Medical Specialties Commons^™

Immunotherapy Of Tsa-1.C4 Or In Combination With Bnz Confers Protection Against Trypanosoma Cruzi Infection With A Distinct Cytokine Response, Landy Magaly Pech-Pisté, Victor Dzul-Huchim, Christian Florian Teh-Poot, Fabian Gusovsky, Kathryn Jones, Peter Hotez, Liliana Estefanía Villanueva-Lizama, Maria Elena Bottazzi, Jaime Ortega-Lopez, Julio Vladimir Cruz-Chan

Faculty, Staff and Students Publications

Chagas disease is a poverty-related neglected tropical disease caused by the protozoan Trypanosoma cruzi affecting approximately 6.3 million people, predominantly in the Americas. Approximately 30% of T. cruzi infections progress to chronic cardiomyopathy and 10% of these cases end in death from cardiac failure. The first-line drug Benznidazole (BNZ) require a prolonged treatment regimen and can be highly toxic. Here, we evaluate a therapeutic vaccine in T. cruzi-infected mice, based on the recombinant Trypomastigote Surface Antigen 1 (TSA-1.C4) protein and the emulsified adjuvant E6020, and in combination with a suboptimal dose of BNZ. We observed a reduced burden parasite in …

The Agreement Between Pulse Oximetry And Measured Arterial Oxygen Saturations In Postoperative Functionally Univentricular Patients, Fabio Savorgnan, Sebastian Acosta, Joshua Prabhu, Pranathi Pilla, Vikram Shah, Saul Flores, Rohit S Loomba

Faculty, Staff and Students Publications

Background: Pulse oximetry is widely used to estimate arterial oxygen saturation, yet accuracy may vary for a number of reasons. Data on children with functionally univentricular circulation are limited. The primary aim of this study was to evaluate the agreement between arterial oxygen saturation measured by blood gas and pulse oximetry in children with functionally univentricular circulations. Methods: A retrospective analysis was performed of paired arterial blood gas and pulse oximetry oxygen saturation measurements following Norwood, Glenn, or Fontan procedures. Signed difference was defined as arterial oxygen saturation by blood gas-arterial oxygen saturation by pulse oximetry. Bland-Altman analyses, …

2026 Clinical Practice Guideline Update By The Infectious Diseases Society Of America And The Pediatric Infectious Diseases Society On The Management Of Community-Acquired Pneumonia In Infants And Children Older Than 3 Months Of Age: The Use Of Tpa And Dnase Or Tpa Alone For Fibrinolysis, Shawn D St Peter, Krow Ampofo, Thomas Brogan, Michael D Cabana, Claudia Espinosa, Todd A Florin, Jeffrey S Gerber, Michelle Gill, Debra L Palazzi, Mark Sawyer, Angela M Statile, Derek J Williams, Sheena Patel, Mark I Neuman, Samir S Shah

Faculty, Staff and Students Publications

This paper is part of a larger clinical practice guideline on the diagnosis and management of parapneumonic effusion and empyema in children, developed by the Pediatric Infectious Diseases Society and the Infectious Diseases Society of America. In this report, the panel provides recommendations for intrapleural fibrinolysis with tissue plasminogen activator (tPA) alone over tPA and dornase alfa (DNase) in children (3 months to 18 years) with complicated parapneumonic effusion and empyema. The panel's recommendations are based upon evidence derived from systematic literature reviews and adhere to a standardized methodology for rating the certainty of evidence and strength of recommendation according …

2026 Clinical Practice Guideline Update By The Infectious Diseases Society Of America And The Pediatric Infectious Diseases Society On The Management Of Community-Acquired Pneumonia In Infants And Children Older Than 3 Months Of Age: The Choice Of Chest Tube Size, Shawn D St Peter, Krow Ampofo, Thomas Brogan, Michael D Cabana, Claudia Espinosa, Todd A Florin, Jeffrey S Gerber, Michelle Gill, Debra L Palazzi, Mark Sawyer, Angela Statile, Derek J Williams, Sheena Patel, Mark I Neuman, Samir S Shah

Faculty, Staff and Students Publications

This paper is part of a larger clinical practice guideline on the diagnosis and management of parapneumonic effusion and empyema in children, developed by the Infectious Diseases Society of America. In this paper, the panel provides recommendations on the appropriate size of thoracostomy tube for drainage. The panel's recommendations are based upon evidence derived from systematic literature reviews and adhere to a standardized methodology for rating the certainty of evidence and strength of recommendation according to the GRADE (Grading of Recommendations Assessment, Development, and Evaluation) approach.

2026 Clinical Practice Guideline Update By The Infectious Diseases Society Of America And The Pediatric Infectious Diseases Society On The Management Of Community-Acquired Pneumonia In Infants And Children Older Than 3 Months Of Age: The Use Of Pleural Fluid Drainage Versus Observation, Shawn D St Peter, Krow Ampofo, Thomas Brogan, Michael D Cabana, Claudia Espinosa, Todd A Florin, Jeffrey S Gerber, Michelle Gill, Debra L Palazzi, Mark Sawyer, Angela M Statile, Derek J Williams, Sheena Patel, Mark I Neuman, Samir S Shah

Faculty, Staff and Students Publications

This paper is part of a larger clinical practice guideline on the diagnosis and management of parapneumonic effusion and empyema in children, developed by the Infectious Diseases Society of America. In this paper, the panel provides recommendations for pleural fluid drainage in children (3 months to 18 years) with parapneumonic effusion and empyema. The panel's recommendations are based upon evidence derived from systematic literature reviews and adhere to a standardized methodology for rating the certainty of evidence and strength of recommendation according to the GRADE (Grading of Recommendations Assessment, Development, and Evaluation) approach.

2026 Clinical Practice Guideline Update By The Infectious Diseases Society Of America And The Pediatric Infectious Diseases Society On The Management Of Community-Acquired Pneumonia In Infants And Children Older Than 3 Months Of Age: The Use Of Chest Ultrasound In Children With Parapneumonic Effusion, Shawn D St Peter, Krow Ampofo, Thomas Brogan, Michael D Cabana, Claudia Espinosa, Todd A Florin, Jeffrey S Gerber, Michelle Gill, Debra L Palazzi, Mark Sawyer, Angela M Statile, Derek J Williams, Sheena Patel, Samir S Shah, Mark I Neuman

Faculty, Staff and Students Publications

This paper is part of a larger clinical practice guideline on the diagnosis and management of parapneumonic effusion and empyema in children, developed by the Pediatric Infectious Diseases Society and the Infectious Diseases Society of America. In this paper, the panel provides recommendations for the role of chest ultrasound to evaluate parapneumonic effusion and empyema. The panel's recommendations are based upon evidence derived from systematic literature reviews and adhere to a standardized methodology for rating the certainty of evidence and strength of recommendation according to the GRADE (Grading of Recommendations Assessment, Development, and Evaluation) approach.

2026 Clinical Practice Guideline Update By The Infectious Diseases Society Of America And The Pediatric Infectious Diseases Society On The Management Of Community-Acquired Pneumonia In Infants And Children Older Than 3 Months Of Age: The Use Of Pleural Fluid Drainage Compared To Surgical Debridement, Shawn D St Peter, Krow Ampofo, Thomas Brogan, Michael D Cabana, Claudia Espinosa, Todd A Florin, Jeffrey S Gerber, Michelle Gill, Debra L Palazzi, Mark Sawyer, Angela M Statile, Derek J Williams, Sheena Patel, Samir S Shah, Mark I Neuman

Faculty, Staff and Students Publications

This paper is part of a larger clinical practice guideline on the diagnosis and management of parapneumonic effusion and empyema in children, developed by the Infectious Diseases Society of America. In this paper, the panel provides recommendations on the choice of pleural fluid drainage by chest tube with fibrinolysis versus mechanical debridement. The panel's recommendations are based upon evidence derived from systematic literature reviews and adhere to a standardized methodology for rating the certainty of evidence and strength of recommendation according to the GRADE (Grading of Recommendations Assessment, Development, and Evaluation) approach.

Clinical Practice Guideline By The Infectious Diseases Society Of America And The Pediatric Infectious Diseases Society: 2026 Guideline Update On The Management Of Community-Acquired Pneumonia In Infants And Children Older Than 3 Months Of Age, Shawn D St Peter, Krow Ampofo, Thomas Brogan, Michael D Cabana, Claudia Espinosa, Todd A Florin, Jeffrey S Gerber, Michelle Gill, Debra L Palazzi, Mark Sawyer, Angela M Statile, Derek J Williams, Sheena Patel, Mark I Neuman, Samir S Shah

Faculty, Staff and Students Publications

As the first part of an update to the clinical practice guideline on the management of community-acquired pneumonia in infants and children older than 3 months of age, we present six updated recommendations. The updated recommendations span the characterization and management of pneumonia with parapneumonic effusion. The panel's recommendations are based on evidence derived from systematic literature reviews and adhere to a standardized methodology for rating the certainty of evidence and strength of recommendation according to the GRADE (Grading of Recommendations Assessment, Development, and Evaluation) approach.

Incidence Of Venous Thromboembolism In Women Receiving Transdermal Oestradiol/Testosterone With Micronised Progesterone In Accordance With Nice Guidelines, Amy Neville, Dan Reisel, John Benton, Mohit Khera, Louise Newson

Faculty, Staff and Students Publications

Background: Despite growing evidence supporting the long-term safety of hormone replacement therapy (HRT), concerns persist about certain adverse health outcomes, including venous thromboembolism (VTE). The aim of the study is to evaluate the risk of VTE in peri- and postmenopausal women treated with transdermal 17β-oestradiol with micronised progesterone or intrauterine progesterone (levonorgestrel) via the Mirena coil, in a real-world clinical setting.

Methods: The study included peri- and postmenopausal women attending a private menopause clinic between January 1, 2023, and December 31, 2023. Patients were actively undergoing treatment with transdermal oestradiol and progesterone in the form of micronised oral progesterone or …

Gut Microbiota Modulation Via Repeated Donor Fecal Transplantation Improves Motor And Gastrointestinal Symptoms In Drug-Naïve Parkinson’S Disease: A Randomized Phase 2 Trial, Rui Zhang, Renyi Feng, Jiuqi Wang, Yongkang Chen, Han Liu, Qingyong Zhu, Haiyan Tian, Chi Qin, Junfang Teng, Beisha Tang, Min Wu, Jinsheng Zeng, Erxi Wu, Xuebing Ding, Xuejing Wang

Faculty, Staff and Students Publications

The gut-brain axis is increasingly recognized as a critical contributor to Parkinson's disease (PD) pathogenesis, yet the therapeutic impact of microbiota modulation remains unclear due to lack of clinical trials in drug-naïve patients. We conducted a randomized, double-blind, placebo-controlled phase 2 trial to evaluate the safety, tolerability, and efficacy of repeated donor fecal microbiota transplantation (dFMT) in de novo PD. FMT was administered for seven days (200 mL on days 1-3; 50 mL on days 4-7) per 4-week cycle. Seventy-two patients were randomized 1:1 to receive dFMT or autologous FMT (aFMT), and 66 completed the trial. At 35 weeks, the …

Quality Of Life In Patients With P16+ Oropharyngeal Cancer Receiving Accelerated Radiation Therapy With Either Cisplatin Or Cetuximab In The Nrg/Rtog 1016 Randomized Controlled Trial, Jolie Ringash, Pedro A Torres-Saavedra, Maura L Gillison, Jimmy J Caudell, David J Adelstein, Paul M Harari, Erich M Sturgis, Ethan Basch, Shlomo A Koyfman, Greg A Krempl, Dukagjin M Blakaj, James Edward Bates, Thomas J Galloway, Christopher U Jones, Beth M Beadle, Jonathan Harris, Quynh-Thu Le, Sue S Yom, Benjamin Movsas

Faculty, Staff and Students Publications

Purpose: NRG/RTOG 1016 was a phase III randomized noninferiority de-escalation trial comparing cetuximab versus cisplatin, concurrent with accelerated radiation 70 Gy/6 weeks, in p16+ oropharyngeal cancer. Quality of life (QOL) was a secondary endpoint.

Methods and materials: Eligible/consenting patients among the first 400 entered completed the EORTC QLQ-C30/H&N35 at baseline, end of treatment, 3, 6, and 12 months posttreatment, to provide 90% power to detect an effect size of 0.5 in the between-arm change in QOL scores from baseline to 6 months. We report completion, responsiveness, and patterns over time across domains between arms, considering a difference of >10 points …

Hematopoietic Cell Transplantation For Wiskott-Aldrich Syndrome: A Pidtc Report, Jessie L Alexander, Blachy J Dávila Saldaña, Ruta Brazauskas, Sravya Gethika Dammalapati, Linda M Griffith, Ami J Shah, Kristin A Shimano, Hans D Ochs, Jack J Bleesing, Christen L Ebens, Malika Kapadia, Andrea Bauchat, Neena Kapoor, Joseph H Oved, Hesham Eissa, Hannah Lust, Michael D Keller, Hilary Haines, Shanmuganathan Chandrakasan, Julie-An Talano, Ahmad Rayes, Lisa Madden, Evan Shereck, Holly K Miller, Lisa Forbes Satter, Caridad Martinez, Jacob Rozmus, Jeffrey J Bednarski, Lolie C Yu, Deepakbabu Chellapandian, Victor M Aquino, Alan Knutsen, Hey Chong, Ashley Chopek, Alfred P Gillio, Avni Joshi, Hemalatha Rangarajan, Theodore B Moore, Jeffrey R Andolina, Kenneth B Desantes, Mark Vander Lugt, Susan E Prockop, David C Shyr, Kathleen E Sullivan, Suhag Parikh, Katja G Weinacht, Troy R Torgerson, Rebecca Marsh, Christopher C Dvorak, Alice Y Chan, Elie Haddad, Jennifer R Heimall, Michael A Pulsipher, Jennifer W Leiding, Donald B Kohn, Jennifer M Puck, Luigi D Notarangelo, David J Rawlings, Morton J Cowan, Aleksandra Petrovic, Sung-Yun Pai, Lauri M Burroughs

Faculty, Staff and Students Publications

Wiskott-Aldrich syndrome (WAS), an X-linked disorder characterized by immunodeficiency, thrombocytopenia, autoimmunity, and malignancy, can be effectively treated with allogeneic hematopoietic cell transplantation (HCT). Older age at HCT and mismatched donors are known to affect overall survival (OS). However, the influence of specific clinical manifestations or WAS variant class on OS and factors associated with event-free survival (EFS) remain incompletely defined. We analyzed outcomes of 308 patients with WAS who underwent HCT at 37 institutions of the Primary Immune Deficiency Treatment Consortium from 1990 to 2018. With a median follow-up of 5.3 years, the 5-year OS and EFS were 87.2% and …

Association Between Bmi Z-Score And Postoperative Complications In Pediatric Neuromuscular Scoliosis Surgery, Alexander K Mihas, Dion Birhiray, Chun Wai Hung, Lorenzo R Deveza, Dallas Vanorny, Frank Gerow, Darrell Hanson, Benny Dahl

Faculty, Staff and Students Publications

Study Design

Single-center retrospective review.

Objective

To investigate the association between BMI Z-scores and postoperative complications in pediatric patients with neuromuscular scoliosis undergoing spinal instrumentation.

Methods

Pediatric patients who underwent spinal instrumentation for neuromuscular scoliosis from July 2012 to June 2016 with a minimum 2-year follow-up were included. BMI and BMI Z-scores were calculated, and patients were stratified into CDC-defined BMI-for-age percentiles: underweight (< 5th percentile), normal (5th to < 85th percentile), overweight (85th to < 95th percentile), and obese (≥95th percentile). Logistic regression assessed associations of BMI, BMI Z-score, and BMI groups with postoperative complications.

Results

In total, 147 patients were included. Average follow-up was 3.2 ± 1.4 years. The most common diagnosis was cerebral palsy (39.5%). Complications occurred in 65 (44.2%) patients: 32 (21.8%) had a surgical site infection (SSI), …

Regulating Ai In Pediatrics-Is Transparency Enough?, R Brandon Hunter, Kolaleh Eskandanian, Chester J Koh

Faculty, Staff and Students Publications

No abstract provided.

Activin A Secretion By Muscle-Repairing Macrophages Induces Heterotopic Ossification In Mice, Wenqiang Yin, Kazuo Okamoto, Asuka Terashima, Warunee Pluemsakunthai, Takehito Ono, Taku Ito-Kureha, Shizuo Akira, Yoshinobu Hashizume, Roland Baron, Satoshi Ueha, Kouji Matsushima, Martin M Matzuk, Yuji Mishina, Hiroshi Takayanagi

Faculty, Staff and Students Publications

The immune system is not only essential for host defense, but it is also involved in tissue maintenance and disease pathogenesis. Macrophages play a key role in tissue repair, fibrosis, and tumorigenesis, but the mechanisms underlying their multifunctionality have not been fully explored. Here, we identified Mrep (Ly6ChiCX3CR1loPDPN+CD9+) as a crucial subset of macrophages for muscle regeneration after muscle injury. Muscle regeneration required Mrep-derived activin A, which was produced via the TLR4/TIR domain-containing adapter-inducing interferon-β/TANK-binding kinase 1/interferon regulatory factor 3/7 signaling pathway in response to muscle injury. Mrep exerted pathological effects by secreting activin A in a model of genetically …

Examining Sleep-Related Problems In Youth With Misophonia, Kevin M Wagner, Matti Cervin, Catherine E Rast, Mered Parnes, Nicholas Murphy, Samuel Spencer, Eric A Storch, Andrew G Guzick

Faculty, Staff and Students Publications

The relationship between misophonia and sleep-related problems (SRPs) in youth is underexplored. This paucity of research is concerning because SRPs might be elevated among youth with misophonia. If left untreated, SRPs can contribute to long-term health consequences. Thus, in this study we examined the link between misophonia and SRPs in youth aged 8 to 17. In this study, we compared SRPs in 102 children and adolescents with clinically significant misophonia to SRPs in a normative youth sample and a sample of 94 youth with anxiety disorders. We also examined the extent to which SRPs were associated with misophonia severity. Approximately …

Risk Factors For Acute Rejection In Pediatric Lung Transplantation And Its Impact On Graft Failure, Sophie Li, Spoorthi Kamepalli, David Moreno Mcneill, Selena Li, Abbas Rana

Faculty, Staff and Students Publications

Background: Although acute rejection is a common complication of lung transplantation, few studies have analyzed risk factors for acute rejection in pediatric lung transplant patients. The purpose of this study was to examine risk and protective factors for acute rejection in pediatric lung transplantation and its impact on graft failure.

Methods: A retrospective analysis was performed on lung transplant data for a cohort of 906 pediatric patients from the Organ Procurement and Transplantation Network (OPTN) Database between October 1, 1987, and June 30, 2023. Multivariable logistic regression was conducted to identify risk and protective factors for acute rejection, adjusting for …

Rural-Urban Differences In Use Of Mental Health Care For Serious Mental Illness At The Veterans Health Administration: A Retrospective Cohort Analysis, Alexandra B Caloudas, Amber B Amspoker, Ashley Smith, Liang Chen, Amy K Cuellar, Giselle Day, Alexandra Zingg, Julianna Hogan, Jan A Lindsay

Faculty, Staff and Students Publications

PURPOSE: Rural Veterans with serious mental illness (SMI) encounter significant barriers to accessing mental health care, related to SMI symptoms and rurality. A 2023 Government Accountability Office report found that rural Veterans used Veterans Health Administration (VHA) intensive mental health care for SMI less than urban Veterans relative to their population distributions. Our purpose was to deepen understanding of rural-urban differences in use of VHA mental health care among Veterans with SMI.

METHODS: Using VHA databases, we conducted a retrospective cohort analysis of 387,477 Veterans diagnosed with SMI, examining rurality as a predictor of intensive care for SMI and other …

Simulation-Free Spine Palliative Radiotherapy Enabled By Ai-Adapted Diagnostic Ct, Yiding Han, Alexander Nicola Hanania, Zaid Ali Siddiqui, Vincent Ugarte, Boran Zhou, Abdallah S R Mohamed, Piyush Pathak, Daniel Allen Hamstra, Baozhou Sun

Faculty, Staff and Students Publications

Background: Radiotherapy planning traditionally requires a dedicated simulation CT (sCT), which can introduce delays in initiating treatment. This is particularly impactful in spinal palliative care, where timely treatment is often important for symptom control and prevention of neurological deterioration. Although diagnostic CT (dCT) is frequently available earlier in the workflow, it can lead to geometric and dosimetric inaccuracies when used directly for treatment planning due to discrepancies in patient positioning, vertebral alignment, and table curvature.

Purpose: To develop and evaluate an AI-based method that transforms dCT into a simulation-equivalent planning CT (AI-pCT), enabling a clinically feasible simulation-free workflow for spinal …

Lower Gyrification Of The Ventromedial Prefrontal Cortex In Posttraumatic Stress Disorder: An Enigma-Ptsd Study., Ahmed Hussain, C Lexi Baird, Ashley A Huggins, Courtney C Russell, Delin Sun, Leonel Rangel-Jimenez, Chadi G Abdallah, Michael Angstadt, Geoffrey May, Hannah Berg, Jennifer U Blackford, Josh Cisler, Judith K Daniels, Nicholas D Davenport, Richard J Davidson, Maria Densmore, Seth G Disner, Wissam El-Hage, Amit Etkin, Negar Fani, Jessie L Frijling, Evan M Gordon, Daniel W Grupe, Ryan A Herringa, Julia I Herzog, David Hofmann, Anna R Hudson, Tanja Jovanovic, Anthony King, Saskia B J Koch, Ruth A Lanius, Amit Lazarov, Gen Li, Israel Liberzon, Shmuel Lissek, Guangming Lu, Antje Manthey, Adi Maron-Katz, Katie A Mclaughlin, Sven C Mueller, Laura Nawijn, Steven M Nelson, Yuval Neria, Richard W J Neufeld, Jack B Nitschke, Bunmi O Olatunji, Miranda Olff, Matthew Peverill, Rongfeng Qi, Yann Quidé, Orren Ravid, Ivan Rektor, Kerry J Ressler, Pavel Říha, Marisa Ross, Kelly Sambrook, Christian Schmahl, Anika Sierk, Scott R Sponheim, Jennifer S Stevens, Thomas Straube, Benjamin Suarez-Jimenez, Jean Théberge, Nic J A Van Der Wee, Steven J A Van Der Werff, Sanne J H Van Rooij, Mirjam Van Zuiden, Dick J Veltman, Robert R J M Vermeiren, Henrik Walter, Li Wang, Xi Zhu, Ye Zhu, Sigal Zilcha-Mano, Neda Jahanshad, Paul M Thompson, Rajendra A Morey

Faculty, Staff and Students Publications

Background: Cortical gyrification involves the formation of folds in the cerebral cortex, coinciding with key neurodevelopmental processes. Its strong correlation with increased cortical surface area and decreased cortical thickness may improve cortical signaling efficiency by decreasing cortico-cortical distance. Differences in brain structure have been found in posttraumatic stress disorder (PTSD), yet few small studies have examined cortical gyrification.

Methods: Gyrification was quantified using FreeSurfer's Local Gyrification Index (lGI), derived from 3-dimensional T1-weighted volumetric brain magnetic resonance imaging in 1876 participants (PTSD n = 789, control n = 1087) across 24 sites from the ENIGMA-PGC (Enhancing Neuro Imaging Genetics through Meta-Analysis …

How Does Frailty Impact Peri-Operative And Speech Recognition Outcomes For Cochlear Implants In Veterans?, Kaitlyn A Brooks, Benjamin D Lovin, Alex D Sweeney, Angela S Peng, Nathan R Lindquist

Faculty, Staff and Students Publications

Objective: To investigate the association of frailty with post-cochlear implant (CI) admission, morbidity, and CI-aided word recognition outcomes in veterans.

Study design: Retrospective cohort.

Setting: Single-institution tertiary care Veterans Health Administration (VHA) hospital.

Methods: Veterans who underwent cochlear implantation between 1998 to 2024 were included. The Modified 5-Item Frailty Index (mFI-5) score was used to characterize preoperative frailty. Outcomes of interest were admission from post-anesthesia care unit (PACU), relative risk (RR) of admission among frail patients, and relationship between frailty and post-CI word recognition score (WRS). Ordinal data were analyzed via logistic regression and Chi square tests; a multivariate linear …

Lean On Me: Social Factors And Diabetes Outcomes Among Young Adults With Type 1 Diabetes Prior To Transitioning To Adult Care, Caroline M Roberts, Samantha A Carreon, Charles G Minard, Sarah K Lyons, Randi Streisand, Barbara J Anderson, Tricia S Tang, Siripoom Mckay, Katherine Gallagher, Sridevi Devaraj, Marisa E Hilliard

Faculty, Staff and Students Publications

Aims: Little is known about how different aspects of social relationships relate to diabetes self-management and glycaemic outcomes in young adults with type 1 diabetes (T1D). We examined relationships of general and diabetes-specific social variables with diabetes self-management and HbA1c. We hypothesized diabetes-specific factors would more strongly associate with self-management and HbA1c than general social factors.

Methods: We analysed baseline data from 100 young adults with T1D (Mage = 19.9 ± 1.3 years) enrolled in a randomized controlled trial of a paediatric to adult care transition intervention. We examined associations of self-reported general and diabetes-specific social factors with self-reported self-management …

Concurrent Vulvar Vitiligo And Lichen Sclerosus: The Importance Of Melanocyte-Specific Immunostains In Resolving Diagnostic Ambiguity, Nabor S Mireles, Kaitlyn Levett, Annie Dai, Abdul Hafeez Diwan, Omid Jalali

Faculty, Staff and Students Publications

Vitiligo and lichen sclerosus (LS) are autoimmune dermatoses that may share overlapping clinical and histopathologic features, posing diagnostic challenges when they occur concurrently. We report a biopsy-confirmed case of vulvar vitiligo and LS in a 61-year-old woman presenting with pruritic, depigmented patches on the labia majora and minora. Histopathologic examination showed epidermal thinning with papillary dermal hyalinization and a dense, band-like lymphocytic infiltrate consistent with LS. Melan-A immunostaining demonstrated a complete absence of epidermal melanocytes, confirming concurrent vitiligo rather than LS-related post-inflammatory hypopigmentation. This case underscores the importance of correlating subtle clinical clues with melanocyte-specific immunostains to distinguish coexistent vitiligo …

Cardiac Catheterization In American Indian/Alaska Native Populations: Empowering Communities, Providers, And Systems Toward Equity, Joseph Burns, Daniel J Penny, Keila N Lopez, Lindsay F Eilers, Athar M Qureshi, Christine J Chung, Jason F Deen

Faculty, Staff and Students Publications

American Indian/Alaska Native (AI/AN) people face numerous health disparities, including alarming rates of cardiovascular morbidity and mortality. These inequities are closely tied to unique social, political, and economic circumstances due to the ongoing consequences of systemic racism. Although poorly described, these cardiovascular inequities extend to adult and pediatric interventional cardiology. AI/AN adults are less likely to undergo cardiac catheterization and are more likely to experience complications. There are no published studies on pediatric interventional cardiology outcomes in AI/AN children. The scarcity of available data underscores the need for novel studies to investigate the epidemiology, sociodemographic associations, and outcomes of AI/AN …

Hes V2.0 Validation And Performance Compared To Galad And Asap In The Heds Cohort, Hashem B El-Serag, Camden Lopez, Michelle Luster, K Rajender Reddy, Neehar Parikh, Amit G Singal, Jorge A Marrero, Aaron P Thrift, Jagpreet Chhatwal, Ziding Feng, Stephanie Page-Lester, Qingchun Jin, Nabihah Tayob, Fasiha Kanwal

Faculty, Staff and Students Publications

Background & aims: We previously developed HES V2.0, a biomarker panel (age, ALT, platelets, etiology, AFP, AFP-L3, DCP, and their 1-year gradients) for early detection of hepatocellular carcinoma (HCC) among patients with cirrhosis. We externally validated HES V2.0 and compared its performance with HES V1.0, GALAD, and ASAP.

Methods: We conducted a PRoBE (prospective specimen-collection, retrospective blinded-evaluation) cohort study in the HEDS (Hepatocellular Carcinoma Early Detection Strategy) cohort of 1,485 patients with cirrhosis, of whom 119 developed HCC. Patient- and test-level true positive rates (TPR) for HCC were calculated at 6, 12, and 24 months before diagnosis, using thresholds at …

Health Care Worker-Reported Barriers And Potential Facilitators Of Acute Lower Respiratory Infection Care Deliver For Children At Mchinji District Hospital In Malawi, Shubhada Hooli, Beatwil Zadutsa, Everlisto Phiri, Karina Hofstee, Nichole Davis, Eric D Mccollum, Charles Makwenda, Carina King

Faculty, Staff and Students Publications

Objectives: We sought to understand barriers to high-quality hospital-based child acute lower respiratory infection (ALRI) care at Mchinji District Hospital in Malawi.

Methods: In 2020, we conducted focus group discussions (FGDs) with clinical officers (COs) who provided direct clinical care after a half-day refresher course on pediatric ALRI case management. The underpinning research methodology of the FGDs was phenomenology, and they were analyzed using inductive and deductive thematic analysis.

Results: We recruited 16 COs to participate in three FGDs. Five themes emerged: lack of confidence in ALRI diagnosis and management, high clinical burden with understaffing, dysfunctional team dynamics, limited physical …

Defining The Society Of Pediatric Liver Transplantation (Split) Research Agenda: A Delphi Consensus Project, Sharad I Wadhwani, Pooja Reddy Spector, Amy G Feldman, Katryn N Furuya, Kayla Hartjes, Chaowapong Jarasvaraparn, Beau Kelly, Stacee M Lerret, Krupa Mysore, Arti Pawaria, Philip Rosenthal, Amy Shui, Voytek Slowik, Sarah Taylor, Jennifer Vittorio, Pamela L Valentino

Faculty, Staff and Students Publications

Background: Despite major advances in surgical and medical management, only one-third of pediatric liver transplant (LT) recipients remain morbidity-free at 10 years. The Society of Pediatric Liver Transplantation (SPLIT) published a research agenda over 10 years ago, and much progress has been made since then. An updated consensus-driven SPLIT research agenda could guide future research and funding priorities. We aimed to identify and prioritize key research needs across the continuum of pediatric LT care using a modified Delphi consensus approach.

Methods: We conducted a three-round modified Delphi process among SPLIT members. In round 1, participants submitted open-ended research priorities. In …

Adenovirus Disease Following Pediatric Liver Transplantation: 10-Year Experience From A Large Pediatric Transplant Program, Anna M Banc-Husu, Sara Hassan, N Thao N Galvan, Elizabeth A Moulton, Jeremy M Schraw, Kathleen Hosek, Dana Cerminara, Flor M Munoz, Krupa Mysore

Faculty, Staff and Students Publications

Background: Human adenovirus in immunocompromised patients can be life-threatening. We describe the prevalence, clinical presentation, and treatment of adenovirus after pediatric liver transplantation at a large transplant center.

Methods: We performed a retrospective cohort study of adenovirus infection in children from 2013 to 2022. We compared incidence, clinical characteristics, and outcomes of post-transplant children by adenovirus treatment status.

Results: Adenovirus disease developed in 26% (84/320) children after liver transplant. Median age at liver transplant was 17.5 months, 48% were female; 50% had biliary atresia. Fever (53%), gastrointestinal symptoms (48%), and hepatitis (41%) were the most common clinical presentations at diagnosis. …

Relationship Between Heart Rate Variability And Sleep Among Girls And Boys With Abdominal Pain-Related Disorders Of Gut-Brain Interaction, Kendra J Kamp, Robert L Burr, Camden E Matherne, Emily Simonds, Tasha Murphy, Margaret M Heitkemper, Rona L Levy, Robert J Shulman, Miranda A L Van Tilburg

Faculty, Staff and Students Publications

Background: Children with abdominal pain-related disorders of gut-brain interaction (AP-DGBI) often experience sleep disturbances which may be linked to the autonomic nervous system. Heart rate variability (HRV) is a non-invasive measure of autonomic nervous system activity. Our aim was to examine if HRV was associated with characteristics of disturbed sleep in children with AP-DGBI in order to elucidate the underlying physiological mechanisms of disturbed sleep.

Methods: This analysis used baseline data from a randomized control trial of 156 children ages 7-12 years with AP-DGBI. HRV was measured after dinner using a Polar monitor. Sleep disturbances were evaluated using the Children's …

Pharmacodynamics, Efficacy, And Safety Of Intraputaminal Eladocagene Exuparvovec Administered To Pediatric Patients With Aromatic L-Amino Acid Decarboxylase Deficiency Using An Mr-Compatible Cannula: 48 Weeks Of Follow-Up, Daniel J Curry, Phillip L Pearl, Scellig S D Stone, Donald L Gilbert, Sudhakar Vadivelu, Bruria Ben-Zeev, Matthew Vestal, Muhammad Zafar, Chun-Hwei Tai, Sheng-Che Chou, Zion Zibly, Lior Ungar, Mered Parnes, Mariam Hull, Lisa Emrick, Christian Werner, Alexis Krolick, Vinay Penematsa, Antonia Wang, Rezwanur Rahman, Lee Golden, Yin-Hsiu Chien, Paul Wuh-Liang Hwu

Faculty, Staff and Students Publications

Aromatic ʟ‐amino acid decarboxylase (AADC) deficiency is a rare pediatric neurotransmitter disorder that typically necessitates lifelong care, and that carries a risk of childhood mortality. Eladocagene exuparvovec gene therapy is designed to restore AADC production. Study GT‐002 (NCT04903288) is a phase 2, multicenter, open‐label trial assessing the pharmacodynamics, safety, and efficacy of eladocagene exuparvovec administered to the putamen bilaterally in pediatric patients with AADC deficiency using a magnetic resonance (MR)‐compatible cannula. Patients received eladocagene exuparvovec at 1.8 × 1011 vector genomes via the SmartFlow MR‐compatible cannula in a single operative session. Endpoints include the change from baseline in …