Department of Neurology, University of Rochester, Rochester, New York 14620, USA.
Mov Disord. 2010;25 Suppl 1(Suppl 1):S152-4. doi: 10.1002/mds.22790.
The focus on disease-modifying treatments and cures for Parkinson's disease (PD) has raised expectations for quantum leaps and overshadowed incremental gains that have been slowly achieved. Large multi-center clinical trials such as DATATOP and PRECEPT keep on generating new knowledge that is relevant to clinical care as well as experimental therapeutics. The largely unforeseen relationship between circulating uric acid and the occurrence and progression of PD was developed and confirmed in these clinical trials. Systematic follow-up of clinical trial cohorts after conclusion of the interventional phase provides added value that continues to inform about natural history, state and trait biomarkers, and genotype-phenotype relationships. These efforts are enhanced by data mining, public reporting, and timely sharing of data and biological samples.
对帕金森病(PD)的疾病修饰治疗和治愈方法的关注,提高了人们对取得巨大突破的期望,而忽视了那些缓慢取得的渐进性进展。DATATOP 和 PRECEPT 等大型多中心临床试验不断产生新的知识,这些知识与临床护理以及实验治疗都相关。在这些临床试验中,发现并证实了循环尿酸与 PD 的发生和进展之间的这种出乎意料的关系。在干预阶段结束后对临床试验队列进行系统的随访提供了额外的价值,这些价值持续提供关于自然史、状态和特征生物标志物以及基因型-表型关系的信息。通过数据挖掘、公开报告以及及时共享数据和生物样本,这些努力得到了加强。