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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...

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Autores principales: Kolovou, Genovefa, Kolovou, Vana, Katsiki, Niki
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
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.
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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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