Home treatment for Fabry disease: practice guidelines based on 3 years experience in The Netherlands

Recently, chronic supplementation with alpha-galactosidase A (alphaGal A) has been approved as a treatment modality for Fabry disease. The aim of the current study was to investigate the feasibility of home therapy for Fabry disease during a follow-up of >3 years and to make a proposal for practice guidelines.

Based on experience in previous clinical trials, an algorithm for home treatment eligibility was developed. The number of successful and uneventful infusions was recorded, as well as adverse and infusion-associated events. The presence and titre of recombinant human (rh)-alphaGal A antibodies were monitored every 3 months.

Thirty of the 36 patients eligible for home treatment received a total of 1418 infusions at home (median 44 infusions, range 1-108), between March 2001 and July 2005. Mean age was 44.7 years (17-71). Seventeen patients receiving home treatment (57%) were male. The majority of patients (27 out of 30, 90%) undergoing home treatment received 0.2 mg/kg agalsidase alpha or beta. Six male patients developed an infusion-associated event, of which three developed these at home. All patients with an infusion-associated event were anti-rh-alphaGal A IgG positive at 3 months, but three patients with rh-alphaGal A antibodies did not develop side effects. Antibody titres between these patients did not differ. None of the events was life-threatening or necessitated urgent admission.

Home treatment with rh-alphaGal A for Fabry disease with 0.2 mg/kg for males and both 1.0 and 2.0 mg/kg for females is feasible and safe, and reduces both the burden related to chronic intravenous therapy and health care costs. Whether this can also be applied for male patients treated with 1.0 mg/kg has not yet been determined.