Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome
| Autor: | Louis O'Dea, Sotirios Tsimikas, Qingqing Yang, Erik S.G. Stroes, Daniel Gaudet, Fernando Civeira, Handrean Soran, Dirk J. Blom, Linda C. Hemphill, Steven D. Freedman, Jean Bergeron, Joseph L. Witztum, Steven Hughes, Richard S. Geary, Eric Bruckert, Andres Digenio, Veronica J. Alexander, Marcello Arca, Karren R Williams |
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| Přispěvatelé: | University of California [San Diego] (UC San Diego), University of California, Université de Montréal (UdeM), Beth Israel Deaconess Medical Center [Boston] (BIDMC), Harvard Medical School [Boston] (HMS), Università degli Studi di Roma 'La Sapienza' = Sapienza University [Rome], Université Laval [Québec] (ULaval), Manchester University NHS Foundation Trust (MFT), University of Zaragoza - Universidad de Zaragoza [Zaragoza], Massachusetts General Hospital [Boston], University of Cape Town, CHU Pitié-Salpêtrière [AP-HP], Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Vascular Medicine, ACS - Atherosclerosis & ischemic syndromes |
| Rok vydání: | 2019 |
| Předmět: |
Adult
Male medicine.medical_specialty [SDV]Life Sciences [q-bio] Injections Subcutaneous/adverse effects 030204 cardiovascular system & hematology Triglycerides blood Hyperlipoproteinemia Type I/blood familial chylomicronemia volanesorsen triglycerides therapy apoCIII familial chylomicronemia Injections Oligonucleotides/administration & dosage Young Adult 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine Double-Blind Method apoCIII Internal medicine Humans Medicine volanesorsen 030212 general & internal medicine triglycerides Aged RNA Messenger/antagonists & inhibitors therapy Analysis of Variance Triglyceride Platelet Count Thrombocytopenia/chemically induced business.industry Genetic disorder General Medicine Middle Aged Familial Chylomicronemia medicine.disease 3. Good health Triglycerides/blood Endocrinology Multicenter study chemistry RNA Female Subcutaneous/adverse effects Apolipoprotein C-III/antagonists & inhibitors business Lipoprotein lipase activity Messenger/antagonists & inhibitors |
| Zdroj: | Zaguán: Repositorio Digital de la Universidad de Zaragoza Universidad de Zaragoza New England Journal of Medicine New England Journal of Medicine, Massachusetts Medical Society, 2019, 381 (6), pp.531-542. ⟨10.1056/NEJMoa1715944⟩ Zaguán. Repositorio Digital de la Universidad de Zaragoza instname New England journal of medicine, 381(6), 531-542. Massachussetts Medical Society |
| ISSN: | 0028-4793 1533-4406 |
| DOI: | 10.1056/NEJMoa1715944⟩ |
| Popis: | Background Familial chylomicronemia syndrome is a rare genetic disorder that is caused by loss of lipoprotein lipase activity and characterized by chylomicronemia and recurrent episodes of pancreatitis. There are no effective therapies. In an open-label study of three patients with this syndrome, antisense-mediated inhibition of hepatic APOC3 mRNA with volanesorsen led to decreased plasma apolipoprotein C-III and triglyceride levels. Methods We conducted a phase 3, double-blind, randomized 52-week trial to evaluate the safety and effectiveness of volanesorsen in 66 patients with familial chylomicronemia syndrome. Patients were randomly assigned, in a 1:1 ratio, to receive volanesorsen or placebo. The primary end point was the percentage change in fasting triglyceride levels from baseline to 3 months. Results Patients receiving volanesorsen had a decrease in mean plasma apolipoprotein C-III levels from baseline of 25.7 mg per deciliter, corresponding to an 84% decrease at 3 months, whereas patients receiving placebo had an increase in mean plasma apolipoprotein C-III levels from baseline of 1.9 mg per deciliter, corresponding to a 6.1% increase (P |
| Databáze: | OpenAIRE |
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