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Lookup NU author(s): Professor Jordi Diaz ManeraORCiD
This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).
© 2024 The AuthorsDuring three previously reported clinical trials of avalglucosidase alfa in patients with Pompe disease, 17 out of 142 participants were considered by the investigators to be appropriate candidates for home infusion. During their respective trials, these participants received a total of 419 avalglucosidase alfa infusions at home under healthcare professional supervision. They were clinically stable with no history of moderate or severe infusion-associated reactions within at least 12 months prior to starting home infusions. As of February 25, 2022, the 15 participants with late-onset Pompe disease (LOPD) had received between 2 and 48 home infusions and the 2 participants with infantile-onset Pompe disease (IOPD) had received 19 and 20 infusions. Adverse events occurred in 8 (53 %) participants with LOPD and neither of the participants with IOPD. Seven participants with LOPD had a total of 15 non-treatment-related, non-serious adverse events. One participant with LOPD experienced infusion-associated reactions of eyelid edema and flushing during the first home infusion; both were non-serious adverse events classified as grade 1 (mild). Home infusion was later resumed for this participant. Among LOPD participants, event rates for home infusions were comparable to those for clinic infusions: overall adverse events (0.028 vs 0.039 participants with events/infusion, respectively) and adverse events classified as infusion-associated reactions (0.003 vs. 0.006, respectively). No medication errors occurred during home infusion. These data suggest that infusion of avalglucosidase alfa at home is feasible and does not compromise safety for patients who have not experienced an infusion-associated reaction during the preceding 12 months of infusions in a clinical setting. Evaluation of real-world experience with avalglucosidase alfa home infusion in countries where it is already approved is ongoing.
Author(s): Diaz-Manera J, Hughes D, Erdem-Ozdamar S, Tard C, Behin A, Bouhour F, Davison J, Hahn SH, Haack KA, Huynh-Ba O, Periquet M, Tammireddy S, Thibault N, Zhou T, van der Ploeg AT
Publication type: Article
Publication status: Published
Journal: Molecular Genetics and Metabolism
Year: 2024
Volume: 143
Issue: 4
Online publication date: 08/11/2024
Acceptance date: 05/11/2024
Date deposited: 05/12/2024
ISSN (print): 1096-7192
ISSN (electronic): 1096-7206
Publisher: Academic Press Inc.
URL: https://doi.org/10.1016/j.ymgme.2024.108608
DOI: 10.1016/j.ymgme.2024.108608
Data Access Statement: Data will be made available on request.
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