Medical Specialties Commons^™

The Nonvesicular Sterol Transporter Aster-C Plays A Minor Role In Whole Body Cholesterol Balance, Ranjan Banerjee, Rachel C. Hohe, Shijie Cao, Bryan M. Jung, Anthony J. Horak, Iyappan Ramachandiran, William J. Massey, Venkateshwari Varadharajan, Natalie I. Zajczenko, Amy C. Burrows, Sumita Dutta, Maryam Goudarzi, Kala Mahen, Abigail Carter, Robert N. Helsley, Scott M. Gordon, Richard E. Morton, Christopher Strauch, Belinda Willard, Camelia Baleanu Gogonea, Valentin Gogonea, Matteo Pedrelli, Paolo Parini, J. Mark Brown

Markey Cancer Center Faculty Publications

Introduction: The Aster-C protein (encoded by the Gramd1c gene) is an endoplasmic reticulum (ER) resident protein that has been reported to transport cholesterol from the plasma membrane to the ER. Although there is a clear role for the closely-related Aster-B protein in cholesterol transport and downstream esterification in the adrenal gland, the specific role for Aster-C in cholesterol homeostasis is not well understood. Here, we have examined whole body cholesterol balance in mice globally lacking Aster-C under low or high dietary cholesterol conditions.

Method: Age-matched Gramd1c^+/+ and Gramd1c^−/− mice were fed either low (0.02%, wt/wt) or high (0.2%, …

Patient Experience Of Head And Neck Surgery With Free Flap Reconstruction., Lillian W. Dattilo, Trinity I. Russell, Chloe B. Warinner, Heather Starmer, Donald J. Annino, Laura A. Goguen, Rosh K. V. Sethi, Ravindra Uppaluri, Melina Windon, Regan W. Bergmark, Eleni M. Rettig

Markey Cancer Center Faculty Publications

IMPORTANCE: Major head and neck surgery with microvascular free tissue transfer reconstruction is complex, with considerable risk of morbidity. Little is known about patients' experiences, including decision-making prior to, and regret following, free flap surgery.

OBJECTIVE: To characterize patient experiences and decision regret of patients undergoing head and neck reconstructive free flap surgery.

DESIGN, SETTING, AND PARTICIPANTS: This mixed-methods cohort study comprising semistructured interviews was conducted June to August 2021 at a single tertiary academic cancer center. Participants underwent head and neck reconstructive surgery with microvascular free tissue transfer (flap) more than 3 months before recruitment (range, 3 months to …

A Dynamic Model Of Inorganic Arsenic-Induced Carcinogenesis Reveals An Epigenetic Mechanism For Epithelial–Mesenchymal Plasticity, Matthew Rea, Greg Kimmerer, Shania Mittendorf, Xiaopeng Xiong, Megan Green, Darrell Chandler, Wesley Saintilnord, Jessica S. Blackburn, Tianyan Gao, Yvonne N. Fondufe-Mittendorf

Markey Cancer Center Faculty Publications

Inorganic arsenic (iAs) causes cancer by initiating dynamic transitions between epithelial and mesenchymal cell phenotypes. These transitions transform normal cells into cancerous cells, and cancerous cells into metastatic cells. Most in vitro models assume that transitions between states are binary and complete, and do not consider the possibility that intermediate, stable cellular states might exist. In this paper, we describe a new, two-hit in vitro model of iAs-induced carcinogenesis that extends to 28 weeks of iAs exposure. Through week 17, the model faithfully recapitulates known and expected phenotypic, genetic, and epigenetic characteristics of iAs-induced carcinogenesis. By 28 weeks, however, exposed …

A Single Hydrogen Bond That Tunes Flavin Redox Reactivity And Activates It For Modification, Debarati Das, Anne-Frances Miller

Markey Cancer Center Faculty Publications

Electron bifurcation produces high-energy products based on less energetic reagents. This feat enables biological systems to exploit abundant mediocre fuel to drive vital but demanding reactions, including nitrogen fixation and CO₂ capture. Thus, there is great interest in understanding principles that can be portable to man-made devices. Bifurcating electron transfer flavoproteins (Bf ETFs) employ two flavins with contrasting reactivities to acquire pairs of electrons from a modest reductant, NADH. The bifurcating flavin then dispatches the electrons individually to a high and a low reduction midpoint potential (E°) acceptor, the latter of which captures most of the energy. Maximum efficiency …

Outcomes Of Multiple Myeloma In Hospitalized Patients With Opioid Use Disorder: A Nationwide Analysis, Saad Javaid, Kelly Frasier, Ammad J. Chaudhary

Journal of Social, Behavioral, and Health Sciences

Multiple myeloma is commonly associated with advanced age. This study aims to investigate how multiple myeloma outcomes are affected by opioid use disorder (OUD) among hospitalized patients. We analyzed the National Inpatient Sample (NIS) for 2019 and 2020 for our retrospective cohort study. International Classification of Diseases Clinical Modification codes (ICD-10-CM) were utilized to identify the population of interest. Primary and secondary outcomes were studied using a multivariate regression model. Among the 38,735 patients hospitalized with multiple myeloma, 350 patients had the concurrent diagnosis of opioid use disorder. OUD patients were found to be at increased risk for major depressive …

An Increased Failure Rate Of Asparaginase Desensitization With Calaspargase Pegol, Jillian Fry, Erin M. Guest, Keith J. August

Posters

Background: In late 2022, pegaspargase (SS-PEG) became unavailable to patients younger than 22 years in the United States, leaving calaspargase pegol (SC-PEG) as the only available long-acting asparaginase formulation. SC-PEG has been compared to SS-PEG in two randomized, pediatric clinical trials and found to have similar rates of adverse events with comparable rates of event-free survival. Our institution uses a desensitization protocol for patients who experience a hypersensitivity reaction to asparaginase. Here we review our experience with desensitization since the transition to SC-PEG from SS-PEG. Objectives: To compare the rate of hypersensitivity reactions, success of desensitization protocols, and need for …

Diagnosis Of Mosaic Rasopathy In A Child With Rhabdomyosarcoma, Meagan Vacek, Paige Johnson, Midhat S. Farooqi, Kristi M. Canty, Dihong Zhou, Brendan Lanpher, Wendy Allen-Rhoades, Erin M. Guest

Posters

Background: Phacomatosis pigmentokeratotica (PPK) is a subtype of epidermal nevus syndrome characterized by the co-existence of a sebaceous nevus and a speckled lentiginous nevus and described in approximately 30 cases in literature. PPK is now recognized as a mosaic RASopathy due a postzygotic mutation in the Ras-Raf-MEK-ERK pathway. RAS variants are also known to contribute to tumorigenesis, in some pediatric cancers, including rhabdomyosarcoma. Objective: Describe the presentation and evaluation of a child with pelvic rhabdomyosarcoma and evolving skin lesions found to have a rare mosaic-RASopathy. Design/Method: Case Report Results: The patient is a former 32-week premature female who presented to …

Anesthetic And Multidisciplinary Management Of A 2-Staged Liver Transplant​, Kasey Brooks, Lisa Conley, Brandon Layton

Posters

We present a case of a 2 staged liver transplant on a 12-month-old 8kg male with newly diagnosed mitochondrial depletion syndrome causing acute liver failure, refractory lactic acidosis, and cardiopulmonary collapse requiring ECMO. The patient’s past medical history at the time of presentation included history of prematurity at 32 weeks, developmental delay, and a brother with an undiagnosed pancreatic disorder. The patient was brought in by parents to the emergency department with a cough, minimal PO intake for 5 days, and increased fatigue who was found to be URI positive. Initial labs were concerning for anemia, elevated bilirubin, elevated AST/ALT, …

Early Cholestasis And Vitamin K Deficiency Secondary To Biliary Atresia Presenting As Coagulopathy And Thymic Hemorrhage, Abbey Elsbernd, Lauren Amos Md

Posters

Background Bleeding and progressive coagulopathy are established symptoms of early cholestasis, including extrahepatic biliary atresia. We present a patient with bleeding secondary to vitamin K deficiency and cholestasis-induced fat malabsorption with unusual presentation of mediastinal mass. Objective Describe a case of late-onset vitamin K deficiency and cholestasis ¬¬presenting as jaundice and coagulopathy with associated bleeding into thymic structures Design/Method Case report Results A 4-week-old term male infant was brought to the ED with fussiness and bruising. Prior to presentation, he was afebrile and followed regularly with his primary care provider for jaundice below phototherapy thresholds. Infant received vitamin K at …

Single Center Retrospective Comparison Of Bivalirudin And Heparin For Therapeutic Anticoagulation In Pediatric Patients, Clayton Habiger, Shannon L. Carpenter

Posters

Background: The pharmacological choice for continuous anticoagulation therapy in pediatric patients has classically been unfractionated heparin. However, with heparin there are risks of treatment resistance in pediatrics given differences in developmental hemostasis, and contemporary medications such as bivalirudin offer a valuable alternative. Bivalirudin binds to thrombin and has shown encouraging results compared to heparin in pediatric patients receiving ECMO, but no comparison has been made in patients receiving therapeutic anticoagulation. Three years ago, Children’s Mercy Pediatric Hematology department encouraged bivalirudin use over heparin use for therapeutic anticoagulation requiring a continuous infusion. Objectives: Compare clinical and laboratory outcomes in patients receiving …

Transition Skills Lab: A Telehealth Intervention For Improving Healthcare Transition In Youth With Special Health Care Needs, Angela Combs Ma, Alaina Linafelter, Jordan Sevart, Michele H. Maddux

Posters

Introduction: Few interventions exist to provide hands-on practice of key health management tasks to youth with special health care needs (YSHCN). The Transition Skills Lab was adapted from Jerlym Porter’s work and piloted as a virtual intervention in YSHCN. Methods: Sixteen young adults participated in the virtual Transition Skills Lab, during which they practiced three key patient competencies pertaining to health care tasks: reading an insurance card, reading a pill bottle label, and scheduling an initial visit with an adult doctor. Participants were then asked to rate their confidence on skills taught within the intervention on a four-point scale from …

Congenital Heart Defects And Autism: Understanding The Breakdown Of Associated Risk Factors In A Clinically Referred Sample, Elizabeth Raines, Amanda Strasser, Amanda Manderfeld, Paul Glasier, Elizabeth J. Willen

Posters

Background: Children with Congenital Heart Disease (CHD) have higher odds of developing social difficulties and/or an Autism Spectrum Disorder (AuSD) than the general population (i.e., ~10% vs. ~1%). However, there is a paucity of nuanced understanding of specific drivers of the increased rates of AuSD in extant literature. The purpose of this study is to identify the rates of co-occurring cardiac, neurological, and genetic conditions to better understand associated risk factors in a patient sample from a medium-size children’s hospital. Methods: Our population includes a clinically referred sample of children (i.e., medical history of CHD and neurodevelopmental risk) under 18 …

Parents’ Perception Of An Intensive Interdisciplinary Pain Treatment For Amplified Pain Syndrome, Jacqueline Pabis Phd, Sabrina Ung, Jenny Scheurich, Dustin Wallace Phd

Posters

Introduction: Intensive interdisciplinary pain treatment (IIPT) is an effective treatment for youth impaired by chronic pain (Claus et al., 2022). IIPT programs incorporate parents in treatment, as parental distress has been found to impact health and functioning in youth with chronic pain. Research has suggested parent satisfaction may be related to increased child adherence, and in turn, reduced pain (Gorodzinsky et al., 2012). There is limited research on parental satisfaction with IIPT and the role of parent-provider communication. Method: Parents (n=99) of children in an IIPT program completed questionnaires related to treatment satisfaction, parent-provider communication (e.g., provider listened carefully, provider …

Ic3d Classification Of Corneal Dystrophies-Edition 3, Jayne Weiss, Christopher Rapuano, Berthold Seitz, Massimo Busin, Tero Kivelä, Nacim Bouheraoua, Cecilie Bredrup, Ken Nischal, Harshvardhan Chawla, Vincent Borderie, Kenneth Kenyon, Eung Kweon Kim, Hans Ulrik Møller, Francis Munier, Tim Berger, Walter Lisch

Wills Eye Hospital Papers

PURPOSE: The International Committee for the Classification of Corneal Dystrophies (IC3D) was created in 2005 to develop a new classification system integrating current information on phenotype, histopathology, and genetic analysis. This update is the third edition of the IC3D nomenclature.

METHODS: Peer-reviewed publications from 2014 to 2023 were evaluated. The new information was used to update the anatomic classification and each of the 22 standardized templates including the level of evidence for being a corneal dystrophy [from category 1 (most evidence) to category 4 (least evidence)].

RESULTS: Epithelial recurrent erosion dystrophies now include epithelial recurrent erosion dystrophy, category 1 ( …

Immunologic Basis For Development Of Keratoconjunctivitis Sicca In Systemic Autoimmune Diseases: Role Of Innate Immune Sensors, Michael E Stern, Argyrios N Theofilopoulos, Philipp Steven, Jerry Y Niederkorn, Robert Fox, Margarita Calonge, Christof Scheid, Stephen C Pflugfelder

Faculty, Staff and Students Publications

The literature is filled with citations reporting an increased incidence of chronic dry eye disease, also known as keratoconjunctivitis sicca, in patients with systemic autoimmune diseases such as rheumatoid arthritis, Sjögren's Syndrome, systemic sclerosis and lupus. As the most environmentally exposed mucosal surface of the body, the conjunctiva constantly responds to environmental challenges which are typically self limited, but when persistent and unresolved may provoke pathogenic innate and adaptive immune reactions. Our understanding of the pathophysiological mechanisms by which systemic autoimmune diseases cause dry eye inducing ocular surface inflammation continues to evolve. Conjunctival immune tone responds to self or foreign …

The Impact Of Cognitive Behavioral Therapy On Sleep Problems In Autistic Children With Co-Occurring Anxiety, Holly K Harris, Minjee Kook, Peter Boedeker, Andrew G Gusick, Ariel M Lyons-Warren, Robin P Goin-Kochel, Chaya Murali, Leandra N Berry, Eric A Storch

Faculty, Staff and Students Publications

Purpose: This study seeks to examine the relationship between anxiety-symptom severity and sleep behaviors in autistic children receiving cognitive behavioral therapy (CBT).

Methods: We conducted a secondary-data analysis from a sample of 93 autistic youth, 4 to 14 years, participating in 24 weeks of CBT. Clinicians completed the Pediatric Anxiety Rating Scale (PARS) and parents completed the Children's Sleep Habits Questionnaire, Abbreviated/Short Form (CSHQ-SF) at baseline, mid-treatment, post-treatment and 3 months post-treatment. Mediation analysis evaluated the role of anxiety symptoms in mediating the effect of time in treatment on sleep.

Results: There was a negative association between time in treatment …

Functional Vision In Patients With Biallelic Ush2a Variants, Elise Heon, Michele Melia, Laura E Bocchino, Lassana Samarakoon, Jacque L Duncan, Allison R Ayala, Isabelle Audo, Chris Bradley, Janet K Cheetham, Gislin Dagnelie, Todd A Durham, Carel B Hoyng, Nieraj Jain, Kanishka T Jayasundera, Mark E Pennesi, Christina Y Weng, Foundation Fighting Blindness Consortium Investigator Group

Faculty, Staff and Students Publications

Purpose: To describe functional vision (FV) and investigate the relationship between FV, visual acuity (VA), and hill of vision (VTOT) at baseline in patients with biallelic USH2A variants.

Design: Multicenter, international, cross-sectional study.

Methods: In individuals with biallelic disease-causing variants in USH2A, clinical diagnosis of Usher syndrome type 2 (USH2) or autosomal recessive nonsyndromic retinitis pigmentosa (ARRP) was based on history of hearing loss and audiology examinations. The VALVVFQ-48 was administered verbally to participants ≥18 years old. VA was measured in both eyes; VTOT was determined from static perimetry in the study eye (better VA). FV scores were calculated using …

Improved Postoperative Speech Recognition And Processor Use With Early Cochlear Implant Activation, Ankita Patro, Nathan R Lindquist, Jourdan T Holder, Michael H Freeman, René H Gifford, Kareem O Tawfik, Matthew R O'Malley, Marc L Bennett, David S Haynes, Elizabeth L Perkins

Faculty, Staff and Students Publications

Objective: To report speech recognition outcomes and processor use based on timing of cochlear implant (CI) activation.

Study design: Retrospective cohort.

Setting: Tertiary referral center.

Patients: A total of 604 adult CI recipients from October 2011 to March 2022, stratified by timing of CI activation (group 1: ≤10 d, n = 47; group 2: >10 d, n = 557).

Main outcome measures: Average daily processor use; Consonant-Nucleus-Consonant (CNC) and Arizona Biomedical (AzBio) in quiet at 1-, 3-, 6-, and 12-month visits; time to peak performance.

Results: The groups did not differ in sex ( p = 0.887), age at CI …

Autophagy Is Required For Stem-Cell-Mediated Endometrial Programming And The Establishment Of Pregnancy, Pooja Popli, Ramakrishna Kommagani

Faculty, Staff and Students Publications

Autophagy plays an important role in the normal growth and morphogenesis of a variety of tissues. Its role in uterine maturation, however, is not fully characterized. Recently, we reported that BECN1 (Beclin1)-dependent autophagy, but not apoptosis, is crucial for stem cell-mediated endometrial programming and the establishment of pregnancy in mice. Upon genetic and pharmacological inhibition of BECN1-mediated autophagy, female mice displayed severe endometrial structural and functional defects leading to infertility. Specifically, conditional loss of Becn1 in the uterus induces apoptosis and results in the gradual loss of endometrial progenitor stem cells. Importantly, the restoration of BECN1-driven autophagy, but not apoptosis …

Current And Future Perspectives On The Consensus Guideline For Food Protein-Induced Enterocolitis Syndrome (Fpies), Sara Anvari, Melanie A Ruffner, Anna Nowak-Wegrzyn

Faculty, Staff and Students Publications

Food protein-induced enterocolitis syndrome (FPIES) is a non-IgE mediated food allergy presenting with delayed onset of projectile vomiting in the absence of cutaneous and respiratory symptoms. The pathophysiology of FPIES remains poorly characterized. The first international consensus guidelines for FPIES were published in 2017 and provided clinicians with parameters on the diagnosis and treatment of FPIES. The guidelines have served as a resource in the recognition and management of FPIES, contributing to an increased awareness of FPIES. Since then, new evidence has emerged, shedding light on adult-onset FPIES, the different phenotypes of FPIES, the recognition of new food triggers, center-specific …

Relevance Of Lymphocyte Proliferation To Pha In Severe Combined Immunodeficiency (Scid) And T Cell Lymphopenia, Roshini S Abraham, Amrita Basu, Jennifer R Heimall, Elizabeth Dunn, Alison Yip, Malika Kapadia, Neena Kapoor, Lisa Forbes Satter, Rebecca Buckley, Richard O'Reilly, Geoffrey D E Cuvelier, Sharat Chandra, Jeffrey Bednarski, Sonali Chaudhury, Theodore B Moore, Hilary Haines, Blachy J Dávila Saldaña, Deepakbabu Chellapandian, Ahmad Rayes, Karin Chen, Emi Caywood, Shanmuganathan Chandrakasan, Mark Thomas Vander Lugt, Christen Ebens, Pierre Teira, Evan Shereck, Holly Miller, Victor Aquino, Hesham Eissa, Lolie C Yu, Alfred Gillio, Lisa Madden, Alan Knutsen, Ami J Shah, Kenneth Desantes, Jessie Barnum, Larisa Broglie, Avni Y Joshi, Gary Kleiner, Jasmeen Dara, Susan Prockop, Caridad Martinez, Talal Mousallem, Joseph Oved, Lauri Burroughs, Rebecca Marsh, Troy R Torgerson, Jennifer W Leiding, Sung Yun Pai, Donald B Kohn, Michael A Pulsipher, Linda M Griffith, Luigi D Notarangelo, Morton J Cowan, Jennifer Puck, Christopher C Dvorak, Elie Haddad

Faculty, Staff and Students Publications

Severe combined immunodeficiency (SCID) is characterized by a severe deficiency in T cell numbers. We analyzed data collected (n = 307) for PHA-based T cell proliferation from the PIDTC SCID protocol 6901, using either a radioactive or flow cytometry method. In comparing the two groups, a smaller number of the patients tested by flow cytometry had < 10% of the lower limit of normal proliferation as compared to the radioactive method (p = 0.02). Further, in patients with CD3+ T cell counts between 51 and 300 cells/μL, there was a higher proliferative response with the PHA flow assay compared to the 3H-T assay (p < 0.0001), suggesting that the method of analysis influences the resolution and interpretation of PHA results. Importantly, we observed many SCID patients with profound T cell lymphopenia having normal T cell proliferation when assessed by flow cytometry. We recommend this test be considered only as supportive in the diagnosis of typical SCID.

Temporary Peripheral Nerve Stimulation For The Treatment Of Greater Occipital Neuralgia, Royce Copeland, Angela Nwankwo, Emanuel Narcis Husu

Faculty, Staff and Students Publications

Background: Greater occipital neuralgia (GON), a cause of chronic headaches, can be debilitating and vastly affect one's quality of life. Recent strides in comprehending the pathophysiology of GON and the ongoing evolution of approaches to the condition's treatment have led to the pursuit of more effective interventions. Among these, temporary peripheral nerve stimulation (PNS) devices have surfaced as an avenue for management, presenting a minimally invasive yet highly efficient alternative in treating other chronic pain conditions. This study discusses a case of GON successfully managed with temporary (60-day) PNS.

Case report: A 35-year-old man presented with a nearly 8-month history …

The Satb1-Mir22-Gba Axis Mediates Glucocerebroside Accumulation Inducing A Cellular Senescence-Like Phenotype In Dopaminergic Neurons, Taylor Russo, Benjamin Kolisnyk, Aswathy B S, Jonathan Plessis-Belair, Tae Wan Kim, Jacqueline Martin, Jason Ni, Jordan A Pearson, Emily J Park, Roger B Sher, Lorenz Studer, Markus Riessland

Faculty, Staff and Students Publications

Idiopathic Parkinson's disease (PD) is characterized by the loss of dopaminergic neurons in the substantia nigra pars compacta, which is associated with neuroinflammation and reactive gliosis. The underlying cause of PD and the concurrent neuroinflammation are not well understood. In this study, we utilize human and murine neuronal lines, stem cell-derived dopaminergic neurons, and mice to demonstrate that three previously identified genetic risk factors for PD, namely SATB1, MIR22HG, and GBA, are components of a single gene regulatory pathway. Our findings indicate that dysregulation of this pathway leads to the upregulation of glucocerebrosides (GluCer), which triggers a cellular senescence-like phenotype …

Novel Approach To Difficult Spinal Reconstruction: Bilateral Simultaneous Rib And Iliac Crest Vascularized Bone Graft Spinoplastic Surgery, Maria Shvedova, Isra Abdulwadood, Daniel M Vernik, Max A Shrout, Jonathan L Jeger, Dylon C Buchanan, Alexander Ropper, Sebastian Winocour, Michael Bohl, Maziyar A Kalani, Edward M Reece

Faculty, Staff and Students Publications

Pseudoarthrosis is a severe complication of spinal fusion surgery with occurrence rates as high as 35%-40%. Current options of revision surgery to correct pseudoarthrosis frequently carry high failure rates and risk of developing junctional kyphosis. Pedicled vascularized bone grafts (VBGs) are an innovative approach to boost spinal fusion rates via improving structural integrity and increasing the delivery of blood to the donor site. This versatile technique can be performed at different spinal levels without additional skin incisions and with minimal added operative time. Here we present the first bilateral rib and iliac crest VBG spinoplastic surgery performed to augment spinal …

Initial Experience With The Anaesthetic Management Of Fetoscopic Spina Bifida Repair At A German University Hospital: A Case Series Of 15 Patients, Nicolas Schmitt, Ann-Kristin Schubert, Hinnerk Wulf, Corinna Keil, Caitlin Dooley Sutton, Ivonne Bedei, Gerald Kalmus

Faculty, Staff and Students Publications

Spina bifida aperta (SBA) is a serious neural tube defect that can lead to a range of disabilities and health complications in affected individuals. In recent years, fetoscopic surgical repair has emerged as a promising new approach to treat spina bifida prenatally, offering the potential for improved outcomes compared with traditional open surgery. As one of the few centres in Europe to offer this innovative technique, the Departments of Obstetrics and Gynaecology, Neurosurgery, and Anaesthesiology and Intensive Care Medicine at the University Medical Centre of Marburg (UKGM Marburg) have faced unique challenges in developing and establishing standards of care for …

Clinical And Echocardiographic Findings Of Intraoperative Inferior Vena Cava Obstruction Following Orthotopic Heart Transplantation, Montserrat Fontanals, Tam T Doan, Iki Adachi, Premal Trivedi

Faculty, Staff and Students Publications

No abstract provided.

Using Starling Murmuration As A Model For Creating A Global Health Community Of Practice To Advance Equity In Scholarship, Diane Nguyen, Rogers N Ssebunya, Kajal Hirani, Anna Mandalakas, Jennifer Benjamin, B Lee Ligon, Satid Thammasitboon

Faculty, Staff and Students Publications

Background: Disparities in scholarship exist between authors in low- or middle-income countries (LMIC) and high-income countries. Recognizing these disparities in our global network providing pediatric, adolescent, and maternal healthcare to vulnerable populations in LMIC, we sought to improve access and provide resources to address educational needs and ultimately impact the broader scholarship disparity.

Methods: We created a virtual community of practice (CoP) program underpinned by principles from starling murmuration to promote interdisciplinary scholarship. We developed guiding principles- autonomy, mastery and purpose- to direct the Global Health Scholarship Community of Practice Program. Program components included a continuing professional development (CPD) program, …

Tachycardia And Atrial Fibrillation-Related Cardiomyopathies: Potential Mechanisms And Current Therapies, Joshua A Keefe, Rebecca Garber, Mark D Mccauley, Xander H T Wehrens

Faculty, Staff and Students Publications

Atrial fibrillation (AF) is associated with an increased risk of new-onset ventricular contractile dysfunction, termed arrhythmia-induced cardiomyopathy (AIC). Although cardioembolic stroke remains the most feared and widely studied complication of AF, AIC is also a clinically important consequence of AF that portends significant morbidity and mortality to patients with AF. Current treatments are aimed at restoring sinus rhythm through catheter ablation and rate and rhythm control, but these treatments do not target the underlying molecular mechanisms driving the progression from AF to AIC. Here, we describe the clinical features of the various AIC subtypes, discuss the pathophysiologic mechanisms driving the …

A Review Of Shared Decision-Making, Published Protocols, And Post-Desensitization Strategies In Oral Immunotherapy (Oit), Susan Laubach, Edwin H Kim, Matthew Greenhawt, Sally Bailey, Aikaterini Anagnostou

Faculty, Staff and Students Publications

PURPOSE OF REVIEW: The aim of this review is to highlight key published oral immunotherapy (OIT) protocols and post-desensitization strategies for the major food allergens and to cover important concepts to consider when evaluating OIT for food-allergic patients. Shared decision-making should help identify patient and family values which will help influence the type of evidence-based protocol and maintenance strategy to use.

RECENT FINDINGS: With food OIT emerging as a treatment option, there is a pressing need for patients, physicians, and other providers to have a nuanced understanding of the management choices available to them. There are now randomized controlled trials …

Load703, An Oncolytic Virus-Based Immunostimulatory Gene Therapy, Combined With Chemotherapy For Unresectable Or Metastatic Pancreatic Cancer (Lokon001): Results From Arm 1 Of A Non-Randomised, Single-Centre, Phase 1/2 Study, Benjamin L Musher, Eric K Rowinsky, Brandon G Smaglo, Wasif Abidi, Mohamed Othman, Kalpesh Patel, Salmaan Jawaid, James Jing, Amanda Brisco, Ann M Leen, Mengfen Wu, Linda C Sandin, Jessica Wenthe, Emma Eriksson, Gustav J Ullenhag, Bambi Grilley, Justyna Leja-Jarblad, Susan G Hilsenbeck, Malcolm K Brenner, Angelica S I Loskog

Faculty, Staff and Students Publications

Background: Pancreatic ductal adenocarcinoma is characterised by low immunogenicity and an immunosuppressive tumour microenvironment. LOAd703, an oncolytic adenovirus with transgenes encoding TMZ-CD40L and 4-1BBL, lyses cancer cells selectively, activates cytotoxic T cells, and induces tumour regression in preclinical models. The aim of this study was to evaluate the safety and feasibility of combining LOAd703 with chemotherapy for advanced pancreatic ductal adenocarcinoma.

Methods: LOKON001 was a non-randomised, phase 1/2 study conducted at the Dan L Duncan Comprehensive Cancer Center, Baylor College of Medicine, Houston, TX, USA, and consisted of two arms conducted sequentially; the results of arm 1 are presented here. …