title: Successful management of difficult infusion-associated reactions in a young patient with mucopolysaccharidosis type VI receiving recombinant human arylsulfatase B (galsulfase [Naglazyme]).
abstract: Our patient with mucopolysaccharidosis type VI received enzyme replacement therapy with recombinant human arylsulfatase B (galsulfase [Naglazyme, BioMarin Pharmaceutical Inc, Novato, CA]) shortly after approval by the US Food and Drug Administration. After 1 month of weekly infusions, the patient developed significant infusion-associated reactions and could not tolerate therapy at the recommended infusion rate. We were able to continue treatment successfully by the addition of steroids to the premedication regimen and by significantly reducing the rate of drug infusion. Over the next several months, the patient's infusion rate was slowly increased and the premedications were weaned. We demonstrate that by significantly reducing the rate of infusions and adjusting the premedication regimen, galsulfase infusions can continue with no additional observance of infusion-associated reactions.
authors.affiliation: Division of Genetics, Birth Defects, and Metabolism, Children's Memorial Hospital, 2300 Children's Plaza, Box 59, Chicago, IL 60614, USA. kkim@childrensmemorial.org
authors.forename: Katherine H
authors.initials: KH
authors.lastname: Kim
authors.forename: Celeste
authors.initials: C
authors.lastname: Decker
authors.forename: Barbara K
authors.initials: BK
authors.lastname: Burton
mesh_terms: D002675:Child, Preschool; D004155:Diphenhydramine / Q000008:administration & dosage*; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D004342:Drug Hypersensitivity / Q000188:drug therapy* / Q000209:etiology* / Q000503:physiopathology; D005500:Follow-Up Studies; D006801:Humans; D007262:Infusions, Intravenous; D008297:Male; D009087:Mucopolysaccharidosis VI / Q000188:drug therapy* / Q000235:genetics; D020051:N-Acetylgalactosamine-4-Sulfatase / Q000009:adverse effects* / Q000627:therapeutic use; D011994:Recombinant Proteins / Q000009:adverse effects / Q000627:therapeutic use; D018570:Risk Assessment; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D004155:Diphenhydramine; D020051:N-Acetylgalactosamine-4-Sulfatase; C508864:galsulfase