High serum neurofilament light chain normalizes after hematopoietic stem cell transplantation for MS.
Thebault, Simon MD; R. Tessier, Daniel PhD; Lee, Hyunwoo PhD; Bowman, Marjorie MScN; Bar-Or, Amit MD; Arnold, Douglas L. MD; L. Atkins, Harold MD; Tabard-Cossa, Vincent PhD; Freedman, Mark S. MD
Neurology Neuroimmunology & Neuroinflammation.
6(5), September 2019.
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Objective: To evaluate neurofilament light chain (NfL) levels in serum and CSF of patients with aggressive MS pre- and post-treatment with immunoablation followed by autologous hematopoietic stem cell transplantation (IAHSCT) and examine associations with clinical and MRI outcomes.
Methods: Paired serum and CSF in addition to MRI and clinical measures were collected on 23 patients with MS at baseline and 1 and 3 years post-IAHSCT. An additional 33 sera and CSF pairs were taken from noninflammatory neurologic controls. NfL levels were quantitated using the Simoa platform (Quanterix).
Results: Baseline MS NfL levels were significantly elevated relative to controls in serum (p = 0.001) and CSF (p = 0.001). Following IAHSCT, high pretreatment NfL levels significantly reduced in serum (p = 0.0023) and CSF (p = 0.0068) and were not significantly different from controls. Serum and CSF NfL levels highly correlated (r = 0.81, p < 0.0001). Baseline NfL levels were associated with worse pretreatment disease measures (Expanded Disability Status Scale [EDSS], relapses, MRI lesions, and MR spectroscopy (MRS) N-acetylaspartate/creatine). Elevated baseline NfL levels were associated with persistently worse indices of disease burden post-IAHSCT (sustained EDSS progression, cognition, quality of life, T1 and T2 lesion volumes, MRS, and brain atrophy).
Conclusion: These data substantiate that serum and CSF NfL levels reflect disease severity and treatment response in patients with MS and may therefore be a useful biomarker. Baseline serum levels associated with markers of pretreatment disease severity and post-treatment outcomes.
Classification of evidence: This study provides Class II evidence that for patients with aggressive MS, serum NfL levels are associated with disease severity.
(C) 2019 American Academy of Neurology