Successful reinstitution of agalsidase beta therapy in Fabry disease patients with previous IgE-antibody or skin-test reactivity to the recombinant enzyme

Genet Med. 2008 May;10(5):353-8. doi: 10.1097/GIM.0b013e318170f868.


Purpose: To determine if enzyme replacement therapy, involving intravenous infusions of recombinant human alpha-galactosidase A (agalsidase beta; Fabrazyme), could be safely continued in patients with Fabry disease who had been withdrawn from a previous clinical trial as a precautionary, protocol-specified measure due to detection of serum IgE antibodies or skin-test reactivity to agalsidase beta.

Methods: The rechallenge infusion protocol specified strict patient monitoring conditions and graded dosing and infusion-rate schemes that were adjusted according to each patient's tolerance to the infusion. Six males (age: 26-66 years) were enrolled.

Results: During rechallenge, five patients received between 4 and 27 infusions; one patient voluntarily withdrew after one infusion because of recurrence of infusion-associated reactions. No anaphylactic reactions occurred. All adverse events, including four serious adverse events, were mild or moderate in intensity. Most treatment-related adverse events occurred during infusions (most commonly urticaria, vomiting, nausea, chills, pruritus, hypertension) and were resolved by infusion rate reductions and/or medication. After participation in the study, all patients, including the one who withdrew after one infusion, transitioned to commercial drug.

Conclusions: Agalsidase beta therapy can be successfully reinstated in patients with Fabry disease who have developed IgE antibodies or skin test reactivity to the recombinant enzyme.

Publication types

  • Clinical Trial
  • Multicenter Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Fabry Disease / drug therapy*
  • Humans
  • Immunoglobulin E / blood
  • Infusions, Intravenous
  • Isoenzymes / adverse effects*
  • Isoenzymes / immunology
  • Isoenzymes / therapeutic use
  • Male
  • Middle Aged
  • Patient Selection
  • Recombinant Proteins / adverse effects
  • Recombinant Proteins / immunology
  • Recombinant Proteins / therapeutic use
  • Skin Tests
  • Treatment Outcome
  • alpha-Galactosidase / adverse effects*
  • alpha-Galactosidase / immunology
  • alpha-Galactosidase / therapeutic use


  • Isoenzymes
  • Recombinant Proteins
  • Immunoglobulin E
  • alpha-Galactosidase
  • agalsidase beta