Enzyme replacement therapy

PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled “Intravenous enzyme replacement therapy: better in home or hospital?” (Br J Nurs. 2006 Mar 23-Apr 12;15(6):330-3). Authors are Milligan A, Hughes D, Goodwin S et al, from the Lysosomal Storage Disorders Unit, Royal Free Hospital, London, UK. The article evaluates satisfaction with enzyme replacement therapy (ERT) at home and at hospital. A questionnaire was developed and sent to 34 patients with Fabry disease who were receiving ERT with agalsidase alfa (Replagal) and to 49 patients with type I Gaucher disease who were receiving ERT with glucocerebrosidase (Cerezyme). Of the 45 returned questionnaires, 20 were from patients with Fabry disease and 25 from patients with Gaucher disease. Nearly all of the patients preferred home-based therapy. Treatment in the home was reported as more comfortable, less stressful, more effective and had less impact on family life. To access the full abstract of the article, click here.