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帕金森病的 CamPaIGN 研究:基于发病人群的队列研究的 10 年展望。

The CamPaIGN study of Parkinson's disease: 10-year outlook in an incident population-based cohort.

机构信息

John Van Geest Centre for Brain Repair, Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK.

出版信息

J Neurol Neurosurg Psychiatry. 2013 Nov;84(11):1258-64. doi: 10.1136/jnnp-2013-305277. Epub 2013 Jun 18.

Abstract

BACKGROUND

Prognosis in Parkinson's disease (PD) remains poorly understood due to a lack of unbiased data on the natural history of treated PD. The CamPaIGN study has been the first to prospectively track disease evolution from diagnosis in an unselected population-representative incident cohort. We now report the 10-year follow-up data, focusing on three key irreversible milestones: postural instability (Hoehn and Yahr 3), dementia and death.

METHODS

The cohort was collected between December 2000 and 2002. Those meeting diagnostic criteria (n=142) were followed-up until 1 January 2012. Clinical, neuropsychological and genetic testing were performed. Progression to key milestones was evaluated using Kaplan-Meier and Cox regression survival analyses.

RESULTS

At 10 years, 55% had died, 68% had postural instability and 46% dementia. 23% had a good outcome at 10 years (surviving free of dementia/postural instability). Death rate was comparable with the UK population (standardised mortality ratio 1.29 (0.97-1.61)). Death certificates indicated PD was a substantial contributor in only 20%, with pneumonia being the commonest cause of death. Age, non-tremor-dominant motor phenotype and comorbidity predicted earlier postural instability. Baseline predictors of dementia were age, motor impairment, 'posterior-cortical' cognitive deficits and MAPT genotype.

CONCLUSIONS

(1) outlook in PD is heterogeneous, with most dying or developing dementia or postural instability by 10 years from diagnosis, but a quarter still doing well, with preserved mobility and intact cognition; (2) death is not directly related to PD in the majority; (3) baseline clinical and genetic variables are predictive of outcome and may be helpful in selecting patients for clinical trials.

摘要

背景

由于缺乏关于治疗帕金森病(PD)自然史的无偏数据,PD 的预后仍然难以理解。CamPaIGN 研究是第一个前瞻性地从未选择的人群代表性发病队列中跟踪疾病演变的研究。我们现在报告了 10 年的随访数据,重点关注三个关键的不可逆里程碑:姿势不稳(Hoehn 和 Yahr 3 期)、痴呆和死亡。

方法

该队列于 2000 年 12 月至 2002 年期间收集。符合诊断标准的患者(n=142)随访至 2012 年 1 月 1 日。进行临床、神经心理学和基因检测。使用 Kaplan-Meier 和 Cox 回归生存分析评估进展至关键里程碑的情况。

结果

10 年后,55%的患者死亡,68%的患者出现姿势不稳,46%的患者出现痴呆。23%的患者在 10 年后有良好的预后(免于痴呆/姿势不稳)。死亡率与英国人口相当(标准化死亡率比为 1.29(0.97-1.61))。只有 20%的死亡证明表明 PD 是主要原因,肺炎是最常见的死亡原因。年龄、非震颤主导型运动表型和合并症预测更早出现姿势不稳。痴呆的基线预测因素包括年龄、运动障碍、“皮质后”认知缺陷和 MAPT 基因型。

结论

(1)PD 的预后是异质的,大多数患者在诊断后 10 年内死亡或出现痴呆或姿势不稳,但仍有四分之一的患者预后良好,保持运动能力和完整的认知能力;(2)大多数患者的死亡与 PD 无直接关系;(3)基线临床和遗传变量可预测结局,可能有助于为临床试验选择患者。

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