Researchers indicated that home-based infusions of alglucosidase alfa (Myozyme®) in late-onset Pompe disease (LOPD) are safe, according to data presented at the 18th Annual WORLDSymposium 2022.

“The COVID-19 pandemic has prompted the need to provide enzyme replacement therapy (ERT) for Pompe patients at home. However, this is not allowed in most countries due to safety concerns related to the risk of infusion associated reactions (IARs),” the authors said.

The data analysis of 13 years of experience from the Erasmus University Medical Center in Rotterdam, Netherlands revealed the occurrence of an IAR in 1% of home infusions and 2% of hospital infusions. Most IARs were mild (73 in hospital infusions and 126 in-home infusions) and did not require additional medical intervention.

Continue Reading

Read more about Pompe disease therapies

Three and 5 moderate IARs were reported among hospital and home infusions, respectively. No severe IARs were reported in any of the contexts.

The study included data on patients’ descriptives, infusion characteristics, and IARs from 114 patients with LOPD starting ERT between 2006 and 2018. IARs from a total of 16,920 infusions with alglucosidase alfa were graded with the Common Terminology Criteria for Adverse Events classification.

Home-Infused Avalglucosidase Alfa Could Be Safe in Pompe Disease

Most (76.6%) infusions were given at home, and most (88.7%) infusions were administered using a standard infusion schedule. Lastly, similar conclusions were recently reported for home-infused avalglucosidase alfa.


Ditters IAM, van Kooten H, van der Beek NAME, van den Hout H, Huidekoper HH, van der Ploeg AT. Safety of home-based infusion of alglucosidase alfa in late onset Pompe disease: 13 years of experience from the Erasmus MC University Medical Center. Mol Genet Metab. 2022;135(2):S39. doi:10.1016/j.ymgme.2021.11.088