van Rumund Anouke, Esselink Rianne A J, Berrevoets-Aerts Marjolein B, Otto Markus, Bloem Bastiaan R, Verbeek Marcel M
Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Center, Nijmegen, The Netherlands.
Radboudumc Center of Expertise for Parkinson & Movement Disorders, Nijmegen, The Netherlands.
NPJ Parkinsons Dis. 2022 Jun 2;8(1):67. doi: 10.1038/s41531-022-00329-4.
Prognosis of patients with parkinsonism varies greatly between the various parkinsonian syndromes. However, it is often difficult to distinguish the different forms, particularly in early stages. We examined predictors of mortality and functional outcome in patients with recent-onset parkinsonism with an initially uncertain diagnosis (n = 156). Patients were recruited between 2003 and 2006, comprehensively investigated, and followed prospectively (up to 15 years, mean 7 years). A final clinical diagnosis was established after follow-up. Independent predictors of mortality were investigated with multivariable Cox regression and combined into a simple prediction model. Model performance to predict 5- and 10-year mortality and functional outcome after 3 years was evaluated and externally validated in a second cohort of 62 patients with parkinsonism with an initially uncertain diagnosis. Ninety-one patients died (58%). Orthostatic hypotension, impaired cognition, abnormal tandem gait, and elevated neurofilament light chain concentration in serum or CSF were associated with mortality. A simple model that combined these factors showed excellent performance for prediction of functional outcome after 3 years and mortality after 5 and 10 years (c-statistic ~0.90 for all models). Model performance was confirmed after external validation: prediction of functional outcome after 3 years (c-statistic 0.89, 95% CI 0.80-0.98) and mortality after 5 years (c-statistic 0.91, 95% CI 0.84-0.99) were comparable to the results in the discovery cohort. These findings help clinicians to estimate a patient's prognosis, irrespective of the specific diagnosis.
帕金森综合征患者的预后在不同的帕金森氏症综合征之间差异很大。然而，区分不同形式往往很困难，尤其是在早期阶段。我们研究了近期发病且最初诊断不明确的帕金森氏症患者（n = 156）的死亡率和功能结局预测因素。患者于2003年至2006年招募，进行了全面调查，并进行前瞻性随访（长达15年，平均7年）。随访后确定最终临床诊断。采用多变量Cox回归研究死亡率的独立预测因素，并将其合并为一个简单的预测模型。对预测3年后5年和10年死亡率及功能结局的模型性能进行评估，并在另一组62例最初诊断不明确的帕金森氏症患者中进行外部验证。91例患者死亡（58%）。体位性低血压、认知障碍、异常串联步态以及血清或脑脊液中神经丝轻链浓度升高与死亡率相关。结合这些因素的简单模型在预测3年后的功能结局以及5年和10年后的死亡率方面表现出色（所有模型的c统计量约为0.90）。外部验证后证实了模型性能：3年后功能结局的预测（c统计量0.89，95%CI 0.80 - 0.98）和5年后死亡率的预测（c统计量0.91，95%CI 0.84 - 0.99）与发现队列中的结果相当。这些发现有助于临床医生评估患者的预后，而无需考虑具体诊断。
