Fibroblast growth factor 21 is a sensitive biomarker of mitochondrial disease

Fibroblast growth factor 21 is a sensitive biomarker of mitochondrial disease
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DOI:
10.1212/01.wnl.0000436068.43384.ef
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发表时间:
2013-11-19
期刊:
影响因子:
9.9
通讯作者:
Sue, Carolyn M.
Sue, Carolyn M.
中科院分区:
医学1区
文献类型:
--
作者:
Davis, Ryan L.;Liang, Christina;Sue, Carolyn M.

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目的:前瞻性地确定血清成纤维细胞生长因子21(FGF-21)作为线粒体疾病生物标志物的可靠性和有效性,该生物标志物在来自专科初级保健和三级转诊诊所的线粒体疾病成人横断面队列中进行。我们招募了140名受试者,包括54名线粒体疾病成人患者,20名非线粒体神经肌肉疾病患者和66名对照受试者,2011年11月至2012年10月期间。我们比较了血清FGF-21浓度与经典生物标志物,血清肌酸激酶,乳酸,丙酮酸和乳酸丙酮酸比,以确定其作为线粒体疾病生物标志物的有效性和可靠性。我们确定的敏感性,比值比(OR),和整体的可靠性FGF-21作为线粒体疾病的标志物使用statistical analysis.Results:中值血清FGF-21浓度显着升高线粒体疾病患者和所有实验组之间的差异显着。与其他生物标志物相比,FGF-21显示出显著更高的诊断OR(45.7 [ 95%置信区间5 12.6-166.5],p,0.0001),根据灵敏度和受试者工作特征曲线分析,FGF-21是疾病的最佳预测因子。多变量logistic回归分析控制潜在的混杂因素后,FGF-21是唯一的测量参数能够预测线粒体diseases.Conclusion:这项前瞻性研究建立了血清FGF-21水平作为线粒体疾病的敏感生物标志物,并表明,他们是最好的预测这种疾病相比,血清水平的经典指标:肌酸激酶、乳酸盐、丙酮酸盐以及乳酸盐与丙酮酸盐的比率。
Objective: To prospectively determine the reliability and validity of serum fibroblast growth factor 21 (FGF-21) as a biomarker for mitochondrial disease in a cross-sectional cohort of adults with mitochondrial disease from a specialist primary care and tertiary referral clinic.Methods: We recruited 140 subjects, including 54 adults with mitochondrial disease, 20 patients with nonmitochondrial neuromuscular disease, and 66 control subjects, between November 2011 and October 2012. We compared serum FGF-21 concentrations to classical biomarkers, serum creatine kinase, lactate, pyruvate, and lactate to pyruvate ratio, to determine its validity and reliability as a biomarker of mitochondrial disease. We determined the sensitivity, odds ratio (OR), and overall reliability of FGF-21 as a marker of mitochondrial disease using statistical analyses.Results: Median serum FGF-21 concentrations were significantly elevated in patients with mitochondrial disease and differed significantly between all experimental groups. FGF-21 showed a markedly higher diagnostic OR (45.7 [ 95% confidence interval 5 12.6-166.5], p, 0.0001) when compared to other biomarkers and was the best predictor of disease according to sensitivity and receiver operating characteristic curve analysis. After multivariate logistic regression analysis controlling for potential confounders, FGF-21 was the only measured parameter capable of predicting mitochondrial disease.Conclusion: This prospective study establishes serum FGF-21 levels as a sensitive biomarker of mitochondrial disease and demonstrates that they are the best predictor of this disorder when compared to serum levels of classical indicators: creatine kinase, lactate, pyruvate, and the lactate to pyruvate ratio.