학술논문
Safety outcomes and patients' preferences for home-based intravenous enzyme replacement therapy (ERT) in pompe disease and mucopolysaccharidosis type I (MPS I) disorder: COVID-19 and beyond
Document Type
Report
Author
Toscano, Antonio; Musumeci, Olimpia; Sacchini, Michele; Ravaglia, Sabrina; Siciliano, Gabriele; Fiumara, Agata; Verrecchia, Elena; Maione, Melania; Gentile, Jennifer; Fischetto, Rita; Crescimanno, Grazia; Taurisano, Roberta; Sechi, Annalisa; Gasperini, Serena; Cianci, Vittoria; Maggi, Lorenzo; Parini, Rossella; Lupica, Antonino; Scarpa, Maurizio
Source
Orphanet Journal of Rare Diseases. October 27, 2023, Vol. 18 Issue 1
Subject
Language
English
ISSN
1750-1172
Abstract
Author(s): Antonio Toscano[sup.1] , Olimpia Musumeci[sup.2] , Michele Sacchini[sup.3] , Sabrina Ravaglia[sup.4] , Gabriele Siciliano[sup.5] , Agata Fiumara[sup.6] , Elena Verrecchia[sup.7] , Melania Maione[sup.8] , Jennifer Gentile[sup.9] , Rita Fischetto[sup.10] [...]
Background The Italian Medicines Agency (AIFA) demands precise information on benefit/risk profile of home-based enzyme replacement therapy (ERT) for the treatment of patients with Pompe disease and Mucopolysaccharidosis type I (MPS I). This passage is necessary to obtain the authorization for ERT home therapy, even after the coronavirus disease-19 (COVID-19) pandemic period. This research intends to evaluate the safety, treatment satisfaction, and compliance of MPS I patients treated with laronidase (Aldurazyme[R]) and Pompe Disease patients treated with alglucosidase alfa (Myozyme[R]) in a homecare setting. Results We report herein an early interim analysis of the HomERT (Home infusions of ERT) study, a multicenter, non-interventional, double-cohort study that retrospectively analyzed 38 patients from 14 sites in Italy: cohort A (Pompe disease - 32 patients) and cohort B (MPS I - 6 patients). Among the selected patients who started home therapy before enrollment, the average number of missed home-based infusions was 0.7 (1.3) in cohort A and 3.8 (6.4) in cohort B with no return to the hospital setting. Irrespective of the treatment location, 3 prior ADRs per cohort were reported. The majority of patients preferred home-based infusions (cohort A: 96.9%; cohort B: 100%): the main reason was attributed to treatment convenience (cohort A: 81.3%; cohort B: 83.3%). Despite the underlying conditions, most patients self-evaluated their health as "good" (cohort A: 50%; cohort B: 83.3%). Conclusions Evidence of favorable safety profile, improved treatment compliance and personal satisfaction validates the use of ERT with laronidase and alglucosidase alfa as a strong candidate for home therapy. Keywords: Enzyme replacement therapy, Pompe disease, Home therapy, Mucopolysaccharidosis, Treatment adherence, Safety
Background The Italian Medicines Agency (AIFA) demands precise information on benefit/risk profile of home-based enzyme replacement therapy (ERT) for the treatment of patients with Pompe disease and Mucopolysaccharidosis type I (MPS I). This passage is necessary to obtain the authorization for ERT home therapy, even after the coronavirus disease-19 (COVID-19) pandemic period. This research intends to evaluate the safety, treatment satisfaction, and compliance of MPS I patients treated with laronidase (Aldurazyme[R]) and Pompe Disease patients treated with alglucosidase alfa (Myozyme[R]) in a homecare setting. Results We report herein an early interim analysis of the HomERT (Home infusions of ERT) study, a multicenter, non-interventional, double-cohort study that retrospectively analyzed 38 patients from 14 sites in Italy: cohort A (Pompe disease - 32 patients) and cohort B (MPS I - 6 patients). Among the selected patients who started home therapy before enrollment, the average number of missed home-based infusions was 0.7 (1.3) in cohort A and 3.8 (6.4) in cohort B with no return to the hospital setting. Irrespective of the treatment location, 3 prior ADRs per cohort were reported. The majority of patients preferred home-based infusions (cohort A: 96.9%; cohort B: 100%): the main reason was attributed to treatment convenience (cohort A: 81.3%; cohort B: 83.3%). Despite the underlying conditions, most patients self-evaluated their health as "good" (cohort A: 50%; cohort B: 83.3%). Conclusions Evidence of favorable safety profile, improved treatment compliance and personal satisfaction validates the use of ERT with laronidase and alglucosidase alfa as a strong candidate for home therapy. Keywords: Enzyme replacement therapy, Pompe disease, Home therapy, Mucopolysaccharidosis, Treatment adherence, Safety