Allergic reactions to enzyme replacement therapy in children with lysosomal storage diseases and their management
Author:
Arik Elif1ORCID, Keskin Özlem1ORCID, Albayrak Serpil2ORCID, Keskin Mehmet3ORCID, Cesur Mahmut1ORCID, Karaoglan Murat2ORCID, Inal Gaye1ORCID, Yildirim Ahmet2ORCID, Kucukosmanoglu Ercan1ORCID
Affiliation:
1. Faculty of Medicine, Department of Pediatric Allergy and Immunology , Gaziantep University , Gaziantep , Türkiye 2. Faculty of Medicine, Department of Pediatric Endocrinology , Gaziantep University , Gaziantep , Türkiye 3. Faculty of Medicine Department of Pediatric Endocrinology and Metabolism , Gaziantep University , Gaziantep , Türkiye
Abstract
Abstract
Objectives
Human recombinant enzyme replacement therapy, given to compensate for genetic enzyme deficiency in lysosomal storage diseases, delays the progression of the disease and improves the quality of life. However, enzyme replacement therapy may cause hypersensitivity reactions. Within the scope of this research, we aimed to elucidate the frequency and clinical features of hypersensitivity reactions against enzyme replacement therapy in children with lysosomal storage diseases and clarify the management of these reactions.
Methods
Medical records of pediatric patients with lysosomal storage disease and receiving enzyme replacement therapy were retrospectively reviewed, and patients who experienced allergic reactions were included in the study. The demographic characteristics of the patients, their diagnosis, the responsible enzyme, the time at which the reaction started and at what dose, the signs and symptoms associated with the reaction, diagnostic tests, the management of the reaction, and the protocol applied for the maintenance of enzyme replacement therapy after the reaction were recorded.
Results
Hypersensitivity reactions developed in 18 of 71 patients (25.3 %) who received enzyme replacement therapy. The most common cutaneous findings were observed. Anaphylaxis developed in 6 of 18 patients. Patients who experienced recurrent hypersensitivity reactions with premedication or a slower infusion rate, those with positive skin test results, and patients who developed anaphylaxis were given enzyme replacement therapy with desensitization.
Conclusions
HSR may develop during enzyme replacement therapy, which are vital in lysosomal storage diseases, and discontinuation of enzyme replacement therapy is a significant loss for patients with metabolic disorders. These reactions can be treated with premedication and long-term infusions, but some patients may require desensitization protocols for continued treatment.
Publisher
Walter de Gruyter GmbH
Reference22 articles.
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