Digital Commons Network^™

Hes V2.0 Validation And Performance Compared To Galad And Asap In The Heds Cohort, Hashem B El-Serag, Camden Lopez, Michelle Luster, K Rajender Reddy, Neehar Parikh, Amit G Singal, Jorge A Marrero, Aaron P Thrift, Jagpreet Chhatwal, Ziding Feng, Stephanie Page-Lester, Qingchun Jin, Nabihah Tayob, Fasiha Kanwal

Faculty, Staff and Students Publications

Background & aims: We previously developed HES V2.0, a biomarker panel (age, ALT, platelets, etiology, AFP, AFP-L3, DCP, and their 1-year gradients) for early detection of hepatocellular carcinoma (HCC) among patients with cirrhosis. We externally validated HES V2.0 and compared its performance with HES V1.0, GALAD, and ASAP.

Methods: We conducted a PRoBE (prospective specimen-collection, retrospective blinded-evaluation) cohort study in the HEDS (Hepatocellular Carcinoma Early Detection Strategy) cohort of 1,485 patients with cirrhosis, of whom 119 developed HCC. Patient- and test-level true positive rates (TPR) for HCC were calculated at 6, 12, and 24 months before diagnosis, using thresholds at …

Health Care Worker-Reported Barriers And Potential Facilitators Of Acute Lower Respiratory Infection Care Deliver For Children At Mchinji District Hospital In Malawi, Shubhada Hooli, Beatwil Zadutsa, Everlisto Phiri, Karina Hofstee, Nichole Davis, Eric D Mccollum, Charles Makwenda, Carina King

Faculty, Staff and Students Publications

Objectives: We sought to understand barriers to high-quality hospital-based child acute lower respiratory infection (ALRI) care at Mchinji District Hospital in Malawi.

Methods: In 2020, we conducted focus group discussions (FGDs) with clinical officers (COs) who provided direct clinical care after a half-day refresher course on pediatric ALRI case management. The underpinning research methodology of the FGDs was phenomenology, and they were analyzed using inductive and deductive thematic analysis.

Results: We recruited 16 COs to participate in three FGDs. Five themes emerged: lack of confidence in ALRI diagnosis and management, high clinical burden with understaffing, dysfunctional team dynamics, limited physical …

Predominant Symptom In Gastroparesis: Relationships To Quality Of Life, Treatments, And Outcome, Henry P Parkman, Laura A Wilson, William L Hasler, Madhusudan Grover, Bruno P Chumpitazi, Helen Burton-Murray, Irene Sarosiek, Baha Moshiree, Geoffrey A Preidis, Robert J Shulman, Laura A Miriel, Dave Shade, Braden Kuo, Richard W Mccallum, Thomas L Abell, Pankaj J Pasricha

Faculty, Staff and Students Publications

Background: Patients with gastroparesis (Gp) can have a variety of symptoms. Apart from use of prokinetics, symptomatic therapies for the predominant symptom (PrS) are used.

Aims: (1) To classify Gp patients based on their PrS; (2) To determine differences in Gp symptoms, gastric emptying (GE), quality of life (QOL), treatments, and symptom improvement among Gp patients with different PrS.

Methods: Gp patients were enrolled in a multicenter registry and followed for 96-weeks. Patients recorded their PrS and completed questionnaires assessing symptoms and QOL at enrollment and at follow up visits.

Results: Of 1013 Gp patients (361 diabetic, 607 idiopathic, 45 …

Linkage Disequilibrium Score Regression Identifies Genetic Correlations Between Hepatocellular Carcinoma And Clinically Relevant Traits, Younghun Han, Vikram R Shaw, Jinyoung Byun, Aaron P Thrift, Catherine Zhu, Donghui Li, Rikita I Hatia, Robin Kate Kelley, Sean P Cleary, Anna S Lok, Paige M Bracci, Jennifer B Permuth, Roxana Bucur, Jennifer Knox, Jian-Min Yuan, Amit G Singal, Prasun K Jalal, R Mark Ghobrial, Yuko Kono, Dimpy P Shah, Mindie H Nguyen, Neehar D Parikh, Richard Kim, Hui-Chen Wu, Hashem El-Serag, Ping Chang, Yun Shin Chun, Jian Gu, Chad Huff, Asif Rashid, Lu-Yu Hwang, Alison P Klein, Saira A Khaderi, Ahmed O Kaseb, Kathrine A Mcglynn, Lewis R Roberts, Manal M Hassan, Christopher I Amos

Faculty, Staff and Students Publications

Hepatocellular carcinoma (HCC) mortality is increasing globally, partly due to the growing prevalence of nonviral liver diseases. Genome-wide association studies (GWAS) have identified genetic variants associated with HCC development. Leveraging GWAS summary statistics and linkage disequilibrium score regression (LDSR), we investigated disease co-development with hepatitis C virus-negative (HCV-negative) HCC to provide unique insights into HCC etiology and prioritize relationships for further causal inquiry. We utilized the LDSR statistical framework to estimate the genetic correlation and heritability between HCV-negative HCC with 901 epidemiologic, behavioral, and clinical traits from the United Kingdom Biobank (UKBB). First, we set the threshold for observed scale …

Preserving Residual Kidney Function In Haemodialysis: Current Practices, Challenges, And Perspectives From Renal Clinicians, Lijun Zhong, Ulrich Steinwandel, Hugh Davies, Sara Bayes, Jagadish Jamboti, Hemant Kulkarni, Doris Chan

Research outputs 2022 to 2026

Background: Residual kidney function in patients receiving haemodialysis is associated with improved survival, reduced cardiovascular risk and better quality of life. Yet, clinical guidelines on its preservation are limited, and local practices remain unclear. Objectives: To explore current practices, perceptions and challenges related to residual kidney function preservation among renal clinicians. Design: Descriptive cross-sectional survey. Participants: Renal physicians and nurses working in hospital-based haemodialysis units across Western Australia. Measurements: Role-specific questionnaires were administered to renal physicians and nurses. Results: Eighty-four completed responses were received (physicians n = 25; nurses n = 59). Over half of physicians (56%, n = 14) …

Clinical Insights Into The Management Of Sleep Disturbances Within Cancer Care: A Qualitative Analysis, Sam Adams, Timothy D. Clay, Mitchell Turner, Christopher Kueh, Kelly Moes, Travis Cruickshank

Research outputs 2022 to 2026

Purpose: The present study aimed to gain a deeper understanding of how oncology healthcare professionals’ (HCPs) manage sleep disturbances. Methods: Semi-structured interviews were conducted with 10 oncology HCPs (medical oncologists, oncology nurse practitioners, clinical nurse consultants) working in metropolitan Perth, Western Australia. Reflexive thematic analysis was used to understand oncology HCPs’ perspectives on treating sleep disturbances, explore challenges and barriers to their management, as well as opportunities for improvement. Results: Four main themes were developed: (1) sleep disturbances are underreported and underassessed, (2) poor sleep can be difficult to manage, (3) limited capacity to address sleep issues, and (4) opportunities …

Pleural Metastases From Acral Lentiginous Melanoma Diagnosed By Medical Thoracoscopy, Ad Rian Chong, Khai Lip Ng, Nai-Chien Huan, Nur Husna Mohd Aminudin, Fazilah Hassan, Kasuma Mohamed Nordin

Research outputs 2022 to 2026

Acral lentiginous melanoma (ALM) with pleural metastases is rare and under-recognised. We report an 86-year-old woman with progressive dyspnoea and pleuritic chest pain. 18 months ago, she was diagnosed with stage IIC ALM of the right heel, and was treated with wide local excision, but she declined adjuvant therapy. Imaging demonstrated a large left pleural effusion with diffuse pleural thickening, pulmonary mass, and hepatic metastases. Brownish pleural fluid, along with thoracoscopic identification of multiple pigmented nodules, led to a histologically confirmed diagnosis. Talc pleurodesis was performed for symptom control, and best supportive care was pursued. This case highlights the diagnostic …

Skeletal Muscle Health In Childhood Cancer Survivors: A Systematic Review And Meta-Analysis, Anna Maria Markarian, Dennis R. Taaffe, Daniel A. Galvão, Carolyn J. Peddle-Mcintyre, Jodie Cochrane Wilkie, Francesco Bettariga, Nicholas G. Gottardo, Mayank Dhamija, Santosh Valvi, Catriona M. Buchanan, Kerrie Graham, Robert U. Newton

Research outputs 2022 to 2026

Purpose: Childhood cancer survivors (CCS) are at risk of long-term skeletal muscle deficits following intensive therapies during critical periods of growth. This review aimed to synthesize approaches for assessing muscle quantity, quality, and function in CCS and to quantify deficits relative to healthy peers. Methods: A systematic search was conducted in CINAHL, Embase, PubMed, SPORTDiscus, and Web of Science from inception to June 2024, with an update in November 2025. Studies including CCS who had completed cancer treatment and reported measures of muscle quantity, quality, or physical function were eligible. A three-level mixed-effects model meta-analysis was conducted. Associations between muscle …

Vibostolimab Coformulated With Pembrolizumab Versus Pembrolizumab Alone As Adjuvant Therapy For High-Risk Stage Iib–Iv Melanoma (Keyvibe-010): A Randomised, Double-Blind, Phase 3 Study, Reinhard Dummer, Jun Guo, Jason J. Luke, Matteo S. Carlino, Dirk Schadendorf, Muhammad A. Khattak, Axel Hauschild, Paolo A. Ascierto, Yu Chen, Sang Joon Shin, Piotr Rutkowski, Zhiguo Luo, Jing Chen, Gareth Rivalland, Corlia Coetzee, Antoni Ribas, Karmele Mujika, Gal Markel, Rodrigo U. Villarroel, Omer Dizdar, Christian Caglevic, Dmitri Grebennik, Kevin Donovan, Clemens Krepler, Georgina V. Long

Research outputs 2022 to 2026

Background Combination therapy with vibostolimab plus pembrolizumab has previously shown promising antitumor activity in melanoma. We aimed to evaluate the efficacy and safety of vibostolimab coformulated with pembrolizumab as adjuvant therapy for high-risk resected melanoma. Methods This randomised, double-blind, phase 3 study was done at 205 global sites (hospitals and cancer centres). Participants aged 12 years or older with surgically resected, stage IIB–IV cutaneous melanoma per the American Joint Committee on Cancer Cancer Staging Manual 2017 (8th edition), with no evidence of metastatic disease after resection, were randomly assigned (1:1) to receive vibostolimab 200 mg coformulated with pembrolizumab 200 mg …

Work Challenges And Cancer-Related Cognitive Impairment: Level Of Education Accounts For Unmet Needs Beyond Cognitive Impairment Severity, Darren Haywood, Susan L. Rossell, Ashley Henneghan, Frank D. Baughman, Jennifer Haywood, Evan Dauer, Annika Hegde, Ahmed A. Moustafa, Nicolas H. Hart

Research outputs 2022 to 2026

Purpose: Cancer-related cognitive impairment (CRCI) can impact cancer survivors’ return to work and occupational functioning for many years following the completion of cancer treatment. Understanding the characteristics of CRCI-related occupational difficulties and unmet supportive care needs is critical for the development of effective interventions. Within occupational roles, a higher level of education is typically associated with greater cognitive load and different demands. The objective of this study was to examine whether the level of education accounted for CRCI-related (a) occupational difficulties and (b) occupational unmet supportive care needs, beyond the contributions of perceived cognitive functioning and employment level. Methods: A …

The Emerging Role Of Tertiary Lymphoid Structures As Predictive And Prognostic Biomarkers Of Immunotherapy In Cancer, Lidia B. Medhin, Rodas Amanuel, Lydia Warburton, Leslie Calapre, Benhur Amanuel, Elin S. Gray

Research outputs 2022 to 2026

Background: Immune checkpoint inhibitors (ICIs) have improved outcomes across several cancers, yet many patients do not respond, highlighting the need for robust predictive biomarkers. Tertiary lymphoid structures (TLS), ectopic lymphoid aggregates that support local antigen presentation and adaptive immune activation, have emerged as potential indicators of favourable prognosis and immunotherapy responsiveness. Methods: This review summarises current clinical and translational evidence examining the prognostic and predictive value of TLS in solid malignancies. Studies assessing TLS presence, organisation, and biological function were identified through searches of major scientific databases and evaluated with respect to their association with patient outcomes and responses to …

The Role Of Top-Down Appetite Self-Regulation In The Development Of Healthy Eating Behaviors Among Children: A Narrative Review And Socialization Framework, David J Bridgett, Sheryl O Hughes, Matthew Broussard, Daniela Mccourt, Christina M Croce, Jennifer O Fisher

Faculty, Staff and Students Publications

Appetite self-regulation (ASR) among children is thought to have a fundamental role in shaping the development of healthy eating behaviors, dietary intake, and growth during childhood. Parallel to developmental frameworks for understanding "general" self-regulation among children, ASR has been described as involving children's use of "top-down" cognitive processes to moderate "bottom-up" biological drives around food approach and avoidance in the interest of achieving desired eating behaviors or outcomes. Whereas bottom-up ASR processes during early childhood are well characterized, particularly in the context of dysregulation and obesity risk, the role of top-down ASR processes in the development of healthy eating behaviors …

Surgical Repair Of Double-Outlet Right Ventricle With Doubly Committed Juxtaarterial Ventricular Septal Defect: The Role Of Virtual Reality, Fumiya Yoneyama, Jonathan Awori, Shagun Sachdeva, Kaimal A Jayakumar, Javier Brenes, Jeffery S Heinle

Faculty, Staff and Students Publications

Double-outlet right ventricle (DORV) with a doubly committed juxtaarterial ventricular septal defect (VSD) presents significant surgical challenges. We report a neonate with DORV, a hypoplastic aortic arch, and a patent ductus arteriosus. Virtual reality (VR) imaging provided detailed visualization, confirming the VSD's position beneath both great arteries. Intraventricular rerouting was unfeasible given the patient's small systemic structures and tricuspid valve chordae. The patient underwent successful VSD closure through a transarterial approach, arterial switch operation, and arch repair. This case highlights the utility of VR-based planning in optimizing surgical strategy for complex congenital heart defects.

Cardiac Catheterization In American Indian/Alaska Native Populations: Empowering Communities, Providers, And Systems Toward Equity, Joseph Burns, Daniel J Penny, Keila N Lopez, Lindsay F Eilers, Athar M Qureshi, Christine J Chung, Jason F Deen

Faculty, Staff and Students Publications

American Indian/Alaska Native (AI/AN) people face numerous health disparities, including alarming rates of cardiovascular morbidity and mortality. These inequities are closely tied to unique social, political, and economic circumstances due to the ongoing consequences of systemic racism. Although poorly described, these cardiovascular inequities extend to adult and pediatric interventional cardiology. AI/AN adults are less likely to undergo cardiac catheterization and are more likely to experience complications. There are no published studies on pediatric interventional cardiology outcomes in AI/AN children. The scarcity of available data underscores the need for novel studies to investigate the epidemiology, sociodemographic associations, and outcomes of AI/AN …

Rural-Urban Differences In Use Of Mental Health Care For Serious Mental Illness At The Veterans Health Administration: A Retrospective Cohort Analysis, Alexandra B Caloudas, Amber B Amspoker, Ashley Smith, Liang Chen, Amy K Cuellar, Giselle Day, Alexandra Zingg, Julianna Hogan, Jan A Lindsay

Faculty, Staff and Students Publications

PURPOSE: Rural Veterans with serious mental illness (SMI) encounter significant barriers to accessing mental health care, related to SMI symptoms and rurality. A 2023 Government Accountability Office report found that rural Veterans used Veterans Health Administration (VHA) intensive mental health care for SMI less than urban Veterans relative to their population distributions. Our purpose was to deepen understanding of rural-urban differences in use of VHA mental health care among Veterans with SMI.

METHODS: Using VHA databases, we conducted a retrospective cohort analysis of 387,477 Veterans diagnosed with SMI, examining rurality as a predictor of intensive care for SMI and other …

Spatial Profiling Reveals Distinct Molecular And Immune Evolution Of Mouse Lung Adenocarcinoma Precancers With Or Without Carcinogen Exposure, Bo Zhu, Muhammad Aminu, Pingjun Chen, Jian-Rong Li, Chuanpeng Dong, Chenyang Li, Yanhua Tian, Shao-Wei Lu, Hong Chen, Chenxi Ma, Xin Hu, Jie Ye, Andrew Y Liu, Beibei Huang, Frank R Rojas, Parra Cuentas Edwin Roger, Ou Shi, Monique B Nilsson, Alissa Poteete, Khaja B Khan, Wei Lu, Luisa M Solis Soto, Junya Fujimoto, Cara Haymaker, Ignacio I Wistuba, Zhubo Wei, Linghua Wang, Don L Gibbons, Ken Chen, Alexandre Reuben, Jason M Schenkel, John V Heymach, Chao Cheng, Jia Wu, Jianjun Zhang

Faculty, Staff and Student Publications

Tumor evolution involves genetic, transcriptional, and phenotypic alterations that shape cancer cell behavior and interactions with the microenvironment. While single‐cell technologies have advanced our understanding of this process, spatial dynamics remain incompletely characterized. Here, whole‐exome sequencing (WES), imaging mass cytometry (IMC), and spatial transcriptomics (ST) were integrated to study molecular evolution and immune responses in two lung adenocarcinoma (LUAD) mouse models: a genetically engineered model (129S4/Sv‐KrasLSL‐G12D, termed 129S4 K) and a carcinogen‐induced precancer model (129S4 U). Compared to 129S4 K, 129S4 U tumors exhibited higher mutational, neoantigen but lower copy number variation (CNV) burdens at matched developmental timepoints, consistent with …

Effect Of Dental Treatments On Reduction Of Preterm Birth: A Systematic Review And Meta-Analysis, Camille Thomas, Shakil Ahmed, Vincenzo Berghella, Romina Brignardello-Petersen, Mohamed El-Rabbany, Catherine Devion, Stefania Ronzoni

Department of Obstetrics and Gynecology Faculty Papers

OBJECTIVE: Periodontal disease is associated with increased risk of preterm birth. Effective management during pregnancy may reduce preterm birth, though clinical trial evidence has been inconsistent. This study aimed to assess whether treating dental disease during pregnancy reduces preterm birth.

DATA SOURCES: A systematic search was performed of EMBASE, MEDLINE, PubMed, Cochrane Library, and trial registries up to December 2023 and re-run in January 2025.

STUDY ELIGIBILITY CRITERIA: Randomized controlled trials (RCTs) enrolling pregnant persons with any dental disease randomized to receive dental treatment vs no treatment were included. No language restriction was applied.

STUDY APPRAISAL AND SYNTHESIS METHODS: Data …

Reply To The Letter-To-Editor On Article "Evaluation Of Left Ventricular Function In Coronary Artery Ectasia: The Added Value Of 2d Speckle-Tracking Echocardiography", Osama Elshaer

Journal of the Saudi Heart Association

No abstract provided.

In Vitro Studies Of The Effects Of Antithrombotic Zn-Dipicolylamine-Harboring Liposomes (Dpals) On Serum Albumin And Human Umbilical Vein Endothelial Cells, Michelle Tanujaya, Gianna Cai, Jia Patel, Zana Moldavsky, Yumna Ejaz, Malia Mahazabin Ahmed, Sangsang Duong, Lawrence E. Goldfinger, Koon Y. Pak, Brian D. Gray, Parkson Lee-Gau Chong

Cardeza Foundation for Hematologic Research

Thrombosis remains a leading cause of cardiovascular morbidity and mortality. During thrombosis, activated platelets and endothelial cells expose phosphatidylserine (PS) on their outer membranes, creating a surface that accelerates clot formation. Current antithrombotic therapies, such as heparin and warfarin, carry significant bleeding risks, highlighting the need for safer alternatives. In response, we developed a PS-targeting liposomal formulation composed of Zn-dipicolylamine (DPA)-cyanine-3[22,22] and 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphocholine (molar ratio 3:97). This DPA-harboring liposome (DPAL) binds selectively to PS-rich surfaces such as activated platelets and has demonstrated efficacy in reducing thrombosis in mouse models, with minimal bleeding. In the present study, we examined the interaction …

Alpha And Beta Emitters In Translational Nuclear Medicine: Clinical Advances, Challenges, And Future Direction, Hanieh Karimi, Thomas H. Shaffer, Erik Stauff, Vinay V. R. Kandula, Heidi H. Kecskemethy, Lauren W. Averill, Xuyi Yue

Department of Radiology Faculty Papers

Radiopharmaceutical therapy (RPT) has emerged as a transformative modality in oncology, particularly for patients with metastatic or inoperable tumors. By leveraging molecularly targeted carriers conjugated to cytotoxic radionuclides, RPT enables precise delivery of ionizing radiation to tumor sites while minimizing off-target effects. Central to this approach are alpha (α) and beta (β) particle-emitting radionuclides. This review aims to provide a comprehensive overview of all clinically relevant alpha and beta emitters and incorporates the most recent advances from 2017-2025, offering a comprehensive and up-to-date perspective. Alpha and beta emitters hold significant promises for the future, especially in nuclear medicine, energy, and …

Prenatal Stress Induces Changes In Behavior, Hpa Axis, Inflammation, And Oxidative Stress In Adult Rats Offspring, Jorge M Aguiar-Geraldo, Jefté Peper-Nascimento, José Henrique Cararo, Taise Possamai-Della, Alexandra I Zugno, Anilkumar Pillai, João Quevedo, Samira S Valvassori

Faculty, Staff and Student Publications

Prenatal stress is related to the development of psychiatric disorders involving inflammation, oxidative stress, and hypothalamic-pituitary-adrenal (HPA) axis. Therefore, the aim of the present study was to evaluate the effects of prenatal stress on behavior, inflammation, oxidative stress, and the HPA-axis in the dams and their offspring treated with lithium. Thirteen pregnant Wistar rats were exposed to a prenatal chronic unpredictable stress protocol from the 14th day of gestation until birth. At the 60th postnatal day (PND), a treatment protocol was carried out in the offspring with lithium (intraperitoneally - 47.5 mg/kg) or saline for seven days (twice a day). …

Profiling The Genomic Landscape And Evolutionary History Of Polyploid Giant Cancer Cells In Undifferentiated Pleomorphic Sarcomas, Amy L Bowes, Sara Waise, Tom Lesluyes, Thomas Butters, Christie English, Haixi Yan, Annelien Verfaillie, Christopher Davies, Jianan Chen, Emma Nye, Richard Stone, Jiten Manji, Adrienne M Flanagan, Jonas Demeulemeester, Maxime Tarabichi, Nischalan Pillay, Peter Van Loo

Faculty, Staff and Student Publications

Polyploid giant cancer cells (PGCCs), characterised by multinucleation and atypical nuclear morphology, are a common feature of undifferentiated pleomorphic sarcomas. While PGCCs may be a critical substrate for cancer evolution, their formation pathways and genomic consequences remain underexplored. In this study, we characterise PGCCs in ten pleomorphic sarcomas and use topographic single-cell DNA sequencing (scDNA-seq) to investigate their genomic landscape. We selected PGCCs based on their nuclear morphology, including mononucleated or multinucleated bizarre, misshapen nuclei, and analysed them at single-cell resolution. Histopathological analysis showed that PGCCs were often randomly distributed throughout the tumour and did not appear in clusters, suggesting …

Profiling The Genomic Landscape And Evolutionary History Of Polyploid Giant Cancer Cells In Undifferentiated Pleomorphic Sarcomas, Amy L Bowes, Sara Waise, Tom Lesluyes, Thomas Butters, Christie English, Haixi Yan, Annelien Verfaillie, Christopher Davies, Jianan Chen, Emma Nye, Richard Stone, Jiten Manji, Adrienne M Flanagan, Jonas Demeulemeester, Maxime Tarabichi, Nischalan Pillay, Peter Van Loo

Faculty, Staff and Student Publications

Polyploid giant cancer cells (PGCCs), characterised by multinucleation and atypical nuclear morphology, are a common feature of undifferentiated pleomorphic sarcomas. While PGCCs may be a critical substrate for cancer evolution, their formation pathways and genomic consequences remain underexplored. In this study, we characterise PGCCs in ten pleomorphic sarcomas and use topographic single-cell DNA sequencing (scDNA-seq) to investigate their genomic landscape. We selected PGCCs based on their nuclear morphology, including mononucleated or multinucleated bizarre, misshapen nuclei, and analysed them at single-cell resolution. Histopathological analysis showed that PGCCs were often randomly distributed throughout the tumour and did not appear in clusters, suggesting …

Aav Gene Therapy For Mps Iva With Induction Of Immune Tolerance Via Oral Administration Of Epitope Peptides Of N-Acetylgalactosamine-6-Sulfate Sulfatase, Sampurna Saikia, Yasuhiko Ago, Fnu Nidhi, Shaukat Khan, Zhengyu Ma, Shunji Tomatsu

Department of Pediatrics Faculty Papers

Mucopolysaccharidosis IVA (MPS IVA) is caused by the accumulation of undegraded glycosaminoglycans due to the deficiency of the N-acetylgalactosamine-6-sulfate sulfatase (GALNS) enzyme. MPS IVA manifests as progressive systemic skeletal dysplasia. Gene therapy (GT) is potentially a one-time treatment in which the enzyme is continuously produced, circulated, and delivered to target tissues. However, immune responses to gene products can diminish therapeutic efficacy. We hypothesized that oral delivery of tolerogenic peptides induces immune tolerance to human GALNS (hGALNS) in MPS IVA mice, enhancing therapeutic efficacy. Neonatal mice deficient in mouse GALNS (mGALNS) were treated orally with three T-cell/B-cell epitope peptides or hGALNS …

From Stemi To Saddle Pulmonary Embolism: Management Challenges And The Role Of Va-Ecmo, Enad Haddad, Irfan Ahsan, Sudeep Nugooru, Gregary D. Marhefka

Division of Internal Medicine Faculty Papers & Presentations

Acute pulmonary embolism and acute myocardial infarction are critical conditions with overlapping presentations, and their coexistence can be catastrophic. We report a 45-year-old man with prior mid LAD stent who presented with chest pain and ventricular fibrillation arrest, diagnosed with inferior/posterior STEMI. Emergent PCI to the mid RCA restored stability. The patient developed persistent hypoxia over the next two days requiring intubation and mechanical ventilation. Worsening hypoxia and shock prompted urgent transesophageal echocardiography to evaluate for mechanical complications, instead revealing a mobile echo density spanning the right and left main pulmonary arteries, consistent with acute saddle pulmonary embolism, and right …

Benign Transient Hyperphosphatasemia In Adults: Case Report And Literature Review, Jose L. Rodriguez Castro, Grettel Gonzalez Garcia, Daniel Heller, Bernardo J. Reyes

HCA Healthcare Journal of Medicine

Background

Benign transient hyperphosphatasemia (BTH) in children is characterized by temporary highly elevated serum alkaline phosphatase (ALP) activity in the absence of liver or bone disease and a return to normal within 3 to 4 months. Since its first description in the 1950s, several cases of BTH in infants and children have been reported. Although there is no known etiology for this illness, it has been associated with viral infections. This condition has rarely been seen in the adult population, and there is scarce available information. The sialylation of the ALP isoenzymes decreases their renal clearance from the circulation; however, …

Presentations Of Cutaneous Disease In Various Skin Pigmentations: Tinea Corporis, Braden Van Alfen, Navya Peddireddy, Marshall Hall, Henry Lim, Christian Scheufele, Garrett Furth, Dustin Wilkes, Michael Carletti, Stephen E. Weis

HCA Healthcare Journal of Medicine

Tinea corporis is a common superficial fungal infection caused by dermatophytes. It typically presents as well-defined, pruritic, annular papules and plaques with central hypopigmentation. The differential diagnosis includes pityriasis rosea, nummular dermatitis, plaque psoriasis, tinea versicolor, granuloma annulare, and discoid lupus. This article compares different presentations of tinea corporis in various skin types as classified by the Fitzpatrick scale. Recognizing varying presentations of tinea in patients of different skin tones will aid in early and appropriate diagnosis and treatment.

Presentations Of Cutaneous Disease In Various Skin Pigmentations: Keratosis Pilaris, Henry Lim, Reem Ayoub, Madelyn Richards, Marshall Hall, Christian Scheufele, Dustin Wilkes, Michael Carletti, Stephen E. Weis

HCA Healthcare Journal of Medicine

Keratosis pilaris (KP) is a common benign disorder involving hyperkeratosis of the skin. It is associated with other common dry skin disorders such as atopic dermatitis and ichthyosis vulgaris. Lesions are clinically characterized as symmetrically distributed, monomorphic, folliculocentric, hyperkeratotic papules with a variable degree of perifollicular erythema. The appearance can be likened to spikey bumps that are commonly located on the arms, legs, and buttocks. Awareness of KP is important as every primary care clinician will see patients with this disorder. Identification can support the diagnosis of other associated skin diseases. Education about KP and its treatment may alleviate psychological …

Impact Of Palliative Care Consultation On Clinical Interventions And Outcomes In A Medical-Surgical Community-Based Intensive Care Unit, Manjot S. Malhi, Jude Tabba, Aunie M. Danyalian, Michele Iguina, Daniel Zapata, Raiko Diaz, Dionne Morgan, Daniel Heller, Sanaz Kashan, Mauricio Danckers

HCA Healthcare Journal of Medicine

Background

Integrating palliative care into intensive care unit (ICU) practice is an essential component of comprehensive patient management, ensuring that critical interventions are aligned with patient-centered care objectives. This study aims to evaluate the impact of palliative care consultation (PCC) on clinical interventions, patient outcomes, and decision-making processes in a community-based medical-surgical ICUs.

Methods

We conducted a prospective, observational, single-center cohort study of critically ill adult patients in a community-based medical-surgical ICU. Patients were grouped by the presence (PCCP) or absence (PCCA) of palliative care consultation. Primary outcomes included differences in clinical interventions (mechanical ventilation, vasoactive medications, renal replacement therapy, …

Replicability Of Highly Cited Comparative Studies In Orthopedic Journals, Cody Ashy, William E. Few, Gabriella A. Rivas, Emily Brennan, Langdon Hartsock, Kristoff R. Reid

HCA Healthcare Journal of Medicine

Introduction

Concerns are rising regarding the quality, validity, and reliability of clinical research findings in medical literature. This investigation sought to identify the most cited orthopedic clinical studies and assess the replicability of the findings reported.

Methods

Web of Science was used to identify the top ten orthopedic surgery journals by impact factor from which primary comparative studies cited at least 250 times were identified. A second literature search identified follow-up studies relevant to the respective primary studies. Studies investigating the same intervention via parallel methodology were summarized and their conclusions compared to their respective highly cited primary study.

Results …