427 results match your criteria: "New York (T.K.); and Karolinska Institute[Affiliation]"

Background And Objectives: Drug development in schizophrenia is limited by the differential scaling of the active treatment and placebo arms of a study, such that, as the number of sites increases, the magnitude of placebo response disproportionately increases. The objective of this article was to identify factors conducive to efficient recruitment as a step towards trial designs allowing recruitment of more participants per site, leading to reduced variability, and potentially a smaller placebo effect.

Patients And Methods: Using the information of 554 individuals, we calculated the percentage of individuals who were screened, consented, and retained in our research, along with rationale for nonconsent.

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High-Resolution Free-Breathing Chemical-Shift-Encoded MRI for Characterizing Lymph Nodes in the Upper Abdomen.

Invest Radiol

January 2025

From the Department of Medical Imaging, Radboud University Medical Center, Nijmegen, the Netherlands (I.T.M., M.C.M., S.Y., R.v.d.E., A.V., E.J.S., J.J.H., T.W.J.S.); and Department of Radiology, NYU Langone Health, New York, NY (T.K.B.).

Objectives: Accurate lymph node (LN) staging is crucial for managing upper abdominal cancers. Ultrasmall superparamagnetic iron oxide (USPIO)-enhanced magnetic resonance imaging effectively distinguishes healthy and metastatic LNs through fat/water and -weighted imaging. However, respiratory motion artifacts complicate detection of abdominal LNs.

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Background: Intravascular ultrasound (IVUS) use in aortic endovascular interventions, including thoracic endovascular aneurysm repair (TEVAR) and endovascular aneurysm repair (EVAR), may have similar benefits to those seen in coronary and peripheral interventions, but limited utilization and outcome data exist.

Methods: Centers for Medicare and Medicaid Services claims data were used to identify patients undergoing TEVAR and EVAR from 2016 to 2023. Utilization trends were stratified by region, urbanicity, distressed communities index, community versus academic center, Medicare versus dual enrollment status, indication, urgency, and presence of dissection with malperfusion.

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Background: Covered stent correction for a sinus venosus atrial septal defect (SVASD) was first performed in 2009. This innovative approach was initially viewed as experimental and was reserved for highly selected patients with unusual anatomic variants. In 2016, increasing numbers of procedures began to be performed, and in several centers, it is now offered as a standard of care option alongside surgical repair.

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Adeno-associated virus-based gene therapy is a promising avenue in heart failure treatment, but has shown limited cardiac virus uptake in humans, requiring new approaches for clinical translation. Using a Yorkshire swine ischemic heart failure model, we demonstrate significant improvement in gene uptake with temporary coronary occlusions assisted by mechanical circulatory support. We first show that mechanical support during coronary artery occlusions prevents hemodynamic deterioration (n = 5 female).

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Background: Underlying intracranial stenosis is the most common cause of failed mechanical thrombectomy in patients with acute ischemic stroke with large vessel occlusion. Adjunct emergent stenting is sometimes performed to improve or maintain reperfusion, despite limited data regarding its safety or efficacy.

Methods: We conducted a prospective multicenter observational international cohort study.

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Background: Percutaneous microaxial ventricular assist devices (pVADs) have the potential to reduce mortality of patients with cardiogenic shock (CS). However, the association between the distribution of pVAD-performing centers and outcomes of CS has not been explored.

Methods: This observational study included Medicare fee-for-service beneficiaries aged 65 to 99 years treated with pVAD for CS from 2016 to 2020.

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Objective: To evaluate whether an antimicrobial stewardship bundle (ASB) can safely empower frontline providers in the treatment of gram-negative bloodstream infections (GN-BSI).

Intervention And Method: From March 2021 to February 2022, we implemented an ASB intervention for GN-BSI in the electronic medical record (EMR) to guide clinicians at the point of care to optimize their own antibiotic decision-making. We conducted a before-and-after quasi-experimental pre-bundle (preBG) and post-bundle (postBG) study evaluating a composite of in-hospital mortality, infection-related readmission, GN-BSI recurrence, and bundle-related outcomes.

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Disruption of cellular plasticity by repeat RNAs in human pancreatic cancer.

Cell

December 2024

Mass General Cancer Center, Harvard Medical School, Charlestown, MA 02129, USA; Department of Medicine, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA. Electronic address:

Article Synopsis
  • Aberrant expression of repeat RNAs in pancreatic ductal adenocarcinoma (PDAC) resembles viral responses, affecting tumor cells and their microenvironment.
  • A study on 46 primary tumors revealed that high repeat RNA levels correlate with changes in cell identity in both PDAC cells and cancer-associated fibroblasts (CAFs).
  • The distinct immune signaling pathways in PDAC and CAFs, particularly involving interferon regulatory factor 3 (IRF3), highlight how these viral-like responses impact cellular flexibility and interactions within the tumor environment.
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The U.S. Latino HIV Crisis - Ending an Era of Invisibility.

N Engl J Med

November 2024

From the Center for Latino Adolescent and Family Health (V.G.-R., M.T.-K., A.B.), and the Institute for Policy Solutions, School of Nursing (V.G.-R., M.T.-K., A.B.), Johns Hopkins University; and the U.S. Presidential Advisory Council on HIV/AIDS (V.G.-R.) - both in Washington, DC; and the Department of Epidemiology, Mailman School of Public Health, Columbia University (M.T.-K.), and the Department of Applied Psychology, Steinhardt School of Culture, Education, and Human Development, New York University (A.B.) - both in New York.

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Anticoagulation vs Antiplatelets Across Subgroups of Embolic Stroke of Undetermined Source: A Meta-Analysis of Randomized Controlled Trials.

Neurology

November 2024

From the Department of Neurology (M.G., Q.N.A., A.A.), University of Iowa Hospitals & Clinics, Iowa City; Department of Neurology (A.M.A.-Q.), University of Pittsburgh Medical Center, PA; Departments of Biostatistics (R.K.), Neurology (W.T.L., D.T.) and Epidemiology (W.T.L.), University of Washington, Seattle; Department of Internal Medicine (G.N.), Faculty of Medicine, School of Health Sciences, University of Thessaly, Larissa, Greece; Hardin Library for the Health Sciences (C.A.C.), The University of Iowa, Iowa City; Department of Cardiology and Angiology (T.K., T.G.), and Institute for Clinical Epidemiology and Applied Biometry (L.M.S.-H.), Faculty of Medicine, Eberhard Karls University Tübingen, Germany; Department of Neurology (K.F., S.Y.), Brown University, Providence, RI; Department of Neurology (J.L.S.), University of California at Los Angeles (UCLA); Department of Neurology (S.E.K.), University of Pennsylvania, PA; Department of Neurology (M.S.V.E.), Vagelos College of Physicians and Surgeons; Department of Epidemiology (M.S.V.E.), Mailman School of Public Health, Columbia University, New York, NY; Department of Neurology & Stroke (S.P.), and Hertie Institute for Clinical Brain Research (S.P.), Eberhard Karls University Tübingen, Germany; and Department of Neurology (H.K.), Weill Cornell Medicine, New York, NY.

Article Synopsis
  • The study focuses on patients with ischemic stroke classified as embolic stroke of undetermined source (ESUS), highlighting the need for personalized treatment options between anticoagulation and antiplatelet therapy for secondary prevention.
  • A meta-analysis of 7 trials with nearly 15,000 patients showed that, overall, anticoagulation did not significantly reduce the risk of recurrent ischemic stroke compared to antiplatelet therapy.
  • However, in patients with a patent foramen ovale (PFO), anticoagulation demonstrated improved outcomes, while results varied for those with left atrial enlargement (LAE) depending on whether cardiac monitoring was allowed after randomization.*
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Policy Points In a recently commissioned report on solutions for eliminating racial and ethnic health care inequities entitled Ending Unequal Treatment, the National Academies of Sciences, Engineering, and Medicine found a health workforce that is representative of the communities it serves is essential for health care equity. The Supreme Court decision to ban race-conscious admission constraints pathways toward health workforce representativeness and equity. This paper draws on the National Academies report's findings that health care workforce representativeness improves care quality, population health, and equity to discuss policy and programmatic options for various participants to promote health workforce representativeness in the context of race-conscious admissions bans.

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Curriculum Research: Disseminating Neuropalliative Care Education Through an Adaptable Curriculum: A Multisite Feasibility Trial.

Neurol Educ

June 2024

From the Division of Geriatric Medicine and Palliative Care (E.H.), Department of Medicine, NYU Langone Health, New York; Department of Neurology (B.L.T., C.J.C.), University of Washington, Seattle; Department of Neurology & Neurological Sciences (H.L.K.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (S.G., K.T.K., T.G.), New York Presbyterian-Columbia University Irving Medical Center, NY; Division of Palliative Care (K.T.B.), Departments of Neurology and Medicine, Massachusetts General Hospital, Boston; and Cambia Palliative Care Center of Excellence (C.J.C.), University of Washington, Seattle.

Introduction And Problem Statement: Neurologic disease is a leading cause of disability and death worldwide. As the global population ages, the burden of these diseases is expected to increase. Despite this increased clinical need, neurology trainees are seldom taught skills and concepts in palliative care.

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Gene-Specific Effects on Brain Volume and Cognition of in Frontotemporal Lobar Degeneration.

Neurology

October 2024

From the VIB Center for Molecular Neurology (M.V., R.R., V.B., S.W.); Department of Biomedical Sciences (M.V., M.V.B., S.W., R.R.), University of Antwerp, Belgium; Department of Neurology (E.M.R., M.F.M.), David Geffen School of Medicine, University of California, Los Angeles; Department of Neurology (N.C.-L., V.K.R., T.K., K.K., B.F.B.); Department of Psychiatry and Psychology (N.C.-L., J.A.F., D.S.K., L.K.F.), Mayo Clinic, Rochester, MN; Department of Epidemiology and Biostatistics (J.K.), University of California, San Francisco; Department of Quantitative Health Sciences (C.M., D.E.B.), Mayo Clinic, Rochester, MN; Department of Neurology (A.M.S., A.A.W.), Memory and Aging Center, University of California, San Francisco; Weill Institute for Neurosciences, San Francisco, California; Institute for Precision Health (D.H.G.), Departments of Neurology, Psychiatry and Human Genetics at David Geffen School of Medicine, UCLA; Department of Neuroscience (T.G., L.P., M.B., N.R.G.-R.), Mayo Clinic, Jacksonville, FL; Alzheimer's Disease and Other Cognitive Disorders Unit (S.B.-É.), Neurology Service, Hospital Clínic de Barcelona, Institut d'Investigacions Biomèdiques August Pi i Sunyer (IDIBAPS), Fundació Clínic per a la Recerca Biomèdica, Uni; Department of Neurology (B.A., B.C.D.), Case Western Reserve University, Cleveland, OH; Department of Neurology (S.B.), University of Michigan, Ann Arbor; Department of Neurology (A.C.B.), University of North Carolina, Chapel Hill; Department of Neurology (D.C.), Indiana University, Indianapolis; Department of Neurology (R.R.D.), Vanderbilt University, Nashville, TN; Department of Neurology (K.D.-R.), University of Washington, Seattle, WA; Department of Neurosciences (D.G., G.C.L., I.L.), University of California, San Diego, La Jolla; Departments of Neurology and Psychiatry (N.G.), Washington University School of Medicine, Washington University, St. Louis, MO; Department of Psychiatry and Behavioral Sciences (I.M.G.), Northwestern Feinberg School of Medicine, Chicago, IL; Taub Institute for Research on Alzheimer's Disease and the Aging Brain (L.S.H.), College of Physicians and Surgeons; Department of Neurology (L.S.H.), Columbia University, New York; Division of Neurology (G.-Y.R.H.), University of British Columbia, Vancouver, Canada; Department of Psychiatry and Human Behavior (E.D.H.), Alpert Medical School of Brown University, Providence, RI; Department of Neurology and Penn Frontotemporal Degeneration Center (D.J.I.), Perelman School of Medicine, University of Pennsylvania, Philadelphia; National Institute of Neurological Disorders and Stroke (J.Y.K., A.S.), National Institutes of Health, Bethesda, MD; Department of Neurology (J.C.M., B.P.), Houston Methodist, TX; Department of Psychiatry and Behavioral Sciences (C.U.O.), Johns Hopkins University, Baltimore, MD; Department of Neurology (P.S.P.), University of Colorado, Aurora; Cleveland Clinic Lou Ruvo Center for Brain Health (A.R., D.W.), Las Vegas, NV; Department of Neurology (E.D.R.), University of Alabama at Birmingham; Glenn Biggs Institute for Alzheimer's & Neurodegenerative Diseases (A.C.S.), UT Health San Antonio; Tanz Centre for Research in Neurodegenerative Diseases (M.C.T.), Division of Neurology, University of Toronto, Ontario, Canada; Department of Neurology (H.W.H., A.L.B., H.J.R.), Memory and Aging Center, University of California, San Francisco; Weill Institute for Neurosciences, San Francisco, CA; and Department of Neuroscience (R.R.), Mayo Clinic, Jacksonville, FL.

Article Synopsis
  • The study investigates the role of the genetic variant rs1990622 as a potential modifier of disease risk in frontotemporal lobar degeneration (FTLD), particularly among those with pathogenic variants.
  • Researchers enrolled participants from the ALLFTD study, analyzing the impact of rs1990622 on gray matter volume and cognitive function across various genetic groups related to FTD.
  • Results indicate that carriers of the minor allele of rs1990622 show increased gray matter volume and better cognitive performance, especially in the thalamus and among presymptomatic individuals.
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Background And Objective: Within recent years, transcatheter tricuspid edge-to-edge repair (T-TEER) has emerged as a safe and effective treatment option in patients with secondary tricuspid regurgitation (TR). However, for primary TR, data on the robustness and durability of TR reduction 1 year following T-TEER is limited.

Methods: All consecutive patients treated with T-TEER for TR at two high-volume centers between September 2018 and December 2022 were enrolled in a registry.

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Intravenous Thrombolysis in Patients With Cervical Artery Dissection: A Secondary Analysis of the STOP-CAD Study.

Neurology

October 2024

From the Department of Neurology (L.S., F. Akpokiere, D.M.M., K.P., V.D., K.B., T.M.B., N.S.K., F. Khan, C.S., N. Mohammadzadeh, E.D.G., K.F., S. Yaghi), Warren Alpert Medical School of Brown University, Providence, RI; Vancouver Stroke Program (T.S.F., L.Z., P.G.), Division of Neurology, University of British Columbia, Vancouver, Canada; Department of Neurology (C.R.L.G.), Atrium Health, Charlotte, NC; Department of Neurology (J. Muppa, N.H.), University of Massachusetts Chan Medical School, Worcester; Department of Neurology (M. Affan, O.U.H.L.), University of Minnesota, Minneapolis; Department of Neurology (M.R.H., K.A., D.J.S., M. Arnold), Inselspital, University Hospital and University of Bern, Switzerland; Department of Neurology (S.S.O., R. Crandall), University of Colorado, Denver; Department of Neurology (E.L.), Weill Cornell Medicine, New York; ; Instituto Nacional de Neurología y Neurocirugía Manuel Velasco Suarez (D.L.-M., A. Arauz), Mexico City, Mexico; Service de neurologie (A.N., M.B., E.T.), Université Caen Normandie, CHU Caen Normandie, France; Department of Neurology (J.A.S., J.S.-F., V.B.), Coimbra University, ; Department of Internal Medicine (P.C.-C., M.T.B.), São João University Hospital, Porto, Portugal; Department of Neurology (M.K., D.M.), Corewell Health, Grand Rapids, MI; Department of Neurology (M.K.), Mayo Clinic, Rochester, MN; Department of Neurology (A.R., O.K.), University of Pennsylvania, Philadelphia; Neurology and Neurorehabilitation (J.E.K., S.T.E., C.T.), University Department of Geriatric Medicine FELIX PLATTER, Department of Clinical Research, University of Basel, and University Hospital Basel, Switzerland; Stroke Center (D.A.d.S.), Centro Hospitalar Universitário Lisboa Central, and Institute of Anatomy, Faculdade de Medicina da Universidade de Lisboa; Department of Neurology (M.D.S.); Department of Neuroradiology (S.B.R.), Centro Hospitalar Universitário Lisboa Central, Lisbon, Portugal; Vancouver Stroke Program (S. Mancini), Division of Neurology, University of British Columbia, Vancouver, Canada; Department of Neurology (I.M., R.R.L.), Hadassah-Hebrew University Medical Center, Jerusalem, Israel; Department of Neurology with Experimental Neurology (R.V.R., C.H.N.), Charite Universitätsmedizin-Berlin and Center for Stroke Research, Berlin, and Berlin Institute of Health, Germany; Department of Neurosciences (R. Choi, J. MacDonald), ChristianaCare, Newark, DE; Department of Neurology (R.B.S.), University of California at San Diego; Department of Neurology (X.G.), Loma Linda University, Loma Linda, CA; Department of Neurology (M. Ghannam, M. Almajali, E.A.S.), University of Iowa, Iowa City; Department of Neurosciences (B.R., F.Z.-E., A.P.), Université de Montréal, Canada; Department of Neurology (A.C.F., M.F.B., D.C.), Hospital de Santa Maria, Centro de Estudos Egas Moniz, Faculdade de Medicina, Universidade de Lisboa, Portugal; Neurology and Stroke Unit (M. Romoli, G.D.M., M.L.), Department of Neuroscience, Bufalini Hospital, Cesena, Italy; Department of Neurology (Z.K., K.J.G.), Mayo Clinic, Rochester, MN; Department of Neurology (L.K., J.A.F.), NYU Langone Health, New York; Department of Neurology (J.Y.A., J.A.G.), Washington University, Saint Louis, MO; Neurology Unit, Stroke Unit (M. Zedde, I.G.), Azienda Unità Sanitaria Locale-IRCCS di Reggio Emilia; Neuroradiology Unit (R.P.), Azienda Unità Sanitaria Locale-IRCCS di Reggio Emilia; Department of Internal Medicine (H.N.), Centro Hospital Universitario do Algarve, Faro, Portugal; Department of Neurology (D.S.L., A.M.), University of California at Los Angeles; Department of Neurology (A.C., B.M.G., R.W.), Duke University, Durham, NC; Department of Neurology (W.K.), University of North Carolina Health Rex, Raleigh; Department of Neurology (S.A.K., M. Anadani), Medical University of South Carolina, Charleston, SC; Department of Neurosurgery (K.P.K.), Medical University of South Carolina, Charleston, SC; Department of Neurology (A.E., L.C., R.C.R., Y.N.A., E.A.M.), University of Cincinnati Medical Center, OH; Department of Neurology (E.B., T.L.T.), University of Alabama at Birmingham; Department of Neurology (M.R.-G., M. Requena), University Hospital Vall d'Hebron, Barcelona, Spain; Department of Neurology (F.G.S.V., J.O.G.), University of Oklahoma; Department of Neurology (V.M.), Einstein-Jefferson Healthcare Network, Philadelphia, PA; Department of Neurology (A.H.), University of Utah, Salt Lake City; Department of Neurology (A.H.); Department of Neurology (S. Sanchez, A.S.Z., Y.K.C., R.S.), Yale New Haven Hospital, New Haven, CT; Department of Neurology (V.Y.V.), All India Institute of Medical Sciences, New Delhi, India; Department of Neurology (S. Yaddanapudi, L.A., A. Browngoehl), Thomas Jefferson University, Philadelphia, PA; Department of Neurology (T.R., R.D., Z.L.), Wake Forest Medical Center, NC; Department of Neurology (M.P., J.E.S.), Cooper University, Camden, NJ; Department of Neurology (S. Mayer, J.Z.W.), Columbia University Medical Center, New York, NY; Department of Neurology (J.P.M., D.K.), Hospital de Egas Moniz, Centro Hospitalar Lisboa Ocidental, Lisbon, Portugal; Department of Neurology (P.K., T.N.N.), Boston Medical Center, MA; Department of Neurology (S.D.A., Z.S., A. Balabhadra, S.P.), Hartford Hospital, CT; Department of Neurology (T.S.), Hospital Moinhos de Vento; Department of Neurology (S.C.M., G.P.M.), Hospital de Clínicas de Porto Alegre, Porto Alegre, Brazil; Department of Neurology (Y.D.K.), Yonsei University, Seoul, South Korea; Department of Neurology (B.K., C.E.), University of Tennessee at Memphis; Department of Neurology (S. Lingam, A.Y.Q.), Kansas University Medical Center, Kansas City; Department of Neurology (S.F., A. Alvarado), Western Ontario University, London, Canada; Department of Neurology (F. Khasiyev, G.L.), Saint Louis University, MO; Department of Neurology and Stroke Unit (M.M., V.T.), AOOR Villa Sofia-V. Cervello, Palermo, Italy; First Department of Neurology (A.T., V.T.-P.), National and Kapodistrian University of Athens, Greece; Department of Neurology (M.M.M.-M., V.C.W.), Centro Médico Nacional Siglo XXI IMSS., México City; Department of Neurology (F.I., S.E.E.J.), The Miriam Hospital, Providence, RI; Department of Neurocritical Care (S. Liu, M. Zhou), The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology, Hefei, China; Department of Neurology (M.M.A., F. Ali, M.S.), West Virginia University, WV; Department of Neurology (R.Z.M., T.K.-H.), University of Chicago, IL; Department of Neurology (F.S., J.Z.), Sir Run Run Shaw Hospital of Zhejiang University Medical School, Hangzhou, China; Department of Neurology (D.S., J.S., N. Mongare), Aga Khan University, Nairobi, Kenya; Department of Neurology (A.N.S., R.G., Shayak Sen), Cedars Sinai Medical Center, Los Angeles, CA; Department of Neurology (M. Ghani, M.E.), University of Louisville, KY; and Department of Economics (H.X.), University of California, Santa Barbara.

Article Synopsis
  • Cervical artery dissection (CeAD) is a leading cause of ischemic strokes in young adults, and this study explored the effects of intravenous thrombolysis (IVT) on patients with CeAD and stroke symptoms.
  • Analyzed data from the STOP-CAD study, it found that IVT significantly improved functional independence after 90 days in patients without increasing the risk of symptomatic intracranial hemorrhage.
  • The results suggest that IVT is a beneficial treatment for eligible patients with CeAD, aligning with current medical guidelines on its use.
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Article Synopsis
  • The genetic factors contributing to stroke risk in South Asians remain largely unstudied, with a recent study examining 75,000 Pakistanis using exome-wide sequencing.
  • A specific genetic variant, NOTCH3 p.Arg1231Cys, was found to be more common in South Asians (0.58%) compared to Western Europeans (0.019%) and was significantly linked to hemorrhagic and overall stroke risk.
  • This variant accounts for about 2.0% of hemorrhagic strokes and 1.1% of all strokes in South Asians, emphasizing the importance of including diverse populations in genetic research for better understanding and treatment of stroke.
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Background: Modulation of glucocorticoid receptor (GR) activity in tumor cells enhances chemotherapy efficacy. We evaluated the selective GR modulator relacorilant plus nab-paclitaxel in patients with metastatic pancreatic ductal adenocarcinoma (mPDAC) who had received at least 2 prior therapy lines.

Patients And Methods: In this open-label, single-arm, phase III study, patients received once-daily oral relacorilant (100 mg, titrated to 150 mg in 25 mg increments/cycle) and nab-paclitaxel (80 mg/m2) on days 1, 8, and 15 of 28-day cycles.

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Framework for Multistakeholder Patient Registries in the Field of Rare Diseases: Focus on Neurogenetic Diseases.

Neurology

September 2024

From the Department of Child Neurology (D.H.S., M.S.v.d.K., N.I.W.), Emma's Children's Hospital, Amsterdam UMC location Vrije Universiteit; Amsterdam Leukodystrophy Center (D.H.S., M.S.v.d.K., N.I.W.), Amsterdam Neuroscience, Cellular & Molecular Mechanisms; Medicine for Society (D.H.S., S.v.d.B., N.R., C.E.M.H.), Platform at Amsterdam UMC location University of Amsterdam; Department of Endocrinology and Metabolism (S.v.d.B., A.B., M.R.D., N.R., C.E.M.H.), Amsterdam UMC location University of Amsterdam; National Health Care Institute (Zorginstituut Nederland) (L.T.), Diemen, the Netherlands; Division of Child Neurology (L.A.A.), Children's Hospital of Philadelphia, PA; Institute of Systems Motor Science (T.B.), CBBM, Universität of Lübeck; Centre of Rare Diseases (T.B.), University Hospital Schleswig Holstein, Lübeck, Germany; Division of Metabolic Diseases (A.B.), Department of Pediatrics, Emma Childrens' Hospital, Amsterdam UMC location University of Amsterdam, the Netherlands; National Health Care Institute RIZIV-INAMI (M.v.d.C.), Brussels, Belgium; VKS (H.D.), Dutch Patient Organization for Metabolic Diseases, Zwolle; United for Metabolic Diseases (UMD) (H.D.), Amsterdam, the Netherlands; International Niemann-Pick Disease Registry (C.D.), Washington, Tyne & Wear, United Kingdom; VSOP-Patient Alliance for Rare and Genetic Diseases (M.H.E.D.), Soest, the Netherlands; Institute for Medical Genetics and Applied Genomics (H.G.), University of Tübingen; Centre for Rare Disease (H.G.), University Hospital Tübingen, Germany; Yaya foundation for 4H Leukodystrophy (V.G.), Minneapolis, MN; Orphanet (T.H.), INSERM US14 Rare Disease Platform, Paris, France; Department of Neurology (G.U.H.), LMU University Hospital, Ludwig-Maximilians-Universität (LMU), Munich; German Center for Neurodegenerative Diseases e.V. (DZNE) (G.U.H., T.K.), Munich; Munich Cluster for Systems Neurology (SyNergy) (G.U.H.), Germany; Department of Pediatrics (H.v.d.H.), Center for Lysosomal and Metabolic Diseases, Erasmus MC University Medical Center, Sophia Children's Hospital, Rotterdam; European Medicines Agency (C.J., K.P.), Amsterdam; Medicines Evaluation Board (C.J.), Utrecht; Department of Endocrinology and Metabolism (M.L.), Amsterdam UMC, Amsterdam Gastroenterology Endocrinology Metabolism (AGEM) Research Institute, University of Amsterdam, the Netherlands; Canadian Agency for Drugs and Health Technology Technologies Agendcy in Health (CADTH) (L.J.L.), Ottawa, Ontario, Canada; CHDI Management, Inc. (E.N.), the company that manages the scientific activities of CHDI Foundation, Inc., New York, NY; National Health Care Institute (M.N., W.G.G.), Diemen, the Netherlands; Department of Neurology (T.K.), University of Bonn, Germany; Department of Integrative Neurophysiology (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; European Commission (A.P.), Joint Research Centre (JRC), Ispra, Italy; Patient Advocate Organization 'Vereniging HCHWA-d' (HCHWA-D Association) (S.v.R.), the Netherlands; European Leukodystrophies Association (E.F.S.-V.), Paris, France; Medical BioSciences Department (B.d.S.V.), Radboud University Medical Center, Nijmegen; and WHO Collaborating Centre for Pharmaceutical Policy and Regulation (W.G.G.), Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht University, the Netherlands.

Article Synopsis
  • Progress in genetic diagnosis and orphan drug legislation has led to new therapies for rare neurogenetic diseases (RNDs), but challenges remain in academia, regulation, and finances.
  • The study aims to create a practical framework for developing patient registries that address these challenges and enhance outcomes in care, research, and drug development for RNDs.
  • A comprehensive approach combining literature review, interviews with existing registries, and feedback from various stakeholders was used to ensure the framework meets diverse needs and emphasizes key principles like accessible, independent, and trustworthy data governance.
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Quantitative Ga-PSMA-11 PET and Clinical Outcomes in Metastatic Castration-resistant Prostate Cancer Following Lu-PSMA-617 (VISION Trial).

Radiology

August 2024

From the University of Arizona, Tucson, Ariz (P.H.K.); Memorial Sloan-Kettering Cancer Center, New York, NY (M.J.M.); Invicro, Needham, Mass (J.H.); Mayo Clinic, Rochester, Minn (A.T.K., O.S.); Department of Nuclear Medicine, University Hospital Münster, Münster, Germany (K.R.); West German Cancer Center, Münster and Essen, Germany (K.R.); Dana-Farber Cancer Institute, Boston, Mass (X.X.W.); Astera Cancer Care, East Brunswick, NJ (B.F.); Indiana University Simon Comprehensive Cancer Center, Indianapolis, Ind (N.A.); Miami Cancer Institute, Baptist Health South Florida, Miami, Fla (R.G.); Washington University, St. Louis, Mo (J.M.M.); British Columbia Cancer Agency, Vancouver, British Columbia, Canada (K.C.); The Institute of Cancer Research and Royal Marsden Hospital, London, United Kingdom (J.d.B.); Gustave Roussy Institute, University of Paris-Saclay, Villejuif, France (K.F.); Rostock University Medical Center, Rostock, Germany (B.K.); Weill Cornell Medicine, New York, NY (S.T.T.); Novartis Pharmaceuticals, East Hanover, NJ (S.G.); Novartis Pharmaceuticals, Indianapolis, Ind (M.B.); Novartis Pharmaceuticals, Cambridge, Mass (C.C.W.); Novartis Pharmaceuticals, Geneva, Switzerland (A.M.C.); Novartis Pharmaceuticals, St. George, Utah (T.B.); Duke Cancer Institute Center for Prostate and Urologic Cancers, Duke University, Durham, NC (A.J.A.); and University Hospital Essen and German Cancer Consortium, Hufelandstr. 55, 45147 Essen, Germany (K.H.).

Article Synopsis
  • Lutetium 177 (Lu-PSMA-617) is a targeted therapy for metastatic castration-resistant prostate cancer (mCRPC), and baseline Ga-PSMA-11 PET/CT parameters may help determine treatment effectiveness.
  • The analysis used data from the VISION trial, where participants received either Lu-PSMA-617 plus standard care or standard care alone, focusing on how various PET parameters related to treatment outcomes like survival and response rates.
  • Results showed that higher whole-body tumor standardized uptake value (SUV) was linked to better treatment outcomes; for every 1-unit increase in SUV, the risk of radiographic progression and death decreased, indicating Lu-PS
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Article Synopsis
  • The 2022 European LeukemiaNet (ELN) classification predicts outcomes for younger acute myeloid leukemia (AML) patients but was tested for those aged 60 and older receiving lower-intensity treatment (LIT), involving 595 patients with varying risk levels.
  • Results showed that while ELN risk is predictive of overall survival, it fails to distinguish between favorable and intermediate risks, prompting further exploration into adverse-risk patients' molecular abnormalities.
  • A new "Beat-AML" risk classification was developed, combining favorable and intermediate risks and integrating mutation scoring, leading to better survival predictions for older AML patients and aiding treatment decisions with clear risk group delineations.
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Assessment of Thrombectomy versus Combined Thrombolysis and Thrombectomy in Patients with Acute Ischemic Stroke and Medium Vessel Occlusion.

Radiology

August 2024

From the Neuroendovascular Program, Massachusetts General Hospital & Brigham and Women's Hospital, Harvard University, Boston, Mass (A.A.D., R.W.R., C.J.S., J.D.R., A.B.P.); Departments of Medical Imaging and Neurosurgery, Neurovascular Centre, St. Michael's Hospital, Toronto, ON, Canada M5B 1W8 (A.A.D., N.M.C., T.R.M., V.M.P.); Departments of Neurologic Surgery & Radiology, Mayo Clinic, Rochester, Minn (S.G., H.K., R.K.); Cooper Neurologic Institute, Cooper University Hospital, Cooper Medical School of Rowen University, Camden, NJ (J.E.S., H.S., J.K., A.J.T., A.G.); Departments of Radiology & Neurology, Boston Medical Center, Boston, Mass (M.A., P. Klein, T.N.N.); Department of Interventional Neuroradiology, Stanford Medical Center, Palo Alto, Calif (J.J.H.); Department of Neurosurgery, Thomas Jefferson University, Philadelphia, Pa (K.E.N., A.A., S.I.T., P.J.); Department of Neurosurgery and Interventional Neuroradiology, Louisiana State University, Shreveport, La (H.A.S., B.M., N.A., H.H.C.S.); Department of Diagnostic and Interventional Neuroradiology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany (L.M., J.F. T.D.F.); Department of Neurology, Hôpital Civil Marie Curie, Charleroi, Belgium (A.D., F.B.); Department of Neuroradiology, University Hospital of Limoges, Université de Limoges, Limoges, France (G.F., A.R., S. Saleme, C.M.); Department of Radiology, Division of Neurointerventional Radiology, University of Massachusetts Medical Center, Worcester, Mass (A.L.K., A.S.P.); Department of Neuroradiology, Sana Kliniken, Lübeck GmbH, Lübeck, Germany (C.D.); Department of Neurosurgery, University of Texas Medical Branch, Galveston, Tex (P.T.K., M.C.); Department of Interventional Neuroradiology, Bordeaux University Hospital, Bordeaux, France (G.M., J.B., X.B.); Department of Neurology, Bordeaux University Hospital, Bordeaux, France (I.S.); Department of Neurology, University of Massachusetts Chan Medical School, Worcester, Mass (S.N., N.H.); Department of Psychiatry, University of Massachusetts Chan Medical School, Worcester, Mass (N.H.); Department of Neurology, Beth Israel Deaconess Medical Center, Harvard Medical School, Boston, Mass (N.H.); Department of Neurosurgery, Tokyo Metropolitan Tama Medical Center, Tokyo, Japan (T.O., S.D.); Department of Medicine, Yong Loo Lin School of Medicine, National University of Singapore, Singapore (L.L.L.Y., B.Y.Q.T.); Department of Medicine, Division of Neurology, National University Hospital, Singapore (B.Y.Q.T.); Department of Neurology, UTHealth McGovern Medical School, Houston, Tex (J.C.M.G., S.S.M.); Interventistica Neurovascolare, Ospedale Careggi di Firenze, Florence, Italy (S. Sheth, L.R., C.C.); Department of Neurologic Surgery, Division of Stroke and Endovascular Neurosurgery, Keck School of Medicine, University of Southern California, Los Angeles, Calif (A.M.); Department of Endovascular Neurosurgery and Neuroradiology, Rutgers University New Jersey Medical School in Newark, Newark, NJ (P. Khandelwal); Department of Neurosurgery, Westchester Medical Center at New York Medical College, Valhalla, NY (A.B.); Department of Neuroradiology, Pitié-Salpêtrière Hospital, GRC BioFast, Sorbonne University, Paris, France (F.C., M.E., K.P.); Neurology Department, Faculty of Medicine, Tanta University, Egypt (M.E.); UOSA Neuroradiologia Interventistica, Fondazione Policlinico Universitario A.Gemelli IRCCS, Rome, Italy (I.V., A.P., A.M.A.); Department of Diagnostic and Interventional Neuroradiology, Centro Hospitalar Universitário do Porto, Porto, Portugal (J.P.F.); Department of Neurology, Centro Hospitalar Universitário do Porto, Porto, Portugal (R.V.); Department of Neurosurgery, Cedars-Sinai Medical Center, Los Angeles, Calif (M.Q.C., N.R.G.); Department of Vascular and Interventional Neuroradiology, Universitätsklinikum Heidelberg, Heidelberg, Germany (M.A.M., J.J., C.W.); Department of Neuroradiology, Gui de Chauliac Hospital, Montpellier University Medical Center, Montpellier, France (V.C., R.A.R.); Department of Neurology, Gui de Chauliac Hospital, Montpellier University Medical Center, Montpellier, France (A.t.S.); Department of Radiology, Division of Neuroradiology, Johns Hopkins Medical Center, Baltimore, Md (V.Y.); Department of Neurology, University of Cincinnati Medical Center, Cincinnati, Ohio (P.H., L.M.C., Y.A.); Department of Interventional Neuroradiology, Nancy University Hospital, Nancy, France (B.G.); INSERM U1254, IADI, Université de Lorraine, Nancy, France (B.G.); Department of Radiology, Section of Interventional Neuroradiology, University Medical Center Münster, Münster, Germany (C.P.S.); Departments of Neurology & Neurosurgery, Christian Doppler Clinic, Paracelsus Medical University, Salzburg, Austria (C.H., M.K.O., C.J.G.); Department of Neurology, Sin-Lau Hospital, Tainan, Taiwan (C.Y.H.); UCLA Stroke Center and Department of Neurology Department, University of California Los Angeles, Los Angeles, Calif (D.S.L.); Department of Medicine, Division of Neurology, The Ottawa Hospital, Ottawa Hospital Research Institute and University of Ottawa, Ottawa, Ontario, Canada (I.T., R.F.); Department of Diagnostic and Interventional Neuroradiology, Erasme University Hospital, Brussels, Belgium (B.L.).

Article Synopsis
  • * Data from 670 patients revealed that while IVT may improve some outcomes, such as higher chances of achieving a modified Rankin Scale score of 0-2 in univariable analysis, this benefit was not consistently observed in more rigorous multivariable analyses.
  • * Overall, the findings suggest that adjunctive IVT may not significantly enhance clinical outcomes or safety compared to MT alone for this patient population.
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