Henricson Erik, Abresch Richard, Han Jay J, Nicorici Alina, Goude Keller Erica, Elfring Gary, Reha Allen, Barth Jay, McDonald Craig M
Department of Physical Medicine & Rehabilitation, University of California, Davis, CA, USA.
PLoS Curr. 2012 Jan 25;4:RRN1297. doi: 10.1371/currents.RRN1297.
We recently described a modified version of the 6-minute walk test (6MWT) for Duchenne muscular dystrophy (DMD) based partly on the American Thoracic Society (ATS) guidelines. This measure has shown reliability, validity and utility as a primary outcome measure in DMD clinical trials. Because loss of muscle function in DMD occurs against the background of normal childhood growth and development, younger children with DMD can show increase in distance walked during 6MWT over ~1 year despite progressive muscular impairment. In this study, we compare 6-minute walk distance (6MWD) data from DMD boys (n=17) and typically developing control subjects (n=22) to existing normative data from age- and sex-matched children and adolescents. An age- and height-based equation fitted to normative data by Geiger and colleagues was used to convert 6MWD to a percent-predicted (%-predicted) value in boys with DMD. Analysis of %-predicted 6MWD data represents a method to account for normal growth and development, and shows that gains in function at early ages represents stable rather than improving abilities in boys with DMD. Boys with DMD from 4-7 years of age maintain a stable 6MWD approximately 80% of that of typically developing peers, with the deficit progressing at a variable rate thereafter.
我们最近描述了一种针对杜氏肌营养不良症(DMD)的6分钟步行测试(6MWT)的改良版本,该版本部分基于美国胸科学会(ATS)的指南。这项测量已显示出作为DMD临床试验主要结局指标的可靠性、有效性和实用性。由于DMD患者的肌肉功能丧失是在正常儿童生长发育的背景下发生的,尽管肌肉功能逐渐受损,但患有DMD的年幼儿童在约1年的时间里6MWT步行距离仍可能增加。在本研究中,我们将DMD男孩(n = 17)和正常发育对照受试者(n = 22)的6分钟步行距离(6MWD)数据与年龄和性别匹配的儿童及青少年的现有规范数据进行比较。使用Geiger及其同事根据规范数据拟合的基于年龄和身高的方程,将DMD男孩的6MWD转换为预测百分比(%-预测)值。对%-预测的6MWD数据进行分析是一种考虑正常生长发育的方法,结果表明,DMD男孩早期功能的改善代表能力稳定而非提高。4至7岁的DMD男孩的6MWD稳定维持在正常发育同龄人6MWD的约80%,此后差距以可变速率扩大。