Division of Physical Medicine and Rehabilitation, University of Utah School of Medicine, Salt Lake City, Utah 84132, USA.
Muscle Nerve. 2010 Nov;42(5):703-8. doi: 10.1002/mus.21838.
Reliable outcome measures that reflect the underlying disease process and correlate with motor function in children with SMA are needed for clinical trials. Maximum ulnar compound muscle action potential (CMAP) data were collected at two visits over a 4-6-week period in children with SMA types II and III, 2-17 years of age, at four academic centers. Primary functional outcome measures included the Modified Hammersmith Functional Motor Scale (MHFMS) and MHFMS-Extend. CMAP negative peak amplitude and area showed excellent discrimination between the ambulatory and non-ambulatory SMA cohorts (ROC = 0.88). CMAP had excellent test-retest reliability (ICC = 0.96-0.97, n = 64) and moderate to strong correlation with the MHFMS and MHFMS-Extend (r = 0.61-0.73, n = 68, P < 0.001). Maximum ulnar CMAP amplitude and area is a feasible, valid, and reliable outcome measure for use in pediatric multicenter clinical trials in SMA. CMAP correlates well with motor function and has potential value as a relevant surrogate for disease status.
需要可靠的能够反映疾病进程且与 SMA 患儿运动功能相关的结局评估指标，以便开展临床试验。在四个学术中心，2-17 岁 SMA Ⅱ型和Ⅲ型患儿接受为期 4-6 周、两次访视的最大尺神经复合肌肉动作电位（CMAP）检测。主要功能结局评估指标包括改良 Hammersmith 运动功能量表（MHFMS）和 MHFMS-伸展。CMAP 负向波峰振幅和面积能够很好地区分可移动和不可移动的 SMA 队列（ROC = 0.88）。CMAP 具有很好的测试-重测信度（ICC = 0.96-0.97，n = 64），与 MHFMS 和 MHFMS-伸展具有中等到高度的相关性（r = 0.61-0.73，n = 68，P < 0.001）。最大尺神经 CMAP 振幅和面积是一种可行、有效且可靠的 SMA 儿科多中心临床试验结局评估指标。CMAP 与运动功能相关性良好，作为疾病状态相关替代指标具有潜在价值。
