RT期刊文章SR电子T1纵向评估肌酸激酶、肌酸和肌酐比和肌肉生长抑制素监测生物标志物在贝克肌营养不良摩根富林明神经病学神经学乔FD Lippincott Williams &威尔金斯SP e975 O首页P e984 10.1212 / WNL。100签证官0000000000201609是9 A1 Nienke m·范·德·A1沙滩Koeks A1 Mirko Signorelli A1尼莎Verwey A1莫里斯Overzier A1 A1他乡巴佬a Gautam Sajeev A1詹姆斯Signorovitch A1也好Ricotti A1 Jan Verschuuren A1克里斯蒂布朗A1 Pietro Spitali A1埃里克·h·尼克年2023 UL //www.ez-admanager.com/content/100/9/e975.abstract AB背景和目标的减缓疾病恶化和可变贝克尔肌肉萎缩症首页(BMD)敦促生物标记的发展促进临床试验。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)使用液体chromatography-tandem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,p < 0.001)。CK与年龄呈负关联(p = 0.0002),但不是与病人的相关性能。 Cr/Crn and myostatin correlated moderately with the average annual change of the 6MWT (rho = −0.532 and 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