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Case report: desensitization of hypersensitivity against the antisense oligonucleotide volanesorsen
Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive metabolic disorder that causes extremely elevated plasma triglyceride levels, with limited therapeutic options. Volanesorsen is an antisense oligonucleotide approved for its treatment. A 24-year-old woman with genetically diagnose...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10285465/ https://www.ncbi.nlm.nih.gov/pubmed/37361109 http://dx.doi.org/10.3389/falgy.2023.1201807 |
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author | Isaac, Rafael H. Gonzalez-Devia, Deyanira Mendivil, Carlos O. Chapman, Edgardo |
author_facet | Isaac, Rafael H. Gonzalez-Devia, Deyanira Mendivil, Carlos O. Chapman, Edgardo |
author_sort | Isaac, Rafael H. |
collection | PubMed |
description | Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive metabolic disorder that causes extremely elevated plasma triglyceride levels, with limited therapeutic options. Volanesorsen is an antisense oligonucleotide approved for its treatment. A 24-year-old woman with genetically diagnosed FCS secondary to a pathogenic variant in APOA5 and a history of recurrent hypertriglyceridemia-induced pancreatitis episodes was being treated with volanesorsen, 285 mg every 2 weeks. Treatment with volanesorsen achieved normalization of triglycerides to <200 mg/dl. However, after the fifth dose of the medication, the patient developed urticaria and volanesorsen was discontinued. In the absence of alternative pharmacological treatments, the patient received a novel desensitization protocol for volanesorsen that allowed continuation of therapy, without evidence of hypersensitivity reactions after subsequent administrations. FCS requires aggressive multimodal therapy and close follow-up. Volanesorsen has shown great efficacy, but a significant rate of discontinuation due to side effects has been observed. Here, the patient presented an immediate hypersensitivity reaction to volanesorsen, but the provision of a desensitization protocol was effective, facilitating continued treatment and impacting the survival and quality of life of the patient. |
format | Online Article Text |
id | pubmed-10285465 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-102854652023-06-23 Case report: desensitization of hypersensitivity against the antisense oligonucleotide volanesorsen Isaac, Rafael H. Gonzalez-Devia, Deyanira Mendivil, Carlos O. Chapman, Edgardo Front Allergy Allergy Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive metabolic disorder that causes extremely elevated plasma triglyceride levels, with limited therapeutic options. Volanesorsen is an antisense oligonucleotide approved for its treatment. A 24-year-old woman with genetically diagnosed FCS secondary to a pathogenic variant in APOA5 and a history of recurrent hypertriglyceridemia-induced pancreatitis episodes was being treated with volanesorsen, 285 mg every 2 weeks. Treatment with volanesorsen achieved normalization of triglycerides to <200 mg/dl. However, after the fifth dose of the medication, the patient developed urticaria and volanesorsen was discontinued. In the absence of alternative pharmacological treatments, the patient received a novel desensitization protocol for volanesorsen that allowed continuation of therapy, without evidence of hypersensitivity reactions after subsequent administrations. FCS requires aggressive multimodal therapy and close follow-up. Volanesorsen has shown great efficacy, but a significant rate of discontinuation due to side effects has been observed. Here, the patient presented an immediate hypersensitivity reaction to volanesorsen, but the provision of a desensitization protocol was effective, facilitating continued treatment and impacting the survival and quality of life of the patient. Frontiers Media S.A. 2023-06-08 /pmc/articles/PMC10285465/ /pubmed/37361109 http://dx.doi.org/10.3389/falgy.2023.1201807 Text en © 2023 Isaac, Gonzalez-Devia, Mendivil and Chapman. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY) (https://creativecommons.org/licenses/by/4.0/) . The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Allergy Isaac, Rafael H. Gonzalez-Devia, Deyanira Mendivil, Carlos O. Chapman, Edgardo Case report: desensitization of hypersensitivity against the antisense oligonucleotide volanesorsen |
title | Case report: desensitization of hypersensitivity against the antisense oligonucleotide volanesorsen |
title_full | Case report: desensitization of hypersensitivity against the antisense oligonucleotide volanesorsen |
title_fullStr | Case report: desensitization of hypersensitivity against the antisense oligonucleotide volanesorsen |
title_full_unstemmed | Case report: desensitization of hypersensitivity against the antisense oligonucleotide volanesorsen |
title_short | Case report: desensitization of hypersensitivity against the antisense oligonucleotide volanesorsen |
title_sort | case report: desensitization of hypersensitivity against the antisense oligonucleotide volanesorsen |
topic | Allergy |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10285465/ https://www.ncbi.nlm.nih.gov/pubmed/37361109 http://dx.doi.org/10.3389/falgy.2023.1201807 |
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