| Title: |
Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome |
| Authors: |
Witztum JL; Gaudet D; Freedman SD; Alexander VJ; Digenio A; Williams KR; Yang Q; Hughes SG; Geary RS; Arca M; Stroes ESG; Bergeron J; Soran H; Civeira F; Hemphill L; Tsimikas S; Blom DJ; O'Dea L; Bruckert E |
| Contributors: |
Witztum, Jl; Gaudet, D; Freedman, Sd; Alexander, Vj; Digenio, A; Williams, Kr; Yang, Q; Hughes, Sg; Geary, R; Arca, M; Stroes, Esg; Bergeron, J; Soran, H; Civeira, F; Hemphill, L; Tsimikas, S; Blom, Dj; O'Dea, L; Bruckert, E |
| Publication Year: |
2019 |
| Collection: |
Sapienza Università di Roma: CINECA IRIS |
| Subject Terms: |
familial chylomicronemia; volanesorsen; triglycerides; therapy; apoCIII |
| Description: |
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 |
| Document Type: |
article in journal/newspaper |
| Language: |
English |
| Relation: |
info:eu-repo/semantics/altIdentifier/wos/WOS:000479273000008; volume:381; firstpage:531; lastpage:542; numberofpages:12; journal:NEW ENGLAND JOURNAL OF MEDICINE; http://hdl.handle.net/11573/1344420 |
| DOI: |
10.1056/NEJMoa1715944 |
| Availability: |
http://hdl.handle.net/11573/1344420; https://doi.org/10.1056/NEJMoa1715944 |
| Rights: |
info:eu-repo/semantics/closedAccess |
| Accession Number: |
edsbas.BC54A47 |
| Database: |
BASE |