CLINICAL TRIAL READINESS IN NON-AMBULATORY BOYS AND MEN WITH DUCHENNE MUSCULAR DYSTROPHY: MDA-DMD NETWORK FOLLOW-UP

被引：32

作者：

Connolly, Anne M. ^{[1
,2
]}

Florence, Julaine M. ^{[1
]}

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

Golumbek, Paul T. ^{[1
,2
]}

Mendell, Jerry R. ^{[4
,5
]}

Flanigan, Kevin M. ^{[4
,5
]}

Karachunski, Peter I. ^{[7
]}

Day, John W. ^{[8
]}

Mcdonald, Craig M. ^{[6
]}

Darras, Basil T. ^{[9
]}

Kang, Peter B. ^{[10
]}

Siener, Catherine A. ^{[1
]}

Gadeken, Rebecca K. ^{[1
]}

Anand, Pallavi ^{[1
]}

Schierbecker, Jeanine R. ^{[1
]}

Malkus, Elizabeth C. ^{[1
]}

Lowes, Linda P. ^{[4
,5
]}

Alfano, Lindsay N. ^{[4
,5
]}

Johnson, Linda ^{[6
]}

Nicorici, Alina ^{[6
]}

Kelecic, Jason M. ^{[7
]}

Quigley, Janet ^{[9
]}

Pasternak, Amy E. ^{[9
]}

Miller, J. Philip ^{[3
]}

机构：

[1] Washington Univ, Sch Med, Dept Neurol, St Louis, MO 63110 USA

[2] Washington Univ, Sch Med, Dept Pediat, St Louis, MO 63110 USA

[3] Washington Univ, Sch Med, Div Biostat, St Louis, MO 63110 USA

[4] Ohio State Univ, Dept Pediat, Columbus, OH 43210 USA

[5] Nationwide Childrens Hosp, Ctr Gene Therapy, Columbus, OH USA

[6] Univ Calif Davis, Med Ctr, Dept Phys Med & Rehabil, Sacramento, CA 95817 USA

[7] Univ Minnesota, Dept Neurol, Minneapolis, MN 55455 USA

[8] Stanford Univ, Dept Neurol, Stanford, CA 94305 USA

[9] Harvard Univ, Boston Childrens Hosp, Dept Neurol, Boston, MA 02115 USA

[10] Univ Florida, Coll Med, Div Pediat Neurol, Gainesville, FL USA

来源：

MUSCLE & NERVE | 2016年 / 54卷 / 04期

基金：

美国国家卫生研究院;

关键词：

Brooke Scale; clinical trial; Duchenne muscular dystrophy; Egen Klassifikation Scale; force vital capacity; non-ambulatory; outcomes; PULMONARY-FUNCTION; GLUCOCORTICOID TREATMENT; NATURAL-HISTORY; PROGRESSION; VALIDATION; PREDNISONE; CHILDREN; SURVIVAL; THERAPY; DISEASE;

D O I：

10.1002/mus.25089

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

100204 [神经病学];

摘要：

Introduction: Outcomes sensitive to change over time in non-ambulatory boys/men with Duchenne muscular dystrophy (DMD) are not well-established. Methods: Subjects (n = 91; 16.8 +/- 4.5 years old) were assessed at baseline and 6-month intervals for 2 years. We analyzed all subjects using an intent-to-treat model and a subset of stronger subjects with Brooke Scale score <= 4, using repeated measures. Results: Eight patients (12-33 years old) died during the study. Sixty-six completed 12-month follow-up, and 51 completed 24-month follow-up. Those taking corticosteroids performed better at baseline, but rates of decline were similar. Forced vital capacity percent predicted (FVC% predicted) declined significantly only after 2 years. However, Brooke and Egen Klassifikation (EK) Scale scores, elbow flexion, and grip strength declined significantly over both 1 and 2 years. Conclusion: Brooke and EK Scale scores, elbow flexion, and grip strength were outcomes most responsive to change. FVC% predicted was responsive to change over 2 years. Corticosteroids benefited non-ambulatory DMD subjects but did not affect decline rates of measures tested here.

引用

页码：681 / 689

页数：9

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