Home infusion experience in patients with Pompe disease receiving avalglucosidase alfa during three clinical trials

• Published in: Molecular genetics and metabolism Vol. 143; no. 4; p. 108608
• Main Authors: Díaz-Manera, Jordi, Hughes, Derralynn, Erdem-Özdamar, Sevim, Tard, Céline, Béhin, Anthony, Bouhour, Françoise, Davison, James, Hahn, Si Houn, Haack, Kristina An, Huynh-Ba, Olivier, Periquet, Magali, Tammireddy, Swathi, Thibault, Nathan, Zhou, Tianyue, van der Ploeg, Ans T.
• Format: Journal Article
• Language: English
• Published: United States Elsevier Inc 01.12.2024; Elsevier
• Series: Molecular Genetics and Metabolism
• Subjects: Adolescent; Adult; alpha-Glucosidases - administration & dosage; alpha-Glucosidases - adverse effects; alpha-Glucosidases - therapeutic use; Avalglucosidase alfa; Child; Child, Preschool; Enzyme Replacement Therapy - adverse effects; Female; Glycogen Storage Disease Type II - drug therapy; Home infusion; Home Infusion Therapy - adverse effects; Humans; Infant; Life Sciences; Male; Middle Aged; Pompe disease; Safety; Young Adult; Pompe disease; Safety; Avalglucosidase alfa; Home infusion
• ISSN: 1096-7192; 1096-7206; 1096-7206
• DOI: 10.1016/j.ymgme.2024.108608

During 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. •We assessed safety of avalglucosidase alfa home infusions in three clinical trials.•Most adverse events during home infusion were non-serious and not treatment related.•Adverse event rates were comparable for home infusions and clinic infusions.•Patient safety was not compromised with home infusion of avalglucosidase alfa.