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Review
. 2021 Oct;18(4):2351-2367.
doi: 10.1007/s13311-021-01136-0. Epub 2021 Oct 18.

Fluid Biomarkers for Monitoring Structural Changes in Polyneuropathies: Their Use in Clinical Practice and Trials

Luuk Wieske  1 , Duncan Smyth  2   3 , Michael P Lunn  2   3 , Filip Eftimov  1 , Charlotte E Teunissen  4
Affiliations
Review

Fluid Biomarkers for Monitoring Structural Changes in Polyneuropathies: Their Use in Clinical Practice and Trials

Luuk Wieske et al. Neurotherapeutics. 2021 Oct.

Erratum in

Abstract

Reliable and responsive tools for monitoring disease activity and treatment outcomes in patients with neuropathies are lacking. With the emergence of ultrasensitive blood bioassays, proteins released with nerve damage are potentially useful response biomarkers for many neurological disorders, including polyneuropathies. In this review, we provide an overview of the existing literature focusing on potential applications in polyneuropathy clinical care and trials. Whilst several promising candidates have been identified, no studies have investigated if any of these proteins can serve as response biomarkers of longitudinal disease activity, except for neurofilament light (NfL). For NfL, limited evidence exists supporting a role as a response biomarker in Guillain-Barré syndrome, vasculitic neuropathy, and chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Most evidence exists for NfL as a response biomarker in hereditary transthyretin-related amyloidosis (hATTR). At the present time, the role of NfL is therefore limited to a supporting clinical tool or exploratory endpoint in trials. Future developments will need to focus on the discovery of additional biomarkers for anatomically specific and other forms of nerve damage using high-throughput technologies and highly sensitive analytical platforms in adequality powered studies of appropriate design. For NfL, a better understanding of cut-off values, the relation to clinical symptoms and long-term disability as well as dynamics in serum on and off treatment is needed to further expand and proceed towards implementation.

Keywords: Biomarkers; Nerve damage; Neurofilament light chain; Polyneuropathies.

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Figures

Fig. 1
Fig. 1
Summary of fold change in blood NfL in various polyneuropathies. This figure displays the fold change in blood NfL levels when comparing patients to healthy controls. Values are presented in Table 1. When reported, patients with active disease were chosen for this comparison. CIDP, chronic inflammatory demyelinating polyneuropathy; CMT, Charcot-Marie Tooth disease; GBS, Guillain-Barré syndrome; hATTR, hereditary transthyretin-related amyloidosis. NB two studies were excluded from this figure because they presented as outliers: Körtveleyessy [74], which reports mean NfL values, and Gaiottino [75] which uses an ECL assay
See this image and copyright information in PMC

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