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

Introduction. Recently, chronic supplementation with α-galactosidase A (αGal 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. Methods. 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... Mehr ...