Digital Commons Network^™

Five-Year Outcomes Of Lenadogene Nolparvovec Gene Therapy In Leber Hereditary Optic Neuropathy, Patrick Yu-Wai-Man, Nancy Newman, Valérie Biousse, Valerio Carelli, Mark Moster, Catherine Vignal-Clermont, Thomas Klopstock, Alfredo Sadun, Robert C Sergott, Rabih Hage, Simona Degli Esposti, Chiara La Morgia, Claudia Priglinger, Rustum Karanja, Magali Taiel, José-Alain Sahel

Department of Neurology Faculty Papers

IMPORTANCE: Limited studies have assessed the long-term benefit/risk of gene therapy for Leber hereditary optic neuropathy (LHON).

OBJECTIVE: To determine the safety and efficacy of lenadogene nolparvovec in patients with LHON due to the MT-ND4 gene variant for up to 5 years after administration.

DESIGN, SETTING, AND PARTICIPANTS: The RESCUE and REVERSE Long-Term Follow-up Study (RESTORE), conducted from 2018 to 2022, is the 5-year follow-up study of the 2 phase 3 clinical studies RESCUE (Efficacy Study of Lenadogene Nolparvovec for the Treatment of Vision Loss Up to 6 Months From Onset in LHON Due to the MT-ND4 Mutation) and REVERSE …

Leptin Receptor Neurons In The Dorsomedial Hypothalamus Require Distinct Neuronal Subsets For Thermogenesis And Weight Loss, Marie Francois, Laura Kaiser, Yanlin He, Yong Xu, J Michael Salbaum, Sangho Yu, Christopher D Morrison, Hans-Rudolf Berthoud, Heike Münzberg

Children’s Nutrition Research Center Staff Publications

The dorsomedial hypothalamus (DMH) receives inputs from the preoptic area (POA), where ambient temperature mediates physiological adaptations of energy expenditure and food intake. Warm-activated POA neurons suppress energy expenditure via brown adipose tissue (BAT) projecting neurons in the dorsomedial hypothalamus/dorsal hypothalamic area (dDMH/DHA). Our earlier work identified leptin receptor (Lepr)-expressing, BAT-projecting dDMH/DHA neurons that mediate metabolic leptin effects. Yet, the neurotransmitter (glutamate or GABA) used by dDMH/DHALepr neurons remains unexplored and was investigated in this study using mice. We report that dDMH/DHALepr neurons represent equally glutamatergic and GABAergic neurons. Surprisingly, chemogenetic activation of glutamatergic and/or GABAergic dDMH/DHA neurons were capable …

Association Between Maternal Stress And Child Sleep Quality: A Nationwide Echo Prospective Cohort Study, Sarah Dee Geiger, Aruna Chandran, Marie L Churchill, Maxwell Mansolf, Cai Zhang, Salma Musaad, Courtney K Blackwell, Stephanie M Eick, Dana E Goin, Susan Korrick, Akram Alshawabkeh, Patricia A Brennan, Carrie V Breton, Jose F Cordero, Sean Deoni, Viren D'Sa, Anne L Dunlop, Amy J Elliott, Assiamira Ferrara, Arlene Keddie, Monique Lebourgeois, Kaja Z Lewinn, Daphne Koinis-Mitchell, Maristella Lucchini, Sara S Nozadi, Thomas O'Connor, Yeyi Zhu, Emily Zimmerman, Susan L Schantz

Children’s Nutrition Research Center Staff Publications

Background: Childhood sleep quality is associated with physical, cognitive, and behavioral health and predicts later sleep quality; it has many determinants, including developmental exposures.

Objectives: To examine associations between maternal stress during pregnancy and childhood sleep quality and determine whether postnatal stress mediates the association.

Method: Data from the Environmental Influences on Child Health Outcomes cohort were used. Perceived Stress Scale (PSS) T-scores were the exposure measure. Outcome measures were preschool Child Behavior Checklist (CBCL) sleep syndrome scale and Patient-Reported Outcomes Measurement Information System Sleep Disturbance Parent Proxy short form 4a (PSD4a) T-scores at ages 4-8 years. Linear mixed-effects regression …

Improvement On The Slopes: The Impact Of An Adaptive Snow Sports Program On Children And Young Adults With Disabilities., Stephanie M. Barton, Matthew J. Mclaughlin, Mark T. Fisher

Manuscripts, Articles, Book Chapters and Other Papers

PurposeThis retrospective cohort study sought to examine participant skill and functional progression in an adaptive snow sports program for children and young adults with disabilities.MethodsOne hundred twelve individuals, majority male, who participated in at least 2 adapted ski or snowboard sessions were evaluated by trained adaptive instructors. The primary outcome measure was standardized skill level ranging from 1-novice to 9-expert. Secondary outcome measures of cognitive, emotional, social, physical, and independence scores, were rated on a scale from 1-5, with 5 being the highest.ResultsThe median number of sessions per participant was 4 (range 2-65 sessions) and median participation duration was 2 …

Analyzing The Creation And Use Of Abbreviations In Cardiology And Cardiac Imaging Society Guidelines., Christopher R. Green, Rui Zhang, Raymond F. Stainback, Sofia W. Ye, Daniel Forsha, Enrique Garcia-Sayan, Jeffrey C. Hill, Carol Mitchell, Vera H. Rigolin, Vandana Sachdev, Partho P. Sengupta, Vincent L. Sorrell, Jordan B. Strom, Alexander M. Ye, Cynthia C. Taub

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Abbreviation use in clinical and academic cardiology is widespread, yet there are few guidelines regulating the creation and utilization of abbreviations. Inconsistent abbreviations can introduce ambiguity and pose challenges to practice and research.

OBJECTIVES: The authors aimed to analyze how abbreviations are created and utilized in general cardiology and cardiac imaging society guidelines in order to assess whether ambiguities and discrepancies exist between societies.

METHODS: Abbreviation data were collected from 7 national and international societies of general cardiology and cardiac imaging over a 6-year span (2018-2023). Data were linguistically coded for abbreviation type, unique occurrence, meaning or sense count, …

Diagnostic Accuracy Prior To Congenital Heart Defect Surgery: A Multicenter Collaboration., Shobha S. Natarajan, Kasey Chaszczewski, Chris Penney, Steve Ampah, Douglas Ryba, Andrea T. Kennedy, Robert Olsen, Shubhika Srivastava, Matthew J. Campbell, Mya Carney, Carol Prospero, Lisa Elliott, Carlie Brewer, Michael Dimaria, Nitin Madan, Seda Tierney, Meaghan Beattie, Ritu Sachdeva, Joan Lipinski, Kenan W D Stern, Grace Kong, Preeta Dhanantwari, Elena N. Kwon, Hari Rajagopal, Carolyn Taylor, Tammy Churchill, Aura A. Sanchez Mejia, Deidra Abenlah Ansah, Anitha Parthiban, Hasti Sanandajifar, Sowmya Balasubramanian, David A. Parra, Kimberly Crum, Corey Stiver, Aarti H. Bhat, Pei-Ni Jone, Stefani Samples, Kathleen Van't Hof, Curt Degroff, Dalia Lopez-Colon, Meryl S. Cohen

Manuscripts, Articles, Book Chapters and Other Papers

BACKGROUND: Echocardiography is the mainstay for diagnosing congenital heart disease (CHD). Diagnostic errors can lead to suboptimal surgical outcomes.

OBJECTIVES: This multicenter pediatric echocardiography collaborative learning initiative explores reasons for diagnostic errors, investigates associations between patient- and center-specific factors and errors, and relays the benefits of a multicenter approach to decrease these errors as a first step to improve CHD surgical outcomes.

METHODS: Participating centers submitted diagnostic evaluations on patients prior to 2-ventricle repair into a central database. We held virtual meetings to revise variables and discuss cases to learn from each other.

RESULTS: Fourteen pediatric echocardiography laboratories entered data …

Minimal Clinically Important Difference In Obsessive-Compulsive Disorder, Matti Cervin, Eric A Storch, Lara Farrell, Erik Andersson, Christian Rück, Daniel A Geller, David Mataix-Cols

Faculty, Staff and Students Publications

No abstract provided.

Exploring Individual Differences In Amygdala-Mediated Memory Modulation, Martina K Hollearn, Joseph R Manns, Lou T Blanpain, Stephan B Hamann, Kelly Bijanki, Robert E Gross, Daniel L Drane, Justin M Campbell, Krista L Wahlstrom, Griffin F Light, Aydin Tasevac, Phillip Demarest, Peter Brunner, Jon T Willie, Cory S Inman

Faculty, Staff and Students Publications

Amygdala activation by emotional arousal during memory formation can prioritize events for long-term memory. Building upon our prior demonstration that brief electrical stimulation to the human amygdala reliably improved long-term recognition memory for images of neutral objects without eliciting an emotional response, our study aims to explore and describe individual differences and stimulation-related factors in amygdala-mediated memory modulation. Thirty-one patients undergoing intracranial monitoring for intractable epilepsy were shown neutral object images paired with direct amygdala stimulation during encoding with recognition memory tested immediately and one day later. Adding to our prior sample, we found an overall memory enhancement effect without …

Gheard: An Open-Access Modular Curriculum To Incorporate Equity, Anti-Racism, And Decolonization Training Into Global Health Education, Amy R L Rule, Heather A Haq, Adelaide Barnes, Demarco Bowen, Msandeni Chiume-Kayuni, Whitney Cameron, S Aya Fanny, Elizabeth Groothuis, Bethany Hodge, Cindy Howard, James C Hudspeth, Sheridan Langford, Megan S Mchenry, Ijeoma Nnodim Opara, Beena D Kamath-Rayne, Parminder S Suchdev, Reena P Tam, Franci Taylor, Victor Musiime

Faculty, Staff and Students Publications

Background Global health (GH) interest is rising among graduate medical education (GME) trainees, yet GH engagement is marred by the impact of colonization or racism, and there remains a lack of training to confront these challenges.

Objective To develop a modular, open-access curriculum that provides training in decolonization for GH GME and evaluate its feasibility and impact on learners’ critical reflection on decolonization.

Methods From 2019 to 2022, 40 GH educators, including international and indigenous scholars from diverse organizations, created the Global Health Education for Equity, Anti-Racism, and Decolonization (GHEARD) curriculum. Using Kern’s 6 steps of curriculum development, critical gaps …

Spectrum Of Venous Thromboembolism In Adult Patients With Ulcerative Colitis In Pakistan: A Single Center Retrospective Study, Masood Muhammad Karim, , Hafsa Shaikh, Faisal Wasim Ismail

Department of Medicine

Background: Patients with inflammatory bowel disease are at a 2-8-fold higher risk of developing venous thromboembolism (VTE) as compared to the general population. Although the exact pathogenesis is unclear, the literature suggests that increased risk of thromboembolic events in such patients occurs as a result of increased coagulation factors, inflammatory cytokines, and reduction in anticoagulants leading to a prothrombotic state.
Aim: To assess the prevalence, risk factors, management, and outcome of ulcerative colitis (UC) patients who develop VTE.
Methods: This was a retrospective chart review done in The Gastroenterology Department of The Aga Khan University Hospital. Data was collected from …

The Effect Of Non-Pharmacological Interventions On Cognitive Function In Cancer: An Overview Of Systematic Reviews, Darren Haywood, Ashley M. Henneghan, Alexandre Chan, Raymond J. Chan, Haryana M. Dhillon, Maryam B. Lustberg, Janette L. Vardy, Moira O’Connor, Norah Elvidge, Evan Dauer, Oscar Y. Franco-Rocha, Shradha Vasan, James Murray, Megan Crichton, Helen Wilding, Susan L. Rossell, Nicolas H. Hart

Research outputs 2022 to 2026

Purpose: A significant number of cancer survivors experience cancer-related cognitive impairment (CRCI), which can impact their ability to think, reason, make decisions, and perform daily actions. In recent years, non-pharmacological interventions for CRCI have gained significant attention. These interventions include exercise, cognitive behavioural therapy, cognitive training/remediation, dietary, mind–body, and multi-modal/complex interventions. This umbrella review provides a critical overview to inform guidelines and current practice, identify the most promising interventions, and uncover gaps in the research literature.

Methods: This umbrella review of systematic reviews was pre-registered on Open Science Framework and PROSPERO. Six databases were searched. Systematic reviews (SR) assessing any …

Trajectories Of Supportive Care Needs For People Who Travel To Receive Cancer Treatment: A Longitudinal Study In Australia, Susannah K. Ayre, Elizabeth A. Johnston, Michael Ireland, Sonja March, Jeff Dunn, Suzanne Chambers, Belinda C. Goodwin

Research outputs 2022 to 2026

Objective: To describe trajectories of change in unmet supportive care needs over a two-year period among people diagnosed with cancer and assess whether these trajectories vary as a function of sociodemographic and clinical characteristics. Methods: This analysis used data from a longitudinal study of people in Queensland, Australia who travelled largely from regional and remote areas to metropolitan centres to receive cancer care (N = 784). Supportive care needs were measured at baseline, then at 3-, 12-, and 24-month post-baseline across five domains (‘psychological’, ‘physical and daily living’, ‘health systems and information’, ‘patient care and support’, ‘sexuality’) using the Supportive …

Cardiac Surgical Bleeding, Transfusion, And Quality Metrics: Joint Consensus Statement By The Enhanced Recovery After Surgery Cardiac Society And Society For The Advancement Of Patient Blood Management, Rawn Salenger, Rakesh C Arora, Arthur Bracey, Mario D'Oria, Daniel T Engelman, Caroline Evans, Michael C Grant, Serdar Gunaydin, Vicki Morton, Sherri Ozawa, Prakash A Patel, Jacob Raphael, Todd K Rosengart, Linda Shore-Lesserson, Pierre Tibi, Aryeh Shander

Faculty, Staff and Students Publications

Background: Excessive perioperative bleeding is associated with major complications in cardiac surgery, resulting in increased morbidity, mortality, and cost.

Methods: An international expert panel was convened to develop consensus statements on the control of bleeding and management of transfusion and to suggest key quality metrics for cardiac surgical bleeding. The panel reviewed relevant literature from the previous 10 years and used a modified RAND Delphi methodology to achieve consensus.

Results: The panel developed 30 consensus statements in 8 categories, including prioritizing control of bleeding, prechest closure checklists, and the need for additional quality indicators beyond reexploration rate, such as time …

Comprehensive Assessment Reveals Numerous Clinical And Neurophysiological Differences Between Mecp2-Allelic Disorders, Davut Pehlivan, Chengjun Huang, Holly K Harris, Christine Coquery, Aditya Mahat, Mirjana Maletic-Savatic, Laurence Mignon, Sukru Aras, Daniel G Glaze, Charles S Layne, Leonardo Sahelijo, Huda Y Zoghbi, Matthew J Mcginley, Bernhard Suter

Faculty, Staff and Students Publications

OBJECTIVE: Rett syndrome (RTT) and MECP2 duplication syndrome (MDS) result from under- and overexpression of MECP2, respectively. Preclinical studies using genetic-based treatment showed robust phenotype recovery for both MDS and RTT. However, there is a risk of converting MDS to RTT, or vice versa, if accurate MeCP2 levels are not achieved. The aim of this study was to identify biomarkers distinguishing RTT from MDS.

MATERIALS AND METHODS: We prospectively enrolled 11 MDS and 6 male RTT like (MRL) individuals for a panel of clinical and neurophysiological assessments over two visits, 8-10 months apart.

RESULTS: We identified numerous clinical and physiological …

Oral Anticoagulant Initiation In Patients With Kidney Failure On Hemodialysis Newly Diagnosed With Atrial Fibrillation (2007-2020): An Observational Study Of Trends And Disparities, Wolfgang C Winkelmayer, Austin Hu, Pascale Khairallah, Medha Airy, Kevin F Erickson, Tara I Chang, Jingbo Niu

Faculty, Staff and Students Publications

Rationale & objective: Atrial fibrillation (AF) is common in patients with kidney failure on hemodialysis (HD), but few patients receive oral anticoagulant (OAC) treatment. Availability of direct-target OACs starting in 2010 may have induced greater OAC initiation, but this has not been systematically studied.

Study design: Retrospective cohort study.

Setting & participants: Using Medicare fee-for-service billing claims (2006-2020), we identified previously OAC-naïve HD patients newly-diagnosed with AF between January 1, 2007, and October 1, 2020.

Exposures: Calendar year; race/ethnicity.

Outcomes: OAC initiation within 90 days from AF diagnosis (any; specific agent).

Analytical approach: We estimated initiation risk ratios for each …

Hospice Patients In The Emergency Department: Challenging High-Risk Encounters With Unanswered Questions And Unquestioned Answers, Jason K Bowman, Joan M Teno, Kei Ouchi

Faculty, Staff and Students Publications

No abstract provided.

Analyzing The Creation And Use Of Abbreviations In Cardiology And Cardiac Imaging Society Guidelines, Christopher R Green, Rui Zhang, Raymond F Stainback, Sofia W Ye, Daniel E Forsha, Enrique Garcia-Sayan, Jeffrey C Hill, Carol Mitchell, Vera H Rigolin, Vandana Sachdev, Partho P Sengupta, Vincent L Sorrell, Jordan B Strom, Alexander M Ye, Cynthia C Taub

Faculty, Staff and Students Publications

BACKGROUND: Abbreviation use in clinical and academic cardiology is widespread, yet there are few guidelines regulating the creation and utilization of abbreviations. Inconsistent abbreviations can introduce ambiguity and pose challenges to practice and research.

OBJECTIVES: The authors aimed to analyze how abbreviations are created and utilized in general cardiology and cardiac imaging society guidelines in order to assess whether ambiguities and discrepancies exist between societies.

METHODS: Abbreviation data were collected from 7 national and international societies of general cardiology and cardiac imaging over a 6-year span (2018-2023). Data were linguistically coded for abbreviation type, unique occurrence, meaning or sense count, …

Disease Activity Of Rheumatoid Arthritis And Kidney Function Decline: A Large Prospective Registry Study, Sho Fukui, Wolfgang C Winkelmayer, Sara K Tedeschi, Javier Marrugo, Hongshu Guan, Leslie Harrold, Heather J Litman, Tomohiro Shinozaki, Daniel H Solomon

Faculty, Staff and Students Publications

Introduction: Chronic kidney disease (CKD) is a common comorbidity of rheumatoid arthritis (RA). The association of longitudinal RA disease activity with long-term kidney function has remained uncertain.

Method: We analysed a multicentre prospective RA registry in the USA from 2001 to 2022. The exposure was updated time-averaged Clinical Disease Activity Index (TA-CDAI) categories from study enrolment. The primary outcome was a longitudinal estimated glomerular filtration rate (eGFR) change. Secondary outcomes included developments of CKD stage G3a (eGFR< 60 mL/min/1.73 m2) and stage G3b (eGFR< 45 mL/min/1.73 m2). Results were adjusted for relevant time-fixed and time-varying covariates.

Results: 31 129 patients (median age: 58.0 years, female: 76.3%, median eGFR: 90.7 mL/min/1.73 m2) contributed 234 973 visits and 146 778 person-years …

Intercostal Nerve Cryoablation In Lung Transplantation: A Single-Center, Propensity Score-Matched Analysis, Marcelo Salan-Gomez, Ramiro Fernandez, Ethan D'Silva, Belia Camarena, Emma Hills, Andres Leon, Syed B Peer, Abdussalam Elsenousi, Puneet Garcha, Gabriel Loor

Faculty, Staff and Students Publications

Background: Achieving adequate pain control after lung transplantation is an essential milestone in a patient's recovery. We compared postoperative opioid use, clinical outcomes, and respiratory function in lung transplant recipients treated with intercostal nerve cryoablation (INC) compared to those receiving standard pain management.

Methods: We reviewed all adult lung transplants performed at our center between January 2016 and December 2022. We excluded cases performed through median sternotomy and patients with prior thoracotomies, multiorgan transplants, and redo transplants. We performed a propensity score-matched analysis, comparing patients who received INC versus those who received standard pain management. The primary outcome was cumulative …

Diagnoses Supported By A Computerised Diagnostic Decision Support System Versus Conventional Diagnoses In Emergency Patients (Ddx-Bro): A Multicentre, Multiple-Period, Double-Blind, Cluster-Randomised, Crossover Superiority Trial, Wolf E Hautz, Thimo Marcin, Stefanie C Hautz, Stefan K Schauber, Gert Krummrey, Martin Müller, Thomas C Sauter, Cornelia Lambrigger, David Schwappach, Mathieu Nendaz, Gregor Lindner, Simon Bosbach, Ines Griesshammer, Philipp Schönberg, Emanuel Plüss, Valerie Romann, Svenja Ravioli, Nadine Werthmüller, Fabian Kölbener, Aristomenis K Exadaktylos, Hardeep Singh, Laura Zwaan

Faculty, Staff and Students Publications

Background: Diagnostic error is a frequent and clinically relevant health-care problem. Whether computerised diagnostic decision support systems (CDDSSs) improve diagnoses is controversial, and prospective randomised trials investigating their effectiveness in routine clinical practice are scarce. We hypothesised that diagnoses made with a CDDSS in the emergency department setting would be superior to unsupported diagnoses.

Methods: This multicentre, multiple-period, double-blind, cluster-randomised, crossover superiority trial was done in four emergency departments in Switzerland. Eligible patients were adults (aged ≥18 years) presenting with abdominal pain, fever of unknown origin, syncope, or non-specific symptoms. Emergency departments were randomly assigned (1:1) to one of two …

Spock2 Controls The Proliferation And Function Of Immature Pancreatic Β-Cells Through Mmp2, Katarzyna Blaszczyk, Anna P Jedrzejak, Natalia Ziojla, Ekaterina Shcheglova, Karolina Szarafin, Artur Jankowski, Christine A Beamish, Jolanta Chmielowiec, Omaima M Sabek, Ashok Balasubramanyam, Sanjeet Patel, Malgorzata Borowiak

Faculty, Staff and Students Publications

Human pluripotent stem cell-derived β-cells (SC-β-cells) represent an alternative cell source for transplantation in diabetic patients. Although mitogens could in theory be used to expand β-cells, adult β-cells very rarely replicate. In contrast, newly formed β-cells, including SC-β-cells, display higher proliferative capacity and distinct transcriptional and functional profiles. Through bidirectional expression modulation and single-cell RNA-seq, we identified SPOCK2, an ECM protein, as an inhibitor of immature β-cell proliferation. Human β-cells lacking SPOCK2 presented elevated MMP2 expression and activity, leading to β-integrin-FAK-c-JUN pathway activation. Treatment with the MMP2 protein resulted in pronounced short- and long-term SC-β-cell expansion, significantly increasing glucose-stimulated insulin …

Hes V20 Outperforms Galad For Detection Of Hcc: A Phase 3 Biomarker Study In The United States, Hashem B El-Serag, Qingchun Jin, Nabihah Tayob, Emad Salem, Michelle Luster, Abeer Alsarraj, Saira Khaderi, Amit G Singal, Jorge A Marrero, Sumeet K Asrani, Fasiha Kanwal

Faculty, Staff and Students Publications

BACKGROUND AND AIMS: The original hepatocellular carcinoma early detection screening (HES) score, which combines alpha-fetoprotein (AFP) with age, alanine aminotransferase, and platelets, has better performance than AFP alone for early HCC detection. We have developed HES V2.0 by adding AFP-L3 and des-gamma-carboxy prothrombin to the score and compared its performance to GALAD and ASAP scores among patients with cirrhosis.

APPROACH AND RESULTS: We conducted a prospective-specimen collection, retrospective-blinded-evaluation phase 3 biomarker cohort study in patients with cirrhosis enrolled in imaging and AFP surveillance. True-positive rate (TPR)/sensitivity and false-positive rate for any or early HCC were calculated for GALAD, ASAP, and …

Management Of Children With Food Allergies By Allergists In The United States, Aikaterini Anagnostou, Matthew Greenhawt, Jay A Lieberman, Christina E Ciaccio, Sayantani B Sindher, Blaine Creasy, Katherine Baran, Sachin Gupta, Anna Nowak-Wegrzyn

Faculty, Staff and Students Publications

BACKGROUND: Management of patients with food allergies is complex, especially in cases of patients with multiple and potentially severe food allergies. Although international guidelines exist for food allergy management, the role of the allergist in the decision-making process is key.

OBJECTIVE: Our aim was to investigate the management patterns and educational needs of practicing allergists treating patients with food allergies.

METHODS: An online survey was e-mailed to United States-based practicing allergists (N = 2833) in November-December 2021. The allergists were screened for managing 1 or more patients (including ≥25% pediatric patients) with food allergies per month. The allergists responded to …

Emapalumab Treatment In Patients With Rheumatologic Disease-Associated Hemophagocytic Lymphohistiocytosis In The United States: A Retrospective Medical Chart Review Study, Shanmuganathan Chandrakasan, Carl E Allen, Deepika Bhatla, John Carter, May Chien, Robert Cooper, Lauren Draper, Olive S Eckstein, Rabi Hanna, J Allyson Hays, Michelle L Hermiston, Ashley P Hinson, Patricia M Hobday, Michael S Isakoff, Michael B Jordan, Jennifer W Leiding, Renee Modica, Taizo A Nakano, Abiola Oladapo, Sachit A Patel, Priti Pednekar, Mona Riskalla, Susmita N Sarangi, Prakash Satwani, Anand Tandra, Kelly J Walkovich, John D Yee, Adi Zoref-Lorenz, Edward M Behrens

Faculty, Staff and Students Publications

Objective: Rheumatologic disease-associated hemophagocytic lymphohistiocytosis (HLH), a rare, life-threatening, systemic hyperinflammatory syndrome, occurs as a complication of underlying rheumatologic disease. Real-world evidence is lacking on emapalumab, a fully human monoclonal antibody that neutralizes the proinflammatory cytokine interferon-γ, approved for treating patients with primary HLH.

Methods: REAL-HLH, a retrospective medical chart review study conducted across 33 US hospitals, assessed real-world treatment patterns and outcomes in patients with HLH treated with one or more dose of emapalumab between November 20, 2018, and October 31, 2021. Data are presented for the subset of patients with rheumatologic disease-associated HLH.

Results: Fifteen of 105 patients …

Advance: A Biomedical Informatics Approach To Investigate Acute Kidney Injury In Infants, Jennifer A Rumpel, Sofia Perazzo, Jonathan Bona, Andrew M South, Matthew W Harer, Daniel Liu, Michelle C Starr, Mona Khattab, Rachel Han, Cara Slagle, Eileen Ciccia, Tasnim Najaf, Matthew Gillen, Mimily Harsono, Arwa Nada, Kiran Dwarakanath, Semsa Gogcu, Tahagod Mohamed, Christine Stoops, Elizabeth Bonachea, Mary Revenis, Jessica Roberts, Robert Michael Lenzini, Anne Debuyserie, Catherine Joseph, Karna Murthy, Patricio Ray, Mario Schootman, Corey Nagel

Faculty, Staff and Students Publications

Background: Acute kidney injury (AKI) occurs in up to half of infants admitted to the neonatal intensive care unit (NICU) and is associated with increased risks of death and more days of mechanical ventilation, hospitalization, and vasopressor drug support. Our objective was to build a granular relational database to study the impact that AKI has on infants admitted to Level-IV NICUs.

Methods: A relational database was created by linking data from the Children's Hospitals Neonatal Database with AKI-focused data from electronic health records from 9 centers.

Results: The current cohort consists of 24,870 infants with a median (IQR) gestational age …

Respiratory Syncytial Virus Disease Burden And Nirsevimab Effectiveness In Young Children From 2023-2024, Heidi L Moline, Ariana P Toepfer, Ayzsa Tannis, Geoffrey A Weinberg, Mary A Staat, Natasha B Halasa, Julie A Boom, Eileen J Klein, John V Williams, Jennifer E Schuster, Leah Goldstein, Erin R Mckeever, Casey Kalman, Clinton Paden, Lydia Atherton, Megha Aggarwal, Pavitra Roychoudhury, Pedro A Piedra, Leila C Sahni, Laura S Stewart, Rangaraj Selvarangan, Marian G Michaels, Elizabeth P Schlaudecker, Peter G Szilagyi, Janet A Englund, Benjamin R Clopper, Natalie J Thornburg, Gordana Derado, Meredith L Mcmorrow, Fatimah S Dawood, New Vaccine Surveillance Network Collaborators

Faculty, Staff and Students Publications

Importance: During the 2023-2024 respiratory syncytial virus (RSV) season in the United States, 2 new RSV prevention products were recommended to protect infants in their first RSV season: nirsevimab and Pfizer's maternal RSV vaccine. Postlicensure studies are needed to assess prevention product impact and effectiveness.

Objective: To compare the epidemiology and disease burden of medically attended RSV-associated acute respiratory illness (ARI) among children younger than 5 years during the 2023-2024 RSV season with 3 prepandemic RSV seasons (2017-2020), estimate nirsevimab effectiveness against medically attended RSV-associated ARI, and compare nirsevimab binding site mutations among circulating RSV in infants with and without …

Podoplanin And Microthrombi In Lung Injury, Jahnavi Gollamudi, Ricardo Gonzalez Delgado, Min Soon Cho, Brianne Wharton, Hani Lee, Animesh Vadaparti, Swapan K Dasgupta, Miguel A Cruz, Perumal Thiagarajan, Vahid Afshar-Kharghan

Faculty, Staff and Students Publications

No abstract provided.

Successful Limb Salvage In A Severe Diabetic Foot Infection: A Case Study Of Aggressive Surgical Intervention And Grafting Technique, Stephanie N. Campbell, Amanda Killeen, Michael Sobolevsky

School of Podiatric Medicine Publications

A 53-year-old male with uncontrolled diabetes (A1c 7.9%), housing insecurity, food insecurity presented with severe necrotizing diabetic foot infection with ascending cellulitis, infectious hemorrhagic bullae, multi-compartment abscesses of the foot and anterior leg. It is known that patients with diabetes are at increased risk of both amputation and surgical interventions than patients without diabetes. This patient underwent aggressive surgical intervention to avoid primary below knee amputation.

Investing In Limb Preservation: A Case Report In Avoiding Bilateral Amputation In A Vasculopathy Patient, Stephanie N. Campbell, Craig J. Verdin, Heather V. Tran, Nikita Gambhir

School of Podiatric Medicine Publications

Limb salvage and prosthetics play vital roles in restoring function and mobility for patients with severe injuries or metabolic conditions. Limb salvage involves surgical techniques aimed at preserving a limb rather than resorting to amputation, while prosthetics provide artificial devices to replace lost limbs. Key considerations for limb salvage include restoring a functional foot, maintaining patient independence, minimizing hospitalizations, and avoiding bilateral lower limb amputations.

Beyond Traditional Body Composition Metrics: Load-Capacity Indices Emerge As Predictors Of Cardiometabolic Outcomes—A Systematic Review And Meta-Analysis, Zhongyang Guan, Marianna Minnetti, Steven B. Heymsfield, Eleonora Poggiogalle, Carla M. Prado, Marc Sim, Blossom C.M. Stephan, Jonathan C.K. Wells, Lorenzo M. Donini, Mario Siervo

Research outputs 2022 to 2026

The adaptive and independent interrelationships between different body composition components have been identified as crucial determinants of disease risk. On the basis of this concept, the load-capacity model of body composition, which utilizes measurements obtained through nonanthropometric techniques such as dual-energy X-ray absorptiometry, was proposed. This model is typically operationalized as the ratio of metabolic load (adipose mass) to metabolic capacity (lean mass). In recent years, a series of load-capacity indices (LCIs) have been utilized to identify abnormal body composition phenotypes such as sarcopenic obesity (SO) and to predict the risk of metabolic, cardiovascular, and cognitive disorders. In this review, …