Data from: Blood NfL: a biomarker for disease severity and progression in Parkinson’s disease

Objective: To examine whether plasma neurofilament light chain (NfL)
levels were associated with motor and cognitive progression in Parkinson’s
disease (PD). Methods: This prospective follow-up study enrolled 178
participants, including 116 with PD, 22 with multiple system atrophy
(MSA), and 40 healthy controls. We measured plasma NfL levels with
electrochemiluminescence immunoassay. Patients with PD received
evaluations of motor and cognition, at baseline and at a mean follow-up
interval of 3 years. Changes in the unified Parkinson's disease
rating scale (UPDRS) part III motor score and Mini-Mental State
Examination (MMSE) score were used to assess motor and cognition
progression. Results: Plasma fL levels were significantly higher in MSA
than in PD and healthy groups (35.8±6.2 pg/ml, 17.6±2.8 pg/ml, and
10.6±2.3 pg/ml, respectively; P<0.001). In the PD group, NfL levels
were significantly elevated in patients with advanced Hoehn-Yahr (H-Y)
stage and patients with dementia (PDD) (P<0.001). NfL levels were
modestly correlated with UPDRS part III scores (r=0.42, 95% CI: 0.46-0.56,
P<0.001). After a mean follow-up of 3.4±1.2 years, a Cox regression
analysis adjusted for age, sex, disease duration and baseline motor or
cognitive status showed that higher baseline NfL levels were associated
with higher risks for either motor or cognition progression (P=0.029 and
P=0.015, respectively). Conclusions: Plasma NfL levels correlated with
disease severity and progression in terms of both motor and cognitive
functions in PD. Classification of evidence: This study provides Class III
evidence that plasma NfL levels distinguish PD and MSA, and is a surrogate
biomarker for PD progression.

研究目的：探讨血浆神经丝轻链（plasma neurofilament light chain, NfL）水平与帕金森病（Parkinson’s disease, PD）患者运动及认知功能进展的相关性。
研究方法：本前瞻性随访研究共纳入178名受试者，包括116名帕金森病患者、22名多系统萎缩（multiple system atrophy, MSA）患者及40名健康对照者。采用电化学发光免疫分析法（electrochemiluminescence immunoassay）检测血浆NfL水平。帕金森病患者于基线及平均随访3年时接受运动及认知功能评估。以统一帕金森病评定量表（unified Parkinson's disease rating scale, UPDRS）第三部分运动评分及简易精神状态检查（Mini-Mental State Examination, MMSE）评分的变化作为运动及认知功能进展的评估指标。
研究结果：多系统萎缩患者的血浆NfL水平显著高于帕金森病患者及健康对照者（分别为35.8±6.2 pg/ml、17.6±2.8 pg/ml及10.6±2.3 pg/ml；P<0.001）。在帕金森病患者亚组中，霍恩-雅尔（Hoehn-Yahr, H-Y）分期较晚的患者及帕金森痴呆（Parkinson’s disease dementia, PDD）患者的血浆NfL水平显著升高（P<0.001）。血浆NfL水平与UPDRS第三部分运动评分呈轻度相关（r=0.42，95%置信区间：0.46~0.56，P<0.001）。在平均随访3.4±1.2年后，校正年龄、性别、病程及基线运动/认知状态的Cox回归分析显示，基线较高的血浆NfL水平与运动及认知功能进展的更高风险均显著相关（分别为P=0.029及P=0.015）。
研究结论：血浆NfL水平与帕金森病患者的疾病严重程度及运动、认知功能进展显著相关。
证据分级：本研究提供了Ⅲ级证据，表明血浆NfL水平可区分帕金森病与多系统萎缩，且可作为帕金森病疾病进展的替代生物标志物。