Multicenter, non-interventional, double cohort study to assess the safety of alglucosidase alfa and laronidase in real-world home infusion setting

ABSTRACT

Background: During Coronavirus Disease-19 (COVID-19) pandemic, the way to guarantee the adherence to treatment for patients with lysosomal disorders (LDs) in Italy, was home therapy, allowed by the temporary and exceptional authorization 341/2020 of the Italian Medicines Agency (AIFA). 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 was considered a good occasion to investigate the safety of home infusions considering that information is still lacking. Methods: This is an Italian, multicenter, non-interventional, double cohort study sponsored by Sanofi Genzyme 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 alglucosidase alfa and of patients with MPS I treated with 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.