Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
Categorising trajectories and individual item changes of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy
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DOI：
10.1371/journal.pone.0221097
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发表时间：
2019-09-03
期刊：
影响因子：
3.7
通讯作者：
Yirrel, J.
中科院分区：
文献类型：
--
作者：
Muntoni, Francesco;Domingos, Joana;Yirrel, J.
Duchenne肌营养不良症(DMD)男孩的功能变异性是公认的，并使临床研究的解释复杂化。我们假设，根据北极星动态评估(NSAA)的总分，患有DMD的男孩可以被分成几组，随着时间的推移，他们的步行功能轨迹相似。我们还探索了与其他变量的关系，如年龄、功能能力和基因。使用NorthStar临床网络数据库，395名进行了NSAA评估的患者被确定。我们利用了NSAA纵向得分的潜在班级轨迹分析，这产生了至少四组男孩按照临床严重程度的降序具有相似的轨迹的证据：25%的男孩属于第一组(NSAA下降到
Functional variability among boys with Duchenne muscular dystrophy (DMD) is well recognised and complicates interpretation of clinical studies. We hypothesised that boys with DMD could be clustered into groups sharing similar trajectories of ambulatory function over time, as measured by the North Star Ambulatory Assessment (NSAA) total score. We also explored associations with other variables such as age, functional abilities, and genotype. Using the NorthStar Clinical Network database, 395 patients with >1 NSAA assessment were identified. We utilised latent class trajectory analysis of longitudinal NSAA scores, which produced evidence for at least four clusters of boys sharing similar trajectories versus age in decreasing order of clinical severity: 25% of the boys were in cluster 1 (NSAA falling to