Klimas Rafael, Kohle Felix, Horstkemper Lea, Benkert Pascal, Rehm Adriana, Seibert Aylin, Riesner Moritz, Sgodzai Melissa, Grüter Thomas, Rilke Niklas, Gisevius Barbara, Beyer Léon, Gerwert Klaus, Kuhle Jens, Schroeter Michael, Lehmann Helmar C, Gold Ralf, Fisse Anna Lena, Motte Jeremias, Pitarokoili Kalliopi
Department of Neurology, St. Josef-Hospital, Ruhr-University Bochum, Germany.
Immunmediated Neuropathies Biobank (INHIBIT), Ruhr-University Bochum, Germany.
Neurol Neuroimmunol Neuroinflamm. 2025 Jul;12(4):e200419. doi: 10.1212/NXI.0000000000200419. Epub 2025 Jun 5.
The aim of this study was to characterize serum neurofilament light chain (sNFL) levels in a large cohort of patients with autoimmune neuropathies to provide every-day clinical practice recommendations.
In this retrospective cohort study, we recruited 191 patients with immune-mediated neuropathies from 2 referral centers. sNFL was measured using the Simoa NF-light kit (Quanterix), and age-corrected and BMI-corrected z-scores (zNFL) were calculated. Clinical data were correlated with zNFL and adjusted for different disease subsets. A receiver operator characteristic analysis was performed. Treatments and longitudinal disease course of patients with typical chronic inflammatory demyelinating polyneuropathy (CIDP) in early disease stage were analyzed.
One hundred ten patients had typical CIDP, and 67 had atypical CIDP. Fourteen patients had other immune neuropathies. zNFL of all patients correlated significantly with the Inflammatory Neuropathy Cause and Treatment Scale-overall disability sum score ( = 0.160), Medical Research Council Scale for Muscle Strength score ( = -0.242), modified Rankin Scale score ( = 0.151), and distal tibial compound muscle action potential ( = -0.151). The correlations remained only in the cohort of typical CIDP. zNFL >2 within the first 24 months of illness differentiated patients with atypical and typical CIDP with a sensitivity of 93%. Patients with early-stage typical CIDP with zNFL >2 (n = 9) presented with the most severe manifestation and did not respond to first-line ( < 0.0001) but to second-line treatments.
We established sNFL as a promising biomarker for assessing disease activity in patients with typical CIDP. Elevated zNFL in early-stage typical CIDP indicate severe inflammatory-mediated axonal damage that requires aggressive immunotherapy.
本研究旨在描述一大群自身免疫性神经病患者的血清神经丝轻链（sNFL）水平，以提供日常临床实践建议。
在这项回顾性队列研究中，我们从2个转诊中心招募了191例免疫介导性神经病患者。使用Simoa NF-light试剂盒（Quanterix）测量sNFL，并计算年龄校正和体重指数校正的z分数（zNFL）。临床数据与zNFL相关，并针对不同疾病亚组进行调整。进行了受试者工作特征分析。分析了早期典型慢性炎性脱髓鞘性多发性神经病（CIDP）患者的治疗及疾病纵向病程。
110例患者患有典型CIDP，67例患有非典型CIDP。14例患者患有其他免疫性神经病。所有患者的zNFL与炎性神经病病因及治疗量表-总体残疾总分（r = 0.160）、医学研究委员会肌力量表评分（r = -0.242）、改良Rankin量表评分（r = 0.151）以及胫神经远端复合肌肉动作电位（r = -0.151）均显著相关。这些相关性仅在典型CIDP队列中存在。发病后24个月内zNFL>2可区分非典型和典型CIDP患者，敏感性为93%。zNFL>2的早期典型CIDP患者（n = 9）表现出最严重的症状，对一线治疗无反应（P<0.0001），但对二线治疗有反应。
我们确定sNFL是评估典型CIDP患者疾病活动度的一个有前景的生物标志物。早期典型CIDP中zNFL升高表明存在严重的炎症介导的轴索损伤，需要积极的免疫治疗。
