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.
enzymotherapy
glycogenosis
switch
treatment management
unmet needs
Journal
European journal of neurology
ISSN: 1468-1331
Titre abrégé: Eur J Neurol
Pays: England
ID NLM: 9506311
Informations de publication
Date de publication:
08 Apr 2024
08 Apr 2024
Historique:
revised:
17
02
2024
received:
10
01
2024
accepted:
14
03
2024
medline:
8
4
2024
pubmed:
8
4
2024
entrez:
8
4
2024
Statut:
aheadofprint
Résumé
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. 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. 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. 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. Switching to avalglucosidase alfa demonstrated varied responses in advanced LOPD patients with failing alglucosidase alfa therapy, with a general improvement in motor stabilization.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e16292Subventions
Organisme : Amicus Therapeutics
Organisme : Sanofi
Investigateurs
D Orlikowski
(D)
M Masingue
(M)
T Stojkovic
(T)
A Béhin
(A)
P Y Garcia
(PY)
F Lallement
(F)
G Bassez
(G)
D Germain
(D)
L Kouton
(L)
A Verschueren
(A)
A Furby
(A)
A L Kaminsky
(AL)
A Lacour
(A)
J Praline
(J)
G Sole
(G)
F Duval
(F)
F Bouibede
(F)
F Chapon
(F)
M C Minot
(MC)
J Hubert
(J)
Y Pereon
(Y)
B Acket
(B)
A Nadaj-Pakleza
(A)
R J Morales
(RJ)
F Esselin
(F)
P Petiot
(P)
R Jaussaud
(R)
S Mohamed
(S)
Y A Echaniz-Laguna
(YA)
L Magy
(L)
E Krim
(E)
F Taithe
(F)
A L Bedat-Millet
(AL)
S Toquet
(S)
E Diab
(E)
M Maillet-Vioud
(M)
Informations de copyright
© 2024 The Authors. European Journal of Neurology published by John Wiley & Sons Ltd on behalf of European Academy of Neurology.
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