Details

Autor(en) / Beteiligte

• Smid, B.E.
• Hoogendijk, S.L.
• Wijburg, F.A.
• Hollak, C.E.M.
• Linthorst, G.E.

Titel

A revised home treatment algorithm for Fabry disease: Influence of antibody formation

Ist Teil von

• Molecular genetics and metabolism, 2013-02, Vol.108 (2), p.132-137

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2013

Quelle

MEDLINE

Beschreibungen/Notizen

• 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. 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. 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 1.0mg/kg/eow than agalsidase alpha or beta 0.2mg/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. 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. ► Home therapy with enzyme replacement therapy for Fabry disease is safe. ► Home therapy can safely be initiated in all male patients after 13 infusions. ► Agalsidase dosage increase in AB+ patients necessitates in-hospital observations.