

The placement of a nasogastric tube (NGT) in a pediatric patient is a common practice that is generally perceived as a benign bedside procedure. There is potential risk for NGT misplacement with each insertion. A misplaced NGT compromises patient safety, increasing the risk for serious and even fatal complications. There is no standardized method for verification of the initial NGT placement or reverification assessment of NGT location prior to use. Measurement of the acidity or pH of the gastric aspirate is the most frequently used evidence-based method to verify NGT placement. The radiograph, when properly obtained and interpreted, is considered …