174 results match your criteria: "NC (F.R.); the Sanford Burnham Prebys Medical Discovery Institute[Affiliation]"

Patient Preferences for Features Associated With Leadless Versus Conventional Transvenous Cardiac Pacemakers.

Circ Cardiovasc Qual Outcomes

December 2024

Duke Clinical Research Institute (S.D.R., J.-C.Y., M.J.W., J.S., F.R.J., S.O., S.M.A.-K.), Duke University School of Medicine, Durham, NC.

Article Synopsis
  • A study examined patient preferences for different types of pacemakers, including new dual-chamber leadless options and traditional transvenous ones, to identify which features are most important to them.
  • Surveying 117 patients, researchers found that half preferred leadless pacemakers while the other half favored conventional ones, highlighting a significant division in choices.
  • Key factors influencing patient decisions included preferred pacemaker type, acceptance of complication and infection risks, and the time since regulatory approval, with many willing to accept higher risks for their preferred options.
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Background: Tobacco smoke may affect atopic dermatitis (AD) because of its known effects on humoral and cellular immunity, but prior studies lack data on disease severity and biomarkers over time.

Objective: We investigated the association between passive and active tobacco smoke exposure (TSE) during childhood and adolescence and the activity and severity of AD.

Methods: A birth cohort of 10,521 individuals was followed through adolescence as part of the Avon Longitudinal Study of Parents and Children.

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Mathematical 3D Liver Model for Surgical versus Ablative Therapy Treatment Planning for Colorectal Liver Metastases: Recommendations from the COLLISION and COLDFIRE Trial Expert Panels.

Radiol Imaging Cancer

November 2024

From the Department of Radiology and Nuclear Medicine, Amsterdam UMC, Vrije Universiteit Amsterdam, De Boelelaan 1117, 1081 HV Amsterdam, the Netherlands (B.A.T.v.d.B., R.S.P., M.R.M.); Cancer Center Amsterdam, Amsterdam, the Netherlands (B.A.T.v.d.B., R.S.P., M.R.M.); Department of Surgical Oncology, OLVG Hospital, Oost, Amsterdam, the Netherlands (B.A.T.v.d.B.); Department of Radiology and Nuclear Medicine, OLVG Hospital, Oost, Amsterdam, the Netherlands (R.S.P.), Department of Medical Physics and Radiation Protection, Haaglanden MC, The Hague, the Netherlands (H. Keijzers); and Department of Surgical Oncology, Medical Center Leeuwarden, Leeuwarden, the Netherlands (P.M.v.d.T.).

Article Synopsis
  • This study aims to define criteria for treating colorectal liver metastases (CRLM) using a three-dimensional liver model created by an expert panel, allowing for better treatment predictions.
  • Data from two multicenter trials involved 202 participants with small CRLM (≤3 cm) and explored treatment options like resection, thermal ablation, and irreversible electroporation (IRE).
  • The findings indicate a clear distinction in treatment preferences based on tumor location, suggesting superficial tumors should be resected, while deeper tumors should be treated with ablation, effectively predicting treatment recommendations in 73.8% of cases.
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Background: The ISCHEMIA trial (International Study of Comparative Health Effectiveness with Medical and Invasive Approaches) demonstrated greater health status benefits with an initial invasive strategy, as compared with a conservative one, for patients with chronic coronary disease and moderate or severe ischemia. Whether these benefits vary globally is important to understand to support global adoption of the results.

Methods: We analyzed participants' disease-specific health status using the validated 7-item Seattle Angina Questionnaire (SAQ: >5-point differences are clinically important) at baseline and over 1-year follow-up across 37 countries in 6 international regions.

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Article Synopsis
  • Disparities in heart failure care quality contribute to varying patient outcomes based on sex, race, ethnicity, and insurance type among hospitalized patients.
  • A study analyzed data from 685,227 patients across 596 hospitals, finding that women generally received worse quality of care than men, but some differences disappeared after adjusting for individual patient factors.
  • Racial and ethnic minorities often performed as well or better than White and insured patients, but specific treatment measures showed significant gaps, particularly for certain groups like Asian, Hispanic, and Black patients, highlighting within-hospital variations and a need for improvement in care equity.
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Contemporary injury tolerance of the lumbar spine for under-body blast references axial compression and bending moments in a limited range. Since injuries often occur in a wider range of flexion and extension with increased moment contribution, this study expands a previously proposed combined loading injury criterion for the lumbar spine. Fifteen cadaveric lumbar spine failure tests with greater magnitudes of eccentric loading were incorporated into an existing injury criterion to augment its applicability and a combined loading injury risk model was proposed by means of survival analysis.

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Article Synopsis
  • SARS-CoV-2 can spread from asymptomatic individuals, posing a greater risk to cancer patients who frequently visit healthcare facilities and are more vulnerable to severe COVID-19 outcomes.* -
  • A study of lung cancer patients revealed that over half of those with evidence of SARS-CoV-2 infection were asymptomatic at diagnosis, and a significant number were never clinically diagnosed.* -
  • The findings indicate that older patients and those with early-stage lung cancer are more likely to have asymptomatic infections, highlighting the need for continued preventive measures in high-risk populations.*
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Reliability of CT Enterography for Describing Fibrostenosing Crohn Disease.

Radiology

August 2024

From the Department of Inflammation and Immunity, Lerner Research Institute, Cleveland Clinic, 9500 Euclid Ave, Cleveland, OH 44195 (F.R.); Department of Gastroenterology, Hepatology, and Nutrition, Digestive Diseases and Surgery Institute and Program for Global Translational Inflammatory Bowel Diseases Research, Cleveland Clinic, Cleveland, Ohio (F.R., M.P., J.N., R.O.); Departments of Medicine and Community Health Sciences, Division of Gastroenterology and Hepatology, University of Calgary, Calgary, Alberta, Canada (C.M.); Alimentiv, London, Ontario, Canada (C.M., J.H., Z.W., L.G., L.M.S., J. Rémillard, G.Z., B.G.F., V.J.); Department of Gastroenterology, University of Ljubljana, University Medical Centre Ljubljana, Ljubljana, Slovenia (J.H.); Department of Radiology, Mayo Clinic, Rochester, Minn (J.G.F.); Section of Abdominal Imaging, Imaging Institute, Digestive Diseases and Surgery Institute and Taussig Cancer Institute, Cleveland Clinic, Cleveland, Ohio (M.E.B.); Department of Radiology, University of California San Diego, San Diego, Calif (C.S.S.); Department of Radiology, IBD Unit, Hospital Clínic de Barcelona, Barcelona, Spain (N.C., J. Rimola); Pediatric and Adult Cardiothoracic and Vascular, Oncohematologic and Emergency Radiology Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Bologna, Italy (N.C.); Centre for Medical Imaging, University College London, London, England (S.A.T.); Division of Gastroenterology and Hepatology, Mayo Clinic College of Medicine and Science, Rochester, Minn (D.H.B.); and Departments of Medicine and Epidemiology and Biostatistics, Schulich School of Medicine and Dentistry, University of Western Ontario, London, Ontario, Canada (B.G.F., V.J.).

Background Standardized methods to measure and describe Crohn disease strictures at CT enterography are needed to guide clinical decision making and for use in therapeutic studies. Purpose To assess the reliability of CT enterography features to describe Crohn disease strictures and their correlation with stricture severity. Materials and Methods A retrospective study was conducted in 43 adult patients with symptomatic terminal ileal Crohn disease strictures who underwent standard-of-care CT enterography at a tertiary care center at the Cleveland Clinic between January 2008 and August 2016.

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Article Synopsis
  • More women of childbearing age are surviving heart transplants and many wish to become pregnant, but there's little information on their counseling experiences or knowledge regarding pregnancy after transplantation.
  • A survey of 64 women who underwent heart transplants revealed that many felt they lacked adequate information about post-transplant pregnancy and contraception before and after the procedure.
  • The results indicate a significant gap in communication between healthcare providers and these women, suggesting a need for improved education and support regarding reproductive health after heart transplantation.*
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Cranioencephalic functional lymphoid units in glioblastoma.

Nat Med

October 2024

German Cancer Consortium (DKTK), partner site Essen/Düsseldorf, a partnership between DKFZ and University Hospital Essen, University Duisburg-Essen, Essen, Germany.

The ecosystem of brain tumors is considered immunosuppressed, but our current knowledge may be incomplete. Here we analyzed clinical cell and tissue specimens derived from patients presenting with glioblastoma or nonmalignant intracranial disease to report that the cranial bone (CB) marrow, in juxtaposition to treatment-naive glioblastoma tumors, harbors active lymphoid populations at the time of initial diagnosis. Clinical and anatomical imaging, single-cell molecular and immune cell profiling and quantification of tumor reactivity identified CD8 T cell clonotypes in the CB that were also found in the tumor.

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Background: Prior studies have found no differences in procedural chest discomfort for patients undergoing manual syringe aspiration or drainage with gravity after thoracentesis. However, whether gravity drainage could protect against chest pain due to the larger negative-pressure gradient generated by wall suction has not been investigated.

Research Question: Does wall suction drainage result in more chest discomfort compared with gravity drainage in patients undergoing large-volume thoracentesis?

Study Design And Methods: In this multicenter, single-blinded, randomized controlled trial, patients with large free-flowing effusions of ≥ 500 mL were assigned at a 1:1 ratio to wall suction or gravity drainage.

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Predicting Cognitive Decline in Amyloid-Positive Patients With Mild Cognitive Impairment or Mild Dementia.

Neurology

August 2024

From the Alzheimer Center and Department of Neurology (P.J.v.d.V., L.N.C.V., A.C.V.H., H.F.R.-M., S.A.M.S., V.V., W.M.V.D.F.), and Department of Epidemiology and Biostatistics (P.J.v.d.V., J.H., L.N.C.V., J.B., W.M.V.D.F.), Amsterdam Neuroscience, VU University Medical Center; Amsterdam Neuroscience (P.J.v.d.V., L.N.C.V., A.C.V.H., H.F.R.-M., V.V., C.E.T., E.G., W.M.V.D.F.), Neurodegeneration the Netherlands; Division of Clinical Geriatrics (L.N.C.V.), Center for Alzheimer Research, Department of Neurobiology, Care Sciences and Society, Karolinska Institutet, Stockholm, Sweden; Medical Psychology (L.N.C.V.), Amsterdam UMC Location AMC, University of Amsterdam; Amsterdam Public Health (L.N.C.V.), Quality of Care, Personalized Medicine; Internal Medicine (H.F.R.-M.), Geriatric Medicine Section, Amsterdam Cardiovascular Sciences Institute, Amsterdam UMC Location VUmc; Department of Clinical, Neuro and Developmental Psychology (S.A.M.S.), Faculty of Movement and Behavioral Sciences, VU University; Department of Radiology & Nuclear Medicine (F.B., E.G.), Amsterdam UMC, Vrije Universiteit, the Netherlands; Queen Square Institute of Neurology and Centre for Medical Image Computing (F.B.), University College London, United Kingdom; and Neurochemistry Laboratory and Biobank (C.E.T.), Department of Clinical Chemistry, Amsterdam Neuroscience, VU University Medical Center, the Netherlands.

Background And Objectives: Cognitive decline rates in Alzheimer disease (AD) vary greatly. Disease-modifying treatments may alter cognitive decline trajectories, rendering their prediction increasingly relevant. We aimed to construct clinically applicable prediction models of cognitive decline in amyloid-positive patients with mild cognitive impairment (MCI) or mild dementia.

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Background: Although cardiovascular morbidity and mortality are substantial in patients with chronic kidney disease (CKD), guideline-directed treatment of cardiovascular risk factors remains a challenge.

Methods: Observational, cross-sectional study including patients aged 30-75 years with CKD stage 1-5 without kidney replacement therapy from a tertiary hospital outpatient clinic. Data were obtained through patient interview, clinical examination, biochemical work-up, and evaluation of medical records and prescription redemptions.

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ChatGPT versus Radiology Institutional Websites: Comparative Analysis of Radiation Protection Information Provided to Patients.

Radiology

June 2024

From the Department of Diagnostic and Interventional Radiology, Lausanne University Hospital (CHUV), Centre Hospitalier Universitaire Vaudois, Rue du Bugnon 46, 1011 Lausanne, Switzerland (S.J., D.R., C.P.); and Center for Virtual Imaging Trials, Carl E. Ravin Advanced Imaging Laboratories, Clinical Imaging Physics Group, Department of Radiology, Duke University Health System, Durham, NC (F.R.).

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Community-Based Cluster-Randomized Trial to Reduce Opioid Overdose Deaths.

N Engl J Med

September 2024

The authors' affiliations are as follows: Boston Medical Center, Boston University Chobanian and Avedisian School of Medicine, Department of Medicine, Section of General Internal Medicine, and Boston University School of Public Health (J.H.S.), Boston Medical Center and Boston University Chobanian and Avedisian School of Medicine, Departments of Medicine and Pediatrics (S.M.B.), Boston Medical Center, Section of General Internal Medicine (T.J.B., P.B., D. Beers, C. Bridden, K.C., J. Carpenter, E.B.G., A. Harris, S.K., Nikki Lewis, R.M.L., M.R., M. Saucier, R.S.C.), Boston University Chobanian and Avedisian School of Medicine (T.A.B.), Boston University School of Public Health, Department of Health Law, Policy and Management (D.D.B., M.D. Stein), Massachusetts Department of Public Health, Bureau of Substance Addiction Services (D. Calvert), Boston University School of Social Work (D. Chassler), Boston University School of Public Health, Department of Biostatistics (D.M.C.), Boston University Chobanian and Avedisian School of Medicine, Department of Medicine, Section of Infectious Diseases, and Boston University School of Public Health, Department of Health Law, Policy and Management (M.-L.D.), Massachusetts HCS Community Advisory Board (J.L.K., K.P.), Boston Medical Center, Section of Infectious Diseases (E.N.K., C.S.), Boston Medical Center and Boston University Chobanian and Avedisian School of Medicine, Department of Medicine, Section of General Internal Medicine (M.R. Larochelle, J.L.T., A.Y.W.), Boston Medical Center, Department of Medicine (H.M.L.), Massachusetts Department of Public Health, Registry of Vital Records and Statistics (S.P.), Tufts University School of Medicine, Department of Public Health and Community Medicine (T.J.S.), and McLean Hospital, Division of Alcohol, Drugs, and Addiction, and Harvard Medical School, Department of Psychiatry (R.W.) - all in Boston; the Social Intervention Group, School of Social Work, Columbia University (N.E.-B., A. Dasgupta, J.L.D., A. Davis, K.H.G., L. Gilbert, D.A.G.-E., D.E.G., J. Hotchkiss, T. Hunt, J.L.N., E.R., S. Rodriguez, E.W.), New York HCS Community Advisory Board (A. Angerame, R. Caldwell, S.M., K.M., J.P., K.R., W.R., M. Salvage), Columbia University Irving Medical Center, Department of Psychiatry (D. Blevins, A.N.C.C., F.R.L., E.V.N.), Albert Einstein College of Medicine, Department of Epidemiology and Population Health (N.B., D.G., D.W.L., B.D.R.), Montefiore Medical Center (J. Chaya), New York State Office of Addiction Services and Supports (C.O.C.), City University of New York (T. Huang, N.S.), Weill Cornell Medicine, Department of Population Health Sciences (B.S.), and the New York Office of Mental Health (A. Sullivan), New York, and the New York State Department of Health, Albany (T.Q.N., E.S.) - all in New York; the Department of Psychiatry and Behavioral Neuroscience, University of Cincinnati College of Medicine (T.J.W.), University of Cincinnati Corrections Institute (T.D.), University of Cincinnati College of Medicine, Department of Emergency Medicine (C.E.F., J. McMullan), University of Cincinnati Medical Center, Emergency Medicine (N.H.D.), University of Cincinnati College of Medicine, Department of Environmental and Public Health Sciences (T.I.), Case Western Reserve University School of Medicine, Department of Family Medicine and Community Health (T.V.P.), Brightview Health (S. Ryan), and University of Cincinnati College of Medicine, Department of Psychiatry and Behavioral Neuroscience (J.S.), Cincinnati, the College of Medicine (R.D.J., S.F., K.H., J.E.L., M.S.L.) and the College of Social Work (B.F.), Center of Health Outcomes and Policy Evaluation Studies (W.F.), Department of Family and Community Medicine (T.R.H., A.S.M., D.M.W.), College of Public Health and Translational Data Analytics Institute (A. Hyder), Department of Emergency Medicine (E.K.), Ohio Colleges of Medicine Government Resource Center (A.M., R.M.), One Ohio Foundation (A.N.), College of Public Health (P.S., E.E.S., A. Shoben), Recovery Ohio (A. Shadwick), and the School of Communication (M.D. Slater), Ohio State University, Columbus, Case Western Reserve University School of Medicine, Department of Population and Quantitative Health Sciences (D.A.F.), and Case Western Reserve University School of Medicine, Clinical and Translational Science Institute (M.W.K.), Cleveland, and Brown County Mental Health and Addiction Services, Georgetown (D.J.V.) - all in Ohio; RTI International, Research Triangle Park, NC (E.A.O., J.A., A. Aldridge, D. Babineau, C. Barbosa, R. Caspar, B.E., L. Glasgow, S.G., M.E.H., J. Holloway, C.K., P.A.L., R.C.L., L.N., N.V., G.A.Z.); the National Institute on Drug Abuse, Bethesda, MD (R.K.C., J.V.); University of Colorado School of Medicine, Divisions of General Internal Medicine and Infectious Diseases, Aurora (J.B.); University of North Texas Health Science Center, Fort Worth (S.T.W.); Kentucky Office of Drug Control Policy, Frankfort (V.L.I.), University of Kentucky, College of Public Health (H.M.B.), University of Kentucky, Kentucky Injury Prevention Research Center (J.L. Bush, S.L.H ), University of Kentucky College of Medicine, Department of Medicine, Division of Infectious Diseases, Center on Drug and Alcohol Research (L.C.F.), University of Kentucky, Department of Pharmacy Practice and Science (P.R.F., D.H., D.R.O.), Commonwealth of Kentucky, Cabinet for Health and Family Services (E.F., K.R.M.), University of Kentucky, Department of Communication (D.W.H., Nicky Lewis), University of Kentucky, Department of Behavioral Science (H.K.K.), University of Kentucky College of Medicine, Center on Drug and Alcohol Research (M.R. Lofwall, S.L.W.), University of Kentucky, Department of Health Management and Policy and Center for Innovation in Population Health (M.L.M.), University of Kentucky, Substance Use Research Priority Area (J. Miles, M.F.R., P.R., D.S.), University of Kentucky College of Medicine, Internal Medicine (D.A.O.), University of Kentucky, Department of Sociology (C.B.O.), University of Kentucky (B.D.R.), University of Kentucky, Department of Biostatistics (S.S., P.M.W.), University of Kentucky, Dr. Bing Zhang Department of Statistics (K.L.T.), University of Kentucky, Department of Behavioral Science (M. Staton, H.L.S.), University of Kentucky, Center for Health Equity Transformation (D.J.S.-W.), University of Kentucky College of Medicine, Institute for Biomedical Informatics (J.C.T.), and University of Kentucky, Department of Epidemiology and Environmental Health (R.A.V.-S., A.M.Y.), Lexington, and the Commonwealth of Kentucky, Clark County Health Department, Winchester (J.G.) - all in Kentucky; Purdue University, Department of Psychological Sciences, Lafayette, IN (J.L. Brown); University of Miami Miller School of Medicine, Department of Public Health Sciences, Miami (D.J.F.); Oak Ridge Associated Universities (ORAU), Health Communications, Marketing, and Promotion Program, Oak Ridge, TN (J.G.R.); and University of Pennsylvania School of Nursing, Department of Family and Community Health, Philadelphia (L.E.S.).

Article Synopsis
  • The study examines a community-based intervention aimed at reducing opioid-related overdose deaths by increasing the adoption of evidence-based practices including overdose education and naloxone distribution, medication treatment for opioid use disorder, and prescription safety.
  • In a cluster-randomized trial, 67 communities across Kentucky, Massachusetts, New York, and Ohio were assigned to either receive the intervention or serve as a control group during a period marked by the COVID-19 pandemic and an increase in fentanyl overdoses.
  • Results showed no significant difference in opioid-related overdose death rates between the intervention and control groups, with both averaging similar rates, indicating that the community-engaged strategies did not have a measurable impact during the study period.
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Article Synopsis
  • * The analysis revealed that higher aortic valve gradients (≥30 mm Hg) correlated with increased rates of serious outcomes like mortality and complications, while gradients under 20 mm Hg showed different relationships, indicating a potential threshold effect.
  • * Overall, the study suggests that monitoring aortic valve gradients post-implantation is crucial, as they can significantly influence patient survival and health outcomes, emphasizing the need for tailored patient management strategies.
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Background: Inactivated whole-virus vaccination elicits immune responses to both SARS-CoV-2 nucleocapsid (N) and spike (S) proteins, like natural infections. A heterologous Ad26.COV2.

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Despite the record-breaking discovery, development and approval of vaccines and antiviral therapeutics such as Paxlovid, coronavirus disease 2019 (COVID-19) remained the fourth leading cause of death in the world and third highest in the United States in 2022. Here, we report the discovery and characterization of PF-07817883, a second-generation, orally bioavailable, SARS-CoV-2 main protease inhibitor with improved metabolic stability versus nirmatrelvir, the antiviral component of the ritonavir-boosted therapy Paxlovid. We demonstrate the pan-human coronavirus antiviral activity and off-target selectivity profile of PF-07817883.

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Metformin and the Liver: Unlocking the Full Therapeutic Potential.

Metabolites

March 2024

Department of Medical and Surgical Sciences, IRCCS Azienda Ospedaliero, Universitaria di Bologna, 40138 Bologna, Italy.

Metformin is a highly effective medication for managing type 2 diabetes mellitus. Recent studies have shown that it has significant therapeutic benefits in various organ systems, particularly the liver. Although the effects of metformin on metabolic dysfunction-associated steatotic liver disease and metabolic dysfunction-associated steatohepatitis are still being debated, it has positive effects on cirrhosis and anti-tumoral properties, which can help prevent the development of hepatocellular carcinoma.

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Article Synopsis
  • * Anticholinergic medications can effectively treat conditions like drug-induced parkinsonism and dystonia but are not suitable for tardive dyskinesia, akathisia, or neuroleptic malignant syndrome.
  • * Caution is advised when prescribing anticholinergics due to their potential serious side effects, especially in older patients; they should be used at the lowest effective dose and tapered off gradually.
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Article Synopsis
  • A study was conducted to compare the perioperative outcomes of robotic liver surgery (RLS) and laparoscopic liver surgery (LLS) across various healthcare settings from 2009 to 2021.
  • The results showed that RLS had better outcomes in terms of "textbook outcomes," lower blood loss, fewer complications, and shorter operative times compared to LLS after matching patient groups for bias.
  • Despite the higher costs generally associated with robotic surgery, this study suggests that RLS may offer specific clinical advantages over LLS in minimally invasive liver procedures.
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A novel method to quantify fibrin-fibrin and fibrin-α-antiplasmin cross-links in thrombi formed from human trauma patient plasma.

J Thromb Haemost

June 2024

Radcliffe Department of Medicine, University of Oxford, Oxford, United Kingdom; Oxford Haemophilia and Thrombosis Centre, Oxford University Hospitals NHS Foundation Trust, Oxford, United Kingdom.

Background: The widespread use of the antifibrinolytic agent, tranexamic acid (TXA), interferes with the quantification of fibrinolysis by dynamic laboratory assays such as clot lysis, making it difficult to measure fibrinolysis in many trauma patients. At the final stage of coagulation, factor (F)XIIIa catalyzes the formation of fibrin-fibrin and fibrin-α-antiplasmin (αAP) cross-links, which increases clot mechanical strength and resistance to fibrinolysis.

Objectives: Here, we developed a method to quantify fibrin-fibrin and fibrin-αAP cross-links that avoids the challenges posed by TXA in determining fibrinolytic resistance in conventional assays.

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Computational drug repositioning identifies niclosamide and tribromsalan as inhibitors of Mycobacterium tuberculosis and Mycobacterium abscessus.

Tuberculosis (Edinb)

May 2024

Collaborations Pharmaceuticals Inc., 840 Main Campus Drive, Lab 3510, Raleigh, NC, 27606, USA. Electronic address:

Tuberculosis (TB) is still a major global health challenge, killing over 1.5 million people each year, and hence, there is a need to identify and develop novel treatments for Mycobacterium tuberculosis (M. tuberculosis).

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Innovations in cardiac imaging have fundamentally advanced the understanding and treatment of cardiovascular disease. These advances in noninvasive cardiac imaging have also expanded the role of the cardiac imager and dramatically increased the demand for imagers who are cross-trained in multiple modalities. However, we hypothesize that there is significant variation in the availability of cardiac imaging expertise and a disparity in the adoption of advanced imaging technologies across the United States.

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Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy.

N Engl J Med

January 2024

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, London (J.D.G., M.F.); the Medical University of South Carolina, Charleston, SC (D.P.J.); Tuscan Regional Amyloidosis Centre, Careggi University Hospital, Florence (F.C.), and the Amyloidosis Research and Treatment Center, IRCCS Fondazione Policlinico San Matteo, Pavia (L.O.) - both in Italy; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Centro de Investigacíon Biomédica en Red Enfermedades Cardiovaculares, and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.) - both in Madrid; European Reference Network for Rare, Low Prevalence and Complex Diseases of the Heart, Amsterdam (P.G.-P.); the Victorian and Tasmanian Amyloidosis Service, Department of Haematology, Monash University Eastern Health Clinical School, Box Hill, VIC, Australia (S.G.); the Department of Cardiovascular Diseases, Mayo Clinic, Rochester, MN (M.G.); the Department of Cardiovascular Medicine, Cleveland Clinic, Cleveland (M.H.); the Sylvester Comprehensive Cancer Center, University of Miami, Miami (J.H.), and the Amyloidosis Program, Department of Transplant, Mayo Clinic, Jacksonville (J.N.-N.) - both in Florida; the Cardiac Amyloidosis Program, Knight Cardiovascular Institute, Oregon Health and Science University, Portland (A.M.); the Cardiac Amyloidosis Program, Division of Cardiology, Columbia College of Physicians and Surgeons, New York (M.S.M.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); Duke Clinical Research Institute (F.R., J.G., K.C., H.X.) and Duke University Medical Center (F.R.) - both in Durham, NC; the Pauley Heart Center, Virginia Commonwealth University, Richmond (K.B.S.); the Division of Cardiology, University of Pittsburgh Medical Center, Pittsburgh (P.S.); and Eidos Therapeutics affiliate of BridgeBio Pharma, San Francisco (X.C., T.L., U.S., J.C.F.).

Article Synopsis
  • - Transthyretin amyloid cardiomyopathy involves the buildup of misfolded proteins in the heart, and acoramidis is a medication designed to stabilize these proteins, showing over 90% effectiveness in laboratory tests.
  • - In a phase 3 trial, 632 patients were randomly assigned to receive either acoramidis or a placebo for 30 months, evaluating outcomes like death rates, hospitalizations, and specific health indicators.
  • - The results demonstrated that acoramidis significantly outperformed the placebo in multiple health measures, with a high win ratio favoring acoramidis, while the frequency of side effects was similar in both groups.
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