Multi-Centre Study to Assess the Safety of Alglucosidasi and of Laronidasi in Home Infusion Setting

ABSTRACT

Background - During Coronavirus Disease-19 (COVID- 19) pandemic, the temporary and exceptional authorization 341/2020 0f the Italian Drug Agency (AIFA), allowed to guarantee the adherence to treatment for patients with lysosomal storage disorders (LSDs) through home therapy. Stable patients affected with Pompe Disease and Mucopolysaccharidosis type I (MPS I) could then receive regular Enzyme Replacement Therapy (ERT) infusions at home. Indeed, a data collection seemed to us a good opportunity to assess the safety of home infusions and thus fill the existing information gap. Methods - This is an Italian, multicenter, non-interventional, double cohort study sponsored by SanofiGenzyme with both retrospective and prospective data collection to obtain safety information on ERT treatment of Pompe Disease and Mucopolysaccharidosis type I (MPS I) patients in a home-care setting. The study will enroll 60 patients at 15 sites. The retrospective observation will start from the first ERT infusions in a homecare setting and the prospective observation will last after 12 months from the enrollment. During the control visits, investigators will administer the questionnaires and will record any documented clinical data occurred during the home infusions. Objectives - This study aims at obtaining safety information on patients with Pompe Disease treated with Myozyme® (alglucosidase alfa) and of patients with MPS I treated with Aldurazyme® (laronidase) in a home-care setting, as well as evaluating personal satisfaction of both cohorts of patients and documenting infusion compliance. Conclusions - The outcomes will mirror real-life management of patients in home-care infusion setting, including safety profile, treatment compliance and quality of life.