The Minimal Clinical Important Difference (MCID) in Annual Rate of Change of Timed Function Tests in Boys with DMD

被引:18
作者
Duong, Tina [1 ]
Canbek, Jennifer [2 ]
Birkmeier, Marisa [3 ]
Nelson, Leslie [4 ]
Siener, Catherine [5 ]
Fernandez-Fernandez, Alicia [2 ]
Henricson, Erik [6 ]
McDonald, Craig M. [6 ]
Gordish-Dressman, Heather [7 ]
机构
[1] Stanford Univ, Dept Neurol, Sch Med, Stanford, CA 94305 USA
[2] Nova Southeastern Univ, Phys Therapy Dept, Ft Lauderdale, FL 33314 USA
[3] George Washington Univ, Sch Med & Hlth Sci, Dept Hlth Human Funct & Rehabil Sci, Washington, DC 20052 USA
[4] Univ Texas Southwestern Med Ctr Dallas, Dept Phys Therapy, Dallas, TX 75390 USA
[5] Washington Univ, Dept Neurol, St Louis, MO 63110 USA
[6] Univ Calif Davis Hlth, Dept Phys Med & Rehabil, Sacramento, CA USA
[7] Childrens Natl Med Ctr, Dept Biostat, Washington, DC 20010 USA
[8] Cooperat Int Neuromuscular Res Grp CINRG, Washington, DC USA
基金
美国国家卫生研究院;
关键词
Duchenne muscular dystrophy; MCID; time function tests; DUCHENNE MUSCULAR-DYSTROPHY; QUALITY-OF-LIFE; MEANINGFUL CHANGE; OUTCOME MEASURES; NATURAL-HISTORY; DISEASE; TRIAL; QUESTIONNAIRE; NUSINERSEN; CHILDREN;
D O I
10.3233/JND-210646
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Background: Duchenne muscular dystrophy (DMD) is a rare x-linked recessive genetic disorder affecting 1 in every 5000-10000 [1, 2]. This disease leads to a variable but progressive sequential pattern of muscle weakness that eventually causes loss of important functional milestones such as the ability to walk. With promising drugs in development to ameliorate the effects of muscle weakness, these treatments must be associated with a clinically meaningful functional change. Objective: The objective of this analysis is to determine both distribution, minimal detectable change (MDC), and anchorbased, minimal clinically important difference, (MCID) of 12-month change values in standardized time function tests (TFT) used to monitor disease progression in DMD. Method: This is a retrospective analysis of prospectively collected data from a multi-center prospective natural history study with the Cooperative International Neuromuscular Research Group (CINRG). This study calculated MDC and MCID values for 3 commonly used timed function tests typically used to monitor disease progression; supine to stand (STS), 10 meter walk/run (10MWT), and 4 stair climb (4SC). MDC used standard error of measurement (SEM) while MCID measurements used the Vignos scale as an anchor to determine clinical change in functional status. Results: All 3 TFT were significantly important clinical endpoints to detect MDC and MCID changes. MDC and MCID 12-month changes were significant in 10MWT (-0.138, -0.212), Supine to Stand (-0.026, -0.023) and 4 stair climb (-0.034, -0.035) with an effect size greater or close to 0.2. Conclusion: The 3 TFT are clinically meaningful endpoints used to establish change in DMD. MCID values were higher than MDC values indicating that an anchor-based approach using Vignos as a clinically meaningful loss of lower extremity abilities is appropriate to assess change in boys with DMD.
引用
收藏
页码:939 / 948
页数:10
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