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:
-
Isaac, Rafael H.
Gonzalez-Devia, Deyanira
Mendivil, Carlos O.
Chapman, Edgardo
- Tipo de recurso:
- Article of journal
- Fecha de publicación:
- 2023
- Institución:
- Universidad El Bosque
- Repositorio:
- Repositorio U. El Bosque
- Idioma:
- eng
- OAI Identifier:
- oai:repositorio.unbosque.edu.co:20.500.12495/11154
- Acceso en línea:
- http://hdl.handle.net/20.500.12495/11154
https://doi.org/10.3389/falgy.2023.1201807
- Palabra clave:
- Reacciones adversas
Síndrome de quilomicronemia familiar
Pancreatitis
Triglicéridos
Urticaria
Volanesorsen
Adverse reactions
Familial chylomicronemia syndrome
Pancreatitis
Triglycerides
Urticaria
Volanesorsen
- Rights
- openAccess
- License
- Atribución 4.0 Internacional
| Summary: | 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. |
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