Argentine program of home infusions with enzyme replacement therapy for lysosomal diseases: Results in safety, quality of life and adherence

Abstract

With over 25 years of experience, enzyme replacement therapies have proven to be safe and effective for patients with Fabry, Gaucher, Pompe, Mucopolysaccharidosis I (MPS I) diseases. As with many chronic conditions, one of the keys to achieving therapeutic goals is to avoid interruptions in infusions to ensure proper adherence (AD). Treatment interruptions are associated with the recurrence of previously resolved clinical manifestations depending on the length of the interruption, and the reaccumulation of substrate at the cellular level. The Argentine home infusion program (HI) was established in 2012 in response to recognizing low treatment adherence. This is a retrospective review of the program's safety, quality of life, and treatment adherence after 11 years. A total of 147 treating physicians enrolled 378 patients from 22 Argentine provinces into the program. A total of 85,060 home infusions were performed during the study period. No patients experienced serious adverse events requiring hospitalization. Throughout the follow-up period, 21,600 infusions were suspended, with the most frequent cause being lack of supply from the payer. Adherence to enzyme replacement therapy showed an annual adherence rate of over 80 % across all pathologies. All patients (100 %) reported that the program had improved their quality of life. The Argentine home infusion program has demonstrated an improvement in adherence and quality of life, which is considered significant for enhancing long-term therapeutic outcomes.