De Sanctis Roberto, Pane Marika, Sivo Serena, Ricotti Valeria, Baranello Giovanni, Frosini Silvia, Mazzone Elena, Bianco Flaviana, Fanelli Lavinia, Main Marion, Corlatti Alice, D'Amico Adele, Colia Giulia, Scalise Roberta, Palermo Concetta, Alfonsi Chiara, Tritto Giovanna, Romeo Domenico M, Graziano Alessandra, Battini Roberta, Morandi Lucia, Bertini Enrico, Muntoni Francesco, Mercuri Eugenio
Department of Paediatric Neurology, Catholic University, Rome, Italy.
Dubowitz Neuromuscular Centre, Institute of Child Health, University College, London, United Kingdom.
Neuromuscul Disord. 2015 Jan;25(1):14-8. doi: 10.1016/j.nmd.2014.09.015. Epub 2014 Oct 6.
The aim of this study was to establish the suitability of the North Star Ambulatory Assessment for use in young boys with Duchenne muscular dystrophy. We studied 147 typically developing and 144 boys affected by Duchenne muscular dystrophy between the ages of 3 and 5 years. More than 85% of the typically developing boys by the age of 4 years had full scores on all the items with total scores ≥33/34. Before the age of 4 years more than 15% of the typically developing boys did not achieve full scores on all the items. Some items, such as standing on one leg, showed significant improvement with age. In contrast, other activities were rarely achieved even in the older boys. Even if there was a progressive increase in scores with age, both total and individual item scores in Duchenne were still far from those obtained in the typically developing children of the same age. Our findings suggest that the North Star Ambulatory Assessment can be reliably used at least from the age of 4 years. Longitudinal natural history data studies are needed to assess possible changes over time and the possible effect of early steroids.
本研究的目的是确定北极星动态评估在患有杜氏肌营养不良症的幼龄男孩中的适用性。我们研究了147名发育正常的男孩以及144名年龄在3至5岁之间患有杜氏肌营养不良症的男孩。超过85%发育正常的4岁男孩在所有项目上都获得了满分，总分≥33/34。4岁之前，超过15%发育正常的男孩在所有项目上未获得满分。一些项目，如单腿站立，随着年龄增长有显著改善。相比之下，其他活动即使在年龄较大的男孩中也很少能完成。尽管随着年龄增长分数有逐渐提高，但杜氏肌营养不良症患儿的总分和单项分数仍远低于同龄发育正常儿童。我们的研究结果表明，北极星动态评估至少从4岁起就可以可靠使用。需要进行纵向自然史数据研究，以评估随时间可能发生的变化以及早期使用类固醇的可能影响。
