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Volanesorsen: A New Era in the Treatment of Severe Hypertriglyceridemia
Introduction: Familial chylomicronemia syndrome (FCS) is a rare inherited disease, mainly due to lipoprotein lipase (LPL) gene mutations, leading to lipid abnormalities. Volanesorsen, a second-generation 2′-O-methoxyethyl (2′-MOE) chimeric antisense therapeutic oligonucleotide, can decrease plasma a...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8880470/ https://www.ncbi.nlm.nih.gov/pubmed/35207255 http://dx.doi.org/10.3390/jcm11040982 |
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author | Kolovou, Genovefa Kolovou, Vana Katsiki, Niki |
author_facet | Kolovou, Genovefa Kolovou, Vana Katsiki, Niki |
author_sort | Kolovou, Genovefa |
collection | PubMed |
description | Introduction: Familial chylomicronemia syndrome (FCS) is a rare inherited disease, mainly due to lipoprotein lipase (LPL) gene mutations, leading to lipid abnormalities. Volanesorsen, a second-generation 2′-O-methoxyethyl (2′-MOE) chimeric antisense therapeutic oligonucleotide, can decrease plasma apolipoprotein C3 and triglycerides (TG) levels through LPL-independent pathways. The European Medicines Agency has approved volanesorsen as an adjunct to diet in adult FCS patients with an inadequate response to TG-lowering therapy. Areas covered: Available clinical data on volanesorsen efficacy and safety are presented. Furthermore, we discuss the yearly treatment with volanesorsen of a 21-year-old female FCS patient with LPL mutation. Volanesorsen was well-tolerated and decreased patient’s TG levels (from >5000 mg/dL (56 mmol/L) to 350–500 mg/dL (4–5.6 mmol/L)) at 12 months. Lipoprotein apheresis (LA) was stopped and there were no episodes of pancreatitis or abdominal pain. Expert opinion: Severe hypertriglyceridemia can potentially be fatal. Until recently, there was no specific treatment for FCS, apart from hypotriglyceridemic diet, fibrates, omega-3 fatty acids, and LA sessions. Therefore, volanesorsen represents a promising therapeutic solution for these patients. The main side effect of volanesorsen therapy is thrombocytopenia, which should be monitored and treated accordingly. Increasing evidence will further elucidate the clinical implications of volanesorsen use in daily practice. |
format | Online Article Text |
id | pubmed-8880470 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-88804702022-02-26 Volanesorsen: A New Era in the Treatment of Severe Hypertriglyceridemia Kolovou, Genovefa Kolovou, Vana Katsiki, Niki J Clin Med Communication Introduction: Familial chylomicronemia syndrome (FCS) is a rare inherited disease, mainly due to lipoprotein lipase (LPL) gene mutations, leading to lipid abnormalities. Volanesorsen, a second-generation 2′-O-methoxyethyl (2′-MOE) chimeric antisense therapeutic oligonucleotide, can decrease plasma apolipoprotein C3 and triglycerides (TG) levels through LPL-independent pathways. The European Medicines Agency has approved volanesorsen as an adjunct to diet in adult FCS patients with an inadequate response to TG-lowering therapy. Areas covered: Available clinical data on volanesorsen efficacy and safety are presented. Furthermore, we discuss the yearly treatment with volanesorsen of a 21-year-old female FCS patient with LPL mutation. Volanesorsen was well-tolerated and decreased patient’s TG levels (from >5000 mg/dL (56 mmol/L) to 350–500 mg/dL (4–5.6 mmol/L)) at 12 months. Lipoprotein apheresis (LA) was stopped and there were no episodes of pancreatitis or abdominal pain. Expert opinion: Severe hypertriglyceridemia can potentially be fatal. Until recently, there was no specific treatment for FCS, apart from hypotriglyceridemic diet, fibrates, omega-3 fatty acids, and LA sessions. Therefore, volanesorsen represents a promising therapeutic solution for these patients. The main side effect of volanesorsen therapy is thrombocytopenia, which should be monitored and treated accordingly. Increasing evidence will further elucidate the clinical implications of volanesorsen use in daily practice. MDPI 2022-02-13 /pmc/articles/PMC8880470/ /pubmed/35207255 http://dx.doi.org/10.3390/jcm11040982 Text en © 2022 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Communication Kolovou, Genovefa Kolovou, Vana Katsiki, Niki Volanesorsen: A New Era in the Treatment of Severe Hypertriglyceridemia |
title | Volanesorsen: A New Era in the Treatment of Severe Hypertriglyceridemia |
title_full | Volanesorsen: A New Era in the Treatment of Severe Hypertriglyceridemia |
title_fullStr | Volanesorsen: A New Era in the Treatment of Severe Hypertriglyceridemia |
title_full_unstemmed | Volanesorsen: A New Era in the Treatment of Severe Hypertriglyceridemia |
title_short | Volanesorsen: A New Era in the Treatment of Severe Hypertriglyceridemia |
title_sort | volanesorsen: a new era in the treatment of severe hypertriglyceridemia |
topic | Communication |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8880470/ https://www.ncbi.nlm.nih.gov/pubmed/35207255 http://dx.doi.org/10.3390/jcm11040982 |
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