Home Enzyme Replacement Therapy in Gaucher Disease: A Review.

Abstract

Since the early 1990s, Gaucher Disease has been a pioneering condition for home-based enzyme replacement therapy (ERT), marking a significant shift in patient care. Since then, many countries have adopted this approach. However, home ERT is not possible in all countries. <b>Objectives:</b> The aim of this article is to explore the implementation of home ERT for Gaucher disease, focusing on patient expectations, safety, compliance, economic benefits, and practical considerations. <b>Methods:</b> The PRISMA reporting protocol was followed, focusing on articles about home ERT for Gaucher Disease. <b>Results:</b> Twenty articles were analyzed in the review, revealing promising outcomes. Home ERT has consistently been shown to be safe, to improve patients' quality of life, to reduce the utilization of hospital resources, and to pose no compliance issues. <b>Conclusions:</b> We believe it is essential to expand the availability of home ERT for Gaucher Disease to all countries where ERT is accessible. Based on the literature review, we present the conditions that must be met before starting home ERT programs.