Longitudinal timed function tests in Duchenne muscular dystrophy: ImagingDMD cohort natural history

被引：58

作者：

Arora, Harneet ^{[1
]}

Willcocks, Rebecca J. ^{[1
]}

Lott, Donovan J. ^{[1
]}

Harrington, Ann T. ^{[2
]}

Senesac, Claudia R. ^{[1
]}

Zilke, Kirsten L. ^{[3
]}

Daniels, Michael J. ^{[4
]}

Xu, Dandan ^{[5
]}

Tennekoon, Gihan I. ^{[2
]}

Finanger, Erika L. ^{[3
]}

Russman, Barry S. ^{[3
]}

Finkel, Richard S. ^{[6
]}

Triplett, William T. ^{[1
]}

Byrne, Barry J. ^{[7
]}

Walter, Glenn A. ^{[8
]}

Sweeney, H. Lee ^{[9
]}

Vandenborne, Krista ^{[1
]}

机构：

[1] Univ Florida, Dept Phys Therapy, UFHSC, Box 100154, Gainesville, FL 32610 USA

[2] Childrens Hosp Philadelphia, Philadelphia, PA 19104 USA

[3] Oregon Hlth & Sci Univ, Portland, OR 97201 USA

[4] Univ Florida, Dept Stat, Gainesville, FL 32611 USA

[5] Univ Texas Austin, Dept Stat & Data Sci, Austin, TX 78712 USA

[6] Nemours Childrens Hosp, Orlando, FL USA

[7] Univ Florida, Powell Gene Therapy Ctr, Dept Pediat & Mol Genet & Microbiol, Gainesville, FL USA

[8] Univ Florida, Dept Physiol & Funct Genom, Gainesville, FL USA

[9] Univ Florida, Dept Pharmacol & Therapeut, Gainesville, FL USA

来源：

MUSCLE & NERVE | 2018年 / 58卷 / 05期

基金：

美国国家卫生研究院;

关键词：

6-minute walk test; ambulatory function; Duchenne muscular dystrophy; functional endpoints; loss of ambulation; outcome measures; 6-MINUTE WALK TEST; OUTCOME MEASURE; END-POINTS; MRI;

D O I：

10.1002/mus.26161

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

Introduction: Tests of ambulatory function are common clinical trial endpoints in Duchenne muscular dystrophy (DMD). Using these tests, the ImagingDMD study has generated a large data set that can describe the contemporary natural history of DMD in 5-12.9-year-olds. Methods: Ninety-two corticosteroid-treated boys with DMD and 45 controls participated in this longitudinal study. Participants performed the 6-minute walk test (6MWT) and timed function tests (TFT: 10-m walk/run, climbing 4 stairs, supine to stand). Results: Boys with DMD had impaired functional performance even at 5-6.9 years old. Boys older than 7 had significant declines in function over 1 year for 10-m walk/run and 6MWT. Eighty percent of participants could perform all functional tests at 9 years old. TFTs appear to be slightly more responsive and predictive of disease progression than the 6MWT in 7-12.9 year olds. Discussion: This study provides insight into the contemporary natural history of key functional endpoints in DMD. Muscle Nerve 58: 631-638, 2018

引用

页码：631 / 638

页数：8

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