title: IgE-Mediated Hypersensitivity and Desensitisation with Recombinant Enzymes in Pompe Disease and Type I and Type VI Mucopolysaccharidosis.
abstract: Enzyme replacement therapy (ERT) is important for the treatment of lysosomal storage disorders. Hypersensitivity reactions with ERT have been reported, and in these cases, desensitisation with the enzyme is necessary. Here we report the cases of 3 patients with lysosomal storage disorders, including Pompe disease and mucopolysaccharidosis type I and VI, who had IgE-mediated hypersensitivity reactions and positive skin tests. Successful desensitisation protocols with the culprit enzyme solution were used for these patients. All 3 patients were able to safely receive ERT with the desensitisation protocol.
authors.affiliation: Department of Pediatric Allergy and Immunology, Ankara Children's Hematology and Oncology Hospital, Ankara, Turkey.
authors.forename: Murat
authors.initials: M
authors.lastname: Capanoglu
authors.forename: Emine
authors.initials: E
authors.lastname: Dibek Misirlioglu
authors.forename: Dilek
authors.initials: D
authors.lastname: Azkur
authors.forename: Emine
authors.initials: E
authors.lastname: Vezir
authors.forename: Hakan
authors.initials: H
authors.lastname: Guvenir
authors.forename: Mehmet
authors.initials: M
authors.lastname: Gunduz
authors.forename: Muge
authors.initials: M
authors.lastname: Toyran
authors.forename: Ersoy
authors.initials: E
authors.lastname: Civelek
authors.forename: Can Naci
authors.initials: CN
authors.lastname: Kocabas
mesh_terms: D000485:Allergens / Q000276:immunology; D002675:Child, Preschool; D003888:Desensitization, Immunologic; D056947:Enzyme Replacement Therapy / Q000009:adverse effects*; D004798:Enzymes / Q000008:administration & dosage / Q000009:adverse effects*; D005260:Female; D006009:Glycogen Storage Disease Type II / Q000150:complications* / Q000175:diagnosis / Q000628:therapy; D006801:Humans; D006969:Hypersensitivity, Immediate / Q000150:complications* / Q000175:diagnosis / Q000628:therapy*; D007223:Infant; D008297:Male; D008059:Mucopolysaccharidosis I / Q000150:complications* / Q000175:diagnosis / Q000628:therapy; D009087:Mucopolysaccharidosis VI / Q000150:complications* / Q000175:diagnosis / Q000628:therapy; D020051:N-Acetylgalactosamine-4-Sulfatase / Q000008:administration & dosage / Q000276:immunology; D011994:Recombinant Proteins / Q000009:adverse effects; D000520:alpha-Glucosidases / Q000008:administration & dosage / Q000276:immunology
chemical_list: D000485:Allergens; D004798:Enzymes; D011994:Recombinant Proteins; D020051:N-Acetylgalactosamine-4-Sulfatase; C509951:GAA protein, human; D000520:alpha-Glucosidases