@article {van de Veldee975作者= {Nienke m·范·德·和咱{\ " \我}da Koeks Mirko Signorelli和尼莎Verwey莫里斯Overzier和他乡巴佬a Gautam Sajeev和詹姆斯Signorovitch瓦Ricotti和Jan Verschuuren克里斯蒂布朗和Pietro Spitali和埃里克·h·尼克},title ={纵向评估肌酸激酶、肌酸/ Creatinineratio,和肌肉生长抑制素作为监测生物标志物在贝克肌营养不良},体积={100}={9},页面= {e975——e984} = {2023}, doi = {10.1212 / WNL。出版商0000000000201609}= {Wolters Kluwer健康,公司代表美国神经病学学会},文摘={背景和目标的减缓疾病恶化和可变贝克尔肌肉萎缩症(BMD)敦促生物标记的发展促进首页临床试验。我们探索的变化3 muscle-enriched BMD患者的血清生物标志物在四年时间和研究协会与疾病严重程度,疾病进展和BMD的肌营养不良蛋白水平。方法定量测定肌酸激酶(CK)使用国际临床化学联合会的参考方法,肌酸/ creatinineratio (Cr / Crn)使用液相色谱{\ textendash}串联质谱,和肌肉生长抑制素在血清ELISA和评估功能性能动态评估使用北极星(NSAA), 10米运行速度(TMRv), 6分钟步行试验(6 mwt)、用力肺活量4潜在自然历史研究。胫骨前肌肌营养不良蛋白水平的量化使用毛细管西方免疫测定。生物标记物之间的关系、年龄、功能性能,年平均变化,预测的并发使用线性混合模型功能性能进行了分析。结果34例包括达106人次。8个病人nonambulant基线。Cr / Crn和肌肉生长抑制素非常耐心具体(组内相关系数= 0.960)。Cr / Crn强烈负相关,而肌肉生长抑制素是强烈与NSAA呈正相关,TMRv 6 mwt (Cr / Crnρ= -0.869 - -0.801和肌肉生长抑制素ρ= 0.792 - 0.842,所有p < 0.001)。CK与年龄呈负关联(p = 0.0002),但不能与患者{\ textquoteright}相关性能。Cr / Crn和肌肉生长抑制素相关的适度的年均变化6 mwt(ρ= -0.532和0.555,p = 0.02)。 Dystrophin levels did not correlate with the selected biomarkers nor with performance. Cr/Crn, myostatin, and age could explain up to 75\% of the variance of concurrent functional performance of the NSAA, TMRv, and 6MWT.Discussion Both Cr/Crn and myostatin could potentially serve as monitoring biomarkers in BMD, as higher Cr/Crn and lower myostatin were associated with lower motor performance and predictive of concurrent functional performance when combined with age. Future studies are needed to more precisely determine the context of use of these biomarkers.6MWT=6-Minute Walking Test; ADP=adenosine diphosphate; ATP=adenosine triphosphate; BMD=Becker muscular dystrophy; CK=creatine kinase; Cr/Crn=creatine/creatinineratio; CV=coefficient of variation; DMD=Duchenne muscular dystrophy; FVC=forced vital capacity; ICC=intraclass correlation coefficient; NSAA=North Star Ambulatory Assessment; TA=tibialis anterior; TMRv=10-meter run velocity}, issn = {0028-3878}, URL = {//www.ez-admanager.com/content/100/9/e975}, eprint = {//www.ez-admanager.com/content/100/9/e975.full.pdf}, journal = {Neurology} }