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从地面定时起身作为杜氏肌营养不良症疾病进展的预测指标:一项观察性研究

Timed Rise from Floor as a Predictor of Disease Progression in Duchenne Muscular Dystrophy: An Observational Study.

作者信息

Mazzone Elena S, Coratti Giorgia, Sormani Maria Pia, Messina Sonia, Pane Marika, D'Amico Adele, Colia Giulia, Fanelli Lavinia, Berardinelli Angela, Gardani Alice, Lanzillotta Valentina, D'Ambrosio Paola, Petillo Roberta, Cavallaro Filippo, Frosini Silvia, Bello Luca, Bonfiglio Serena, De Sanctis Roberto, Rolle Enrica, Forcina Nicola, Magri Francesca, Vita Gianluca, Palermo Concetta, Donati Maria Alice, Procopio Elena, Arnoldi Maria Teresa, Baranello Giovanni, Mongini Tiziana, Pini Antonella, Battini Roberta, Pegoraro Elena, Torrente Yvan, Previtali Stefano C, Bruno Claudio, Politano Luisa, Comi Giacomo P, D'Angelo Maria Grazia, Bertini Enrico, Mercuri Eugenio

机构信息

Department of Paediatric Neurology, Catholic University, Rome, Italy.

Biostatistics Unit, Department of Health Sciences, University of Genoa, Genoa, Italy.

出版信息

PLoS One. 2016 Mar 16;11(3):e0151445. doi: 10.1371/journal.pone.0151445. eCollection 2016.

Abstract

BACKGROUND

The role of timed items, and more specifically, of the time to rise from the floor, has been reported as an early prognostic factor for disease progression and loss of ambulation. The aim of our study was to investigate the possible effect of the time to rise from the floor test on the changes observed on the 6MWT over 12 months in a cohort of ambulant Duchenne boys.

SUBJECTS AND METHODS

A total of 487 12-month data points were collected from 215 ambulant Duchenne boys. The age ranged between 5.0 and 20.0 years (mean 8.48 ±2.48 DS).

RESULTS

The results of the time to rise from the floor at baseline ranged from 1.2 to 29.4 seconds in the boys who could perform the test. 49 patients were unable to perform the test at baseline and 87 at 12 month The 6MWT values ranged from 82 to 567 meters at baseline. 3 patients lost the ability to perform the 6mwt at 12 months. The correlation between time to rise from the floor and 6MWT at baseline was high (r = 0.6, p<0.01).

CONCLUSIONS

Both time to rise from the floor and baseline 6MWT were relevant for predicting 6MWT changes in the group above the age of 7 years, with no interaction between the two measures, as the impact of time to rise from the floor on 6MWT change was similar in the patients below and above 350 m. Our results suggest that, time to rise from the floor can be considered an additional important prognostic factor of 12 month changes on the 6MWT and, more generally, of disease progression.

摘要

背景

计时项目的作用,更具体地说,从地面起身的时间,已被报道为疾病进展和行走能力丧失的早期预后因素。我们研究的目的是调查在一组能行走的杜氏肌营养不良男孩中,从地面起身测试时间对12个月内6分钟步行试验(6MWT)所观察到变化的可能影响。

受试者与方法

从215名能行走的杜氏肌营养不良男孩中总共收集了487个12个月的数据点。年龄范围在5.0至20.0岁之间(平均8.48±2.48标准差)。

结果

在能够进行测试的男孩中,基线时从地面起身的时间结果为1.2至29.4秒。49名患者在基线时无法进行测试,12个月时有87名患者无法进行测试。基线时6MWT值范围为82至567米。3名患者在12个月时失去了进行6MWT的能力。基线时从地面起身时间与6MWT之间的相关性很高(r = 0.6,p<0.01)。

结论

对于预测7岁以上组的6MWT变化,从地面起身时间和基线6MWT均具有相关性,且这两种测量方法之间没有相互作用,因为从地面起身时间对6MWT变化的影响在350米以下和以上的患者中相似。我们的结果表明,从地面起身时间可被视为6MWT 12个月变化以及更普遍的疾病进展的另一个重要预后因素。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/77e7/4794120/6a5f460af22b/pone.0151445.g001.jpg

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