Smith R A, Newcombe R G, Sibert J R, Harper P S
Institute of Medical Genetics, University of Wales College of Medicine, Cardiff.
Muscle Nerve. 1991 May;14(5):462-9. doi: 10.1002/mus.880140513.
Thirty-three young boys (mean age 3.42 years) with Duchenne muscular dystrophy (DMD) and 21 normal control boys (mean age 3.51 years) were studied prospectively to determine whether it is possible to objectively assess locomotor function in young boys with DMD so that they can be included in treatment trials. An initial reproducibility study using a hand-held myometer demonstrated that this method was not useful. The Hammersmith Motor Ability Score demonstrated an increase in developmental abilities with age which was markedly different from normal. The locomotor quotient of the Griffiths' Scales demonstrated a deterioration of quotient scores and is a useful method of assessment that could be used in treatment trials involving young boys with DMD. Sample size planning for treatment trials is discussed.
对33名患有杜氏肌营养不良症（DMD）的男童（平均年龄3.42岁）和21名正常对照男童（平均年龄3.51岁）进行了前瞻性研究，以确定是否有可能客观评估患有DMD的男童的运动功能，以便将他们纳入治疗试验。最初使用手持式肌动计进行的重复性研究表明，该方法并无用处。哈默史密斯运动能力评分显示，随着年龄增长，发育能力有所提高，这与正常情况明显不同。格里菲斯量表的运动商显示商数得分有所下降，这是一种有用的评估方法，可用于涉及患有DMD的男童的治疗试验。文中还讨论了治疗试验的样本量规划。
