Real-world data of in-hospital administration of alglucosidase alfa in French patients with Pompe disease: results from the National Claims Database.

INTRODUCTION: Pompe disease is caused by a rare biallelic mutation in the GAA gene resulting in acid α-glucosidase deficiency and glycogen accumulation. AIM: We analyzed hospital admissions associated with the administration of Myozyme®, utilizing the French hospital discharge database, known in France as the Programme de Médicalisation des Systèmes d'Information (PMSI), which comprehensively captures all hospital activity within the country. METHODS: In this observational study, we examined hospitalization records from April 4, 2012, to December 31, 2019, within the PMSI database, focusing on admissions where Myozyme® was administered. We particularly investigated the incidence of critical care admissions and adverse events (AEs) related to Myozyme®. RESULTS: From 2012 to 2019, approximately 26,714 hospital stays involving Myozyme® administration were recorded for 239 patients. Most (96.6%) of these were outpatient stays, with only 3.2% in critical care. Furthermore, hospitalizations without critical care needs increased from 96% in 2012 to 99% in 2019. Of the patients receiving at least one infusion, 997 critical care admissions were recorded, with 781 (78.3%) occurring concurrent with or the day after the Myozyme® treatment without directly correlating to adverse effects of enzyme therapy. CONCLUSIONS: The analysis of the French hospital discharge database indicated that Myozyme® was associated with a low incidence of AEs and complications in a hospital context, supporting the consideration of its safe use in home-infusion settings.

[Opportunities and challenges of blockchain technology in the healthcare industry]. / Opportunités et enjeux de la technologie blockchain dans le secteur de la santé.

Often described as a tool to build trust among stakeholders with divergent interests, blockchain technology has been of interest to many sectors since it was first used in 2008. Initially designed to record financial transactions between individuals, its applications have largely evolved with technological advances and the growing interest of international companies. In the healthcare sector, blockchain is interesting for many of its features: its immutability which makes it an excellent support for authenticating sensitive data such as clinical trials consents, the possibility of publishing smart contracts that automate and facilitate many processes or the constitution of a network that agrees on the state of the information. Much acclaimed, blockchain technology is still to be tested in real-life conditions and adapted to a particularly complex regulatory and economic context in the healthcare sector.