Department of Neurodegeneration, Hertie Institute for Clinical Brain Research, Tübingen, Germany.
Eur J Neurol. 2013 Jan;20(1):102-8. doi: 10.1111/j.1468-1331.2012.03798.x. Epub 2012 Aug 1.
Screening batteries to narrow down a target-at-risk population are essential for trials testing neuroprotective compounds aiming to delay or prevent onset of Parkinson's disease (PD).
The PRIPS study focuses on early detection of incident PD in 1847 at baseline PD-free subjects, and assessed age, male gender, positive family history, hyposmia, subtle motor impairment and enlarged substantia nigra hyperechogenicity (SN+).
After 3 years follow-up 11 subjects had developed PD. In this analysis of the secondary outcome parameters, sensitivity and specificity of baseline markers for incident PD were calculated in 1352 subjects with complete datasets (10 PD patients). The best approach for prediction of incident PD comprised three steps: (i) prescreening for age, (ii) primary screening for positive family history and/or hyposmia, and (iii) secondary screening for SN+.
With this approach, one out of 16 positively screened participants developed PD compared to one out of 135 in the original cohort. This corresponds to a sensitivity of 80.0%, a specificity of 90.6% and a positive predictive value of 6.1%. These values are higher than for any single screening instrument but still too low for a feasible and cost-effective screening strategy which might require longer follow-up intervals and application of additional instruments.
筛选能够缩小目标风险人群的检测工具，对于旨在延缓或预防帕金森病（PD）发病的神经保护化合物的临床试验至关重要。
PRIPS 研究专注于在基线时无 PD 的 1847 名受试者中早期发现新发 PD，并评估了年龄、男性、阳性家族史、嗅觉减退、轻微运动障碍和黑质超声高回声（SN+）。
在 3 年的随访后，有 11 名受试者发展为 PD。在对次要结局参数的分析中，对 1352 名具有完整数据集（10 名 PD 患者）的基线标志物进行了新发 PD 的敏感性和特异性计算。预测新发 PD 的最佳方法包括三个步骤：（i）年龄预筛查，（ii）阳性家族史和/或嗅觉减退的主要筛查，以及（iii）SN+的次要筛查。
采用这种方法，与原始队列中的 135 人相比，16 名经阳性筛选的参与者中有 1 人发展为 PD。这对应于 80.0%的敏感性、90.6%的特异性和 6.1%的阳性预测值。这些值高于任何单一筛查工具，但仍然太低，无法实现可行且具有成本效益的筛查策略，这可能需要更长的随访间隔和应用其他工具。
