From the Department of Pediatrics and Adolescent Medicine (M.-C.R., P.H., W.S., J.G.), Division of Pediatric Neurology, University Medical Centre Göttingen, Georg August University Göttingen, Germany; Clinical Trial Unit (P.B.), Department of Clinical Research, University Hospital Basel, University of Basel; Medical Image Analysis Centre Basel (MIAC AG) (J.W., E.R.); Department of Biomedical Engineering (J.W.), University Basel; Neurologic Clinic and Policlinic (Z.M., C.B., D.L., J.K.), Departments of Medicine, Biomedicine and Clinical Research, University Hospital Basel, University of Basel; Novartis Pharma AG (H.K., D.T.), Basel, Switzerland; and Institute of Neuropathology (W.B.), University Medical Centre Göttingen, Georg August University Göttingen, Germany.
Neurol Neuroimmunol Neuroinflamm. 2020 May 13;7(4). doi: 10.1212/NXI.0000000000000749. Print 2020 Jul.
To investigate serum neurofilament light chain (sNfL) as a potential biomarker for disease activity and treatment response in pediatric patients with multiple sclerosis (MS).
In this retrospective cohort study, sNfL levels were measured in a pediatric MS cohort (n = 55, follow-up 12-105 months) and in a non-neurologic pediatric control cohort (n = 301) using a high-sensitivity single-molecule array assay. Association of sNfL levels and treatment and clinical and MRI parameters were calculated.
Untreated patients had higher sNfL levels than controls (median 19.0 vs 4.6 pg/mL; CI [4.732, 6.911]), < 0.001). sNfL levels were significantly associated with MRI activity (+9.1% per contrast-enhancing lesion, CI [1.045, 1.138], < 0.001; +0.6% per T2-weighted lesion, CI [1.001, 1.010], = 0.015). Higher values were associated with a relapse <90 days ago (+51.1%; CI [1.184, 1.929], < 0.001) and a higher Expanded Disability Status Scale score (CI [1.001, 1.240], = 0.048). In patients treated with interferon beta-1a/b (n = 27), sNfL levels declined from 14.7 to 7.9 pg/mL after 6 ± 2 months (CI [0.339, 0.603], < 0.001). Patients with insufficient control of clinical or MRI disease activity under treatment with interferon beta-1a/b or glatiramer acetate who switched to fingolimod (n = 18) showed a reduction of sNfL levels from 16.5 to 10.0 pg/mL 6 ± 2 months after switch (CI [0.481, 0.701], < 0.001).
sNfL is a useful biomarker for monitoring disease activity and treatment response in pediatric MS. It is most likely helpful to predict disease severity and to guide treatment decisions in patients with pediatric MS. This study provides Class III evidence that sNfL levels are associated with disease activity in pediatric MS.
探讨血清神经丝轻链（sNfL）作为多发性硬化症（MS）儿科患者疾病活动和治疗反应的潜在生物标志物。
在这项回顾性队列研究中，使用高灵敏度单分子阵列法测量了 55 名儿科 MS 队列（随访 12-105 个月）和 301 名非神经儿科对照组患者的 sNfL 水平。计算 sNfL 水平与治疗以及临床和 MRI 参数的相关性。
未经治疗的患者 sNfL 水平高于对照组（中位数 19.0 比 4.6 pg/ml；CI [4.732, 6.911]， < 0.001）。sNfL 水平与 MRI 活动性显著相关（增强病变每增加 1 个，增加 9.1%，CI [1.045, 1.138]， < 0.001；T2 加权病变每增加 1 个，增加 0.6%，CI [1.001, 1.010]， = 0.015）。更高的值与 90 天内的复发（+51.1%；CI [1.184, 1.929]， < 0.001）和更高的扩展残疾状况量表评分（CI [1.001, 1.240]， = 0.048）相关。在接受干扰素β-1a/b（n=27）治疗的患者中，治疗 6±2 个月后 sNfL 水平从 14.7 降至 7.9 pg/ml（CI [0.339, 0.603]， < 0.001）。在接受干扰素β-1a/b 或聚乙二醇干扰素治疗但疾病活动度控制不佳的患者中，有 18 例患者转为使用芬戈莫德，转换后 6±2 个月 sNfL 水平从 16.5 降至 10.0 pg/ml（CI [0.481, 0.701]， < 0.001）。
sNfL 是监测儿科 MS 疾病活动和治疗反应的有用生物标志物。它很可能有助于预测疾病严重程度，并指导儿科 MS 患者的治疗决策。本研究提供了 III 级证据，表明 sNfL 水平与儿科 MS 的疾病活动有关。
Zotero 插件
