Deflazacort vs prednisone treatment for Duchenne muscular dystrophy: A meta-analysis of disease progression rates in recent multicenter clinical trials.
Anti-Inflammatory Agents
/ therapeutic use
Child
Disease Progression
Humans
Male
Multicenter Studies as Topic
Muscular Dystrophy, Duchenne
/ drug therapy
Prednisolone
/ therapeutic use
Prednisone
/ therapeutic use
Pregnenediones
/ therapeutic use
Randomized Controlled Trials as Topic
Treatment Outcome
Walking
Duchenne muscular dystrophy
ambulatory function
deflazacort
meta-analysis
prednisone/prednisolone
Journal
Muscle & nerve
ISSN: 1097-4598
Titre abrégé: Muscle Nerve
Pays: United States
ID NLM: 7803146
Informations de publication
Date de publication:
01 2020
01 2020
Historique:
received:
01
02
2019
revised:
02
10
2019
accepted:
03
10
2019
pubmed:
11
10
2019
medline:
31
3
2020
entrez:
11
10
2019
Statut:
ppublish
Résumé
In this study we characterized disease progression over 48 weeks among boys receiving deflazacort vs prednisone/prednisolone placebo arm treatment in two recent Duchenne muscular dystrophy (DMD) clinical trials. Ambulatory boys with DMD receiving placebo in the phase 3 ataluren (N = 115) and tadalafil (N = 116) trials were included. The trials required at least 6 months of prior corticosteroid use and stable baseline dosing. Associations between corticosteroid use and 48-week changes in ambulatory function were estimated using mixed models. Adjusted differences between corticosteroid groups were pooled in a meta-analysis. In the meta-analysis, deflazacort-treated patients vs prednisone/prednisolone-treated patients experienced, on average, lower declines of 28.3 meters on 6-minute walk distance (95% confidence interval [CI], 5.7, 50.9; 2.9 seconds on rise from supine [95% CI, 0.9, 4.9 seconds]; 2.3 seconds on 4-stair climb [95% CI, 0.5, 4.1 seconds]; and 2.9 [95% CI, 0.1, 5.8] points on the North Star Ambulatory Assessment linearized score). Deflazacort-treated patients experienced significantly lower functional decline over 48 weeks.
Identifiants
pubmed: 31599456
doi: 10.1002/mus.26736
pmc: PMC6973289
doi:
Substances chimiques
Anti-Inflammatory Agents
0
Pregnenediones
0
Prednisolone
9PHQ9Y1OLM
deflazacort
KR5YZ6AE4B
Prednisone
VB0R961HZT
Types de publication
Journal Article
Meta-Analysis
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
26-35Informations de copyright
© 2019 The Authors. Muscle & Nerve published by Wiley Periodicals, Inc.
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