Department of Paediatric Neurology, Catholic University, Rome, Italy.
PLoS One. 2013;8(1):e52512. doi: 10.1371/journal.pone.0052512. Epub 2013 Jan 11.
The aim of the study was i) to assess the spectrum of changes over 24 months in ambulant boys affected by Duchenne muscular dystrophy, ii) to establish the difference between the first and the second year results and iii) to identify possible early markers of loss of ambulation.
One hundred and thirteen patients (age range 4.1-17, mean 8.2) fulfilled the inclusion criteria, 67 of the 113 were on daily and 40 on intermittent steroids, while 6 were not on steroids. All were assessed using the 6 Minute Walk Test (6MWT), the North Star Ambulatory Assessment (NSAA) and timed test.
On the 6MWT there was an average overall decline of -22.7 (SD 81.0) in the first year and of -64.7 (SD 123.1) in the second year. On the NSAA the average overall decline was of -1.86 (SD 4.21) in the first year and of -2.98 (SD 5.19) in the second year. Fourteen children lost ambulation, one in the first year and the other 13 in the second year of the study. A distance of at least 330 meters on the 6MWT, or a NSAA score of 18 at baseline reduced significantly the risk of losing ambulation within 2 years.
These results can be of help at the time of using inclusion criteria for a study in ambulant patients in order to minimize the risk of patients who may lose ambulation within the time of the trial.
本研究旨在：i）评估 24 个月内患杜氏肌营养不良症的能行走男童的变化情况；ii）确定第 1 年和第 2 年结果之间的差异；iii）确定丧失行走能力的早期标志物。
113 名患者（年龄范围 4.1-17 岁，平均 8.2 岁）符合纳入标准，113 名患者中有 67 名每天服用类固醇，40 名间歇性服用类固醇，6 名未服用类固醇。所有患者均接受 6 分钟步行测试（6MWT）、北极星步行评估（NSAA）和计时测试评估。
在 6MWT 中，第 1 年平均总下降 22.7（SD 81.0），第 2 年平均总下降 64.7（SD 123.1）。在 NSAA 中，第 1 年平均总下降为 1.86（SD 4.21），第 2 年平均总下降为 2.98（SD 5.19）。14 名儿童丧失行走能力，其中 1 名在第 1 年，13 名在研究的第 2 年。6MWT 至少 330 米或基线时 NSAA 评分为 18 分可显著降低 2 年内丧失行走能力的风险。
这些结果可用于制定能行走患者研究的纳入标准，以尽量减少试验期间丧失行走能力的患者风险。
