Medical Specialties Commons^™

Surgical Success Following Robotic Upper Urinary Tract Reconstruction, Results From A Global Network Of Healthcare Organizations, Zachary Prebay, Adam Schneider, Sohan S. Shah, Mauro Dispagna, Mihir S. Shah

Department of Urology Faculty Papers

INTRODUCTION: Available data on robotic ureteral reconstruction procedures is concentrated from high volume centers. To better understand generalizability of success, we used a network of healthcare organizations (HCOs) to evaluate surgical success following robotic-assisted pyeloplasty (RP), ureteral reimplantation (RR), and ureteroureterostomy/ureteroplasty (RU).

METHODS: We searched the TriNetX database for adult (>18 years old) patients undergoing RP, RR and RU. Our primary outcome was need for drain placement (nephrostomy or ureteral stent) from 4 weeks to 10 years postoperatively. We evaluated risk factors (RF) for drain placement (vascular disease, kidney disease, diabetes (DM), smoking history, radiation history, age, body mass …

Brief Report: Lung Cancer Diagnoses Among Lung Cancer Screening Program Participants With Family History Of Lung Cancer, Julia G. Katcher, Christine S. Shusted, Padmanabh Bhatt, Brooke M. Ruane, Jenna Markle, Gregory C. Kane, Kuang-Yi Wen, Hee-Soon Juon, Julie A. Barta

Division of Pulmonary, Allergy, and Critical Care Medicine Faculty Papers

Clinical Practice Points

• Among screening-eligible adults, lung cancer is diagnosed more often in those with family history of lung cancer
• Patients with family history had higher frequency of stage IV disease
• Considering family history in risk assessment could improve lung cancer screening strategies

Standardizing And Scaffolding Health Care Ai-Chatbot Evaluation: Systematic Review., Yining Hua, Winna Xia, David Bates, George Hartstein, Hyungjin Tom Kim, Michael Li, Benjamin Nelson, Charles Stromeyer Iv, Darlene King, Jina Suh, Li Zhou, John Torous

Department of Psychiatry and Human Behavior Faculty Papers

BACKGROUND: Health care chatbots are rapidly proliferating, while generative artificial intelligence (AI) outpaces existing evaluation standards.

OBJECTIVE: We aimed to develop a structured, stakeholder-informed framework to standardize evaluation of health care chatbots.

METHODS: PRISMA (Preferred Reporting Items for Systematic reviews and Meta-Analyses)-guided searches across multiple databases identified 266 records; 152 were screened, 21 full texts were assessed, and 11 frameworks were included. We extracted 356 questions (refined to 271 by deduplication and relevance review), mapped items to Coalition for Health AI constructs, and organized them with iterative input from clinicians, patients, developers, epidemiologists, and policymakers.

RESULTS: We developed the Health …

Pathways And Challenges In The Clinical Translational Of Radiopharmaceuticals For Pediatric Investigations, Erik Stauff, Hanieh Karimi, Heidi H. Kecskemethy, Thomas H. Shaffer, Reza Vali, Lauren W. Averill, Xuyi Yue

Department of Radiology Faculty Papers

Radiopharmaceutical development and clinical translation face numerous scientific, ethical, and regulatory challenges, particularly within the pediatric population. Although molecular imaging holds significant promise for improving diagnosis and treatment across a spectrum of diseases, including pediatric-specific conditions like Kawasaki disease, autism spectrum disorders, attention-deficit/hyperactivity disorder, and neuroblastoma, the path from discovery to clinical application remains problematic. The U.S. Food and Drug Administration (FDA) provides three primary pathways-traditional Investigational New Drug (IND) applications, exploratory Investigational New Drug application (eIND), and the Radioactive Drug Research Committee (RDRC) mechanism-to facilitate clinical translation of radiotracers. However, these frameworks are not specifically tailored to pediatric needs. …

Beyond Distraction: Music Therapy For Chronic Pain Management In People With Advanced Cancer, Joke Bradt, Lynn Gumert, Carrie Cottone, Ming Yuan Low, Clarissa Lacson, Kate Myers-Coffman, Karolina Bryl, Preethi Selvan, Jacelyn Biondo, Allison Millstein, Anna Cephas, Stephenie Sofield, Brooke Worster, Amy Leader

Department of Medical Oncology Faculty Papers

OBJECTIVE: Effective, non-pharmacological approaches to pain management for people with advanced cancer that can enhance quality of life while minimizing adverse effects are urgently needed. While music therapy has been demonstrated to be effective in reducing pain and improving pain-related outcomes across various patient populations, it has not been specifically examined in addressing chronic pain among people with advanced cancer. This study aims to enhance understanding of how people with advanced cancer experience music therapy for pain management.

METHODS: As part of a mixed methods intervention trial, we conducted semi-structured interviews with a subsample of 25 outpatients with advanced cancer …

Aberrant Expression Of A Disintegrin And Metalloproteinase With Thrombospondin Motifs 13 (Adamts13) In Pancreatic Cancer Leads To Dichotomic Functions, Stephanie Allmang, Hagen R. Witzel, Anne Hausen, Simone Marquard, Christoph Eckert, Nicole Marnet, Nina Hörner, Philipp Mayer, Stefan Heinrich, Hien Dang, Wilfried Roth, Matthias M. Gaida

Department of Surgery Faculty Papers

Pancreatic ductal adenocarcinoma (PDAC) is one of the most aggressive cancers characterized by highly invasive growth into the surrounding peripancreatic fat tissue, where tumor cells can directly interact with adipocytes. Due to poor response to the currently available (radio)chemotherapies, there is an urgent need for advanced therapy concepts. The present study shows that ADAMTS13 (a disintegrin and metalloproteinase with thrombospondin motifs 13), a key factor in blood coagulation, is significantly overexpressed in human PDAC. Immunohistochemical analysis revealed that ADAMTS13 expression is associated with prolonged survival and negatively correlated with vascular density. In vitro and in vivo experiments demonstrate its partial …

Use Of An Expert Panel For Symptomatic Patients With Grade I Degenerative Lumbar Spondylolisthesis: A Randomized Clinical Trial, Zoher Ghogawala, Tasneem Rizvi, Zhibang Lin, Adam Kanter, Praveen Mummaneni, Erica Bisson, Todd Albert, Daniel Resnick, Michael Wang, Steven Glassman, David Polly, Mohamad Bydon, Subu Magge, Luis Tumialan, Michael Fehlings, Michael Steinmetz, Robert Whitmore, Vedantam Rajshekhar, James Harrop, Roger Härtl, Elnasri Ahmed, Dom Coric, Paul Mccormick, Richard Assaker, Abdul Msaddi, Langston Holly, Yoshiharu Kawaguchi, Asdrubal Falavigna, Fred Barker, Edward Benzel

Department of Neurosurgery Faculty Papers

BACKGROUND AND OBJECTIVES: The appropriate utilization of lumbar fusion when performing laminectomy for lumbar spondylolisthesis is uncertain. The objective was to determine whether the use of a surgical expert review panel recommending fusion might improve patient selection, possibly reducing surgical failures.

METHODS: Randomized clinical trial of patients with symptomatic degenerative lumbar stenosis with spondylolisthesis enrolled from 14 North American hospitals was conducted with patients randomized to receive an expert panel review of their case or not. Spinal expert review consisted of 10 to 15 surgeons' review of patient images and clinical data with voting on the appropriateness of fusion. Primary …

Development Of A Clinically Relevant Rabbit Model Of Acute Laryngeal Injury, Ryan Stepp, Naushin Ali, Areli A. Rodriguez, Ethan Nicklow, Noah Thornton, Adithya Reddy, Hannah L. Kenny, Patrick S. Cottler, Donald R. Griffin, James J. Daniero

Department of Otolaryngology - Head and Neck Surgery Faculty Papers

OBJECTIVES: Acute laryngeal injury (ALgI) is created as a result of endotracheal tube pressure ulcer formation leading to fibrosis and inflammation. This condition often leads to airway obstruction and voice and swallowing dysfunction. This study demonstrates a reliable animal model of ALgI to reproduce the acute wound process seen clinically, to explore the pathophysiology of this disease process, and to serve as a reproducible injury suitable for the evaluation of therapeutic interventions.

METHODS: An ALgI model was developed in New Zealand White rabbits using precise mucosal stripping of the posterior larynx, followed by intubation with an oversized 4.0 endotracheal tube …

A Rare Case Of Chilaiditi Syndrome As A Driver Of Chronic Respiratory Failure, Paige C. Adams, Daniel Kramer

Division of Pulmonary, Allergy, and Critical Care Medicine Faculty Papers

Chilaiditi sign is a rare radiographic finding demonstrating interposition of the colon between the liver and the diaphragm; Chilaiditi syndrome is the manifestation of gastrointestinal or respiratory symptoms secondary to this finding. This syndrome rarely can be associated with acute and chronic respiratory failure. We report a case of a 66-year-old woman with chronic hypercapnic and hypoxemic respiratory failure due to severe restrictive pulmonary mechanics in the setting of Chilaiditi syndrome. Imaging showed an elevated right hemidiaphragm initially concerning for hemidiaphragm paralysis; however a fluoroscopic Sniff test suggested normal diaphragm function. Despite hospitalisation for aggressive bowel decompression combined with noninvasive …

Outcomes Of Relapse After Teclistamab Therapy In Multiple Myeloma, Kyle L. Yu, Matthew Ho, Luca Paruzzo, Federico Stella, Heta Patel, Zainul S Hasanali, Shivani Kapur, Adam J. Waxman, Dan T. Vogl, Edward A. Stadtmauer, Joseph A. Fraietta, Marco Ruella, Adam D. Cohen, Alfred L. Garfall, Sandra Susanibar-Adaniya

Student Papers, Posters & Projects

No abstract provided.

Evaluation Of Integrative Oncology Modalities For Symptom Management: A Mascc/Sio Global Survey, Alexandre Chan, Reem Nasr, Daniela Arcos, Dalia Kagramanov, Chioma Asuzu, Ting Bao, Yin Ting Cheung, Jung Hye Kwon, Judith Lacey, Richard T. Lee, Maryam Lustberg, Beatrice M. Ohaeri, Santosh Rao, Enrique Soto-Perez-De-Celis, Claudia M. Witt, Ana M. Lopez

Kimmel Cancer Center Faculty Papers

BACKGROUND: With growing evidence pointing towards the potential of integrative oncology modalities (IOM) in addressing cancer and cancer-treatment related symptoms, research on IOM utilization and implementation is warranted. This study examines global stakeholder perspectives on integrative oncology (IO) utilization for supportive cancer care.

METHODS: Members of the Multinational Association of Supportive Care in Cancer (MASCC) and the Society for Integrative Oncology (SIO) completed a survey on the utilization of IOM for supportive cancer care. Descriptive statistics were used to assess demographic data, IOM usage patterns, IOM education, and financial considerations for utilizing IOM.

RESULTS: Among 344 participants representing eight geographical …

Risk Of Late-Onset Depression In Long-Term Survivors Of Breast, Prostate, And Colorectal Cancer, Melissa Taylor, Sarah Westvold, Jessica Long, Terry Hyslop, Andrea Silber, Rebecca Forman, Faiza Yasin, Tendai Kwaramba, Shi-Yi Wang, Michael Leapman, Michael Cecchini, Ira Leeds, Lisa Spees, Stephanie Wheeler, Cary Gross, Kevin Oeffinger, Michaela Dinan

Kimmel Cancer Center Faculty Papers

IMPORTANCE: The prevalence of depression is higher in cancer survivors than in the general population. As the long-term cancer survivor population increases, it is increasingly important to understand risk factors for late-onset depression.

OBJECTIVE: To identify risk factors associated with late-onset depression in long-term (5-year) survivors of breast, prostate, or colorectal cancer.

DESIGN, SETTING, AND PARTICIPANTS: This retrospective cohort study used 2022 linkage of Surveillance, Epidemiology, and End Results (SEER) and Medicare data to identify 5-year survivors of breast, prostate, or colorectal cancer 66 years or older who were enrolled in fee-for-service Medicare and had no previous depression diagnosis. Long-term …

Human Kallikrein 2: A Novel Lineage-Specific Surface Target In Prostate Cancer, Fei Shen, Ryan Smith, Theresa Mcdevitt, Krista Menard, Shaozhou Tian, Gerald Chu, Ruchi Chaudhary, Jennifer Mccann, Halley Oyer, Sherry C. Wang, Steven Max, Peter Francis, William K. Kelly, Charles G. Drake

Kimmel Cancer Center Faculty Papers

PURPOSE: Targeted therapies for metastatic prostate cancer are limited, highlighting the need for novel drug targets and mechanisms of action (MoA). Human kallikrein 2 (KLK2) is a prostate-specific antigen expressed across the prostate cancer disease continuum. However, it was not recognized as a therapeutic target for prostate cancer in the past due to limited evidence of its cell surface expression. In this study, we systematically characterized KLK2 expression in prostate cancer, confirmed its cell surface expression, and demonstrated the preclinical efficacy of three KLK2-targeting therapeutics with distinct MoA.

EXPERIMENTAL DESIGN: The KLK2 expression profile in different stages of prostate cancer …

Genomic Profiling Of Intraocular Leiomyomas Reveals Recurrent Copy Number Alterations, Vivian Tang, Yubai Chou, Cuyan Demirkesen, Michele M. Bloomer, Joseph B. Crawford, Ahmet M. Sarici, Carol L. Shields, Ralph C. Eagle Jr., Codrin E. Iacob, Walter P. Devine, Tatyana Milman, Melike Pekmezci

Wills Eye Hospital Papers

PURPOSE: Leiomyomas are benign smooth muscle tumors that commonly present in the uterus, soft tissue, skin, and gastrointestinal tract but in rare cases can also arise within the eye. Notably, intraocular leiomyomas often show slightly different histopathologic and immunohistochemical features, referred to as mesectodermal morphology, given their presumed neural crest origin. Genetic and cytogenetic alterations of intraocular leiomyomas, as well as their association with various clinical and histopathologic features, have not been previously studied.

METHODS: We identified eight patients diagnosed with intraocular leiomyoma and performed targeted next-generation sequencing, whole transcriptome RNA sequencing, and chromosomal copy number analysis on those with …

National Alliance Of Sickle Cell Centers Consensus Recommendations On Sickle Cell Disease Health Maintenance: A Consensus Statement, Seethal A. Jacob, Melissa Frei-Jones, Sana Saif-Ur-Rehman, Monica L. Hulbert, Andrew R. W. O'Brien, Crawford Strunk, Anthony Villella, Ravi Talati, Amma Owusu-Ansah, Francis Coyne, Patrick T. Mcgann, Parul Rai, Robin Miller, Angeli Rampersad, Vishwas Sakhalkar, Neha Bhasin, Deepa Manwani, Sophie Lanzkron, Julie Kanter

Cardeza Foundation for Hematologic Research

IMPORTANCE: The most common inherited blood disorder in the world, sickle cell disease (SCD) is a known global health concern. While survival of children into adulthood has improved significantly over the last few decades in high-resource countries, substantial gaps in care standards and limited practice harmonization persist.

OBJECTIVE: To provide up-to-date recommendations for preventive care and treatment of all people living with SCD and its related comorbidities.

EVIDENCE REVIEW: As part of the National Alliance of Sickle Cell Centers (NASCC), 41 pediatric and adult sickle cell disease experts developed consensus standards and recommendations based on available evidence and expert consensus …

Avasopasem Manganese Treatment For Severe Oral Mucositis From Chemoradiotherapy For Locally Advanced Head And Neck Cancer: Phase 3 Randomized Controlled Trial (Roman), Carryn Anderson, Christopher M. Lee, Joseph Randall Kelley, Gary V. Walker, Neal E. Dunlap, Voichita Bar-Ad, Douglas A. Miller, Vernon J. King, Abhinand V. Peddada, Douglas F. Ciuba, Francois Vincent, Brian C. Muzyka, Amanda Lynn Gillespie-Twardy, Stephen T. Sonis, Jon Holmlund, Robert A Beardsley, Eugene P Kennedy, Deborah Saunders

Jefferson Hospital Staff Papers and Presentations

BACKGROUND: Of patients who receive standard concomitant chemoradiation (CRT; intensity-modulated radiation therapy [IMRT] plus cisplatin) for locally advanced head and neck cancer (HNC), approximately two-thirds will develop severe oral mucositis (SOM), limiting their ability to eat solids (WHO grade 3) or drink liquids (WHO grade 4). In a randomized, double-blind phase 2b trial, avasopasem manganese substantially reduced duration and incidence of SOM versus placebo. This phase 3 trial further assessed avasopasem's reduction of SOM due to CRT.

METHODS: In this double-blind, placebo-controlled trial (Clinicaltrials.gov: NCT03689712), patients receiving 60-72 Gy IMRT (>50 Gy to ≥2 oral mucosal sites) plus cisplatin …

J. Robert Gladden Orthopaedic Society Research Grants: What Is The Return On Invesments, Lancelot Benn, Susan Stephens, Mark Ehioghae, Rishi Balkissoon, Brendan A. Williams, Shaina Lipa, David N. Bernstein, Gbolabo Sokunbi, Barrett I. Woods, Wakenda Tyler, Jaysson Brooks, Randall C. Morgan, Kwadwo Owusu-Akyaw, Wayne Johnson, Eric Carson, George V. Russell, Addisu Mesfin

Rothman Institute Papers

INTRODUCTION: The J. Robert Gladden Orthopaedic Society (JRGOS) aims to increase diversity in orthopaedic surgery and promote high-quality musculoskeletal care for all. Through targeted research funding, JRGOS supports projects aligned with its mission. Despite awarding over $300,000 in research grants since 2011, the return on investment (ROI) of this program has not been analyzed. This study evaluates the ROI of JRGOS research grants from 2011 to 2023.

METHODS: We conducted a retrospective review of all JRGOS research grants awarded between 2011 and 2023. Data included recipient demographics, grant titles, award year, institution, and amount. PubMed and Google Scholar were used …

A House Divided: A Mixed Method Investigation Of Primary Care Interprofessional Teams Using The Core Principles And Values Of Teamwork Framework, Erin L. Kelly, Catherine Guariglia, Jeanne Felter, Dhruvi Shah, Brooke Mauriello, Amy Cunningham, Chris Arenson, Randa Sifri

Department of Family & Community Medicine Faculty Papers

Context

Primary care teams are facing increased demands, turnover, and escalating rates of burnout. A primary care team aligned in its core values and organized according to common teamwork principles is essential for delivering patient-centered, coordinated, and effective healthcare.

Methods

As part of a series of studies on workplace factors and burnout among primary care practice team members, we selected an urban practice with high rates of burnout to assess their teamwork via a practice-wide survey and six focus groups divided by role. The interview guide focused on identifying areas for potential intervention around teamwork. Transcripts were open-coded using a …

Porokeratosis Ptychotropica Mimicking A Herpes Simplex Virus Infection, Erin R. Pomerantz, Jayci G. Rhein, Jason B. Lee, Sylvia Hsu

Department of Dermatology and Cutaneous Biology Faculty Papers

Introduction

Porokeratosis encompasses a group of skin disorders characterized by abnormal keratinization, with the defining histologic feature of cornoid lamellae, which are stacks of parakeratotic cells that overlie dyskeratotic keratinocytes. Several subtypes of porokeratosis have been identified, including disseminated superficial actinic porokeratosis, porokeratosis of Mibelli, linear porokeratosis, porokeratosis palmaris et plantaris disseminata, punctate porokeratosis, and porokeratosis ptychotropica (PP).¹ Although risk factors for other forms of porokeratosis, such as UV exposure, immunosuppression, and genetic predisposition, have been identified, PP remains less understood. Clinically, PP typically presents as hyperkeratotic, verrucous plaques found bilaterally in the gluteal cleft and buttocks.^{2, …}

An Immunocompromised Patient With Cicatricial Alopecia, Dominique Revan, Victoria Stoj, Sherry Yang

Department of Dermatology and Cutaneous Biology Faculty Papers

Case description: A 37-year-old female with hemophagocytic lymphohistiocytosis presented with sudden onset hair loss that evolved over several hours during admission for neutropenic fever. Examination revealed a well-defined, tender, violaceous alopecic patch with intact follicular ostia (Fig 1). A punch biopsy showed ulcer with nonspecific changes. All fungal, bacterial, and mycobacterial stains were negative. During hospitalization, she was diagnosed with methicillin-resistant staphylococcus aureus (MRSA) bacteremia and discharged on intravenous daptomycin. She presented to the dermatology clinic 1 week later with worsening pain and hair loss, now with loss of follicular ostia.

Botulinum Toxin In Stiff Person Syndrome: How Useful Is It In Helping Muscle Stiffness And Spasms?, Marinos Dalakas

Department of Neurology Faculty Papers

No abstract provided.

Redefining Central Nervous System Multiple Myeloma: From Rare Phenomenon To Emerging Entity, Ian Landry, Adam F. Binder, Marc Yorker, Sarah Ramirez

Department of Medical Oncology Faculty Papers

BACKGROUND: Central nervous system involvement in multiple myeloma (CNS-MM) is considered a rare but devastating manifestation of extra-medullary disease (EMD) associated with aggressive biology and dismal outcomes. Treatment advances over the past few decades have improved survival in patients with MM, however, it has not translated into a similar survival benefit for those with CNS-MM. Early recognition and targeted management of CNS-MM remain major clinical challenges.

OBJECTIVE: This literature review summarizes the current literature on epidemiology, risk factors, clinical features, diagnostic modalities, and therapeutic approaches to CNS-MM. It focuses on emerging biomarkers and new treatment options that may affect the …

Reply To Fernández Et Al. Comment On "Goudra Et Al. Anesthesia For Bronchoscopy-An Update, Basavana Goudra, Lalitha Sundararaman, Prarthna Chandar, Michael Green

Department of Medicine Faculty Papers

No abstract provided.

Cmc Arthroplasty For Treatment Of The Painful And Arthritic Thumb: A Review Of Surgical Treatment Options With Preferred Technique, Jon E. Hammarstedt, Yehuda A. Masturov, Sebastian D. Arango, Jacob Zeitlin, Samuel H. Payne, Mark S. Rekant

Department of Medicine Faculty Papers

Arthritis of the first carpometacarpal joint is a prevalent and often disabling condition for many older adults. The diagnosis is typically established through a comprehensive patient history, physical examination, and confirmatory radiographs. Nonsurgical management with splinting and anti-inflammatory medication has demonstrated effectiveness. Several surgical options have proven effectiveness for those who fail nonsurgical management. Surgical options include silicone implantation, resection arthroplasty, tendon interposition, ligament reconstruction, denervation, and joint implant arthroplasty. In the following review, we will elaborate upon the array of therapeutic approaches, culminating in the delineation of our preferred method.

Patient Satisfaction Among Opioid Use Disorder Treatment Sample In An Opioid Treatment Program: A Mixed Method Research Study, Stanley Nkemjika

Department of Psychiatry and Human Behavior Faculty Papers

BACKGROUND/OBJECTIVES: Patient satisfaction is increasingly recognized as a key indicator of the effectiveness of substance use disorder (SUD) treatment. While some studies suggest disparities in satisfaction across treatment settings, there remains limited research examining these differences, particularly among vulnerable populations. This study aimed to assess patient perceptions of satisfaction with opioid use treatment services and explore how demographic and socioeconomic factors influence these experiences.

METHODS: Conducted between 1 February and 31 March 2025, the study took place at a longstanding Opioid Treatment Program in Philadelphia. A total of 217 participants receiving treatment were recruited through convenience sampling during routine clinic …

Multinucleate Cell Angiohistiocytoma, Connor A. Sheehan, Jayci G. Rhein, Jordan Hyde, Jason B. Lee, Sylvia Hsu

Department of Dermatology and Cutaneous Biology Faculty Papers

No abstract provided.

Does Cognitive Frame Affect Clinical Reasoning Among Medical Students? A Cross-Sectional Study, Xiao C. Zhang, Sara A. Belko, Andrew J Monick

Department of Emergency Medicine Faculty Papers

Background Diagnostic errors affect patient safety and lead to high costs. Cognitive bias is a key source of diagnostic error, and the framing effect poses a particular clinical challenge. The extent to which individual factors affect susceptibility to the framing bias is unclear, and the role of expertise in particular is contested across the literature. Objectives This study investigated the effects of framing bias on diagnostic reasoning among medical students. We hypothesised that the effects attributable to framing would be mediated by the number of years of medical education completed. Methods Participants were randomly assigned on an automated basis with …

Long-Term Liver-Targeted Aav8 Gene Therapy For Mucopolysaccharidosis Iva, Shaukat A. Khan, Eliana Benincore-Florez, Fnu Nidhi, Jose Victor Álvarez, Dione A. Holder, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidosis IVA (MPS IVA) is a lysosomal storage disease with an autosomal recessive trait caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS) enzyme, which leads to the accumulation of chondroitin-6-sulfate and keratan sulfate, primarily in cartilage and its extracellular matrix, resulting in a direct impact on cartilage and bone development, as well as subsequent systemic skeletal dysplasia. ERT and HSCT are current treatment options, but they have a limited effect on bone lesions. In this article, we investigated liver-specific AAV8 vectors with a thyroxine-binding globulin promoter in the MPS IVA murine model to evaluate the long-term (24 weeks in males …

Managing Anticoagulation In A Patient With Calciphylaxis And Mechanical Valve: A Therapeutic Dilemma, Qutaiba Qafisheh, Abdalhakim Shubietah, Mohanad Qwaider, Muath A. Baniowda, Abdalrahman Assaassa, Roaa Aljunaidi, Abubakar Nazir

Division of Cardiology Faculty Papers

BACKGROUND: Calciphylaxis is a rare, life-threatening vasculopathic syndrome most commonly seen in end-stage renal disease (ESRD) and associated with high mortality. Vitamin K antagonists complicate management in patients with mechanical valves by potentially exacerbating vascular calcification while remaining essential for thromboprophylaxis.

CASE PRESENTATION: A 50-year-old woman with ESRD on hemodialysis and a mechanical mitral valve on warfarin presented with severe abdominal pain and rapidly progressive necrotic ulcers. Despite broad-spectrum antibiotics and sodium thiosulfate, lesions worsened. Biopsies were nondiagnostic; however, the clinical phenotype was consistent with calciphylaxis. A multidisciplinary team transitioned anticoagulation from warfarin to subcutaneous unfractionated heparin (UFH) (333 U/kg …

Use Of Emr Automated Fib-4 As A Tool For Masld Screening In The Primary Care Setting, Maria Winte, Md, Rebecca Loh, Md, Sanjna Shelukar, Md, Ananya Venkatesh, Md, Bryan O'Connell, Md, Dina Halegoua-De Marzio, Md

Division of Gastroenterology and Hepatology Posters

Introduction

• MASLD affects up to 40% of the population in North America and is increasing.
• Screening for advanced fibrosis is now widely recommended, including formal guidelines from the AGA, AASLD, and ADA.
• We implemented an automated Fibrosis-4 (FIB-4) Index for liver fibrosis screening as a tool within our electronic medical record (EMR) to increase screening in patients with risk factors for MASLD.
• Our study examines the impact of this intervention on the appropriate MASLD screening rates among 50–65- year-old patients in the primary care clinic.