OUTCOME RELIABILITY IN NON-AMBULATORY BOYS/MEN WITH DUCHENNE MUSCULAR DYSTROPHY

被引：61

作者：

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

Malkus, Elizabeth C. ^{[1
]}

Mendell, Jerry R. ^{[3
,4
]}

Flanigan, Kevin M. ^{[3
,4
]}

Miller, J. Philip ^{[5
]}

Schierbecker, Jeanine R. ^{[1
]}

Siener, Catherine A. ^{[1
]}

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

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

McDonald, Craig M. ^{[6
]}

Johnson, Linda ^{[6
]}

Nicorici, Alina ^{[6
]}

Karachunski, Peter I. ^{[7
]}

Day, John W. ^{[8
]}

Kelecic, Jason M. ^{[8
]}

Lowes, Linda P. ^{[3
,4
]}

Alfano, Lindsay N. ^{[3
,4
]}

Darras, Basil T. ^{[9
]}

Kang, Peter B. ^{[10
]}

Quigley, Janet ^{[9
]}

Pasternak, Amy E. ^{[9
]}

Florence, Julaine M. ^{[1
]}

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

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

[5] Washington Univ, Sch Med, Dept Biostat, St Louis, MO 63110 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, Dept Neurol, Boston Childrens Hosp, Boston, MA 02115 USA

[10] Univ Florida, Coll Med, Dept Neurol, Gainesville, FL 32611 USA

来源：

MUSCLE & NERVE | 2015年 / 51卷 / 04期

基金：

美国国家卫生研究院;

关键词：

corticosteroids; Duchenne muscular dystrophy; non-ambulatory; pulmonary function; quality of life; strength; HAND FUNCTION; NEUROMUSCULAR DISEASE; MUSCLE STRENGTH; CHILDREN; VALIDATION; SCOLIOSIS; SURVIVAL; TRIALS; SLEEP;

D O I：

10.1002/mus.24346

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Introduction: Therapeutic trials in Duchenne muscular dystrophy (DMD) often exclude non-ambulatory individuals. Here we establish optimal and reliable assessments in a multicenter trial. Methods: Non-ambulatory boys/men with DMD (N=91; 16.7 +/- 4.5 years of age) were assessed by trained clinical evaluators. Feasibility (percentage completing task) and reliability [intraclass correlation coefficients (ICCs) between morning and afternoon tests] were measured. Results: Forced vital capacity (FVC), assessed in all subjects, showed a mean of 47.8 +/- 22% predicted (ICC 0.98). Brooke Upper Extremity Functional Rating (Brooke) and Egen Klassifikation (EK) scales in 100% of subjects showed ICCs ranging from 0.93 to 0.99. Manual muscle testing, range of motion, 9-hole peg test, and Jebsen-Taylor Hand Function Test (JHFT) demonstrated varied feasibility (99% to 70%), with ICCs ranging from 0.99 to 0.64. We found beneficial effects of different forms of corticosteroids for the Brooke scale, percent predicted FVC, and hand and finger strength. Conclusions: Reliable assessment of non-ambulatory boys/men with DMD is possible. Clinical trials will have to consider corticosteroid use. Muscle Nerve 51: 522-532, 2015

引用

页码：522 / 532

页数：11

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