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Promises, Pitfalls, And Paths Forward For The Pediatric Research Equity Act., Alexa L. Pagano, Danielle J. Green, Jennifer Goldman, Anjali Deshmukh May 2026

Promises, Pitfalls, And Paths Forward For The Pediatric Research Equity Act., Alexa L. Pagano, Danielle J. Green, Jennifer Goldman, Anjali Deshmukh

Manuscripts, Articles, Book Chapters and Other Papers

IMPORTANCE: Since its enactment in 2003, the Pediatric Research Equity Act (PREA) has significantly increased the number of pediatric drug studies performed and expanded pediatric drug labeling. Despite these advancements, many drugs used in children still lack pediatric-specific US Food and Drug Administration (FDA) labeling, even when pediatric studies are required by law. Recent legislative changes strengthened the FDA's enforcement authority over PREA. As these changes are implemented, persistent gaps in pediatric drug development warrant examination. Addressing these gaps may help ensure that children are systematically included in clinical research and that medications used in children are supported by rigorous …


Defining The Qualities Of A Surgical Resident Who "Gets It": A Qualitative Analysis Of Faculty And Resident Perceptions, Shaleen V Sathe, Ravi R Agrawal, Jennifer Yu, Paul E Wise, Michael Awad, Kevin Y Pei May 2026

Defining The Qualities Of A Surgical Resident Who "Gets It": A Qualitative Analysis Of Faculty And Resident Perceptions, Shaleen V Sathe, Ravi R Agrawal, Jennifer Yu, Paul E Wise, Michael Awad, Kevin Y Pei

2020-Current year OA Pubs

BACKGROUND: The phrase "getting it" is frequently used in surgical trainee assessment, yet its meaning remains undefined. This study explores how residents and faculty define the concept and whether perceptions align.

METHODS: Semi-structured interviews were conducted with 11 residents and 13 faculty at a single institution and analyzed using reflexive thematic analysis.

RESULTS: Residents described "getting it" as clinical competence, social awareness, feedback responsiveness, and team integration, while noting challenges with interpreting implicit expectations and the influence of faculty bias. Faculty described "getting it" as a gestalt recognition of initiative, insight, and sound clinical judgment. Both agreed that the concept …


Computational Fluid Dynamics-Based Risk Stratification Of Modified Blalock-Taussig-Thomas Shunt Thrombogenicity, Yi Qiao, Ethan Penn, Jacob Miller, Scott Bugenhagen, Ram Rohatgi, Kelsey Mercer, Blaire Kulp, Jinli Wang, Pirooz Eghtesady, Guy M Genin, Edon J Rabinowitz, David Bark May 2026

Computational Fluid Dynamics-Based Risk Stratification Of Modified Blalock-Taussig-Thomas Shunt Thrombogenicity, Yi Qiao, Ethan Penn, Jacob Miller, Scott Bugenhagen, Ram Rohatgi, Kelsey Mercer, Blaire Kulp, Jinli Wang, Pirooz Eghtesady, Guy M Genin, Edon J Rabinowitz, David Bark

2020-Current year OA Pubs

BACKGROUND: The modified Blalock-Taussig-Thomas shunt (mBTTS) is a critical palliative procedure for infants with single-ventricle physiology, but thrombosis-related occlusion affects 8% to 12% of infants and carries nearly 50% mortality. Meanwhile, existing antithrombotic strategies fail to address the hemodynamic factors driving thrombosis, highlighting the need for a deeper understanding of flow dynamics in shunt failure.

OBJECTIVES: This study aims to identify how mBTTS geometry influences hemodynamics and thrombosis risk, providing quantitative guidance for surgical planning and shunt design optimization.

METHODS: We used patient-specific imaging data to test 54 idealized mBTTS configurations, systematically varying key geometric factors: pulmonary artery diameter, shunt …


Best Practice: Ct-Guided Adaptive Radiotherapy Reference Planning On The Ethos Platform, Xenia Ray, Joseph Harms, Alex Price, Mu-Han Lin, Dennis N Stanley May 2026

Best Practice: Ct-Guided Adaptive Radiotherapy Reference Planning On The Ethos Platform, Xenia Ray, Joseph Harms, Alex Price, Mu-Han Lin, Dennis N Stanley

2020-Current year OA Pubs

Online adaptive radiotherapy (ART) enables treatment plan modifications based on the patient's anatomy at each fraction, addressing limitations of conventional radiotherapy that relies on a static pre-treatment plan. CT-guided ART (CTgART), as implemented on the Varian Ethos platform, has gained increased clinical adoption over the past several years; however, its success is highly dependent on reference plan design due to limited on-table planning flexibility. As a result, clinicians creating and reviewing reference plans will greatly benefit from both understanding how upstream planning decisions affect the downstream adaptive process and proactively incorporating strategies that improve optimization robustness and efficiency on-treatment. We …


Adapt-On-Demand: Enabling Flexible And Scalable Adaptive Radiotherapy Through Workflow Automation, Sean Domal, Min Geon Choi, Justin Visak, Ruiqi Li, Taoran Li, Boon-Keng Kevin Teo, Xiaodong Zhao, Matthew Schmidt, Eric Laugeman, David Parsons, Yang Kyun Park, Shahed Badiyan, David Sher, Mu-Han Lin May 2026

Adapt-On-Demand: Enabling Flexible And Scalable Adaptive Radiotherapy Through Workflow Automation, Sean Domal, Min Geon Choi, Justin Visak, Ruiqi Li, Taoran Li, Boon-Keng Kevin Teo, Xiaodong Zhao, Matthew Schmidt, Eric Laugeman, David Parsons, Yang Kyun Park, Shahed Badiyan, David Sher, Mu-Han Lin

2020-Current year OA Pubs

PURPOSE: CT-guided adaptive radiotherapy (CTgART) offers significant clinical benefits, but its utilization is often limited by resource demands and rigid workflows. This study presents a flexible and scalable "Adapt-on-Demand" (AOD) framework that integrates the adaptive capabilities of the Varian Ethos platform with the high-throughput IGRT delivery of the Varian Halcyon, supported by automated scripting.

METHODS: We developed an end-to-end dual-platform workflow that dynamically transitions between ART and IGRT delivery based on patient needs. Key components include Eclipse Scripting API (ESAPI) automation to streamline adapted plan ingestion from the Ethos Treatment Planning and Management System (TPMS) export directory, compatibility conversion for …


Utility Of Routine Histopathologic And Culture Analysis In Wrist Tenosynovectomy, Andrew Straszewski, Stockton Troyer, Martin I Boyer, Charles A Goldfarb May 2026

Utility Of Routine Histopathologic And Culture Analysis In Wrist Tenosynovectomy, Andrew Straszewski, Stockton Troyer, Martin I Boyer, Charles A Goldfarb

2020-Current year OA Pubs

PURPOSE: Routine practices without a clear impact on patient care merit continuous reassessment. This retrospective study evaluated the value of routine histopathologic and culture analysis in patients who underwent flexor or extensor tendon tenosynovectomy.

METHODS: Current Procedural Terminology codes for isolated tenosynovectomy procedures were queried from an institutional database from 2018 to 2023, including all patients aged ≥18 treated by one of seven fellowship-trained hand surgeons. Demographics, past medical and surgical history, clinic notes, operative notes, imaging, pathology, and culture data were reviewed. We assessed for changes to the treatment course of each patient based on the pathology and culture …


Sympathetic And Parasympathetic Ganglion Blocks For Treatment Of Long Covid-19 Symptoms: A Scoping Review, Abhay Tyagi, Khushboo Singh, Preet Mohinder Singh, Frederic J Gerges May 2026

Sympathetic And Parasympathetic Ganglion Blocks For Treatment Of Long Covid-19 Symptoms: A Scoping Review, Abhay Tyagi, Khushboo Singh, Preet Mohinder Singh, Frederic J Gerges

2020-Current year OA Pubs

BACKGROUND: Long COVID-19 affects approximately 10-30% of individuals who are infected with SARS-CoV-2 and is associated with persistent functional impairment and reduced quality of life. The heterogeneous, multisystemic nature and nonspecific symptomatology of long COVID-19 makes its treatment challenging. This scoping review evaluates existing evidence on sympathetic and parasympathetic ganglion blocks as potential interventions for patients suffering from long COVID-19.

OBJECTIVE: Our primary objective was to assess the effectiveness of sympathetic and parasympathetic ganglion blocks in treating patients with long COVID-19 by identifying the most commonly reported symptoms, symptom response to different block regimens, and any adverse effects associated with …


Treatment Of Haemophilia A Without Inhibitors: Real-World Treatment Patterns And Clinical Outcomes In The Us., Allison P. Wheeler, Lauren Amos Md, Shveta Gupta, Sylvie Bozzi, Gandarvaka Miles, Kalyani Hawaldar, Andrew Wilson, Gillian Hanson, Emily Cibelli, Amanda Wilson, Siddhi Umarje, Anne-Laure Tardy, Jennifer Dumont, Graham Neill, Alix Arnaud May 2026

Treatment Of Haemophilia A Without Inhibitors: Real-World Treatment Patterns And Clinical Outcomes In The Us., Allison P. Wheeler, Lauren Amos Md, Shveta Gupta, Sylvie Bozzi, Gandarvaka Miles, Kalyani Hawaldar, Andrew Wilson, Gillian Hanson, Emily Cibelli, Amanda Wilson, Siddhi Umarje, Anne-Laure Tardy, Jennifer Dumont, Graham Neill, Alix Arnaud

Manuscripts, Articles, Book Chapters and Other Papers

INTRODUCTION: Despite available prophylactic therapies for haemophilia A, breakthrough bleeding and consequent pain and joint damage still occur.

AIM: To provide insights into the unmet treatment needs of people with moderate-to-severe haemophilia A in the US.

METHODS: For this descriptive, observational cohort study, eligible participants diagnosed with moderate-to-severe haemophilia A were enrolled in the PicnicHealth database on or before 3 October 2022. Three patient groups were established to identify: (1) treatment patterns; (2) clinical outcomes by therapy class (standard half-life [SHL] factor VIII [FVIII]; extended half-life FVIII [EHL]; emicizumab); (3) health-related quality of life (HRQoL) using the Patient-Reported Outcomes Measuring …


Racial, Ethnic, And Neighborhood Disparities In Acute Kidney Injury In Pediatric Acute Leukemia, Wendy C Bravo, Yimei Li, Tamara P Miller, Kelly Getz, Lusha Cao, Edward Krause, Mark Ramos, Judy Lee, Maria Monica Gramatges, Karen R Rabin, Michael Scheurer, Richard Aplenc, Michelle Denburg May 2026

Racial, Ethnic, And Neighborhood Disparities In Acute Kidney Injury In Pediatric Acute Leukemia, Wendy C Bravo, Yimei Li, Tamara P Miller, Kelly Getz, Lusha Cao, Edward Krause, Mark Ramos, Judy Lee, Maria Monica Gramatges, Karen R Rabin, Michael Scheurer, Richard Aplenc, Michelle Denburg

Faculty, Staff and Students Publications

Introduction: We sought to identify racial/ethnic and neighborhood disparities in acute kidney injury (AKI) in children with acute lymphoblastic or myeloid leukemia (ALL, AML).

Methods: Electronic health record data were retrospectively collected from patients treated for de novo pediatric ALL or AML in the multicenter Leukemia Electronic Abstraction of Records Network. AKI was defined by modified Kidney Disease Improving Global Outcomes criteria. Neighborhood-level resources were defined by the Child Opportunity Level (COL). Prevalence ratios (PRs) for AKI at diagnosis by race/ethnicity and COL were calculated using modified Poisson regression. Cox regression was used to calculate hazard ratios (HRs) of first …


Interplay Between Genetic Ancestry, Self-Reported Race And Ethnicity, And Clinical Factors In Pediatric Acute Lymphoblastic Leukemia: A Redial Consortium Report, Pragati Kore, Jennifer M Geris, Maria Leon-Camarena, Maria I Castellanos, Olga A Taylor, Veronica Garcia-Morales, Austin L Brown, Pagna Sok, Jeremy M Schraw, Van Huynh, Kathleen F Ludwig, Laura J Klesse, Sandi L Pruitt, Amy E Hughes, Kevin Tien, Kenneth M Heym, Timothy Griffin, Rodrigo Erana, Juan Carlos Bernini, Maria M Gramatges, Elizabeth G Atkinson, Philip J Lupo, Michael E Scheurer, Karen R Rabin May 2026

Interplay Between Genetic Ancestry, Self-Reported Race And Ethnicity, And Clinical Factors In Pediatric Acute Lymphoblastic Leukemia: A Redial Consortium Report, Pragati Kore, Jennifer M Geris, Maria Leon-Camarena, Maria I Castellanos, Olga A Taylor, Veronica Garcia-Morales, Austin L Brown, Pagna Sok, Jeremy M Schraw, Van Huynh, Kathleen F Ludwig, Laura J Klesse, Sandi L Pruitt, Amy E Hughes, Kevin Tien, Kenneth M Heym, Timothy Griffin, Rodrigo Erana, Juan Carlos Bernini, Maria M Gramatges, Elizabeth G Atkinson, Philip J Lupo, Michael E Scheurer, Karen R Rabin

Faculty, Staff and Students Publications

Background: Pediatric acute lymphoblastic leukemia (ALL) is the most common cancer in children, and its incidence and outcomes vary by race and ethnicity.

Methods: The Reducing Ethnic Disparities in Acute Leukemia (REDIAL) Consortium has assembled a cohort of patients < 25 years at diagnosis to examine genetic and neighborhood-level factors influencing ALL. As the cohort is ongoing, for this report, we examined sociodemographic and cytogenetic factors that might be associated with ALL outcome disparities.

Results: Among 2,512 patients diagnosed between 2005 and 2020, we observed distinct patterns of genetic ancestry and residential characteristics across self-reported race and ethnicity. Latino children, who comprised 55.3% of the cohort, had a mean Amerindigenous ancestry proportion of 50.9%, whereas non-Latino White children exhibited a predominantly European ancestry (mean = 78%). Neighborhood-level analysis revealed significant socioeconomic and geographic differences by …


2-Aminobutyrate Mediates The Impact Of Air Pollution On Blood Biomarkers Of Alzheimer’S Disease, Anum Saeed, Chris Mckennan, Jane E Clougherty, Peidi Zhou, Sheila Tripathy, Ellen J Kinnee, Jiaxuan Duan, Kevin Kip, Xuemei Zeng, Victor Villemagne, Tharick Pascoal, Mark Mapstone, Eric A Whitsel, Oscar I Lopez, Christie Ballantyne, Thomas K Karikari, Bing Yu, Steven Reis, Annie Cohen May 2026

2-Aminobutyrate Mediates The Impact Of Air Pollution On Blood Biomarkers Of Alzheimer’S Disease, Anum Saeed, Chris Mckennan, Jane E Clougherty, Peidi Zhou, Sheila Tripathy, Ellen J Kinnee, Jiaxuan Duan, Kevin Kip, Xuemei Zeng, Victor Villemagne, Tharick Pascoal, Mark Mapstone, Eric A Whitsel, Oscar I Lopez, Christie Ballantyne, Thomas K Karikari, Bing Yu, Steven Reis, Annie Cohen

Faculty, Staff and Students Publications

Introduction: We examined the impact of mid-life metabolites on late-life Alzheimer's disease (AD) plasma biomarkers and whether these metabolites mediate air pollution effects.

Methods: In the Heart Strategies Concentrating on Risk Evaluation (Heart SCORE) cohort, we applied high-dimensional regression and meditation models, adjusting for observed and latent confounders, with replication in the Atherosclerosis Risk in Communities (ARIC) study. Exposures included mid-life fine particulate matter (PM2.5), black carbon, metals, and metabolites. Outcomes were: late-life plasma p-tau(181, 217, and 231) and amyloid beta 42/40 (Simoa assays).

Results: In the Heart SCORE study, seven lipid- and amnio acid-pathway metabolites measured in mid-life were …


Risk Factors For The Development Of Food Allergy In Infants And Children: A Systematic Review And Meta-Analysis, Nazmul Islam, Alexandro W L Chu, Falana Sheriff, Farid Foroutan, Gordon H Guyatt, Romina Brignardello-Petersen, Paul Oykhman, Alfonso Iorio, Ariel Izcovich, Katherine M Morrison, Yetiani Roldan Benitez, Rachel J Couban, Dorota Borovsky, Yiming Zhang, Leonardo Ologundudu, Keerthana Pasumarthi, Syed Fahad Farooq, Kyle Tong, Wang-Choi Tang, Haseeb Faisal, Muhammad Faran Khalid, Mohammad Saad Asif, Shannon French, Susan Waserman, R Sharon Chinthrajah, Hugh A Sampson, S Shahzad Mustafa, Jay A Lieberman, Kirsi M Järvinen, Sally Bailey, Philippe Bégin, Scott H Sicherer, Jennifer Gerdts, Melanie Carver, Lynda Mitchell, Kelly Cleary, Matthew J Greenhawt, Julie Wang, Aikaterini Anagnostou, Marcus S Shaker, Anita Chandra-Puri, Patricia C Fulkerson, Robert A Wood, Derek K Chu May 2026

Risk Factors For The Development Of Food Allergy In Infants And Children: A Systematic Review And Meta-Analysis, Nazmul Islam, Alexandro W L Chu, Falana Sheriff, Farid Foroutan, Gordon H Guyatt, Romina Brignardello-Petersen, Paul Oykhman, Alfonso Iorio, Ariel Izcovich, Katherine M Morrison, Yetiani Roldan Benitez, Rachel J Couban, Dorota Borovsky, Yiming Zhang, Leonardo Ologundudu, Keerthana Pasumarthi, Syed Fahad Farooq, Kyle Tong, Wang-Choi Tang, Haseeb Faisal, Muhammad Faran Khalid, Mohammad Saad Asif, Shannon French, Susan Waserman, R Sharon Chinthrajah, Hugh A Sampson, S Shahzad Mustafa, Jay A Lieberman, Kirsi M Järvinen, Sally Bailey, Philippe Bégin, Scott H Sicherer, Jennifer Gerdts, Melanie Carver, Lynda Mitchell, Kelly Cleary, Matthew J Greenhawt, Julie Wang, Aikaterini Anagnostou, Marcus S Shaker, Anita Chandra-Puri, Patricia C Fulkerson, Robert A Wood, Derek K Chu

Faculty, Staff and Students Publications

Importance: The incidence and risk (predictive) factors for early life food allergy development remain uncertain.

Objective: To estimate the incidence and quantify risk factors for food allergy development.

Data sources: MEDLINE and Embase were systematically searched to January 1, 2025. Data were analyzed from June 1, 2025, to November 25, 2025.

Study selection: Incidence estimates included studies confirming food allergy via food challenge. Risk factor analyses included cohort, case-control, and cross-sectional studies in any language assessing children younger than 6 years using multivariable analyses.

Data extraction and synthesis: Paired reviewers independently extracted data. Random-effects meta-analyses pooled incidence and adjusted odds …


Plasma Proteins Associated With Psychosocial Factors And Heart Disease: The Jackson Heart Study, Sara N O'Brien, Madeline G Gillman, Michael D Green, Daniel E Cruz, James S Floyd, Susan Hankinson, Daniel H Katz, Xiaojuan Liu, Michelle C Odden, Bruce M Psaty, Alexander P Reiner, Jerome I Rotter, Stephen S Rich, Robert E Gerszten, Amil Shah, Mario Sims, Usman A Tahir, Lesley F Tinker, Alexis C Wood, Bing Yu, Anthony S Zannas, Laura M Raffield, Láshauntá M Glover May 2026

Plasma Proteins Associated With Psychosocial Factors And Heart Disease: The Jackson Heart Study, Sara N O'Brien, Madeline G Gillman, Michael D Green, Daniel E Cruz, James S Floyd, Susan Hankinson, Daniel H Katz, Xiaojuan Liu, Michelle C Odden, Bruce M Psaty, Alexander P Reiner, Jerome I Rotter, Stephen S Rich, Robert E Gerszten, Amil Shah, Mario Sims, Usman A Tahir, Lesley F Tinker, Alexis C Wood, Bing Yu, Anthony S Zannas, Laura M Raffield, Láshauntá M Glover

Faculty, Staff and Students Publications

Background: Knowledge of proteomic mechanisms explaining the link between psychosocial stress and cardiovascular disease is limited. This study aimed to (1) identify plasma proteins associated with psychosocial factors and (2) assess associational pathways between psychosocial factors, identified proteins, and incident cardiovascular disease events in a discovery cohort, JHS (Jackson Heart Study), and 2 replication cohorts, the CHS (Cardiovascular Health Study), and the MESA (Multi-Ethnic Study of Atherosclerosis).

Methods: JHS participants from exam 1 (2000-2004) with SomaScan 1.3k platform proteomics data were included (n=2143, mean age=55.3). Depressive symptoms and perceived stress scores were measured via the 20-item Center for Epidemiological Studies …


Stance And Gait Analysis Of Apollo Astronauts On The Moon, Faye Y Chiou-Tan, Conrad Li, Jason P Caffrey, Millard Reschke, Timothy R Macaulay May 2026

Stance And Gait Analysis Of Apollo Astronauts On The Moon, Faye Y Chiou-Tan, Conrad Li, Jason P Caffrey, Millard Reschke, Timothy R Macaulay

Faculty, Staff and Students Publications

Introduction/aim: As humans return to the Moon, safe efficient ambulation and fall prevention on the lunar surface are key concerns. The analysis of gait from archived Apollo mission video footage is now possible with pose estimation video analysis software. Thus, this study aims to retrospectively quantify parameters of Apollo astronauts' stance and gait on the Moon.

Methods: Publicly available National Aeronautics and Space Administration (NASA) footage of astronauts ambulating on the Lunar surface was examined using body pose analysis software. Goniometric lower limb joint/torso angles and width of base of stance and gait were derived. Gait cycle stance phase, swing …


Optical Coherence Tomography As An Emerging Technique For Identifying Pediatric Middle Ear Effusion, Roger Neuberger, Jeffrey Carter, Adriana Dives, Ana Sofia C Ibarrola, Maritsa Ixba, Elton Lambert, Yi-Chun Liu, Anna H Messner May 2026

Optical Coherence Tomography As An Emerging Technique For Identifying Pediatric Middle Ear Effusion, Roger Neuberger, Jeffrey Carter, Adriana Dives, Ana Sofia C Ibarrola, Maritsa Ixba, Elton Lambert, Yi-Chun Liu, Anna H Messner

Faculty, Staff and Students Publications

Objective: Acute otitis media (AOM) and otitis media with effusion (OME) rely on accurate characterization of middle ear effusion (MEE). Optical coherence tomography (OCT) is a potential modality for noninvasive analysis of MEE, thereby assisting with otitis media diagnosis. This study aims to compare the diagnostic performance of OCT with otoscopy and tympanometry in pediatric patients.

Methods: A prospective convenience sample of 164 pediatric patients aged 6 months to 12 years was recruited from two tertiary care pediatric otolaryngology clinics. During their visit, each child underwent otoscopy, tympanometry, and OCT evaluation. Scans were reviewed by both a fellowship-trained pediatric otolaryngologist …


Exploring The Alignment Between Medical School Outcomes With Residency Competencies: A Modified Delphi Study, Sally A. Santen, Luan Lawson, Jose A. Negrete Manriquez, Marjorie Westerveld, Laurah Turner, Anita Wilson, Phd, Yoon Soo Park, Eric S. Holmboe, Michael S. Ryan, Medical School Outcomes Milestone Study Group May 2026

Exploring The Alignment Between Medical School Outcomes With Residency Competencies: A Modified Delphi Study, Sally A. Santen, Luan Lawson, Jose A. Negrete Manriquez, Marjorie Westerveld, Laurah Turner, Anita Wilson, Phd, Yoon Soo Park, Eric S. Holmboe, Michael S. Ryan, Medical School Outcomes Milestone Study Group

JeffMD Academic Affairs

PURPOSE: Facilitation of competency-based medical education adoption would benefit from alignment between medical school (undergraduate medical education [UME]) and residency (graduate medical education [GME]) assessments. This study explores the strength of an a priori alignment of medical school assessments with residency competency domains to guide programmatic assessment and research.

METHOD: In fall 2023, the authors (US faculty with UME and GME expertise) used a 2-phase Delphi consensus approach (9 participants in phase 1 and 16 in phase 2) to independently map medical school assessments to the Accreditation Council for Graduate Medical Education competency domains (0, no association; 1, some or …


Population Pharmacokinetics And Dosing Simulations Of Pentobarbital In The Pediatric Population, Victoria Etges Helfer, Lorena Medina-Aymerich, William J. Muller, Marisa Meyer, Amira Al-Uzri, Russell Mcculloh, Chi D. Hornik, Stephen J. Balevic, Rachel G. Greenberg, Daniel K. Benjamin, Sarah G. Anderson, Daniel Gonzalez May 2026

Population Pharmacokinetics And Dosing Simulations Of Pentobarbital In The Pediatric Population, Victoria Etges Helfer, Lorena Medina-Aymerich, William J. Muller, Marisa Meyer, Amira Al-Uzri, Russell Mcculloh, Chi D. Hornik, Stephen J. Balevic, Rachel G. Greenberg, Daniel K. Benjamin, Sarah G. Anderson, Daniel Gonzalez

Department of Pediatrics Faculty Papers

Pentobarbital is a barbiturate with sedative and anticonvulsant properties, occasionally used in pediatric patients for preoperative sedation, deep sedation during mechanical ventilation, and seizure control. Despite its long-standing clinical use, age-appropriate dosing remains challenging due to variability across studies and limited pediatric pharmacokinetic (PK) data. This study (NCT01431326) aimed to characterize the population pharmacokinetics of pentobarbital in pediatric patients (birth to 21 years) and evaluate dosing strategies using model-based simulations. A two-compartment population PK model, allometrically scaled to total body weight, adequately described pentobarbital plasma concentrations, with no additional covariates identified as significant. Simulations were conducted across four age groups: …


A Population-Based Assessment Of Cancer Risk In Children With Vacterl, Ji Yun Tark, Alexander Renwick, Giorgio Tettamanti, Rachel D Harris, Tania A Desrosiers, Andrew F Olshan, Amanda E Janitz, Michael E Scheurer, Charles J Shumate, Angela E Scheuerle, Sharon E Plon, Chad D Huff, Ann Nordgren, Barbara Luke, Philip J Lupo, Jeremy M Schraw May 2026

A Population-Based Assessment Of Cancer Risk In Children With Vacterl, Ji Yun Tark, Alexander Renwick, Giorgio Tettamanti, Rachel D Harris, Tania A Desrosiers, Andrew F Olshan, Amanda E Janitz, Michael E Scheurer, Charles J Shumate, Angela E Scheuerle, Sharon E Plon, Chad D Huff, Ann Nordgren, Barbara Luke, Philip J Lupo, Jeremy M Schraw

Faculty, Staff and Students Publications

Cancer risk in children with VACTERL, a nonrandom co-occurrence of ≥ 3 defects (vertebral, anal, cardiac, tracheoesophogeal fistula, renal, and limb), remains unclear. We evaluated this association in a population-based study. We analyzed data from the Genetic Overlap Between Anomalies and Cancer in Kids (GOBACK) Study, a US registry linkage cohort. VACTERL was defined as the presence of ≥ 3 associated defects. Cox regression was applied to estimate hazard ratios (HRs) and 95% confidence intervals (CIs) for cancer risk before age 18 in children with VACTERL compared to children without birth defects. Kaplan-Meier analyses were used to estimate cumulative incidence …


High-Penetrance Rare Variants Underlying Familial Lung Cancer Risk: Insights From Genetic Epidemiology Of Lung Cancer Consortium, Yanhong Liu, Yafang Li, Jinyoung Byun, Vikram R Shaw, Claudio Pikielny, Bo Peng, Chao Cheng, Spiridon Tsavachidis, Xiangjun Xiao, Dakai Zhu, Younghun Han, Ivan P Gorlov, Olga Y Gorlova, Michael Cole, Colette R Gaba, Erin L Crawford, Kristen Purrington, Ellen L Goode, Ping Yang, James Mckay, John K Field, Geoffrey Liu, Rayjean J Hung, Jun Xia, Jiyeon Choi, Matthew B Schabath, Jaclyn Lopiccolo, David C Christiani, Joan Bailey-Wilson, Ann G Schwartz, James C Willey, Diptasri Mandal, Susan M Pinney, Christopher I Amos May 2026

High-Penetrance Rare Variants Underlying Familial Lung Cancer Risk: Insights From Genetic Epidemiology Of Lung Cancer Consortium, Yanhong Liu, Yafang Li, Jinyoung Byun, Vikram R Shaw, Claudio Pikielny, Bo Peng, Chao Cheng, Spiridon Tsavachidis, Xiangjun Xiao, Dakai Zhu, Younghun Han, Ivan P Gorlov, Olga Y Gorlova, Michael Cole, Colette R Gaba, Erin L Crawford, Kristen Purrington, Ellen L Goode, Ping Yang, James Mckay, John K Field, Geoffrey Liu, Rayjean J Hung, Jun Xia, Jiyeon Choi, Matthew B Schabath, Jaclyn Lopiccolo, David C Christiani, Joan Bailey-Wilson, Ann G Schwartz, James C Willey, Diptasri Mandal, Susan M Pinney, Christopher I Amos

Faculty, Staff and Students Publications

Introduction: Rare, deleterious germline variants are key contributors to inherited lung cancer (LC) risk. The Genetic Epidemiology of LC Consortium (GELCC) has curated valuable high-risk LC families and is uniquely positioned to uncover rare, high-penetrance variants underlying familial LC (FLC).

Methods: We performed whole-genome and exome sequencing on germline DNA from 120 high-risk LC families (177 FLC cases, 309 unaffected relatives). We prioritized rare (allele frequency < 1% in the genome aggregation database), potentially deleterious variants present in two or more FLC cases. These variants were then validated in 10,085 sporadic LC (SLC) cases and 612,970 controls.

Results: We identified 118 candidate variants, 28 of which were validated in SLC with strong statistical support. We discovered a novel pathogenic axis of three truncating variants in GALNT6, MUC4, and ERBB3 genes, which …


A Smoking-Related Plasma Protein Score And Smoking-Related Cancer Risk And Mortality In The Atherosclerosis Risk In Communities Study, Meng Ru, Christopher Douville, Aghiles Guenoun, Hana Zahed, Christie M Ballantyne, Kenneth R Butler, Josef Coresh, David J Couper, Panagis Galiatsatos, Marc J Gunter, Ron C Hoogeveen, Mattias Johansson, Corinne E Joshu, P Martijn Kolijn, Christina M Lill, Jiayun Lu, Michael T Marrone, Giovanna Masala, David C Muller, Anna E Prizment, Raul Zamora-Ros, Nilanjan Chatterjee, Adrienne Tin, Elizabeth A Platz May 2026

A Smoking-Related Plasma Protein Score And Smoking-Related Cancer Risk And Mortality In The Atherosclerosis Risk In Communities Study, Meng Ru, Christopher Douville, Aghiles Guenoun, Hana Zahed, Christie M Ballantyne, Kenneth R Butler, Josef Coresh, David J Couper, Panagis Galiatsatos, Marc J Gunter, Ron C Hoogeveen, Mattias Johansson, Corinne E Joshu, P Martijn Kolijn, Christina M Lill, Jiayun Lu, Michael T Marrone, Giovanna Masala, David C Muller, Anna E Prizment, Raul Zamora-Ros, Nilanjan Chatterjee, Adrienne Tin, Elizabeth A Platz

Faculty, Staff and Students Publications

Background: Self-reported smoking may not fully capture individualized risk of smoking-related cancer, but circulating proteins may reflect biological consequences of smoking. Thus, we developed a score based on smoking-related proteins and evaluated its association with smoking-related cancer.

Methods: This prospective cohort study included 10 563 participants aged 47-70 years in the Atherosclerosis Risk in Communities study. Plasma proteins were measured using the SomaScan Platform (SomaLogic Operating Co, Inc). The score was constructed from proteins associated with current smoking, pack-years, or recent quitting identified by linear regression and elastic net regression. Cox regression was used to estimate adjusted hazard ratios and …


Screening For Hypertension And Diabetes In Laundromats In The Largest Hispanic-Majority City In The U.S, Nicholas A Mccann, Vanessa Schick, Jack Tsai May 2026

Screening For Hypertension And Diabetes In Laundromats In The Largest Hispanic-Majority City In The U.S, Nicholas A Mccann, Vanessa Schick, Jack Tsai

Faculty, Staff and Student Publications

Purpose: This study aimed to assess the prevalence of hypertension and diabetes among laundromat users in San Antonio, Texas, and explore laundromats as a novel setting for health interventions regarding these 2 conditions.

Design: This descriptive, cross-sectional study involved demographic, blood pressure and HbA1c screenings to assess the health status of laundromat users.

Setting: The study took place in 7 laundromats in low-income areas of San Antonio.

Subjects: A total of 150 adults aged 18 and older who regularly used the laundromats and spoke either English or Spanish were eligible to participate.

>Measures: Hypertension was defined as a systolic BP …


An Integrated Framework For Early Detection And Transmissibility Assessment Of Emerging Variants In Wastewater, Xingwen Chen, Tin Phan, Wei Lin Lee, Katie Clark, Steven F Rhode, Samantha Brozak, Bruce Pell, Tsultrim Palden, Mats Leifels, Anna Gitter, Yang Kuang, Stefan Wuertz, Janelle Thompson, Kristina D Mena, Eric J Alm, Fuqing Wu May 2026

An Integrated Framework For Early Detection And Transmissibility Assessment Of Emerging Variants In Wastewater, Xingwen Chen, Tin Phan, Wei Lin Lee, Katie Clark, Steven F Rhode, Samantha Brozak, Bruce Pell, Tsultrim Palden, Mats Leifels, Anna Gitter, Yang Kuang, Stefan Wuertz, Janelle Thompson, Kristina D Mena, Eric J Alm, Fuqing Wu

Faculty, Staff and Student Publications

Tracking the emergence of new viral variants is critical for epidemic preparedness, but remains challenging because variants typically circulate at low prevalence during the early phase of outbreaks. Here, we present an integrated framework that combines sensitive variant detection in wastewater with quantitative assessment of transmissibility through dynamic epidemic modeling. Using the SARS-CoV-2 Omicron variant as a proof of concept, we developed a novel nested allele-specific RT-qPCR assay (NAS-PCR) that achieved a thousand-fold increase in sensitivity compared with allele-specific RT-qPCR (limit of detection: 0.5 copies/μl). NAS-PCR detected Omicron in wastewater samples from the Greater Boston area starting in September 2021, …


Associations Of Blood Biomarkers Of Glial Cell Dysfunction And Neuronal Injury With Future Cognitive Decline And Incident Dementia, Srishti Shrestha, Xiaoqian Zhu, B Gwen Windham, Kevin J Sullivan, Priya Palta, Rebecca F Gottesman, Russell P Tracy, Clifford R Jack, Petrice M Cogswell, Prashanthi Vemuri, Sudha Seshadri, Thomas H Mosley, Michael E Griswold, Myriam Fornage May 2026

Associations Of Blood Biomarkers Of Glial Cell Dysfunction And Neuronal Injury With Future Cognitive Decline And Incident Dementia, Srishti Shrestha, Xiaoqian Zhu, B Gwen Windham, Kevin J Sullivan, Priya Palta, Rebecca F Gottesman, Russell P Tracy, Clifford R Jack, Petrice M Cogswell, Prashanthi Vemuri, Sudha Seshadri, Thomas H Mosley, Michael E Griswold, Myriam Fornage

Faculty, Staff and Student Publications

Introduction: Prospective studies of blood-based biomarkers reflecting pathogenic processes, other than Alzheimer-specific pathologies, that contribute to dementia in diverse cohorts are lacking.

Methods: We jointly fitted linear mixed-effect models and proportional hazards models to examine associations of midlife glial fibrillary acidic protein (GFAP), chitinase-3-like protein 1 (YKL-40), soluble cluster of differentiation-14 (sCD14), neurofilament light chain (NfL), total tau (t-tau), and ubiquitin C-terminal hydrolase L1 (UCHL1) levels in serum (collected in 1993 to 1995) with cognitive decline and incident dementia (ascertained over 29 years through 2022) in the community-based Atherosclerosis Risk in Communities Study. sCD14 and YKL-40 were measured in 3082 …


Perceived Neighborhood Environment And Objectively Assessed, Habitual Physical Activity Among Us Adults: An All Of Us Research Program Study, Natalia I Heredia, Michael C Robertson, Christopher D Pfledderer, Ethan T Hunt, Timothy J Walker, Deborah Salvo May 2026

Perceived Neighborhood Environment And Objectively Assessed, Habitual Physical Activity Among Us Adults: An All Of Us Research Program Study, Natalia I Heredia, Michael C Robertson, Christopher D Pfledderer, Ethan T Hunt, Timothy J Walker, Deborah Salvo

Faculty, Staff and Student Publications

Introduction: The purpose of this study was to assess the associations between perceived socioenvironmental neighborhood attributes and objectively assessed habitual physical activity among US adults as well as whether associations vary by sociodemographic characteristics.

Methods: We used data from the All of Us research program, a longitudinal cohort study of a diverse sample of Americans. Perceived neighborhood social cohesion, physical and social disorder, walkability, and sociodemographic characteristics were self-reported. Habitual physical activity was ascertained using Fitbit-derived step counts over ≥24 weeks. Generalized additive mixed-effects models were used to test the associations between perceived socioenvironmental neighborhood attributes and daily step counts. …


The Current Status Of Infant Oral Health Instruction Within Predoctoral Dental Education, Beau D Meyer, Priyanshi Ritwik, Jean Star, Timothy Followell, Mikhail Jones, Leola Royston, Brenda Bohaty, Martina Majstorovic, Adriana Modesto Gomes Da Silva, Katherine Chin, James R Boynton, Chelsea Fosse May 2026

The Current Status Of Infant Oral Health Instruction Within Predoctoral Dental Education, Beau D Meyer, Priyanshi Ritwik, Jean Star, Timothy Followell, Mikhail Jones, Leola Royston, Brenda Bohaty, Martina Majstorovic, Adriana Modesto Gomes Da Silva, Katherine Chin, James R Boynton, Chelsea Fosse

Faculty, Staff and Student Publications

A pediatric dental home is a patient-provider relationship that establishes compassionate, comprehensive care that is continually accessible. For more than two decades, professional organizations have recommended children establish a dental home by their first birthday. In 2024, the American Academy of Pediatric Dentistry (AAPD) Council on Pre-Doctoral Education had a governance charge approved by the AAPD Board of Trustees to evaluate the status of infant oral health (IOH) instruction in pre-doctoral dental education. This study was designed to describe the current state of didactic and clinical clock hours dedicated to IOH in U.S. pre-doctoral curricula. Following IRB approval, a 25-item …


Derivation And Validation Of The Pediatric Community-Acquired Pneumonia Severity (Pedcaps) Score: A Prospective Cohort Study, Todd A Florin, Ron Reeder, Lilliam Ambroggio, Richard M Ruddy, Samir S Shah, Allison Cator, Matthew J Lipshaw, Geoffrey A Capraro, Laura F Sartori, Amy Y Cheng, Leah Tzimenatos, Patrick S Walsh, Claudia R Morris, Chris A Rees, Son H Mclaren, Tamar R Lubell, Chari D Larsen, Justin Moher, Eileen J Klein, Shubhada Hooli, Nathan Kuppermann May 2026

Derivation And Validation Of The Pediatric Community-Acquired Pneumonia Severity (Pedcaps) Score: A Prospective Cohort Study, Todd A Florin, Ron Reeder, Lilliam Ambroggio, Richard M Ruddy, Samir S Shah, Allison Cator, Matthew J Lipshaw, Geoffrey A Capraro, Laura F Sartori, Amy Y Cheng, Leah Tzimenatos, Patrick S Walsh, Claudia R Morris, Chris A Rees, Son H Mclaren, Tamar R Lubell, Chari D Larsen, Justin Moher, Eileen J Klein, Shubhada Hooli, Nathan Kuppermann

Faculty, Staff and Students Publications

Introduction: Community-acquired pneumonia (CAP) is a frequent and costly cause of pediatric emergency department (ED) visits and hospitalizations. Previous prognostic tools for CAP are limited by small samples, single-center or retrospective designs, lack of generalizability to ED settings, lack of biomarkers, or limited objective data. To overcome these limitations, we will derive and externally validate a prediction rule for pediatric CAP severity in a large, multicenter prospective cohort.

Methods: This is a prospective cohort study of children 3 months to 18 years old with CAP who present to EDs within the Pediatric Emergency Care Applied Research Network. Enrollment began 8/2023 …


Risk-Stratified Transitional Care And Cardiovascular Hospitalizations By Sex: A Secondary Analysis Of A Randomized Clinical Trial., Douglas S Lee, Chang Nancy Wang, Peter C Austin, Sharon E Straus, Michael E Farkouh, Alice Chong, Monica Taljaard, Stephanie Poon, Stuart Smith, Robert S Mckelvie, Robert M Iwanochko, Andrea Macdougall, Esam Elbarasi, Peter M Cram, Jiming Fang, Clare L Atzema, Jacob A Udell, Paula A Rochon, Michael J Schull, Susanna Mak, Heather J Ross May 2026

Risk-Stratified Transitional Care And Cardiovascular Hospitalizations By Sex: A Secondary Analysis Of A Randomized Clinical Trial., Douglas S Lee, Chang Nancy Wang, Peter C Austin, Sharon E Straus, Michael E Farkouh, Alice Chong, Monica Taljaard, Stephanie Poon, Stuart Smith, Robert S Mckelvie, Robert M Iwanochko, Andrea Macdougall, Esam Elbarasi, Peter M Cram, Jiming Fang, Clare L Atzema, Jacob A Udell, Paula A Rochon, Michael J Schull, Susanna Mak, Heather J Ross

Heart and Vascular Articles

IMPORTANCE: Female patients with heart failure (HF) are older and more often present with preserved left ventricular ejection fraction (LVEF), whereas male patients present with more ischemic disease. Despite these differences, an emergency department-based acute HF strategy may be equally applicable to both sexes.

OBJECTIVE: To determine whether the strategy for acute HF management in the Comparison of Outcomes and Access to Care for Heart Failure (COACH) trial differed by sex.

DESIGN, SETTING, AND PARTICIPANTS: This prespecified secondary analysis of the multicenter COACH stepped-wedge, cluster-randomized clinical trial included 10 acute care hospitals in Ontario, Canada. Data were collected from January …


Robot-Assisted Resection Of Diaphragmatic And Hepatorenal Recess Peritoneal Endometriosis: Two Cases And A Narrative Review, Xiaoming Guan, Qiannan Yang, Daniel Y Lovell, Chunhua Zhang May 2026

Robot-Assisted Resection Of Diaphragmatic And Hepatorenal Recess Peritoneal Endometriosis: Two Cases And A Narrative Review, Xiaoming Guan, Qiannan Yang, Daniel Y Lovell, Chunhua Zhang

Faculty, Staff and Students Publications

In this report, we describe two cases of diaphragmatic endometriosis with concurrent hepatorenal recess peritoneal involvement managed using robotic-assisted laparoscopic surgery with the da Vinci Xi platform. In both patients, diaphragmatic implants and hepatorenal recess peritoneal lesions were excised en bloc, followed by resection of additional pelvic and abdominal lesions. Case 1 involved a 45-year-old woman whose pathology confirmed endometriosis in 28 of 46 sites, including the diaphragm, umbilicus, bowel, hepatorenal recess peritoneum, and pelvic peritoneum. Case 2 involved a 32-year-old woman with histopathologic evidence of endometriosis in 26 of 34 resected sites, including the appendix, diaphragm, hepatorenal recess peritoneum, …


Maternal Fish Intake In The Year Prior To Conception And Birth Defects, National Birth Defects Prevention Study, 1997-2011, Dorothy Kim Waller, Zeyu Miao, Renata H Benjamin, Richard Finnell, Nithya Lakshmi Mohan Dass, Eirini Nestoridi, Marcia C De Oliveira Otto, Julie Peterson, Tunu Ramadhani, Mark A Canfield May 2026

Maternal Fish Intake In The Year Prior To Conception And Birth Defects, National Birth Defects Prevention Study, 1997-2011, Dorothy Kim Waller, Zeyu Miao, Renata H Benjamin, Richard Finnell, Nithya Lakshmi Mohan Dass, Eirini Nestoridi, Marcia C De Oliveira Otto, Julie Peterson, Tunu Ramadhani, Mark A Canfield

Faculty, Staff and Students Publications

Background: Epidemiologic data on the association between maternal fish intake and birth defects are sparse. Our objective was to assess associations between maternal fish intake and 52 different birth defects, most of which have not been assessed previously.

Methods: Using logistic regression, data was analyzed for 29,242 mothers of infants with birth defects and 10,973 mothers of control infants who participated in the National Birth Defects Prevention Study (NBDPS) and delivered between 1997 and 2011. We focused on associations between mothers who reported high fish intake of 2 or more servings of fish per week for the year prior to …


Multidisciplinary Approach To Complex Lower Extremity Limb Salvage In Pyogenic Arthritis, Pyoderma Gangrenosum, And Acne (Papa) Syndrome: A Case Report, Michael M Talanker, Anthony V Basta, Tien Do, Robert Tung, Addison Allen, Morgan Martin, Bryan L Ruygrok, Patrick J Kellam, Jeffrey G Trost, Mohin A Bhadkamkar May 2026

Multidisciplinary Approach To Complex Lower Extremity Limb Salvage In Pyogenic Arthritis, Pyoderma Gangrenosum, And Acne (Papa) Syndrome: A Case Report, Michael M Talanker, Anthony V Basta, Tien Do, Robert Tung, Addison Allen, Morgan Martin, Bryan L Ruygrok, Patrick J Kellam, Jeffrey G Trost, Mohin A Bhadkamkar

Faculty, Staff and Student Publications

Limb salvage using free tissue transfer in patients with rare autoinflammatory syndromes is poorly described, and the safety of microvascular reconstruction in this population remains uncertain. Hereditary pyogenic arthritis, pyoderma gangrenosum, and acne (PAPA) syndrome is characterized by dysregulated inflammation, pathergy, and impaired wound healing, raising concern for flap compromise and postoperative complications. We report a rare case of lower extremity free tissue transfer in a patient with PAPA syndrome. A 66-year-old woman with rheumatoid arthritis and PAPA syndrome presented with rapid wound breakdown, violaceous ulceration, exposed hardware, and periprosthetic joint infection following left total knee arthroplasty (TKA). Examination revealed …