Medicine and Health Sciences Commons^™

Prospective Study To Characterize Adalimumab Exposure In Pediatric Patients With Rheumatic Diseases, Tatjana Welzel, Klervi Golhen, Andrew Atkinson, Verena Gotta, David Ternant, Jasmin B Kuemmerle-Deschner, Christine Michler, Gilbert Koch, Johannes N Van Den Anker, Marc Pfister, Andreas Woerner

2020-Current year OA Pubs

BACKGROUND: In pediatric rheumatic diseases (PRD), adalimumab is dosed using fixed weight-based bands irrespective of methotrexate co-treatment, disease activity (DA) or other factors that might influence adalimumab pharmacokinetics (PK). In rheumatoid arthritis (RA) adalimumab exposure between 2-8 mg/L is associated with clinical response. PRD data on adalimumab is scarce. Therefore, this study aimed to analyze adalimumab PK and its variability in PRD treated with/without methotrexate.

METHODS: A two-center prospective study in PRD patients aged 2-18 years treated with adalimumab and methotrexate (G

RESULTS: Twenty-eight patients (14 per group), diagnosed with juvenile idiopathic arthritis (71.4%), non-infectious uveitis (25%) or chronic recurrent …

Pharmacokinetic-Pharmacodynamic Evidence From A Phase 3 Trial To Support Flat-Dosing Of Rifampicin For Tuberculosis, Huy X. Ngo, Ava Y. Xu, Gustavo E. Velásquez, Nan Zhang, Vincent K. Chang, Ekaterina V. Kurbatova, William C. Whitworth, Erin Sizemore, Kia Bryant, Wendy Carr, Marc Weiner, Kelly E. Dooley, Melissa Engle, Susan E. Dorman, Payam Nahid, Susan Swindells, Richard E. Chaisson, Pheona Nsubuga, Madeleine Lourens, Rodney Dawson, Radojka M. Savic

Journal Articles: Infectious Diseases

BACKGROUND: The optimal dosing strategy for rifampicin in treating drug-susceptible tuberculosis (TB) is still highly debated. In the phase 3 clinical trial Study 31/ACTG 5349 (NCT02410772), all participants in the control regimen arm received 600 mg rifampicin daily as a flat dose. Here, we evaluated relationships between rifampicin exposure and efficacy and safety outcomes.

METHODS: We analyzed rifampicin concentration time profiles using population nonlinear mixed-effects models. We compared simulated rifampicin exposure from flat- and weight-banded dosing. We evaluated the effect of rifampicin exposure on stable culture conversion at 6 months; TB-related unfavorable outcomes at 9, 12, and 18 months using …

Pyrazinamide Safety, Efficacy, And Dosing For Treating Drug-Susceptible Pulmonary Tuberculosis: A Phase 3, Randomized Controlled Clinical Trial, Ava Y. Xu, Gustavo E. Velásquez, Nan Zhang, Vincent K. Chang, Patrick P. J. Phillips, Payam Nahid, Susan E. Dorman, Ekaterina V. Kurbatova, William C. Whitworth, Erin Sizemore, Kia Bryant, Wendy Carr, Nicole E. Brown, Melissa L. Engle, Nguyen Viet Nhung, Pheona Nsubuga, Andreas Diacon, Kelly E. Dooley, Richard E. Chaisson, Susan Swindells, Radojka M. Savic

Journal Articles: Infectious Diseases

Optimizing pyrazinamide dosing is critical to improve treatment efficacy while minimizing toxicity during tuberculosis treatment. Study 31/AIDS Clinical Trials Group A5349 represents the largest phase 3 randomized controlled therapeutic trial to date for such an investigation. Objectives: We sought to report pyrazinamide pharmacokinetic parameters, risk factors for lower pyrazinamide exposure, and relationships between pyrazinamide exposure and efficacy and safety outcomes. We aimed to determine pyrazinamide dosing strategies that optimize risks and benefits. Methods: We analyzed pyrazinamide steady-state pharmacokinetic data using population nonlinear mixed-effects models. We evaluated the contribution of pyrazinamide exposure to long-term efficacy using parametric time-to-event models and safety …

Safety And Efficacy Of Peptide Receptor Radionuclide Therapy In Neuroendocrine Tumors: A Single Center Experience., Vineeth Sukrithan, Heather Armbruster, Sherise Rogers, Sherry Mori Vogt, Cassandra Grenade, Claire Verschraegen, Ye Zhou, Ashima Goyal, Mona Natwa, Akram Hussein, Hallie Barr, Dramane Konate, Rochelle Batdorf, Andrew Brown, Bonnie Williams, Songzhu Zhao, Lai Wei, Menglin Xu, Manisha H Shah, Bhavana Konda

Oncology Articles

Peptide receptor radionucleotide therapy (PRRT) with 177Lu-dotatate is widely used for the treatment of patients with neuroendocrine tumors (NETs). We analyzed data from 104 patients with NETs treated with 177Lu -dotatate at a US academic center between December 2017 and October 2020 to better understand patterns of long-term efficacy, safety, and toxicity in the real-world setting. 177Lu-dotatate (200 mCi) was administered every eight weeks for four doses. The most common sites of primary disease were small intestine NETs (n = 49, 47%), pancreatic NETs (n = 32, 31%), and lung NETs (n = 7, 7%). Twenty-seven percent had Ki-6720%. The …

Risk-Stratified Treatment For Drug-Susceptible Pulmonary Tuberculosis., Vincent K. Chang, Marjorie Z. Imperial, Patrick P. J. Phillips, Gustavo E. Velásquez, Payam Nahid, Andrew Vernon, Ekaterina V. Kurbatova, Susan Swindells, Richard E. Chaisson, Susan E. Dorman, John L. Johnson, Marc Weiner, Amina Jindani, Thomas Harrison, Erin E. Sizemore, William Whitworth, Wendy Carr, Kia E. Bryant, Deron Burton, Kelly E. Dooley, Melissa Engle, Pheona Nsubuga, Andreas H. Diacon, Nguyen Viet Nhung, Rodney Dawson, Radojka M. Savic, Aids Clinical Trial Group, Tuberculosis Trials Consortium

Journal Articles: Infectious Diseases

The Phase 3 randomized controlled trial, TBTC Study 31/ACTG A5349 (NCT02410772) demonstrated that a 4-month rifapentine-moxifloxacin regimen for drug-susceptible pulmonary tuberculosis was safe and effective. The primary efficacy outcome was 12-month tuberculosis disease free survival, while the primary safety outcome was the proportion of grade 3 or higher adverse events during the treatment period. We conducted an analysis of demographic, clinical, microbiologic, radiographic, and pharmacokinetic data and identified risk factors for unfavorable outcomes and adverse events. Among participants receiving the rifapentine-moxifloxacin regimen, low rifapentine exposure is the strongest driver of tuberculosis-related unfavorable outcomes (HR 0.65 for every 100 µg∙h/mL increase, …

High-Grade B-Cell Lymphoma, Not Otherwise Specified: Cns Involvement And Outcomes In A Multi-Institutional Series, Narendranath Epperla, Adam S. Zayac, Daniel J. Landsburg, Allison M. Bock, Grzegorz S. Nowakowski, Emily C. Ayers, Mark Girton, Marie Hu, Amy Beckman, Shaoying Li, L Jeffrey Medeiros, Julie E. Chang, Habibe Kurt, Jose Sandoval-Sus, Mohammad Ali Ansari-Lari, Shalin K. Kothari, Anna Kress, Mina L. Xu, Pallawi Torka, Suchitra Sundaram, Stephen D. Smith, Kikkeri N. Naresh, Yasmin Karimi, David A. Bond, Andrew M. Evens, Seema G. Naik, Manali Kamdar, Bradley M. Haverkos, Reem Karmali, Umar Farooq, Julie M. Vose, Paul Rubinstein, Amina Chaudhry, Adam J. Olszewski

Journal Articles: Oncology and Hematology

Little is known about the central nervous system (CNS) risk in high-grade B-cell lymphoma, not otherwise specified (HGBL NOS). Hence, we sought to describe the rates of baseline CNS involvement, risk of CNS recurrence after primary therapy, and management strategies in HGBL NOS. In this multicenter retrospective study, we included 160 adults with newly diagnosed HGBL NOS treated between 2016 and 2021 at 20 US institutions. Eleven patients (7%) had baseline CNS involvement at diagnosis (leptomeningeal = 6, parenchymal = 4, and both = 1). Baseline CNS involvement was significantly associated only with MYC rearrangement (OR = 3.5) and testicular …

A Proposal For The Use Of A Fixed Low-Energy Selective Laser Trabeculoplasty For Open Angle Glaucoma, Zachary Sacks, L. Jay Katz, Gus Gazzard, Sarah H. Van Tassel, Eytan Z. Blumenthal, Fabian S. Lerner, Augusto Azuara-Blanco, Gregory John Roy Spooner, Yoram Solberg, Thomas Samuelson, Michael Belkin

Wills Eye Hospital Papers

Selective laser trabeculoplasty (SLT) has been in routine clinical use for over 20 years with millions of patients successfully treated and a low rate of clinically significant complications. The procedure requires the clinician to manually position the laser beam on the trabecular meshwork using a gonioscopy lens and to titrate the SLT laser energy based on the amount of pigmentation in the angle, as well as the observation of small bubbles produced by the laser effect. We propose that SLT energy titration is unnecessary either to achieve intraocular pressure (IOP) reduction or to minimize potential side effects. Ample evidence to …

Effects Of Bioprosthetic Valve Fracturing On Valve-In-Valve Transcatheter Aortic Valve Implantation Transvalvular Gradients, Osama Hallak, Karley Fischer, Shaina Ailawadi, Damian Valencia, Yan Yatsynovich, Raja Nazir, Brian Schwartz

The Texas Heart Institute Journal

BACKGROUND: Valve-in-valve (ViV) transcatheter aortic valve implantation (TAVI) is quickly becoming a routine and effective means by which to treat degenerated bioprosthetic valves. A known complication of ViV-TAVI is patient-prosthesis mismatch, which substantially affects survival. Bioprosthetic valve fracture is a method by which to reduce the risk of patient-prosthesis mismatch and post-ViV-TAVI transvalvular gradients. This study sought to determine the safety and efficacy of post-ViV-TAVI bioprosthetic valve fracture.

METHODS: Patients with a history of surgical aortic valve replacement undergoing ViV-TAVI bioprosthetic valve fracture (N = 25) at the corresponding institution from 2015 to 2022 were cataloged for a retrospective analysis. …

Mitral Valve Regurgitation In Klippel-Feil Syndrome With Related Thoracic Deformity, Yuki Endo, Yoshiei Shimamura, Hiroshi Niinami

The Texas Heart Institute Journal

Klippel-Feil syndrome, characterized by congenital fusion of any 2 or more cervical vertebrae, is a rare disorder in which skeletal and other organ system-related abnormalities have been reported. This article reports a case of mitral valve regurgitation in a patient with Klippel-Feil syndrome and related thoracic deformity who underwent mitral valvuloplasty. Postoperatively, the mitral valve regurgitation disappeared, and there has been no recurrence for 3 years. This case highlights mitral valvuloplasty via median sternotomy as an excellent treatment for mitral valve regurgitation in a patient with thoracic deformity related to Klippel-Feil syndrome.

Investigation Of Setmelanotide, An Mc4r Agonist, For Obesity In Individuals With Smith-Magenis Syndrome, Julia Lazareva, Stephanie R Sisley, Sheila M Brady, Ann C M Smith, Sarah H Elsea, Jeremy J Pomeroy, Christian L Roth, Jennifer E Sprague, Martin Wabitsch, Jill Garrison, Jack A Yanovski

Children’s Nutrition Research Center Staff Publications

Background: Smith Magenis Syndrome (SMS) is a rare genetic disorder caused by RAI1 haploinsufficiency. Obesity in people with SMS is believed partially due to dysfunction of the proximal melanocortin 4 receptor (MC4R) pathway. We therefore studied effects of treatment with the MC4R agonist setmelanotide on obesity and hunger, as well as metabolic, cardiac and safety, in individuals with SMS.

Methods: People with SMS received once-daily setmelanotide injections, with the dose titrated bi-weekly to a maximum of 3 mg over ∼1 month; and a full-dose treatment duration of 3mo. The primary outcome was percent change in body weight. Secondary outcomes included …

Use Of Extracorporeal Membrane Oxygenation-Facilitated Large-Bore Catheter Embolectomy In The Treatment Of Acute Pulmonary Embolism Complicated By Shock, Hugh A Glazier, Amir Kaki

The Texas Heart Institute Journal

This article reports the case of a 42-year-old man who presented with a saddle pulmonary embolus complicated by normotensive cardiogenic shock. The patient was first stabilized with venoarterial extracorporeal membrane oxygenation. Then, while the patient was still on extracorporeal membrane oxygenation, thrombectomy with a large-bore catheter device was performed that resulted in a large decrease in pulmonary artery pressures and a clinically significant increase in cardiac index, with rapid clinical improvement. Complete recovery of the patient's cardiopulmonary status has been maintained at intermediate-term follow-up. This treatment strategy should be considered favorably in the treatment of patients presenting with pulmonary embolism …

Assessing The Acceptability And Effectiveness Of A Novel Therapeutic Footwear In Reducing Foot Pain And Improving Function Among Older Adults: A Crossover Randomized Controlled Trial, M G Finco, Abderrahman Ouattas, Nesreen El-Refaei, Anmol Salim Momin, Mehrnaz Azarian, Bijan Najafi

Center on Aging Staff Publications

Introduction: Nearly, a quarter of older adults suffer from frequent foot pain, impacting their quality of life. While proper footwear can alleviate this, design issues often hinder regular use. This study evaluated novel therapeutic footwear, designed for aesthetics and custom fit, to reduce foot pain. We hypothesized that older adults would experience less foot pain and favor the new footwear over their own.

Methods: This 12-week crossover randomized controlled trial evaluated the effectiveness of OrthoFeet therapeutic footwear on reducing foot pain in older adults (n = 50, age = 65 ± 5, 18% male) with moderate to severe pain. Participants …

Impact Of P53-Associated Acute Myeloid Leukemia Hallmarks On Metabolism And The Immune Environment, Monika Chomczyk, Luca Gazzola, Shubhankar Dash, Patryk Firmanty, Binsah S George, Vakul Mohanty, Hussein A Abbas, Natalia Baran

Faculty, Staff and Student Publications

Acute myeloid leukemia (AML), an aggressive malignancy of hematopoietic stem cells, is characterized by the blockade of cell differentiation, uncontrolled proliferation, and cell expansion that impairs healthy hematopoiesis and results in pancytopenia and susceptibility to infections. Several genetic and chromosomal aberrations play a role in AML and influence patient outcomes. TP53 is a key tumor suppressor gene involved in a variety of cell features, such as cell-cycle regulation, genome stability, proliferation, differentiation, stem-cell homeostasis, apoptosis, metabolism, senescence, and the repair of DNA damage in response to cellular stress. In AML, TP53 alterations occur in 5%-12% of de novo AML …

Coronary Laser With Simultaneous Contrast Injection For The Treatment Of Stent Underexpansion, Mohsen Mohandes, Alberto Pernigotti, Cristina Moreno, Luis Mauricio Torres, Francisco Fernández, Diego Zambrano, Alfredo Bardají

Faculty, Staff and Student Publications

BACKGROUND: Stent underexpansion is a challenge in interventional cardiology. Some off-label treatments, such as rotational atherectomy, intravascular lithotripsy (IVL) and coronary lasing, have been used to overcome the problem. The purpose of this study is to evaluate the safety and efficacy of coronary laser atherectomy with simultaneous contrast injection and subsequent balloon dilation to optimize stent expansion.

METHODS: Coronary laser atherectomy with simultaneous contrast injection was used. After lasing, non-compliant balloon dilation at high pressure was performed to overcome the underexpanded point. The average increase in the minimum stent area (MSA) was measured by intravascular ultrasound (IVUS), and any complication …

Outcomes Of Simultaneous Lengthening And Acl Reconstruction In Fibular Hemimelia: A Retrospective Case Series, Bradley Reeves, Brennan Roper, Reba Salton, Ava Baumann, Radomir Dimovski, Nancy Hadley-Miller, Jay Albright

Faculty, Staff and Student Publications

BACKGROUND: Fibular hemimelia is the most common congenital long bone deficiency. It is often associated with femoral and tibial deficiencies which result in a clinically evident leg length discrepancy. The primary soft tissue concern is ACL/PCL deficiency. If treatment includes bony lengthening, joint stability is imperative to avoid complications. In this study, we detail a novel technique for long bone lengthening and ACL reconstruction in a single, cohesive surgery. This consolidates the need for multiple procedures and offers improved limb length symmetry and knee stability for this patient population. Clinical outcomes of pediatric patients with hemimelia who underwent either femoral …

Characterizing Treatment Non-Responders And Responders In Completed Alzheimer's Disease Clinical Trials, Dulin Wang, Yaobin Ling, Kristofer Harris, Paul E Schulz, Xiaoqian Jiang, Yejin Kim

Faculty, Staff and Student Publications

Characterizing differential responses to Alzheimer's disease (AD) drugs will provide better insights into personalized treatment strategies. Our study aims to identify heterogeneous treatment effects and pre-treatment features that moderate the treatment effect of Galantamine, Bapineuzumab, and Semagacestat from completed trial data. The causal forest method can capture heterogeneity in treatment responses. We applied causal forest modeling to estimate the treatment effect and identify efficacy moderators in each trial. We found several patient's pretreatment conditions that determined treatment efficacy. For example, in Galantamine trials, whole brain volume (1092.54 vs. 1060.67 ml, P < .001) and right hippocampal volume (2.43e-3 vs. 2.79e-3, P < .001) are significantly different between responsive and non-responsive subgroups. Overall, our implementation of causal forests in AD clinical trials reveals the heterogeneous treatment effects and different moderators for AD drug responses, highlighting promising personalized treatment based on patient-specific characteristics in AD research and drug development.

Patient-Reported Outcomes Following Severe Abdominal Trauma: A Secondary Analysis Of The Damage Control Laparotomy Trial, Shah-Jahan M Dodwad, Kayla D Isbell, Krislynn M Mueck, James M Klugh, David E Meyer, Charles E Wade, Lillian S Kao, John A Harvin

Faculty, Staff and Student Publications

Introduction: Little is known about patient-reported outcomes (PROs) following abdominal trauma. We hypothesized that patients undergoing definitive laparotomy (DEF) would have better PROs compared to those treated with damage control laparotomy (DCL).

Methods: The DCL Trial randomized DEF versus DCL in abdominal trauma. PROs were measured using the European Quality of Life-5 Dimensions-5 Levels (EQ-5D) questionnaire at discharge and six months postdischarge (1 = perfect health, 0 = death, and < 0 = worse than death) and Posttraumatic Stress Disorder (PTSD) Checklist-Civilian. Unadjusted Bayesian analysis with a neutral prior was used to assess the posterior probability of achieving minimal clinically important difference.

Results: Of 39 randomized patients (21 DEF versus 18 DCL), 8 patients died (7 DEF versus 1 DCL). Of those who survived, 28 completed the EQ-5D at discharge (12 DEF …

Highly Variable Timing Renders Immunotherapy Efficacy And Toxicity Impractical Biomarkers Of One Another In Clinical Practice, Mitchell S Von Itzstein, Yuqiu Yang, Yiqing Wang, David Hsiehchen, Thomas Y Sheffield, Farjana Fattah, Vinita Popat, Murtaza Ahmed, Jade Homsi, Jonathan E Dowell, Sawsan Rashdan, Jay Lohrey, Hans J Hammers, Randall S Hughes, Tao Wang, Yang Xie, David E Gerber

Faculty, Staff and Student Publications

Background: A useful clinical biomarker requires not only association but also a consistent temporal relationship. For instance, chemotherapy-induced neutropenia and epidermal growth-factor inhibitor-related acneiform rash both occur within weeks of treatment initiation, thereby providing information prior to efficacy assessment. Although immune checkpoint inhibitor (ICI)-associated immune-related adverse events (irAE) have been associated with therapeutic benefit, irAE may have delayed and highly variable onset. To determine whether ICI efficacy and irAE could serve as clinically useful biomarkers for predicting each other, we determined the temporal relationship between initial efficacy assessment and irAE onset in a diverse population treated with ICI.

Methods: Using …

Effect Of Pioglitazone On Inflammatory Response And Clinical Outcome In T2dm Patients With Covid-19: A Randomized Multicenter Double-Blind Clinical Trial, Khaled Baagar, Thamer Alessa, Mohamed Abu-Farha, Jehad Abubaker, Heba Alhumaidi, Jose Antonio Franco Ceruto, Mohammad Khair Hamad, Ali Omrani, Salma Abdelrahman, Muhammad Zaka-Ul Haq, Abdul Wajid Safi, Bassem Alhariri, Manish Barman, Alaaeldin Abdelmajid, Humberto Vidal Denis Cancio, Eman Elmekaty, Irina Al-Khairi, Preethi Cherian, Lina Jayyousi, Mohammed Ahmed, Mohammed Qaddoumi, Sulaiman Hajji, Ahmad Esmaeel, Ali Al-Andaleeb, Arshad Channanath, Sriraman Devarajan, Hamad Ali, Thangavel Alphonse Thanaraj, Salman Al-Sabah, Fahd Al-Mulla, Muhammad Abdul-Ghani, Amin Jayyousi

Faculty, Staff and Student Publications

Background: Coronavirus disease 2019 (COVID-19) caused by the coronavirus SARS-CoV-2, has emerged as a rapidly spreading contagious disease across the globe. Recent studies showed that people with diabetes mellitus, severe obesity, and cardiovascular disease are at higher risk of mortality from COVID-19. It has been suggested that the increased risk is due to the chronic inflammatory state associated with type 2 diabetes. This study aimed to evaluate the efficacy of pioglitazone, a strong insulin sensitizer with anti-inflammatory properties, in improving the clinical outcomes of patients with type 2 diabetes admitted with moderate-severe COVID-19.

Method: We enrolled 350 patients with type …

Pathologic Processing Of Lung Cancer Resection Specimens After Neoadjuvant Therapy, Annikka Weissferdt, Cheuk H Leung, Heather Lin, Boris Sepesi, William N William, Stephen G Swisher, Tina Cascone, J Jack Lee, Abujiang Pataer

Faculty, Staff and Student Publications

Neoadjuvant treatment of non-small cell lung cancer challenges the traditional processing of pathology specimens. Induction therapy before resection allows evaluation of the efficacy of neoadjuvant agents at the time of surgery. Many clinical trials use pathologic tumor response, measured as major pathologic response (MPR, ≤10% residual viable tumor [RVT]) or complete pathologic response (CPR, 0% RVT) as a surrogate of clinical efficacy. Consequently, accurate pathologic evaluation of RVT is crucial. However, pathologic assessment has not been uniform, which is particularly true for sampling of the primary tumor, which instead of the traditional processing, requires different tissue submission because the focus …

Responsive Neurostimulation For People With Drug-Resistant Epilepsy And Autism Spectrum Disorder, Madeline C Fields, Christina Marsh, Onome Eka, Emily A Johnson, Lara V Marcuse, Churl-Su Kwon, James J Young, Maite Lavega-Talbott, Mohankumar Kurukumbi, Gretchen Von Allmen, John Zempel, Daniel Friedman, Nathalie Jette, Anuradha Singh, Ji Yeoun Yoo, Leah Blank, Fedor Panov, Saadi Ghatan

Faculty, Staff and Student Publications

PURPOSE: Individuals with autism spectrum disorder (ASD) have comorbid epilepsy at much higher rates than the general population, and about 30% will be refractory to medication. Patients with drug-resistant epilepsy (DRE) should be referred for surgical evaluation, yet many with ASD and DRE are not resective surgical candidates. The aim of this study was to examine the response of this population to the responsive neurostimulator (RNS) System.

METHODS: This multicenter study evaluated patients with ASD and DRE who underwent RNS System placement. Patients were included if they had the RNS System placed for 1 year or more. Seizure reduction and …

Plain Language Summary Of The Foenix-Cca2 Study: Futibatinib For People With Advanced Bile Duct Cancer, Lipika Goyal, Funda Meric-Bernstam, Antoine Hollebecque, Juan W Valle, Chigusa Morizane, Thomas B Karasic, Thomas A Abrams, Junji Furuse, Robin Katie Kelley, Philippe A Cassier, Heinz-Josef Klümpen, Heung-Moon Chang, Li-Tzong Chen, Josep Tabernero, Do-Youn Oh, Amit Mahipal, Markus Moehler, Yoshito Komatsu, Daniel H Ahn, Robert S Epstein, Abdel-Baset Halim, Volker Wacheck, Yaohua He, Mei Liu, Karim A Benhadji, John A Bridgewater, Foenix-Cca2 Study Investigators

Faculty, Staff and Student Publications

What is this summary about?: This summary describes the results from a phase 2 study called FOENIXCCA2. The study evaluated treatment with futibatinib in people with a rare form of advanced bile duct cancer called intrahepatic cholangiocarcinoma (or iCCA), where the tumors have changes in the structure of a gene called FGFR2. These changes include FGFR2 gene fusions. Bile duct cancer often returns after surgery or cannot be treated by surgery because the tumor has spread, so it requires treatment with chemotherapy. People live for a median of 1 year after their first chemotherapy treatment and 6 months after their …

Outcomes For Patients With Head And Neck Sarcoma Treated Curatively With Radiation Therapy And Surgery, Alison K Yoder, Ahsan Farooqi, Devarati Mitra, J Andrew Livingston, Dejka M Araujo, Erich M Sturgis, Ryan Goepfert, Andrew J Bishop, B Ashleigh Guadagnolo

Faculty, Staff and Student Publications

PURPOSE: Soft tissue sarcomas (STSs) of the head and neck (H&N) are rare malignancies that are challenging to manage. We sought to describe the outcomes of patients treated with curative intent using combined surgery and radiation therapy (RT) for H&N STS.

METHODS AND MATERIALS: We performed a single-institution retrospective review of patients with nonmetastatic STS of the H&N who were treated from 1968 to 2020. The Kaplan-Meier method was used to estimate disease-specific survival (DSS) and local control (LC). Multivariable analyses (MVAs) were conducted using Cox proportional hazards model.

RESULTS: One hundred ninety-two patients had a median follow-up of 82 …

Development And Initial Validation Of A Hospital Stress Questionnaire, Daniel M Ford, Rebecca Lawton, Elizabeth Travis, Elizabeth A Teale, Daryl B O'Connor

Faculty, Staff and Student Publications

BACKGROUND: Hospitalisation can be a traumatic experience, where inpatients are exposed to an abundance of physical and psychological stressors. Evidence suggests that these hospital-related stressors negatively impact health: a phenomenon known as post-hospital syndrome. The current study aimed to identify hospital-related stressors, and to develop and provide initial validation for a new measure of in-hospital stress.

METHODS: Measure development occurred in three stages: (i) semi-structured interviews, (ii) item generation, and (iii) pilot testing. Twenty-one patients were interviewed regarding their recent hospital experiences, and a list of hospital-related stressors was produced. These stressors were compiled into a questionnaire and piloted on …

Efficacy Of Etripamil Nasal Spray For Acute Conversion Of Supraventricular Tachycardia: A Network Meta-Analysis, Adam Macech, Nicola Luigi Bragazzi, Francesco Chirico, Basar Cander, Michal Pruc, Zubaid Rafique, William Frank Peacock, Arash Ziapour, Lukasz Szarpak, Anna Salak, Milosz J Jaguszewski

Faculty, Staff and Students Publications

No abstract provided.

The 2023 Society For Vascular Surgery, American Venous Forum, And American Vein And Lymphatic Society Clinical Practice Guidelines For The Management Of Varicose Veins Of The Lower Extremities. Part Ii: Endorsed By The Society Of Interventional Radiology And The Society For Vascular Medicine., Peter Gloviczki, Peter F Lawrence, Suman M Wasan, Mark H Meissner, Jose Almeida, Kellie R Brown, Ruth L Bush, Michael Di Iorio, John Fish, Eri Fukaya, Monika L Gloviczki, Anil Hingorani, Arjun Jayaraj, Raghu Kolluri, M Hassan Murad, Andrea T Obi, Kathleen J Ozsvath, Michael J Singh, Satish Vayuvegula, Harold J Welch

Heart and Vascular Articles

The Society for Vascular Surgery, the American Venous Forum, and the American Vein and Lymphatic Society recently published Part I of the 2022 clinical practice guidelines on varicose veins. Recommendations were based on the latest scientific evidence researched following an independent systematic review and meta-analysis of five critical issues affecting the management of patients with lower extremity varicose veins, using the patients, interventions, comparators, and outcome system to answer critical questions. Part I discussed the role of duplex ultrasound scanning in the evaluation of varicose veins and treatment of superficial truncal reflux. Part II focuses on evidence supporting the prevention …

Characterization And Z-Score Calculation Of Cardiovascular Magnetic Resonance Imaging Parameters In Patients After The Fontan Operation: A Fontan Outcome Registry Using Cardiovascular Magnetic Resonance Examinations Study, Tarek Alsaied, Manish Aggarwal, Et Al.

2020-Current year OA Pubs

BACKGROUND: Cardiac magnetic resonance (CMR) offers valuable hemodynamic insights post-Fontan, but is limited by the absence of normative single ventricle data. The Fontan Outcomes Registry using CMR Examinations (FORCE) is a large international Fontan-specific CMR registry. This study used FORCE registry data to evaluate expected CMR ventricular size/function and create Fontan-specific z-scores adjusting for ventricular morphology (VM) in healthier Fontan patients.

METHODS: "Healthier" Fontan patients were defined as patients free of adverse outcomes, who are New York Heart Association class I, have mild or less valve disease, and < 30% aortopulmonary collateral burden. General linear modeling was performed on 70% of the dataset to create z-scores for volumes and function. Models were tested using the remainder (30%) of the data. The z-scores were compared between children and adults. The z-scores were also compared between "healthier" Fontan and patients with adverse outcomes (death, listing for transplantation, or multiorgan disease).

RESULTS: The "healthier" Fontan population included 885 patients (15.0 ± 7.6 years) …

Efficacy And Safety Of Zuranolone Co-Initiated With An Antidepressant In Adults With Major Depressive Disorder: Results From The Phase 3 Coral Study, Sagar V Parikh, Scott T Aaronson, Sanjay J Mathew, Gustavo Alva, Charles Debattista, Stephen Kanes, Robert Lasser, Amy Bullock, Mona Kotecha, Jungah Jung, Fiona Forrestal, Jeff Jonas, Theresa Vera, Bridgette Leclair, James Doherty

Staff and Researcher Publications

Major depressive disorder (MDD) is a mental health disorder that can cause disability and functional impairment that standard-of-care (SOC) antidepressant therapies (ADTs) can take weeks to treat. Zuranolone is a neuroactive steroid and positive allosteric modulator of synaptic and extrasynaptic γ-aminobutyric acid (GABA) type A receptors approved as an oral, once-daily, 14-day treatment course in adults with postpartum depression and under investigation in adults with MDD. The phase 3 CORAL Study (NCT04476030) evaluated the efficacy and safety of zuranolone 50 mg co-initiated with SOC ADT (zuranolone+ADT) vs placebo co-initiated with SOC ADT (placebo+ADT) in adults with MDD. Patients were randomized …

Self-Correcting Method For Highly Effective Office-Based Helicobacter Pylori Therapy Using Cumulative Test Of Cure Data, Maria Pina Dore, Ruben Hernaez, David Y Graham

Faculty, Staff and Students Publications

Background and aim: Helicobacter pylori infections have become resistant to many previously highly effective antimicrobial regimens resulting in clarithromycin, metronidazole, or fluoroquinolone-containing therapies becoming unsuccessful. Pretreatment susceptibility testing is only widely available in the United States but is still rarely done. Here, we propose a framework to monitor H. pylori eradication in small clinical settings by routinely assessing the effectiveness of therapy.

Methods: Because of the small sample size in individual practice's, we assume an acceptable cure rate of ≥80% (preferred cure rate ≥85%) in adherent patients, with a dichotomous outcome (cured vs. failed) and consecutive patient enrollment. To obtain …

Severity-Adjusted Evaluation Of Initial Dialysis On Short-Term Health Outcomes In Urea Cycle Disorders, Matthias Zielonka, Stefan Kölker, Sven F Garbade, Florian Gleich, Sandesh C S Nagamani, Andrea L Gropman, Ann-Catrin Druck, Nesrine Ramdhouni, Laura Göde, Georg F Hoffmann, Roland Posset, Urea Cycle Disorders Consortium (Ucdc)

Faculty, Staff and Students Publications

Objective: In individuals with urea cycle disorders (UCDs) and neonatal disease onset, extracorporeal detoxification by continuous kidney replacement therapy is considered the therapeutic method of choice in addition to metabolic emergency treatment to resolve hyperammonemic decompensation. However, the indications for the initiation of dialysis are heterogeneously implemented transnationally, thereby hampering our understanding of (optimal) short-term health outcomes.

Methods: We performed a retrospective comparative analysis evaluating the therapeutic effects of initial dialysis on survival as well as neurocognitive outcome parameters in individuals with UCDs in comparison to a severity-adjusted non-dialyzed control cohort. Overall, 108 individuals with a severe phenotype of male …