Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

被引：8

作者：

McDonald, Craig M. ^{[1
,2
]}

Marden, Jessica R. ^{[3
]}

Shieh, Perry B. ^{[4
]}

Wong, Brenda L. ^{[5
]}

Lane, Henry ^{[3
]}

Zhang, Adina ^{[3
]}

Nguyen, Ha ^{[3
]}

Frean, Molly ^{[3
]}

Trifillis, Panayiota ^{[6
]}

Koladicz, Karyn ^{[6
]}

Signorovitch, James ^{[3
]}

机构：

[1] Univ Calif Davis, Dept Phys Med & Rehabil, Davis, CA 95616 USA

[2] Univ Calif Davis, Dept Pediat, Davis, CA 95616 USA

[3] Anal Grp Inc, Boston, MA 02199 USA

[4] UCLA Hlth, Los Angeles, CA 90095 USA

[5] Univ Massachusetts, Dept Pediat, Mem Med Ctr, Worcester, MA 01605 USA

[6] PTC Therapeut Inc, South Plainfield, NJ 07080 USA

来源：

JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH | 2023年 / 12卷 / 04期

关键词：

clinical outcomes; deflazacort; disease milestones; Duchenne muscular dystrophy; efficacy; prednisone; prednisolone; WALK TEST; DEFLAZACORT; PLACEBO; GLUCOCORTICOIDS; PREDNISONE; MANAGEMENT; SURVIVAL; BENEFITS; ATALUREN; TRIAL;

D O I：

10.57264/cer-2022-0190

中图分类号：

R19 [保健组织与事业（卫生事业管理）];

学科分类号：

100404 [儿少卫生与妇幼保健学];

摘要：

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.

引用

页数：12

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