Case report: desensitization of hypersensitivity against the antisense oligonucleotide volanesorsen

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2023

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Frontiers in Allergy, 2673-6101, 4, 2023, 1-5

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Frontiers in Allergy

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Abstract

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.

Palabras clave

Reacciones adversas, Síndrome de quilomicronemia familiar, Pancreatitis, Triglicéridos, Urticaria, Volanesorsen

Keywords

Adverse reactions, Familial chylomicronemia syndrome, Pancreatitis, Triglycerides, Urticaria, Volanesorsen

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