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Limosilactobacillus Reuteri Promotes Melatonin Release From Human Intestinal Organoids Via 5′Ectonucleotidase Activity, Micah D Forshee, Erika J Nachman, Esha R Shenoy, Heather A Danhof, Ludwig Ermann Lundberg, Stefan Roos, Robert A Britton Dec 2026

Limosilactobacillus Reuteri Promotes Melatonin Release From Human Intestinal Organoids Via 5′Ectonucleotidase Activity, Micah D Forshee, Erika J Nachman, Esha R Shenoy, Heather A Danhof, Ludwig Ermann Lundberg, Stefan Roos, Robert A Britton

Faculty, Staff and Students Publications

Strains of Limosilactobacillus reuteri have been used to prevent or treat various conditions; however, the mechanisms by which they exert beneficial effects are not completely understood. Infant colic is one example in which L. reuteri DSM 17938 reduces clinical symptoms. While the etiology of colic is unknown, abnormal melatonin levels in infants have been suggested as a possible contributor. L. reuteri DSM 17938 has been shown to produce adenosine from AMP via production of the extracellular enzyme 5′ectonucleotidase (5′NT). Adenosine is a potent signaling molecule that impacts several important aspects of host physiology, including the release of melatonin from the …


Optimizing Human Fcrn Mouse Models To Improve Pharmacokinetic Evaluation Of Antibody Drug Candidates., Gregory J. Christianson, Zachary M Howard, Samantha Kenney, Emily Lowell, Nijaguna Bethur, Derry C. Roopenian, Elena Gonzalo-Gil Dec 2026

Optimizing Human Fcrn Mouse Models To Improve Pharmacokinetic Evaluation Of Antibody Drug Candidates., Gregory J. Christianson, Zachary M Howard, Samantha Kenney, Emily Lowell, Nijaguna Bethur, Derry C. Roopenian, Elena Gonzalo-Gil

Faculty Research 2026

The use of animal models that can reliably predict drug performance in human patients is critical to antibody therapeutic development. Along with assessing toxicity and efficacy, determining the pharmacokinetic (PK) properties of therapeutics in Tg32 and Tg276 mice is essential to preclinical characterization. While Tg32 mice have been well established as indispensable in their ability to model the PK properties of antibody therapeutics, their intact immunity leaves them capable of mounting anti-drug antibody responses that interfere with PK interpretation. Here, we demonstrate the negative impact anti-drug responses can have on PK parameters derived from Tg32 mice, and provide strong evidence …


Optimizing Human Fcrn Mouse Models To Improve Pharmacokinetic Evaluation Of Antibody Drug Candidates., Gregory J. Christianson, Zachary M Howard, Samantha Kenney, Emily Lowell, Nijaguna Bethur, Derry C. Roopenian, Elena Gonzalo-Gil Dec 2026

Optimizing Human Fcrn Mouse Models To Improve Pharmacokinetic Evaluation Of Antibody Drug Candidates., Gregory J. Christianson, Zachary M Howard, Samantha Kenney, Emily Lowell, Nijaguna Bethur, Derry C. Roopenian, Elena Gonzalo-Gil

Faculty Research 2026

The use of animal models that can reliably predict drug performance in human patients is critical to antibody therapeutic development. Along with assessing toxicity and efficacy, determining the pharmacokinetic (PK) properties of therapeutics in Tg32 and Tg276 mice is essential to preclinical characterization. While Tg32 mice have been well established as indispensable in their ability to model the PK properties of antibody therapeutics, their intact immunity leaves them capable of mounting anti-drug antibody responses that interfere with PK interpretation. Here, we demonstrate the negative impact anti-drug responses can have on PK parameters derived from Tg32 mice, and provide strong evidence …


When Probiotics Guidelines Differ: A Practical Guide For Clinicians And Researchers, Geoffrey A Preidis, Hania Szajewska, Marla Cunningham, Howard Bauchner, Daniel J Merenstein Dec 2026

When Probiotics Guidelines Differ: A Practical Guide For Clinicians And Researchers, Geoffrey A Preidis, Hania Szajewska, Marla Cunningham, Howard Bauchner, Daniel J Merenstein

Faculty, Staff and Students Publications

Many professional organizations publish clinical practice guidelines for the use of probiotics in gastrointestinal disorders. Generally, no two guidelines align perfectly, and some differ markedly. These discrepancies occur because clinical practice guidelines are not purely mechanical outputs of data synthesis. They are shaped by structured interpretation, value judgments, and predefined thresholds for uncertainty. These elements become particularly visible when effect sizes are small, heterogeneity is high, and outcomes vary in clinical relevance-as is the case with probiotic research. This article examines why discordance among probiotic guidelines is predictable and provides practical guidance for clinicians who must decide among conflicting recommendations …


Using Randomization To Compare Ai And Expert-Generated Formative Assessment Questions In Medical Education, Akshat Kumar, Katie Stinson, Litao Wang, Elizabeth Nugent, Yongsu Lee, Mark Hormann, Latanya Jones Love, Toufeeq Ahmed Syed Dec 2026

Using Randomization To Compare Ai And Expert-Generated Formative Assessment Questions In Medical Education, Akshat Kumar, Katie Stinson, Litao Wang, Elizabeth Nugent, Yongsu Lee, Mark Hormann, Latanya Jones Love, Toufeeq Ahmed Syed

Faculty, Staff and Student Publications

Background: AI-generated content is being used across the education spectrum and is beneficial for creating complex multiple-choice questions, such as those used in medical education. However, evaluating AI-generated content is challenging, and existing testing and evaluation methods are falling short. This study uses randomization to compare medical students' performance on and subjective evaluation of AI versus expert-generated questions. We hypothesized that there would be no difference in student performance or subjective evaluation of AI vs expert-generated questions.

Methods: We designed a single-center, randomized study where medical student participants received either one AI- or expert-generated question per day over four weeks. …


Ambulatory Blood Pressure Variability In Prediction Of Target Organ Injury: The Ship Ahoy Study, Coral D Hanevold, Jang Dong Seo, Stephen R Daniels, Bonita E Falkner, Michael A Ferguson, Joseph T Flynn, Julie R Ingelfinger, Philip R Khoury, Marc B Lande, Kevin E Meyers, Mark Mitsnefes, Joshua Samuels, Elaine M Urbina Dec 2026

Ambulatory Blood Pressure Variability In Prediction Of Target Organ Injury: The Ship Ahoy Study, Coral D Hanevold, Jang Dong Seo, Stephen R Daniels, Bonita E Falkner, Michael A Ferguson, Joseph T Flynn, Julie R Ingelfinger, Philip R Khoury, Marc B Lande, Kevin E Meyers, Mark Mitsnefes, Joshua Samuels, Elaine M Urbina

Faculty, Staff and Student Publications

Background: Ambulatory hypertension is associated with elevated left ventricular mass index (LVMI), cardiac dysfunction, and increased arterial stiffness in adolescents. Whether the addition of measures of BP variability improves the prediction of subclinical cardiovascular target organ damage (TOD) over mean BP measures is not known.

Methods: We assessed clinic and ambulatory BP (ABP), anthropometrics, and TOD in 397 adolescents. ABP means standard deviation (SD), BP, and heart rate (HR) dipping were calculated; coefficients of variability (CV) were calculated (SD/mean) to assess ABP variabilities. Measures of TOD included LVMI, left ventricular hypertrophy (LVH), LV systolic shortening, LV diastolic function (e'/a'), and …


In-Hospital Mortality Patterns And Readmissions In Patients With Chronic Obstructive Pulmonary Disease: An Analysis Of The Role Of Pulmonary Hypertension, Saad Afzal Khan, Trishna Parikh, Adishwar Rao, Akriti Agrawal, Aarohi Parikh, Farah Kazzaz, Sarah Shin, Harry Karmouty-Quintana, Maulin Patel, Kha Dinh, Bela Patel, Bindu Akkanti Dec 2026

In-Hospital Mortality Patterns And Readmissions In Patients With Chronic Obstructive Pulmonary Disease: An Analysis Of The Role Of Pulmonary Hypertension, Saad Afzal Khan, Trishna Parikh, Adishwar Rao, Akriti Agrawal, Aarohi Parikh, Farah Kazzaz, Sarah Shin, Harry Karmouty-Quintana, Maulin Patel, Kha Dinh, Bela Patel, Bindu Akkanti

Faculty, Staff and Student Publications

Chronic obstructive pulmonary disease (COPD) may be complicated by pulmonary hypertension (PH). We aimed to understand the impact of PH on in-hospital mortality and quantify the 30-day readmission rate among patients with COPD. For this cross-sectional study, we used the Nationwide Readmissions Database from 2017-2020 to identify adults ≥18 years with COPD. Patients were stratified according to PH diagnosis. Baseline characteristics between groups were compared using the Pearson chi-square test and two-sample t-test. Predictors of in-hospital mortality were determined using multivariate logistic regression analysis adjusted for demographics and confounders. The 30-day readmission rate and prevalence of PH subgroups by baseline …


Differences In Infection Prevention And Control Staffing Between Free-Standing And System-Affiliated Hospitals: A National Survey., Lucas Philip, Vittorio Maio, Pamela De Cordova, Elizabeth Monsees, Raegan Davis, Pat Moeller, Andrew Dick, Scott W Keith, Monika Pogorzelska-Maziarz Nov 2026

Differences In Infection Prevention And Control Staffing Between Free-Standing And System-Affiliated Hospitals: A National Survey., Lucas Philip, Vittorio Maio, Pamela De Cordova, Elizabeth Monsees, Raegan Davis, Pat Moeller, Andrew Dick, Scott W Keith, Monika Pogorzelska-Maziarz

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Infection prevention and control (IPC) staffing varies across US hospitals, and traditional bed-based benchmarks may no longer reflect contemporary workloads or organizational structures. With most hospitals now system-affiliated, understanding how affiliation influences infection preventionist (IP) staffing is critical for modernizing guidance.

METHODS: A national survey was distributed to US acute care hospitals participating in the National Healthcare Safety Network (NHSN) from August to December 2023, assessing IPC staffing, resource availability, and organizational structure. Hospitals were categorized as free-standing or system-affiliated; differences were assessed with Wilcoxon rank-sum and chi-square tests.

RESULTS: Of 741 responding hospitals, 550 (74%) were system-affiliated and …


Emotional Urgency And Lack Of Perseverance Are Linked To Anxiety Sensitivity And Improve During Early Recovery From Opioid Use Disorder., Breanna A. Mcnaughton-Long, Abigail J. Pleiman, Carmen Buchfink, Chrysantha B. Davis, Rayus Kuplicki, Martin P. Paulus, Hung-Wen Yeh, Maëlle C M Gueguen, Jennifer L. Stewart Nov 2026

Emotional Urgency And Lack Of Perseverance Are Linked To Anxiety Sensitivity And Improve During Early Recovery From Opioid Use Disorder., Breanna A. Mcnaughton-Long, Abigail J. Pleiman, Carmen Buchfink, Chrysantha B. Davis, Rayus Kuplicki, Martin P. Paulus, Hung-Wen Yeh, Maëlle C M Gueguen, Jennifer L. Stewart

Manuscripts, Articles, Book Chapters and Other Papers

Few studies have identified how facets of impulsivity relate to recovery in opioid use disorder (OUD). Treatment-enrolled individuals with OUD (n = 167) and healthy controls (CTL; n = 46) completed the Urgency, Premeditation, Perseverance, Sensation Seeking, Positive Urgency (UPPS-P) Impulsive Behavior Scale along with demographic and clinical measures at visit 1. Over the next three months, abstinence and return to use were documented for participants with OUD (via self-report, alcohol breathalyzer, urine drug screen, and secondary contact communication). Participants with OUD who returned to use (n = 59) did not complete further study visits, whereas those who remained abstinent …


Interdisciplinary, Delphi-Driven Consensus Guidelines On The Use Of Intravenous Ketamine Infusions For Depressive Disorders From The American Society Of Ketamine Physicians, Psychotherapists, And Practitioners (Askp3), David S Mathai, Madeleine Cluck, Amna M Aslam, Erin Amato, Arsalan Azam, Michael Banov, Kathleen A Barrett, Carl J Bonnett, David Feifel, Nicolas Grundmann, H Samuel Ko, Rupert Mcshane, Sandhya Prashad, Tatiana Santini, Lowan H Stewart, Patrick Sullivan, Stefany D Wolfsohn, Jill O Robinson, Amy L Mcguire, L Alison Mcinnes Oct 2026

Interdisciplinary, Delphi-Driven Consensus Guidelines On The Use Of Intravenous Ketamine Infusions For Depressive Disorders From The American Society Of Ketamine Physicians, Psychotherapists, And Practitioners (Askp3), David S Mathai, Madeleine Cluck, Amna M Aslam, Erin Amato, Arsalan Azam, Michael Banov, Kathleen A Barrett, Carl J Bonnett, David Feifel, Nicolas Grundmann, H Samuel Ko, Rupert Mcshane, Sandhya Prashad, Tatiana Santini, Lowan H Stewart, Patrick Sullivan, Stefany D Wolfsohn, Jill O Robinson, Amy L Mcguire, L Alison Mcinnes

Center for Medical Ethics and Health Policy Staff Publications

Background: Off-label use of intravenous ketamine (IVK) for depression is a widespread practice with limited regulation in the United States. There is an urgent need for current, evidence-informed treatment guidelines to facilitate patient safety, clinical decision-making, and care quality in real-world contexts.

Methods: The American Society of Ketamine Physicians, Psychotherapists and Practitioners (ASKP3) convened an interdisciplinary working committee tasked with creating guidelines for use of IVK for depression in outpatient settings. Guideline development followed a two-stage process: 1) a hybrid systematic and targeted evidence review and preliminary drafting by the committee, and 2) consensus refinement through a modified Delphi method …


Birth Cohort Effects In Adults Associated With Influenza A(H1n1)Pdm09 Vaccine Effectiveness, Feng Liu, Stacey L House, Tamair Curley, Et Al. Oct 2026

Birth Cohort Effects In Adults Associated With Influenza A(H1n1)Pdm09 Vaccine Effectiveness, Feng Liu, Stacey L House, Tamair Curley, Et Al.

2020-Current year OA Pubs

BACKGROUND: In 2023-2024 influenza season, adults in the 1957-1985 birth cohort had the lowest vaccine effectiveness (VE) against A(H1N1)pdm09 viruses than other age groups, including those in the 1986-2005 birth cohort. We investigated A(H1N1)pdm09 antibody levels in sera collected from influenza cases and controls to explain the birth cohort effect of the low VE.

METHODS: Acute and convalescent sera were collected from adults (1986-2005 and 1957-1985 birth cohorts) during 2023-2024. Hemagglutination inhibiting (HI) and binding antibodies were assessed against representative A(H1N1) viruses and hemagglutinin antigens.

RESULTS: A(H1N1)pdm09-infected cases in the 1957-1985 birth cohort had significantly lower HI antibodies in the …


Integrating Dietitians Into Primary Dental Care For Paediatric Populations: Developing A Conceptual Service Pathway Through A Professional Stakeholder Workshop, Lauren Hallewell, Raul Bescos, Zoe Brookes, Robert Witton, Patricia Casas-Agustench Oct 2026

Integrating Dietitians Into Primary Dental Care For Paediatric Populations: Developing A Conceptual Service Pathway Through A Professional Stakeholder Workshop, Lauren Hallewell, Raul Bescos, Zoe Brookes, Robert Witton, Patricia Casas-Agustench

School of Health Professions

BACKGROUND: Diet is a common risk factor influencing both oral and systemic health, yet dietary support within dental care is often limited to brief advice focused on sugar intake. Integrating dietitians into primary dental care for the paediatric population may support earlier identification of dietary concerns and provide the opportunity for dietetic care. However, no established integrated dietetic-dental service pathway for paediatric populations has been identified within primary dental care settings in the United Kingdom.

OBJECTIVE: To explore how selected full-consensus agreement statements could be configured into a conceptual dietetic-dental service pathway for paediatric populations attending primary dental care services …


Artificial Intelligence, Machine Learning, And Deep Learning Approaches For Attention-Deficit/Hyperactivity Disorder Diagnosis: Protocol For A Scoping Review, Benedict Onochie Ibe, Amir Aly, Shaymaa S Al-Juboori, Emmanuel Ifeachor, Rohit Shankar Oct 2026

Artificial Intelligence, Machine Learning, And Deep Learning Approaches For Attention-Deficit/Hyperactivity Disorder Diagnosis: Protocol For A Scoping Review, Benedict Onochie Ibe, Amir Aly, Shaymaa S Al-Juboori, Emmanuel Ifeachor, Rohit Shankar

School of Engineering, Computing and Mathematics

BACKGROUND: Attention-deficit/hyperactivity disorder (ADHD) affects over 366 million adults and 139 million children worldwide, yet diagnosis remains fundamentally subjective, relying on clinical interviews, behavioral observations, and rating scales that yield inconsistent results across practitioners and settings. Artificial intelligence (AI), machine learning (ML), and deep learning (DL) offer a paradigm shift toward objective, data-driven diagnosis by detecting complex patterns across neuroimaging, electrophysiology, and digital biomarkers that elude conventional assessment. Although AI-based ADHD research has grown exponentially, no comprehensive synthesis examines the full spectrum of data modalities, validation practices, and clinical translation readiness. This gap limits our understanding of which approaches are …


Real-World Experience Of Hepatitis C Treatment With Glecaprevir-Pibrentasvir During Pregnancy, L Madeline Mccrary, Megan R Curtis, Jeannie C Kelly, Jessica Elrod-Gallegos, Patrick Kojima, Patricia Werner, Jennifer L Mullersman, Tracey Habrock-Bach, Michael J Durkin, Laura R Marks Oct 2026

Real-World Experience Of Hepatitis C Treatment With Glecaprevir-Pibrentasvir During Pregnancy, L Madeline Mccrary, Megan R Curtis, Jeannie C Kelly, Jessica Elrod-Gallegos, Patrick Kojima, Patricia Werner, Jennifer L Mullersman, Tracey Habrock-Bach, Michael J Durkin, Laura R Marks

2020-Current year OA Pubs

Guidelines endorse antenatal hepatitis C virus treatment following shared decision making. No clinical trial or real-world data exist for glecaprevir-pibrentasvir (GLE/PIB). We report our experience with prenatal GLE/PIB in 14 patients. Ten patients completed treatment; all achieved virologic response by or at delivery, with no maternal or neonatal safety concerns.


A Framework For Aligning 5-Year And Lifetime Breast Cancer Risk In Women Without High-Penetrance Mutations, Graham A Colditz, Debbie L Bennett, Kala Visvanathan, Bernie A Rosner, Shu Jiang Oct 2026

A Framework For Aligning 5-Year And Lifetime Breast Cancer Risk In Women Without High-Penetrance Mutations, Graham A Colditz, Debbie L Bennett, Kala Visvanathan, Bernie A Rosner, Shu Jiang

2020-Current year OA Pubs

OBJECTIVE: Reconciling cutoff thresholds for short-term (5-year) and long-term (lifetime) breast cancer risk could support tailored and evidence-based approaches to supplemental screening and risk management most relevant to short-term clinical actions. This study aims to consistently classify women at increased risk and provide 5-year risk cutoff that corresponds to a 20% lifetime risk.

METHODS: Using U.S. Surveillance, Epidemiology and End Results (SEER) program population incidence data for women 40 to 74 years of age, this study reports both lifetime and 5-year population-based risk estimates controlling for competing risk and age varying breast cancer incidence. A cut point for 5-year risk …


Social Responsiveness As A Mediator In Adapted Cognitive Behavioral Therapy For Autistic Youth With Maladaptive And Interfering Anxiety, Jeffrey J Wood, Eric A Storch, Katherine Sung, Karen S Wood, Philip C Kendall Oct 2026

Social Responsiveness As A Mediator In Adapted Cognitive Behavioral Therapy For Autistic Youth With Maladaptive And Interfering Anxiety, Jeffrey J Wood, Eric A Storch, Katherine Sung, Karen S Wood, Philip C Kendall

Faculty, Staff and Students Publications

Numerous adaptations to interventions have been included in cognitive behavioral therapy (CBT) for autistic youth. This study examines the degree to which CBT adapted to the social needs of autistic youth confers significant benefit by promoting social responsiveness. A secondary analysis was conducted on a multisite randomized clinical trial (Wood et al. in JAMA Psychiatry 77:474-483, 2020) comparing adapted CBT with standard-of-practice CBT and treatment-as-usual (TA). Autistic youth (N = 167; aged 7-13) with maladaptive and interfering anxiety participated. The adapted CBT (BIACA) uses a modular format, with an emphasis on supplementing common CBT practice elements (reframing and graded exposure) …


Burden Of Disease In Us Patients With Skeletal Versus Muscular Or Pain Manifestations Of Hypophosphatasia: An Analysis From The Global Hpp Registry., Priya S. Kishnani, Steven Ing, Julie Hoover-Fong, J Carl Pallais, Angie Voskopoja, Shona Fang, William R Mowrey, Eric Rush, Kathryn Dahir Oct 2026

Burden Of Disease In Us Patients With Skeletal Versus Muscular Or Pain Manifestations Of Hypophosphatasia: An Analysis From The Global Hpp Registry., Priya S. Kishnani, Steven Ing, Julie Hoover-Fong, J Carl Pallais, Angie Voskopoja, Shona Fang, William R Mowrey, Eric Rush, Kathryn Dahir

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Hypophosphatasia (HPP) is a metabolic disorder characterized by deficient alkaline phosphatase (ALP) activity, defective mineralization of bones and teeth, and non-skeletal manifestations. We compared disease burden between patients with skeletal and non-skeletal manifestations.

METHODS: Data from US adults enrolled in the Global HPP Registry through July 2024 were analyzed to compare baseline characteristics, genetic testing results, functional outcomes, and patient-reported outcomes between participants with and without overt skeletal manifestations. Patients who had only dental manifestations were excluded.

RESULTS: Of 283 participants, 234 (82.7%) had skeletal manifestations (79.1% female; 89.3% White; median age 48.7 years) and 49 (17.3%) had only …


Ceftaroline Pharmacokinetics On Ecmo, A Pediatric Case Report., Kathryn Pavia, Ashton Bellamy, Alaina Burns, Allison Scott, Ethan A. Poweleit Oct 2026

Ceftaroline Pharmacokinetics On Ecmo, A Pediatric Case Report., Kathryn Pavia, Ashton Bellamy, Alaina Burns, Allison Scott, Ethan A. Poweleit

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: In-vitro studies suggest that patients on extracorporeal membrane oxygenation (ECMO) may struggle to achieve therapeutic ceftaroline exposure to treat infections due to antibiotic sequestration within the ECMO oxygenator; however, there are no published data regarding ceftaroline concentrations from patients supported by ECMO.

PATIENT PRESENTATION: Here we present an analysis of ceftaroline pharmacokinetics in a 5-y-old patient supported on ECMO due to influenza-associated necrotizing pneumonia.

RESULTS: A total of nine concentrations were analyzed, including three concentrations prior to cannulation and six on ECMO. ECMO cannulation was associated with a relatively small change in the volume of distribution (0.17 L/kg to …


Ambulatory Blood Pressure Thresholds And Left Ventricular Hypertrophy In Children Aged 6-12 Years: A Pediatric Nephrology Research Consortium Study., Afsana Jahan, Matthew Schuchman, Mahie Abdullah, Meghan Mclaughlin, Emilia Maria Cadiz, Eshanika Manchanda, Aftab Chishti, Maggie Murphy, Sarah Twichell, Alexandra Mazo, Dmitry Samsonov, Aesha Maniar, Hannah Kim, Francis Lomanta, Ashok Chakravarthy Patibandla, Rachel Frank, Tarak Srivastava, Ikuyo Yamaguchi, Christine Sethna Oct 2026

Ambulatory Blood Pressure Thresholds And Left Ventricular Hypertrophy In Children Aged 6-12 Years: A Pediatric Nephrology Research Consortium Study., Afsana Jahan, Matthew Schuchman, Mahie Abdullah, Meghan Mclaughlin, Emilia Maria Cadiz, Eshanika Manchanda, Aftab Chishti, Maggie Murphy, Sarah Twichell, Alexandra Mazo, Dmitry Samsonov, Aesha Maniar, Hannah Kim, Francis Lomanta, Ashok Chakravarthy Patibandla, Rachel Frank, Tarak Srivastava, Ikuyo Yamaguchi, Christine Sethna

Manuscripts, Articles, Book Chapters and Other Papers

OBJECTIVE: To evaluate the diagnostic performance of ambulatory blood pressure monitoring (ABPM) thresholds for predicting left ventricular hypertrophy (LVH) in children aged 6-12 years.

STUDY DESIGN: A retrospective, multicenter study was conducted across 11 Pediatric Nephrology Research Consortium sites and included children aged 6-12 years who underwent ABPM and 2-dimensional echocardiography for diagnostic assessment of elevated BP. The diagnostic accuracy of fixed ABPM thresholds and the current 95th percentile for sex and height were assessed using area under the receiver operating characteristic, sensitivity, specificity, and Youden index. Multivariable logistic regression models, adjusted for age, sex, and body mass index z-score, …


A Rare Recurring Gain-Of-Function Variant In Bmpr2 Causes Neurodevelopmental Phenotypes In Humans And Flies, Jung-Wan Mok, Carrie L Welch, Haley A Dostalik, Zhi Tan, Ariel Brautbar, Maria Descartes, Erica T Gray, Alejandro Iglesias, Scott D Mclean, Morgan Nutter, Olivia Daugherty, Amber Begtrup, Ingrid M Wentzensen, Yufeng Shen, Damian W Young, Martin M Matzuk, Oguz Kanca, Michael F Wangler, Hugo J Bellen, Wendy K Chung, Shinya Yamamoto Oct 2026

A Rare Recurring Gain-Of-Function Variant In Bmpr2 Causes Neurodevelopmental Phenotypes In Humans And Flies, Jung-Wan Mok, Carrie L Welch, Haley A Dostalik, Zhi Tan, Ariel Brautbar, Maria Descartes, Erica T Gray, Alejandro Iglesias, Scott D Mclean, Morgan Nutter, Olivia Daugherty, Amber Begtrup, Ingrid M Wentzensen, Yufeng Shen, Damian W Young, Martin M Matzuk, Oguz Kanca, Michael F Wangler, Hugo J Bellen, Wendy K Chung, Shinya Yamamoto

Faculty, Staff and Students Publications

Bone morphogenetic protein receptor type 2 (BMPR2) encodes an evolutionarily conserved serine/threonine kinase that phosphorylates type-1 BMP receptors to mediate intercellular communication upon ligand binding. Loss-of-function variants in BMPR2 are known to cause pulmonary arterial hypertension and other cardiovascular disorders. Here, we report a rare recurrent missense variant in BMPR2 (c.1126G>A [GenBank: NM_001204] [p.Glu376Lys]) identified in six individuals, who all present with neurodevelopmental phenotypes including autism spectrum disorder and global developmental delay. Trio analysis demonstrates de novo occurrence of this variant in at least five of the individuals. Using the fruit fly Drosophila melanogaster, we show that this variant …


Evaluating Large-Scale Propensity Score Adjustment When Sample Size Is Small, Fleur Vereijken, Jenna M Reps, Marc A Suchard, Linying Zhang, George Hripcsak, Peter R Rijnbeek, Ross D Williams, Martijn J Schuemie Oct 2026

Evaluating Large-Scale Propensity Score Adjustment When Sample Size Is Small, Fleur Vereijken, Jenna M Reps, Marc A Suchard, Linying Zhang, George Hripcsak, Peter R Rijnbeek, Ross D Williams, Martijn J Schuemie

2020-Current year OA Pubs

BACKGROUND: Large-scale propensity score (LSPS) models are increasingly used to control confounding in observational studies, but their reliability in small-sample settings is unclear. Small samples can limit a study's ability to estimate propensity scores accurately and achieve adequate covariate balance, raising concerns about insufficient confounding adjustment. Resulting in small datasets being excluded, despite potentially containing valid information.

METHODS: We evaluated the balancing and bias reduction performance of LSPS models using six target-comparator pairs. To emulate a distributed data network analysis setting, we partitioned two large real-world data sources into smaller subsets. Within each subset, LSPS models were fit locally and …


Early Prediction Of The Breast Cancer Response To Neoadjuvant Chemotherapy Based On Intratumoral Interstitial Fluid Pressure Using 3-D Subharmonic-Aided Pressure Estimation, Sung In Choi, Basak Dogan, Corinne E. Wessner, Jessica H. Porembka, Priscilla Machado, B. Bersu Ozcan, Nisha Unni, Maysa Abu-Khalaf, Kenneth Hoyt, Flemming Forsberg, Kibo Nam Oct 2026

Early Prediction Of The Breast Cancer Response To Neoadjuvant Chemotherapy Based On Intratumoral Interstitial Fluid Pressure Using 3-D Subharmonic-Aided Pressure Estimation, Sung In Choi, Basak Dogan, Corinne E. Wessner, Jessica H. Porembka, Priscilla Machado, B. Bersu Ozcan, Nisha Unni, Maysa Abu-Khalaf, Kenneth Hoyt, Flemming Forsberg, Kibo Nam

Department of Radiology Faculty Papers

Elevated intratumoral interstitial fluid pressure (IFP) may negatively impact the efficacy of neoadjuvant chemotherapy (NAC). This study aimed to predict the breast cancer response to NAC based on intratumoral IFP using 3D contrast-enhanced ultrasound (CEUS) with subharmonic-aided pressure estimation (SHAPE). This prospective study enrolled breast cancer patients undergoing NAC from two clinical sites. 3D CEUS was performed at 0%, 10%, and 30% of NAC completion using a GE Logiq E9 with Definity. The pressure gradients between the tumor and adjacent normal tissue were estimated by SHAPE, utilizing the inverse linear relationship between the ultrasound contrast agent’s subharmonic signal amplitude and …


Efficacy And Safety Of Frail Patients Treated With Ciltacabtagene Autoleucel In The Real World: A Center For International Blood And Marrow Transplant Research Analysis., Hira Mian, Muhammad Salman Faisal, Tiening Chen, Ruta Brazauskas, Temitope Oloyede, Nausheen Ahmed, Aimaz Afrough, Larry D Anderson, Rahul Banerjee, Jesus Berdeja, Aram Bidikian, Jakob Devos, Binod Dhakal, Ajoy Dias, Danai Dima, Yvonne A Efebera, Lohith Gowda, Doris K Hansen, Hamza Hashmi, Heather J Landau, Lazaros Lekakis, Abu-Sayeef Mirza, Ravi Narra, Krina Patel, Ashley E Rosko, Mark Schroeder, Surbhi Sidana, Saad Usmani, Marcelo C Pasquini, Taiga Nishihori, Othman Salim Akhtar, Meera Mohan Oct 2026

Efficacy And Safety Of Frail Patients Treated With Ciltacabtagene Autoleucel In The Real World: A Center For International Blood And Marrow Transplant Research Analysis., Hira Mian, Muhammad Salman Faisal, Tiening Chen, Ruta Brazauskas, Temitope Oloyede, Nausheen Ahmed, Aimaz Afrough, Larry D Anderson, Rahul Banerjee, Jesus Berdeja, Aram Bidikian, Jakob Devos, Binod Dhakal, Ajoy Dias, Danai Dima, Yvonne A Efebera, Lohith Gowda, Doris K Hansen, Hamza Hashmi, Heather J Landau, Lazaros Lekakis, Abu-Sayeef Mirza, Ravi Narra, Krina Patel, Ashley E Rosko, Mark Schroeder, Surbhi Sidana, Saad Usmani, Marcelo C Pasquini, Taiga Nishihori, Othman Salim Akhtar, Meera Mohan

Oncology Articles

Background: Ciltacabtagene autoleucel (cilta-cel), an anti-B-cell maturation antigen (BCMA) chimeric antigen receptor T-cell (CAR-T) therapy, is approved for relapsed/refractory multiple myeloma (RRMM).

Methods: Using the Center for International Blood and Marrow Transplant Research registry, this study evaluated outcomes of frail patients receiving commercial cilta-cel from March 2022 to December 2023. Frailty was defined by an adapted simplified score incorporating age, performance status, and comorbidities.

Results: Among 541 patients with available frailty status, 183 (33.8%) were frail and 358 (66.2%) were nonfrail. Overall response rates were comparable (frail 82.8% vs. nonfrail 88.5%). However, frail patients had inferior progression-free survival (PFS) (12-month …


Emergency Medicine Updates: The Physiologically Difficult Airway, Aaron J Lacy, Brit Long, Michael Gottlieb, Jarrod M Mosier Oct 2026

Emergency Medicine Updates: The Physiologically Difficult Airway, Aaron J Lacy, Brit Long, Michael Gottlieb, Jarrod M Mosier

2020-Current year OA Pubs

INTRODUCTION: Airway management is an integral skill for emergency clinicians. While most of the focus is often on anatomic challenges, clinicians must also consider the unique physiologic challenges that risk decompensation during airway management, often termed the physiologically difficult airway.

OBJECTIVE: This paper evaluates key evidence-based updates concerning management of the physiologically difficult airway for the emergency clinician.

DISCUSSION: Peri-intubation complications in the emergency department are often driven by physiologic instability rather than anatomic difficulty. Key phenotypes of the physiologically difficult airway include hypotension, hypoxemia, metabolic acidemia, and right ventricular dysfunction, and are associated with high rates of physiologic collapse …


Training Apart, Together: Team Dynamics In Individual Sports And Surgical Residency, W. Jack Palmer, Dylan Bertoni, Elizabeth E. Cottrill, Colin T. Huntley Oct 2026

Training Apart, Together: Team Dynamics In Individual Sports And Surgical Residency, W. Jack Palmer, Dylan Bertoni, Elizabeth E. Cottrill, Colin T. Huntley

Department of Otolaryngology - Head and Neck Surgery Faculty Papers

Surgeons in training share notable parallels with competitive athletes, including rigorous training, skill specialization, and functioning under pressure. Surgical educators have leveraged this comparison, incorporating techniques from sports psychology, such as mental imagery and mindfulness, to improve trainees' individual performance and well-being. Surgical trainees and athletes both rely on effective teamwork for optimal outcomes, suggesting that residency leaders can glean valuable team-level insights from sports psychology. This article examines how the structure of surgical residency influences team dynamics, using concepts from sports psychology to identify evidence-based strategies for improving program cohesion and resident well-being. The surgical literature typically conceptualizes surgery …


Early Hands-On Surgical Exposure: Outcomes Of A Student-Led Surgical Externship Program For Preclinical Students, Meryem C. Guler, Elizabeth Spitz, Jennifer E. Geller, Talar Tatarian, Renee Tholey Oct 2026

Early Hands-On Surgical Exposure: Outcomes Of A Student-Led Surgical Externship Program For Preclinical Students, Meryem C. Guler, Elizabeth Spitz, Jennifer E. Geller, Talar Tatarian, Renee Tholey

Department of Surgery Faculty Papers

Objective: Early surgical exposure may potentially influence medical students' decision to pursue a surgical career. At our urban, academic medical school, senior medical students developed a Surgical Externship Program (SEP) to provide first-years with hands-on OR experience. This program is a structured 1- to 2-week immersive experience in which first-year students scrubbed into operations, participated in rounds, and engaged with surgical teams under resident and attending supervision. This study evaluates the SEP's impact on surgical interest, OR confidence, and clinical exposure.

Setting: Academic tertiary care hospital.

Participants: First-year medical students.

Design: First-year students participated in a 1- or 2-week SEP …


Streamlining Diagnosis Of Bardet-Biedl Syndrome: New Diagnostic Algorithm With Updated Criteria., Jeremy J. Pomeroy, Jesse Richards, Brooke Sweeney, Seema Kumar, Katie E. Queen, Joshua Zaritsky, Carl H. Cramer, Elias I. Traboulsi, Brittni A. Scruggs, Erica E. Davis, Ekaterina Keifer, Emma Mcgibbon, Timothy Ogden, Bendert De Graaf, Tonia Hymers, Elizabeth Forsythe, Philip Beales Oct 2026

Streamlining Diagnosis Of Bardet-Biedl Syndrome: New Diagnostic Algorithm With Updated Criteria., Jeremy J. Pomeroy, Jesse Richards, Brooke Sweeney, Seema Kumar, Katie E. Queen, Joshua Zaritsky, Carl H. Cramer, Elias I. Traboulsi, Brittni A. Scruggs, Erica E. Davis, Ekaterina Keifer, Emma Mcgibbon, Timothy Ogden, Bendert De Graaf, Tonia Hymers, Elizabeth Forsythe, Philip Beales

Manuscripts, Articles, Book Chapters and Other Papers

Considerable advances have been made in our understanding of Bardet-Biedl syndrome (BBS), particularly in its core clinical features and molecular genetics, warranting an update to the existing diagnostic criteria framework. Using a rigorous, evidence-based, and consensus-driven process, a multidisciplinary group of international experts and patient-led organizations developed an updated diagnostic algorithm. This algorithm provides practical, updated guidance for clinicians, including a pathway for accurately incorporating genetic findings into the diagnostic process. We recommend that a clinical diagnosis requires either 4 major criteria or 3 major and 2 minor criteria. Revised major criteria are retinal dystrophy, obesity (or overweight in individuals <  2 years old), congenital anomalies of the kidney and urinary tract or chronic kidney disease, hypogonadism/genital anomalies, neurodevelopmental/neurocognitive manifestations, and postaxial polydactyly. The diagnosis can also be established with a positive genetic testing result in patients exhibiting ≥ 1 major criterion, provided that genetic findings should be interpreted in the context of the patient's clinical presentation, age, family history, and overlap with related ciliopathies. These consensus criteria offer a simple algorithm incorporating updated definitions for major and minor criteria and genetic testing to support a timely and accurate diagnosis of patients with BBS, inform genetic counseling, and potentially facilitate earlier access to treatment. Trial Registration: CRIBBS Registry; ClinicalTrials.gov: NCT02329210.


A Deep-Learning Based Biomarker Of Systemic Cellular Senescence Burden To Predict Mortality And Health Outcomes, Shangshu Zhao, Chia-Ling Kuo, Eric J Lenze, Julie L Wetherell, Laura Haynes, Perla El-Ahmad, Richard Fortinsky, George Kuchel, Trevor Harris, Breno S Diniz Oct 2026

A Deep-Learning Based Biomarker Of Systemic Cellular Senescence Burden To Predict Mortality And Health Outcomes, Shangshu Zhao, Chia-Ling Kuo, Eric J Lenze, Julie L Wetherell, Laura Haynes, Perla El-Ahmad, Richard Fortinsky, George Kuchel, Trevor Harris, Breno S Diniz

2020-Current year OA Pubs

The accumulation of senescent cells is a recognized hallmark of biological aging and is associated with the onset of multiple chronic medical conditions. Senescent cells exhibit a distinct secretory profile, known as the senescence-associated secretory phenotype (SASP), which can propagate cellular senescence to neighboring and distant tissues. Measuring SASP factors in blood serves as a practical proxy for cellular senescence burden and may help track disease states and intervention outcomes. We developed and validated a composite SASP Score by integrating large-scale population proteomics data with a semi-supervised deep learning framework. The analytical workflow included: (1) selection of biologically curated SASP …


Agentic Ai For Clinical Outcomes Research, Population Health Management Analyses With Large Administrative Databases, And Generating Epidemiological Estimates Of Diseases: Feasibility And Validation Study, Daniel Frisch, Michael Fronstin, Carla Zema Sep 2026

Agentic Ai For Clinical Outcomes Research, Population Health Management Analyses With Large Administrative Databases, And Generating Epidemiological Estimates Of Diseases: Feasibility And Validation Study, Daniel Frisch, Michael Fronstin, Carla Zema

Division of Cardiology Faculty Papers

BACKGROUND: There is tremendous enthusiasm for the use of AI in health care because of the ability to analyze existing data for preventative, diagnostic, and treatment support. Agentic AI can feasibly provide access to large real-world datasets for the generation of real-world evidence for health care and clinical applications to health care providers, researchers, and administrators without access to large analytic programming resources.

OBJECTIVE: The objective of this study was to understand the feasibility of using agentic AI for clinical outcome research and population health management. Specifically, this study used an agentic AI evidence-generation platform to obtain epidemiological estimates of …


Palopegteriparatide For Adults With Chronic Hypoparathyroidism: Skeletal Dynamics Through 3 Years Of The Phase 2 Path Forward Trial, Mishaela Rubin, Bart Clarke, Lorenz Hofbauer, Aliya Khan, Peter Schwarz, Tamara Vokes, Intekhab Ahmed, Andrea Palermo, Filomena Cetani, Uberto Pagotto, Carol Zhao, Michael Ominsky, Bryant Lai, Jenny Ukena, Aimee Shu, Lars Rejnmark Sep 2026

Palopegteriparatide For Adults With Chronic Hypoparathyroidism: Skeletal Dynamics Through 3 Years Of The Phase 2 Path Forward Trial, Mishaela Rubin, Bart Clarke, Lorenz Hofbauer, Aliya Khan, Peter Schwarz, Tamara Vokes, Intekhab Ahmed, Andrea Palermo, Filomena Cetani, Uberto Pagotto, Carol Zhao, Michael Ominsky, Bryant Lai, Jenny Ukena, Aimee Shu, Lars Rejnmark

Division of Endocrinology, Diabetes and Metabolic Diseases Faculty Papers

Hypoparathyroidism is an endocrine disease caused by insufficient levels of PTH, which acts directly on bone and kidney and indirectly on the intestine to regulate calcium and phosphate balance. In clinical trials, palopegteriparatide (TransCon PTH) treatment enabled independence from conventional therapy (no active vitamin D, ≤600 mg/d calcium) and maintained serum biochemistries within normal ranges. The current analyses describe patterns of change in BMD, serum bone turnover markers, and serum and urine calcium in adults with chronic hypoparathyroidism treated with palopegteriparatide through 3 years of the PaTH Forward trial. Baseline BMD Z-scores for the LS, TH, FN, and 1/3 distal …