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Evolocumab in metastatic castration-resistant prostate cancer: study protocol for a single-arm, phase II trial, and initial experience with use of a validated lipid biomarker to direct therapy.
Ther Adv Med Oncol
December 2024
Medical Oncology, Chris O'Brien Lifehouse, 119-143 Missenden Rd, Camperdown, NSW 2050, Australia.
Background: Despite advances in the treatment of metastatic castration-resistant prostate cancer (mCRPC), primary and secondary resistance to current therapies remains. Elevated circulating sphingolipids are associated with poor outcomes in patients with mCRPC, including therapeutic resistance and shorter overall survival. PCPro is a clinically accessible, regulatory compliant plasma lipid biomarker of poor prognosis in mCRPC, which incorporates prognostic sphingolipids.
View Article and Find Full Text PDFProprotein Convertase Subtilisin Kexin 9 Inhibitor in Severe Sepsis and Septic Shock Patients in a Phase II Prospective Cohort Study-Preliminary Results.
Infect Dis Rep
October 2024
Intensive Care Department, E. Wolfson Medical Center, Holon 5822012, Israel.
Sepsis is a life-threatening organ dysfunction syndrome caused by a dysregulated host response to infection that has a high mortality rate. Proprotein convertase subtilisin kexin 9 (PCSK9) is a serine protease secreted by the liver. Its binding to the low-density lipoprotein (LDL) receptor enhances its degradation, causing an increase in LDL levels in the blood.
View Article and Find Full Text PDFPerformance of LDL-C only compared to the Dutch Lipid Clinic Network Score for screening of familial hypercholesterolaemia: the Austrian experience and literature review.
Eur J Prev Cardiol
November 2024
Division of Endocrinology and Metabolism, Medical University of Vienna, Währinger Gürtel 18-20, 1090 Vienna, Austria.
Aims: Familial hypercholesterolaemia (FH) is a severely underdiagnosed, inherited disease, causing dyslipidaemia and premature atherosclerotic cardiovascular disease. In order to facilitate screening in a broad clinical spectrum, we aimed to analyse the current yield of routine genetic diagnostics for FH and to evaluate the performance of the Dutch Lipid Clinic Network Score (DLCNS) compared to a single value, the off-treatment LDL-cholesterol exceeding 190 mg/dL.
Methods And Results: We investigated all patients that underwent molecular genotyping routinely performed for FH over a 4-year period in two Austrian specialist lipid clinics.
Challenges in the management of familial hypercholesterolemia: a case report.
Front Cardiovasc Med
September 2024
First Department of Cardiology, Medical University of Warsaw, Warsaw, Poland.
Article Synopsis
- - Familial hypercholesterolemia (FH) is a genetic disorder causing high LDL cholesterol levels, which increases the risk of heart disease; the heterozygous form affects about 1 in 500 people worldwide.
- - A case study reported a woman with HeFH whose LDL levels did not adequately decrease with statin and ezetimibe therapies; genetic testing revealed a mutation in the LDL receptor gene.
- - Inclisiran therapy, which uses a specialized RNA to lower LDL cholesterol, significantly reduced her cholesterol levels after three months and may be a viable option for patients who cannot tolerate traditional treatments.
Real-world experience of inclisiran prescription at the University of Pennsylvania Health Systems.
J Clin Lipidol
July 2024
Division of Translational Medicine & Human Genetics, University of Pennsylvania, Philadelphia, PA. Electronic address:
Inclisiran is a novel small interfering RNA targeting proprotein convertase subtilisin/kexin type 9 (PCSK9) that was approved by the US FDA in December 2021. After two doses three months apart, it is administered biannually as a subcutaneous injection and has been shown to lower LDL-C by ∼50 % in clinical trials. Here, we present real-world data on the prescription and administration of inclisiran at the University of Pennsylvania Health Systems.
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