Abbreviation of Desensitization Protocol for Pediatric Patients with Lysosomal Storage Diseases Receiving Enzyme Replacement Therapy
Lysosomal storage diseases (LSDs) constitute a group of metabolic disorders characterized by the accumulation of substrates within lysosomes. For their treatment, disease-specific enzyme replacement therapy (ERT) is employed. In cases of hypersensitivity reactions that may occur during these treatme...
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Published in | International archives of allergy and immunology p. 1 |
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Main Authors | , , , , , |
Format | Journal Article |
Language | English |
Published |
Switzerland
17.07.2024
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Abstract | Lysosomal storage diseases (LSDs) constitute a group of metabolic disorders characterized by the accumulation of substrates within lysosomes. For their treatment, disease-specific enzyme replacement therapy (ERT) is employed. In cases of hypersensitivity reactions that may occur during these treatments, desensitization of enzyme therapy becomes necessary. Repeated desensitization procedures may result some degree of tolerance. This study presents cases of patients received abbreviated desensitization protocols following repeated desensitization procedures.
During the period between September 2019 and January 2024, pediatric patients who experienced anaphylactic reactions to ERT and whose desensitization protocols were abbreviated after receiving uneventful treatment with desensitization for at least a year were included in the study.
Six patients, four with Pompe disease, one with mucopolysaccharidosis type 2, and one with mucopolysaccharidosis type 4, had been receiving uninterrupted ERT by desensitization for at least 1 year. The mean age of the patients was 117.6 months (median: 104.5, IQR: 85.2-144). All patients experienced anaphylaxis as the initial reaction. Skin and intradermal tests were repeated on patients prior to protocol abbreviation. Premedication previously given to all patients was discontinued, and desensitization protocols were subsequently shortened by increasing the infusion rate and/or reducing the number of steps.
The study investigated patients whose desensitization protocols were abbreviated. It demonstrated that some level of tolerance could be attained through repeated applications. This approach aims to identify concise, safe, and efficient protocols, thereby reducing hospitalizations, nosocomial infections, and treatment expenses. |
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AbstractList | Lysosomal storage diseases (LSDs) constitute a group of metabolic disorders characterized by the accumulation of substrates within lysosomes. For their treatment, disease-specific enzyme replacement therapy (ERT) is employed. In cases of hypersensitivity reactions that may occur during these treatments, desensitization of enzyme therapy becomes necessary. Repeated desensitization procedures may result some degree of tolerance. This study presents cases of patients received abbreviated desensitization protocols following repeated desensitization procedures.
During the period between September 2019 and January 2024, pediatric patients who experienced anaphylactic reactions to ERT and whose desensitization protocols were abbreviated after receiving uneventful treatment with desensitization for at least a year were included in the study.
Six patients, four with Pompe disease, one with mucopolysaccharidosis type 2, and one with mucopolysaccharidosis type 4, had been receiving uninterrupted ERT by desensitization for at least 1 year. The mean age of the patients was 117.6 months (median: 104.5, IQR: 85.2-144). All patients experienced anaphylaxis as the initial reaction. Skin and intradermal tests were repeated on patients prior to protocol abbreviation. Premedication previously given to all patients was discontinued, and desensitization protocols were subsequently shortened by increasing the infusion rate and/or reducing the number of steps.
The study investigated patients whose desensitization protocols were abbreviated. It demonstrated that some level of tolerance could be attained through repeated applications. This approach aims to identify concise, safe, and efficient protocols, thereby reducing hospitalizations, nosocomial infections, and treatment expenses. |
Author | Kasapkara, Çiğdem Seher Yilmaz Topal, Özge Küçükçongar Yavaş, Aynur Şengül Emeksiz, Zeynep Yörüsün, Gökhan Dibek Mısırlıoğlu, Emine |
Author_xml | – sequence: 1 givenname: Gökhan surname: Yörüsün fullname: Yörüsün, Gökhan organization: Department of Pediatric Allergy and Immunology, Ankara City Hospital, Ankara, Turkey – sequence: 2 givenname: Özge surname: Yilmaz Topal fullname: Yilmaz Topal, Özge organization: Department of Pediatric Allergy and Immunology, Ankara City Hospital, Ankara, Turkey – sequence: 3 givenname: Zeynep surname: Şengül Emeksiz fullname: Şengül Emeksiz, Zeynep organization: Department of Pediatric Allergy and Immunology, Ankara City Hospital, Ankara, Turkey – sequence: 4 givenname: Aynur surname: Küçükçongar Yavaş fullname: Küçükçongar Yavaş, Aynur organization: Department of Pediatric Metabolic Diseases Clinic, Ankara City Hospital, Ankara, Turkey – sequence: 5 givenname: Çiğdem Seher surname: Kasapkara fullname: Kasapkara, Çiğdem Seher organization: Department of Pediatric Metabolic Diseases Clinic, Ankara City Hospital, Ankara, Turkey – sequence: 6 givenname: Emine surname: Dibek Mısırlıoğlu fullname: Dibek Mısırlıoğlu, Emine organization: Department of Pediatric Allergy and Immunology, Health Sciences University, Ankara City Hospital, Ankara, Turkey |
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Keywords | Abbreviation Lysosomal storage diseases Desensitization protocols Hypersensitivity reactions Enzyme replacement therapy |
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