Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status.
J Comp Eff Res
; 12(4): e220190, 2023 04.
Article
em En
| MEDLINE
| ID: mdl-36749302
ABSTRACT
Aim:
To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease progression.Methods:
Data from daily steroid users (placebo-treated) were pooled from four phase 2b/3 trials in DMD. Outcomes assessed overall and among subgroups included changes from baseline to 48 weeks in six-minute walk distance (6MWD), timed function tests and North Star Ambulatory Assessment total score.Results:
Among 231 patients receiving deflazacort (n = 127) or prednisone (n = 104), observed differences in 6MWD favoring deflazacort over prednisone were significant for patients with relatively older age (≥8-years-old), greater disease progression (baseline timed stand from supine ≥5 s), or longer corticosteroid use (>3 years).Conclusion:
Daily deflazacort had greater benefits than daily prednisone particularly among older/more progressed patients.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Distrofia Muscular de Duchenne
Tipo de estudo:
Clinical_trials
Limite:
Child
/
Humans
Idioma:
En
Revista:
J Comp Eff Res
Ano de publicação:
2023
Tipo de documento:
Article
País de afiliação:
Estados Unidos