Safety of enzyme replacement therapy

Excerpt

One of the main aims of FOS – the Fabry Outcome Survey – is to monitor the long-term safety of enzyme replacement therapy with agalsidase alfa in patients with Fabry disease. Detailed reporting within FOS has revealed few serious adverse events. Only two of these were classified as possibly related to treatment, out of a total of 401 treated patients, corresponding to 940 patient-years. Most adverse events were mild infusion reactions, occurring in approximately 13% of patients (associated with about 1% of the estimated total number of infusions). Infusion-related reactions occurred much less frequently in female than in male patients. In most of the affected patients the infusion reactions occurred soon after the initiation of treatment and normally disappeared after the first few infusions. No IgE antibodies have been detected. The data in FOS confirm that agalsidase alfa has a good safety profile and is well tolerated in adults and children when used in a wide range of patients and in daily clinical practice, including home therapy.