Phenylalanine blood levels and clinical outcomes in phenylketonuria: A systematic literature review and meta-analysis
作者: Susan E. WaisbrenKay NoelKyle FahrbachCatherine CellaDiana FrameAlex DorenbaumHarvey Levy
作者单位: 1Children’s Hospital Boston, 1 Autumn Street, Room 525, Boston, MA 02115, USA
2United BioSource Corporation, 101 Station Landing, Medford, MA 02155, USA
3BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA
刊名: Molecular Genetics and Metabolism, 2007, Vol.92 (1), pp.63-70
来源数据库: Elsevier Journal
DOI: 10.1016/j.ymgme.2007.05.006
关键词: BiomarkerPredictive biomarkerIntelligence quotient
英文摘要: Abstract(#br)Blood phenylalanine (Phe) levels provide a practical and reliable method for the diagnosis and monitoring of metabolic status in patients with phenylketonuria (PKU). To assess the reliability of blood Phe levels as a predictive biomarker of clinical outcomes in the development of treatments for PKU, a systematic literature review and meta-analysis of published trials of PKU, which included Phe level and neurological and dietary compliance outcome measures, was conducted. Within-study correlations between Phe level and intelligence quotient (IQ) were extracted from 40 studies. Significant, proportional correlations were found during critical periods (from 0 to 12 years of age) for early-treated patients with PKU ( r =−0.35; 95% confidence interval [CI]:...
全文获取路径: Elsevier  (合作)
影响因子:2.834 (2012)

  • literature 文献
  • quotient 
  • systematic 有系统的
  • blood 血液
  • review 复审
  • analysis 分析
  • clinical 临床的
  • biomarker 生物标志化合物