Department of Physical Therapy, Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Neuromuscul Disord. 2010 Mar;20(3):155-61. doi: 10.1016/j.nmd.2009.11.014. Epub 2010 Jan 13.
The motor skills of patients with spinal muscular atrophy, type I (SMA-I) are very limited. It is difficult to quantify the motor abilities of these patients and as a result there is currently no validated measure of motor function that can be utilized as an outcome measure in clinical trials of SMA-I. We have developed the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders ("CHOP INTEND") to evaluate the motor skills of patients with SMA-I. The test was developed following the evaluation of 26 infants with SMA-I mean age 11.5 months (1.4-37.9 months) with the Test of Infant Motor Performance and The Children's Hospital of Philadelphia Test of Strength in SMA, a newly devised motor assessment for SMA. Items for the CHOP INTEND were selected by an expert panel based on item mean and standard deviation, item frequency distribution, and Chronbach's alpha. Intra-rater reliability of the resulting test was established by test-retest of 9 infants with SMA-I over a 2 month period; Intraclass correlation coefficient (ICC) (3,1)=0.96. Interrater reliability was by video analysis of a mixed group of infants with neuromuscular disease by 4 evaluators; ICC (3,4)=0.98 and in a group of 8 typically developing infants by 5 evaluators ICC (3,5)=0.93. The face validity of the CHOP INTEND is supported by the use of an expert panel in item selection; however, further validation is needed. The CHOP INTEND is a reliable measure of motor skills in patients with SMA-I and neuromuscular disorders presenting in infancy.
脊髓性肌萎缩症 I 型(SMA-I)患者的运动技能非常有限。很难对这些患者的运动能力进行量化,因此目前尚无经过验证的运动功能测量方法可用于 SMA-I 的临床试验作为结果测量。我们开发了费城儿童医院婴儿神经肌肉疾病测试(“CHOP INTEND”)来评估 SMA-I 患者的运动技能。该测试是在对 26 名 SMA-I 患儿(平均年龄 11.5 个月(1.4-37.9 个月))进行评估后开发的,评估方法包括婴儿运动表现测试和费城儿童医院 SMA 力量测试,这是一种新设计的 SMA 运动评估方法。CHOP INTEND 的项目是由一个专家小组根据项目平均值和标准差、项目频率分布以及 Cronbach's alpha 选择的。9 名 SMA-I 患儿在 2 个月的时间内进行了测试-重测,从而确定了该测试的内部评估者可靠性;组内相关系数(ICC)(3,1)=0.96。通过由 4 名评估员对患有神经肌肉疾病的混合组婴儿进行视频分析,以及由 5 名评估员对 8 名正常发育婴儿进行视频分析,确定了该测试的外部评估者可靠性;ICC(3,4)=0.98 和 ICC(3,5)=0.93。CHOP INTEND 的表面效度得到了项目选择专家小组的使用支持;但是,还需要进一步验证。CHOP INTEND 是一种可靠的 SMA-I 患者和在婴儿期出现神经肌肉疾病患者运动技能的测量方法。