Department of Neuromuscular Diseases, University College London Queen Square Institute of Neurology, London, UK.
Department of Neurosciences, Central Clinical School, Monash University, Alfred Centre, Melbourne, Victoria, Australia.
Eur J Neurol. 2022 Nov;29(11):3347-3357. doi: 10.1111/ene.15496. Epub 2022 Jul 25.
This study was undertaken to explore associations between plasma neurofilament light chain (pNfL) concentration (pg/ml) and disease activity in patients with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) and examine the usefulness of pNfL concentrations in determining disease remission.
We examined pNfL concentrations in treatment-naïve CIDP patients (n = 10) before and after intravenous immunoglobulin (IVIg) induction treatment, in pNfL concentrations in patients on maintenance IVIg treatment who had stable (n = 15) versus unstable disease (n = 9), and in clinically stable IVIg-treated patients (n = 10) in whom we suspended IVIg to determine disease activity and ongoing need for maintenance IVIg. pNfL concentrations in an age-matched healthy control group were measured for comparison.
Among treatment-naïve patients, pNfL concentration was higher in patients before IVIg treatment than healthy controls and subsequently reduced to be comparable to control group values after IVIg induction. Among CIDP patients on IVIg treatment, pNfL concentration was significantly higher in unstable patients than stable patients. A pNFL concentration > 16.6 pg/ml distinguished unstable treated CIDP from stable treated CIDP (sensitivity = 86.7%, specificity = 66.7%, area under receiver operating characteristic curve = 0.73). Among the treatment withdrawal group, there was a statistically significant correlation between pNfL concentration at time of IVIg withdrawal and the likelihood of relapse (r = 0.72, p < 0.05), suggesting an association of higher pNfL concentration with active disease.
pNfL concentrations may be a sensitive, clinically useful biomarker in assessing subclinical disease activity.
本研究旨在探讨慢性炎症性脱髓鞘性多发性神经病（CIDP）患者血浆神经丝轻链（pNfL）浓度（pg/ml）与疾病活动之间的关系，并探讨 pNfL 浓度在确定疾病缓解中的作用。
我们检测了 10 例未经治疗的 CIDP 患者静脉注射免疫球蛋白（IVIg）诱导治疗前后的 pNfL 浓度，9 例病情不稳定与 15 例病情稳定的接受 IVIg 维持治疗的患者的 pNfL 浓度，以及 10 例临床稳定但暂停 IVIg 以确定疾病活动和持续需要维持 IVIg 的患者的 pNfL 浓度。同时测量了年龄匹配的健康对照组的 pNfL 浓度作为比较。
在未经治疗的患者中，IVIg 治疗前患者的 pNfL 浓度高于健康对照组，随后在 IVIg 诱导后降低至与对照组值相当。在接受 IVIg 治疗的 CIDP 患者中，病情不稳定的患者的 pNfL 浓度明显高于病情稳定的患者。pNfL 浓度 >16.6pg/ml 可将不稳定的 CIDP 与稳定的 CIDP 相区分（敏感性为 86.7%，特异性为 66.7%，ROC 曲线下面积为 0.73）。在停药组中，IVIg 停药时的 pNfL 浓度与复发的可能性呈统计学显著相关（r=0.72，p <0.05），提示较高的 pNfL 浓度与活动性疾病相关。
pNfL 浓度可能是评估亚临床疾病活动的一种敏感、具有临床意义的生物标志物。
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