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Abstract
Background Several prognostic models predict clinical outcomes in Guillain-Barré syndrome (GBS). Recently, neurofilament light chain (NfL) has emerged as a prognostic biomarker. We investigated the added prognostic value of NfL in serum (sNfL) and cerebrospinal fluid (cNfL) to models based on clinical factors predicting respiratory failure and inability to walk in GBS.
Methods We included patients from a randomised placebo-controlled trial (second intravenous immunoglobulin dose in GBS). Serum was acquired at entry and week 1, 2, 4 and 12 and cerebrospinal fluid at entry. NfL levels were determined on a single molecule array. The additional prognostic value of NfL to the (modified) Erasmus GBS Outcome Score ((m)EGOS) and (modified) Erasmus GBS Respiratory Insufficiency Score was evaluated using logistic regression analyses.
Results In total, 293 patients were included (74 (25%) mechanically ventilated, 38/275 (13%) unable to walk at 26 weeks). Higher sNfL at entry, week 1 and week 2 and cNfL at entry were associated with inability to walk at 4 and 26 weeks. Neither sNfL nor cNfL levels at entry were associated with respiratory failure. The EGOS and mEGOS improved after adding NfL (∆C-statistic range: 0.01–0.11), especially the models predicting outcome at 26 weeks. A new model predicting inability to walk at 26 weeks consisting of sNfL at entry, GBS disability score at entry and Medical Research Council sum score at week 2 performed best (C-statistic: 0.88 (95% CI 0.83 to 0.94)).
Conclusions Addition of NfL may improve clinical prognostic models for the prediction of inability to walk, but not of respiratory failure.
Trial registration number NTR2224/NL2107.
- GUILLAIN-BARRE SYNDROME
- Patient Outcome Assessment
- Prognosis
Data availability statement
Data are available upon reasonable request.
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Data availability statement
Data are available upon reasonable request.
Footnotes
Contributors RCMT contributed to the conceptualisation and design of the study, execution of data analysis, interpretation of results and drafting of the manuscript. LWGL contributed to the conceptualisation and design of the study, interpretation of results and critical review of the manuscript. SJvT contributed to the conceptualisation and design of the study, interpretation of results and critical review of the manuscript. EJAW contributed to the interpretation of results and critical review of the manuscript. CET contributed to data acquisition and critical review of the manuscript. LV contributed to data acquisition and critical review of the manuscript. PAvD contributed to data acquisition and critical review of the manuscript. RH contributed to data acquisition, conceptualisation and design of the study, interpretation of results and critical review of the manuscript. BCJ contributed to data acquisition, conceptualisation and design of the study, interpretation of results and critical review of the manuscript and supervised the study/served as the guarantor.
Funding This study was funded by Prinses Beatrix Spierfonds W.OR19-24.
Competing interests RCMT, LWGL, SJT, EJAW, CET and LV report no competing interests. PAvD reports grants from Prinses Beatrix Spierfonds, The Netherlands Organisation for Health Research and Development (ZonMW), Sanquin Blood supplySupply, Takeda and Grifols, he. He is a member of the Scientific Advisory Committee/Steering Committee Trials for Annexon, Argenx, Hansa, Octapharma, Sanofi and Roche, all. All grants and fees were paid to his institution. RH reports grants from the GBS-CIDP Foundation International, Stichting GBS, NIH and the PANDIA collaboration project co-funded by BÜHLMANN Laboratories AG and the PPP Allowance made available by Health~Holland, Top Sector Life Sciences & Health, to Prinses Beatrix Spierfonds to stimulate public–private partnerships under project number LSHM23017-SGF. BCJ reports institutional funding for research related to GBS from Prinses Beatrix Spierfonds, Zon-MW, GBS-CIDP Foundation International, Stichting GBS, Horizon 2020, Grifols, CSL-Behring, Annexon, Roche and Hansa Biopharma. BCJ is in the Clinical TrailTrial Committee of Annexon. BCJ serves as the chair of the Steering Committee of the International GBS Outcome Study (IGOS) and is a member of the Global Medical Advisory Board of the GBS-CIDP Foundation International.
Provenance and peer review Not commissioned; externally peer reviewed.
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