title: A revised home treatment algorithm for Fabry disease: influence of antibody formation.
abstract: BACKGROUND
  Enzyme replacement therapy for Fabry disease, consisting of biweekly infusions, interferes daily life. Home treatment proved beneficial. We evaluated a previously reported home treatment algorithm aiming to shorten the period of in-hospital infusions, while ascertaining patient safety.
  
  
  METHODS
  Retrospective analysis on clinical records of treated Fabry patients. Potentially predictive factors for infusion associated reactions (IARs) were studied: agalsidase antibodies, agalsidase product and dose, FOS-SSI scores, and GLA activity and mutation. A questionnaire evaluated patient satisfaction and compliance.
  
  
  RESULTS
  Seventy-nine patients were included (41 males, 46% agalsidase antibody positive (AB+)). 85% received home treatment. Home treatment complications were erroneous fast infusion rates (n=4) causing IARs and, rarely, venous access problems. The single SAE was unrelated to home treatment. IgG antibody status was significantly associated with IARs (89% vs. 26% p-value<0.01). Negative antibody status did not preclude IARs. Except for three AB+ patients, all first IARs occurred within 13 infusions. IARs occurred more frequently in patients using agalsidase beta 1.0 mg/kg/eow than agalsidase alpha or beta 0.2 mg/kg/eow, but the time to first IAR did not differ between groups. Four AB+ males experienced IARs after a dose increase. Compliance between home and in-hospital treatment was similar. Most patients preferred home treatment.
  
  
  CONCLUSIONS
  In this study home therapy for Fabry disease was safe and improved patient satisfaction. We propose a revised algorithm which allows safe home-treatment in all male patients after 13 instead of 26 infusions, irrespective of ERT preparation or dose. Furthermore, AB+ patients with dosage increase may experience new or increased IARs, necessitating in-hospital observations.
authors: Department of Internal Medicine, Division of Endocrinology and Metabolism, Academic Medical Centre, Amsterdam, The Netherlands. b.e.smid@amc.uva.nl
authors: B E
authors: BE
authors: Smid
authors: S L
authors: SL
authors: Hoogendijk
authors: F A
authors: FA
authors: Wijburg
authors: C E M
authors: CE
authors: Hollak
authors: G E
authors: GE
authors: Linthorst
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000465:Algorithms; D000906:Antibodies / Q000097:blood / Q000276:immunology; D000917:Antibody Formation; D002648:Child; D056947:Enzyme Replacement Therapy / Q000009:adverse effects; D000795:Fabry Disease / Q000276:immunology / Q000628:therapy*; D005260:Female; D018718:Home Infusion Therapy / Q000009:adverse effects; D006801:Humans; D007074:Immunoglobulin G / Q000097:blood / Q000276:immunology; D007527:Isoenzymes / Q000276:immunology; D008297:Male; D008875:Middle Aged; D055815:Young Adult; D000519:alpha-Galactosidase / Q000276:immunology
chemical_list: D000906:Antibodies; D007074:Immunoglobulin G; D007527:Isoenzymes; D000519:alpha-Galactosidase; C459420:agalsidase beta