Titre :

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

Auteur(s) :

Tard, Celine [Auteur]
Lille Neurosciences & Cognition (LilNCog) - U 1172
Bouhour, F. [Auteur]
Hospices Civils de Lyon [HCL]
Michaud, M. [Auteur]
Service de neurologie [CHRU Nancy]
Beltran, S. [Auteur]
Fournier, M. [Auteur]
CHU Caen
Demurger, F. [Auteur]
Centre hospitalier Bretagne Atlantique (Morbihan) [CHBA]
Lagrange, E. [Auteur]
Centre Hospitalier Universitaire [CHU Grenoble] [CHUGA]
Nollet, S. [Auteur]
Centre Hospitalier Régional Universitaire de Besançon [CHRU Besançon]
Sacconi, S. [Auteur]
Université Côte d'Azur [UniCA]
Noury, J. B. [Auteur]
Lymphocytes B, Autoimmunité et Immunothérapies [LBAI]
Centre Hospitalier Régional Universitaire de Brest [CHRU Brest]
Magot, A. [Auteur]
Centre Hospitalier Universitaire de Nantes = Nantes University Hospital [CHU Nantes]
Cintas, P. [Auteur]
Centre Hospitalier Universitaire de Toulouse [CHU Toulouse]
Renard, D. [Auteur]
Université de Montpellier [UM]
Centre Hospitalier Universitaire de Nîmes [CHU Nîmes]
Deibener-Kaminsky, J. [Auteur]
Centre Hospitalier Universitaire de Nancy [CHU Nancy]
Lefeuvre, C. [Auteur]
Hôpital Raymond Poincaré [AP-HP]
Davion, Jean Baptiste [Auteur]
Lille Neurosciences & Cognition - U 1172 [LilNCog]
Centre de référence des maladies rares neuromusculaires
Salort-Campana, E. [Auteur]
Marseille medical genetics - Centre de génétique médicale de Marseille [MMG]
Arrassi, A. [Auteur]
CHU Pitié-Salpêtrière [AP-HP]
Taouagh, N. [Auteur]
Spinazzi, M. [Auteur]
Centre Hospitalier Universitaire d'Angers [CHU Angers]
Attarian, S. [Auteur]
Laforêt, P. [Auteur]

Titre de la revue :

European Journal of Neurology

Nom court de la revue :

Eur J Neurol

Numéro :

31

Pagination :

e16292

Date de publication :

2024-04-09

ISSN :

1468-1331

Mot(s)-clé(s) en anglais :

enzymotherapy
glycogenosis
switch
treatment management
unmet needs

Discipline(s) HAL :

Sciences du Vivant [q-bio]

Résumé en anglais : [en]

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 ...
Lire la suite >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.Lire moins >

Langue :

Anglais

Audience :

Internationale

Vulgarisation :

Non

Établissement(s) :

Université de Lille
Inserm
CHU Lille

Collections :

• Lille Neurosciences & Cognition (LilNCog) - U 1172

Date de dépôt :

2024-05-06T22:33:39Z
2024-09-05T08:45:30Z