title: Management of infusion-related reactions to enzyme replacement therapy in a cohort of patients with mucopolysaccharidosis disorders.
abstract: OBJECTIVE
  Enzyme replacement therapy (ERT) is currently available for the treatment of mucopolysaccharidosis (MPS) Type I, MPS II and MPS VI. Hypersensitivity reactions have been reported in some patients receiving ERT, but these can usually be easily managed.
  
  
  METHODS
  In this retrospective study, we evaluated the manifestations and management of hypersensitivity reactions in patients at a single center who were receiving ERT for either MPS I, MPS II or MPS VI between 2002 and 2008.
  
  
  RESULTS
  Hypersensitivity reactions were observed in 28 (36%) out of 77 patients, and were most common in children with severe disease. When an infusion-related reaction occurred, ERT was immediately suspended until the patient's symptoms had resolved. Antihistamines and antipyretics were administered to treat the acute symptoms of hypersensitivity reactions. In some patients, low-dose corticosteroids were administered to attenuate late-phase or biphasic reactions. There were no instances in which resuscitation was necessary. When ERT was restarted, patients were given premedication in the form of antihistamines and antipyretics, and ERT was administered at a slower rate. In most cases, this approach overcame the hypersensitivity. After gradually increasing the infusion rate, patients were generally able to resume a normal infusion schedule without premedication after a period ranging from 8 weeks to 3.5 years.
  
  
  CONCLUSIONS
  Close monitoring of patients receiving ERT is essential. Use of an adapted ERT infusion regimen with premedication resulted in improvement of signs and symptoms of hypersensitivity in most of the patients who experienced infusion-related reactions.
authors.affiliation: Villa Metabolica, Children's Hospital, University of Mainz, Mainz, Germany. miebach@kinder.klinik.uni-mainz.de
authors.forename: E
authors.initials: E
authors.lastname: Miebach
mesh_terms: D000293:Adolescent; D000328:Adult; D018926:Anti-Allergic Agents / Q000008:administration & dosage / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D002985:Clinical Protocols; D056947:Enzyme Replacement Therapy / Q000009:adverse effects* / Q000379:methods*; D005260:Female; D006801:Humans; D006967:Hypersensitivity / Q000188:drug therapy* / Q000517:prevention & control*; D007066:Iduronate Sulfatase / Q000627:therapeutic use; D007262:Infusions, Intravenous / Q000009:adverse effects*; D008297:Male; D008875:Middle Aged; D009083:Mucopolysaccharidoses / Q000188:drug therapy*; D008059:Mucopolysaccharidosis I / Q000188:drug therapy; D016532:Mucopolysaccharidosis II / Q000188:drug therapy; D009087:Mucopolysaccharidosis VI / Q000188:drug therapy; D020051:N-Acetylgalactosamine-4-Sulfatase / Q000627:therapeutic use; D011292:Premedication; D011994:Recombinant Proteins / Q000627:therapeutic use
chemical_list: D018926:Anti-Allergic Agents; D011994:Recombinant Proteins; D020051:N-Acetylgalactosamine-4-Sulfatase; C508864:galsulfase; D007066:Iduronate Sulfatase; C517982:idursulfase