College of Public Health, University of Arizona, Tucson, Arizona, USA.
Muscle Nerve. 2014 Jun;49(6):814-21. doi: 10.1002/mus.24078.
The correlation of markers of disease severity among brothers with Duchenne or Becker muscular dystrophy has implications for clinical guidance and clinical trials.
Sibling pairs with Duchenne or Becker muscular dystrophy (n = 60) were compared for ages when they reached clinical milestones of disease progression, including ceased ambulation, scoliosis of ≥ 20°, and development of cardiomyopathy.
The median age at which younger brothers reached each milestone, compared with their older brothers ranged from 25 months younger for development of cardiomyopathy to 2 months older for ceased ambulation. For each additional month of ambulation by the older brother, the hazard of ceased ambulation by the younger brother decreased by 4%.
The ages when siblings reach clinical milestones of disease vary widely between siblings. However, the time to ceased ambulation for older brothers predicts the time to ceased ambulation for their younger brothers.
杜氏肌营养不良症或贝克型肌营养不良症兄弟患者间疾病严重程度标志物的相关性对临床指导和临床试验具有重要意义。
将杜氏肌营养不良症或贝克型肌营养不良症的兄弟患者（n=60）进行配对，比较他们达到疾病进展的临床里程碑时的年龄，包括停止行走、脊柱侧弯≥20°和发展为心肌病。
与他们的哥哥相比，弟弟达到心肌病、停止行走和脊柱侧弯≥20°等各个临床里程碑的年龄中位数分别小 25 个月、大 2 个月和小 2 个月。哥哥每多行走一个月，弟弟停止行走的风险就会降低 4%。
兄弟姐妹达到疾病临床里程碑的年龄差异很大。然而，哥哥停止行走的时间可以预测弟弟停止行走的时间。
