1. Real‐life effectiveness 1 year after switching to avalglucosidase alfa in late‐onset Pompe disease patients worsening on alglucosidase alfa therapy: A French cohort study.
- Author
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Tard, Céline, Bouhour, Françoise, Michaud, Maud, Beltran, Stephane, Fournier, Maxime, Demurger, Florence, Lagrange, Emmeline, Nollet, Sylvain, Sacconi, Sabrina, Noury, Jean‐Baptiste, Magot, Armelle, Cintas, Pascal, Renard, Dimitri, Deibener‐Kaminsky, Joëlle, Lefeuvre, Claire, Davion, Jean‐Baptiste, Salort‐Campana, Emmanuelle, Arrassi, Azzeddine, Taouagh, Nadjib, and Spinazzi, Marco
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ENZYME replacement therapy , *COHORT analysis , *VITAL capacity (Respiration) , *GLYCOGEN storage disease type II - Abstract
Introduction: Late‐onset Pompe disease (LOPD) is characterized by a progressive myopathy resulting from a deficiency of acid α‐glucosidase enzyme activity. Enzyme replacement therapy has been shown to be effective, but long‐term treatment results vary. Avalglucosidase alfa demonstrated non‐inferiority to alglucosidase alfa in a phase 3 study, allowing in France compassionate access for advanced LOPD patients unresponsive to alglucosidase alfa. Methods: Data from the French Pompe registry were analyzed for patients who benefited from a switch to avalglucosidase alfa with at least 1 year of follow‐up. Respiratory (forced vital capacity [FVC]) and motor functions (Six‐Minute Walk Test [6MWT]) were assessed before and 1 year after switching. Individual changes in FVC and 6MWT were expressed as slopes and statistical analyses were performed to compare values. Results: Twenty‐nine patients were included (mean age 56 years, 11 years of prior treatment). The FVC and 6MWT values remained stable. The individual analyses showed a stabilization of motor worsening: –1 m/year on the 6MWT after the switch versus –63 m/year the year before the switch (i.e., a worsening of 33%/year before vs. an improvement of 3%/year later). Respiratory data were not statistically different. Discussion: At the group level, gait parameters improved slightly with a stabilization of previous worsening, but respiratory parameters showed limited changes. At the individual level, results were discordant, with some patients with a good motor or respiratory response and some with further worsening. Conclusion: Switching to avalglucosidase alfa demonstrated varied responses in advanced LOPD patients with failing alglucosidase alfa therapy, with a general improvement in motor stabilization. [ABSTRACT FROM AUTHOR]
- Published
- 2024
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