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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.
- Source :
-
European journal of neurology [Eur J Neurol] 2024 Jul; Vol. 31 (7), pp. e16292. Date of Electronic Publication: 2024 Apr 08. - Publication Year :
- 2024
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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.<br />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.<br />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.<br />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.<br />Conclusion: Switching to avalglucosidase alfa demonstrated varied responses in advanced LOPD patients with failing alglucosidase alfa therapy, with a general improvement in motor stabilization.<br /> (© 2024 The Authors. European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.)
- Subjects :
- Humans
Male
Middle Aged
Female
France
Aged
Adult
Cohort Studies
Treatment Outcome
Registries
Disease Progression
Walk Test
Drug Substitution
Glycogen Storage Disease Type II drug therapy
Glycogen Storage Disease Type II complications
alpha-Glucosidases therapeutic use
Enzyme Replacement Therapy methods
Subjects
Details
- Language :
- English
- ISSN :
- 1468-1331
- Volume :
- 31
- Issue :
- 7
- Database :
- MEDLINE
- Journal :
- European journal of neurology
- Publication Type :
- Academic Journal
- Accession number :
- 38587143
- Full Text :
- https://doi.org/10.1111/ene.16292