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Introduction <p>Guillain–Barré syndrome (GBS) is an acute immune-mediated disorder of the peripheral nervous system, marked by rapid onset of neurological symptoms. Despite progress in understanding the etiology and improving clinical management, no validated biomarkers are currently available to predict disease severity or treatment response during the acute phase. This meta-analysis aims to evaluate the role of serum neurofilament light chain (NfL) as a biomarker of acute disease activity and prognostic outcomes in GBS.</p> Methods <p>A systematic review and meta-analysis was conducted using PubMed, Scopus, and Cochrane Library databases to identify studies assessing NfL levels in patients with GBS. In addition, we included data from our own cohort of patients with GBS–whose NfL levels were measured at disease onset—and from healthy controls. The primary outcome was the difference in NfL levels—both in serum and cerebrospinal fluid (CSF)—between patients with GBS and controls. Secondary outcomes included the correlations between acute-phase NfL levels, clinical severity at admission as measured by the Guillain–Barré Disability Scale (GBDS) or the Hughes Functional Scale (HFS), and long-term outcomes such as the inability to walk or run 1&#xa0;year after disease onset.</p> Results <p>In this meta-analysis of nine studies, which also included data from our cohort, serum NfL levels were significantly higher in patients with GBS compared with controls (mean difference 143.17&#xa0;pg/mL, 95%&#xa0;CI 67.7–218.6; <i>p</i> &lt; 0.01; <i>I</i><sup>2</sup> = 83%). In contrast, the difference in CSF NfL levels only approached statistical significance (mean difference 2091.1&#xa0;pg/mL, 95%&#xa0;CI 171.2–4353.4; <i>p</i> = 0.07, <i>I</i><sup>2</sup> = 92.1%). These findings were corroborated in our cohort, where median serum NfL concentrations were markedly higher in patients with GBS compared to controls (97&#xa0;pg/mL, IQR 79–194 vs. 15&#xa0;pg/mL, IQR 13–20; <i>p</i> &lt; 0.05, Wilcoxon rank-sum test). Serum NfL levels were higher in patients with the acute motor axonal neuropathy (AMAN) compared to those with acute inflammatory demyelinating polyneuropathy (AIDP) (MD 531.9&#xa0;pg/mL, 95%&#xa0;CI 32.8–1031.01; <i>I</i><sup>2</sup> = 81.1%; <i>p</i> = 0.04). Moreover, NfL levels positively correlated with disease severity at admission (<i>r</i> = 0.38; <i>p</i> &lt; 0.001) and poor long-term outcomes (OR 3.74, 95%&#xa0;CI 1.05–13.37; <i>p</i> &lt; 0.001).</p> Conclusion <p>Serum NfL is a promising biomarker for early diagnosis and prognosis in GBS and may support risk stratification at hospital admission.</p>

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Neurofilament Light Chain Levels as Diagnostic and Prognostic Biomarkers in Guillain–Barré Syndrome: An Updated Systematic Review and Meta-Analysis

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  • 发表日期：

    2025年9月21日

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  • Giovanni Siconolfi,
  • Francesca Vitali,
  • Maria Ausilia Sciarrone,
  • Valeria Guglielmino,
  • Guido Primiano,
  • Marco Luigetti

摘要

Introduction

Guillain–Barré syndrome (GBS) is an acute immune-mediated disorder of the peripheral nervous system, marked by rapid onset of neurological symptoms. Despite progress in understanding the etiology and improving clinical management, no validated biomarkers are currently available to predict disease severity or treatment response during the acute phase. This meta-analysis aims to evaluate the role of serum neurofilament light chain (NfL) as a biomarker of acute disease activity and prognostic outcomes in GBS.

Methods

A systematic review and meta-analysis was conducted using PubMed, Scopus, and Cochrane Library databases to identify studies assessing NfL levels in patients with GBS. In addition, we included data from our own cohort of patients with GBS–whose NfL levels were measured at disease onset—and from healthy controls. The primary outcome was the difference in NfL levels—both in serum and cerebrospinal fluid (CSF)—between patients with GBS and controls. Secondary outcomes included the correlations between acute-phase NfL levels, clinical severity at admission as measured by the Guillain–Barré Disability Scale (GBDS) or the Hughes Functional Scale (HFS), and long-term outcomes such as the inability to walk or run 1 year after disease onset.

Results

In this meta-analysis of nine studies, which also included data from our cohort, serum NfL levels were significantly higher in patients with GBS compared with controls (mean difference 143.17 pg/mL, 95% CI 67.7–218.6; p < 0.01; I2 = 83%). In contrast, the difference in CSF NfL levels only approached statistical significance (mean difference 2091.1 pg/mL, 95% CI 171.2–4353.4; p = 0.07, I2 = 92.1%). These findings were corroborated in our cohort, where median serum NfL concentrations were markedly higher in patients with GBS compared to controls (97 pg/mL, IQR 79–194 vs. 15 pg/mL, IQR 13–20; p < 0.05, Wilcoxon rank-sum test). Serum NfL levels were higher in patients with the acute motor axonal neuropathy (AMAN) compared to those with acute inflammatory demyelinating polyneuropathy (AIDP) (MD 531.9 pg/mL, 95% CI 32.8–1031.01; I2 = 81.1%; p = 0.04). Moreover, NfL levels positively correlated with disease severity at admission (r = 0.38; p < 0.001) and poor long-term outcomes (OR 3.74, 95% CI 1.05–13.37; p < 0.001).

Conclusion

Serum NfL is a promising biomarker for early diagnosis and prognosis in GBS and may support risk stratification at hospital admission.