Outcome measures and biomarkers in chronic inflammatory demyelinating polyradiculoneuropathy: from research to clinical practice

References

• Lunn MP, Manji H, Choudhary PP, et al. Chronic inflammatory demyelinating polyradiculoneuropathy: a prevalence study in south east England. J Neurol Neurosurg Psychiatry. 1999;66(5):677–680.
   PubMed Web of Science ®Google Scholar
• Laughlin RS, Dyck PJ, Melton LJ 3rd, et al. Incidence and prevalence of CIDP and the association of diabetes mellitus. Neurology. 2009;73(1):39–45.
   PubMed Web of Science ®Google Scholar
• Köller H, Kieseier BC, Jander S, et al. Chronic inflammatory demyelinating polyneuropathy. N Engl J Med. 2005;352(13):1343–1356.
   PubMed Web of Science ®Google Scholar
• Van den Bergh PY, Hadden RD, Bouche P, et al. European Federation of Neurological Societies/Peripheral Nerve Society guideline on management of chronic inflammatory demyelinating polyradiculoneuropathy: report of a joint task force of the European Federation of Neurological societies and the Peripheral Nerve Society - first revision. Eur J Neurol. 2010;17(3):356–363. Erratum in: Eur J Neurol. 2011;18(5):796.
   Web of Science ®Google Scholar
• Mathey EK, Park SB, Hughes RA, et al. Chronic inflammatory demyelinating polyradiculoneuropathy: from pathology to phenotype. J Neurol Neurosurg Psychiatry. 2015;86(9):973–985.
   PubMed Web of Science ®Google Scholar
• Doneddu PE, Cocito D, Manganelli F, et al. Atypical CIDP: diagnostic criteria, progression and treatment response. Data from the Italian CIDP database. J Neurol Neurosurg Psychiatry. 2019;90(2):125–132.
   PubMed Web of Science ®Google Scholar
• Ikeda S, Koike H, Nishi R, et al. Clinicopathological characteristics of subtypes of chronic inflammatory demyelinating polyradiculoneuropathy. J Neurol Neurosurg Psychiatry. 2019;90(9):988–996.
   PubMed Web of Science ®Google Scholar
• Vallat JM, Mathis S, Vegezzi E, et al. Antibody- and macrophage-mediated segmental demyelination in chronic inflammatory demyelinating polyneuropathy: clinical, electrophysiological, immunological and pathological correlates. Eur J Neurol. 2020;27(4):692–701.
   PubMed Web of Science ®Google Scholar
• Querol L, Devaux J, Rojas-Garcia R, et al. Autoantibodies in chronic inflammatory neuropathies: diagnostic and therapeutic implications. Nat Rev Neurol. 2017;13(9):533–547.
   PubMed Web of Science ®Google Scholar
• Koike H, Kadoya M, Kaida KI, et al. Paranodal dissection in chronic inflammatory demyelinating polyneuropathy with anti-neurofascin-155 and anti-contactin-1 antibodies. J Neurol Neurosurg Psychiatry. 2017;88(6):465–473.
   PubMed Web of Science ®Google Scholar
• Delmont E, Manso C, Querol L, et al. Autoantibodies to nodal isoforms of neurofascin in chronic inflammatory demyelinating polyneuropathy. Brain. 2017;140(7):1851–1858.
   PubMedGoogle Scholar
• Manso C, Querol L, Lleixà C, et al. Anti-neurofascin-155 IgG4 antibodies prevent paranodal complex formation in vivo. J Clin Invest. 2019;129(6):2222–2236.
   PubMed Web of Science ®Google Scholar
• Doppler K, Schuster Y, Appeltshauser L, et al. Anti-CNTN1 IgG3 induces acute conduction block and motor deficits in a passive transfer rat model. J Neuroinflammation. 2019;16(1):73.
   PubMedGoogle Scholar
• Koike H, Nishi R, Ikeda S, et al. Ultrastructural mechanisms of macrophage-induced demyelination in CIDP. Neurology. 2018;91(23):1051–1060.
   PubMed Web of Science ®Google Scholar
• Van Rhijn I, Van Den BergLH, Bosboom WM, et al. Expression of accessory molecules for T-cell activation in peripheral nerve of patients with CIDP and vasculitic neuropathy. Brain. 2000;123(Pt 10):2020–2029.
   PubMedGoogle Scholar
• Allen JA. The misdiagnosis of CIDP: a review. Neurol Ther. 2020;9(1):43–54.
   PubMed Web of Science ®Google Scholar
• Hughes RA, Mehndiratta MM, Rajabally YA. Corticosteroids for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev. 2017 Nov 29;11(11):CD002062.
   PubMedGoogle Scholar
• Oaklander AL, Lunn MP, Hughes RA, et al. Treatments for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP): an overview of systematic reviews. Cochrane Database Syst Rev. 2017 Jan 13;1(1):CD010369.
   PubMedGoogle Scholar
• Mehndiratta MM, Hughes RA, Pritchard J. Plasma exchange for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Cochrane Database Syst Rev. 2015 Aug 25;2015(8):CD003906.
   Google Scholar
• Eftimov F, Winer JB, Vermeulen M, et al. Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev. 2013 Dec;30(12):CD001797.
   Google Scholar
• Cocito D, Paolasso I, Antonini G, et al. A nationwide retrospective analysis on the effect of immune therapies in patients with chronic inflammatory demyelinating polyradiculoneuropathy. Eur J Neurol. 2010;17(2):289–294.
   PubMed Web of Science ®Google Scholar
• Kuitwaard K, Hahn AF, Vermeulen M, et al. Intravenous immunoglobulin response in treatment-naïve chronic inflammatory demyelinating polyradiculoneuropathy. J Neurol Neurosurg Psychiatry. 2015;86(12):1331–1336.
   PubMed Web of Science ®Google Scholar
• van Lieverloo GGA, Peric S, Doneddu PE, et al. Corticosteroids in chronic inflammatory demyelinating polyneuropathy: a retrospective, multicentre study, comparing efficacy and safety of daily prednisolone, pulsed dexamethasone, and pulsed intravenous methylprednisolone. J Neurol. 2018;265(9):2052–2059.
   PubMedGoogle Scholar
• Liberatore G, Manganelli F, Doneddu PE, et al. Chronic inflammatory demyelinating polyradiculoneuropathy: can a diagnosis be made in patients not fulfilling electrodiagnostic criteria? Eur J Neurol. 2021;28(2):620–629.
   PubMed Web of Science ®Google Scholar
• Glasziou P, Rose P, Heneghan C, et al. Diagnosis using “test of treatment.” BMJ. 2009;338:b1312.
   PubMedGoogle Scholar
• Allen JA, Lewis RA. CIDP diagnostic pitfalls and perception of treatment benefit. Neurology. 2015;85(6):498–504.
   PubMed Web of Science ®Google Scholar
• Dyck PJ, O’Brien P, Swanson C, et al. Combined azathioprine and prednisone in chronic inflammatory-demyelinating polyneuropathy. Neurology. 1985;35(8):1173–1176.
   PubMed Web of Science ®Google Scholar
• Hadden RD, Sharrack B, Bensa S, et al. Randomized trial of interferon beta-1a in chronic inflammatory demyelinating polyradiculoneuropathy. Neurology. 1999;53(1):57–61.
   PubMed Web of Science ®Google Scholar
• Hughes RA, Donofrio P, Bril V, et al. Intravenous immune globulin (10% caprylate-chromatography purified) for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy (ICE study): a randomised placebo-controlled trial. Lancet Neurol. 2008;7:136–144.
   PubMed Web of Science ®Google Scholar
• RMC Trial Group. Randomised controlled trial of methotrexate for chronic inflammatory demyelinating polyradiculoneuropathy (RMC trial): a pilot, multicentre study. Lancet Neurol. 2009;8(2):158–164.
   PubMed Web of Science ®Google Scholar
• Hughes RA, Gorson KC, Cros D, et al. Intramuscular interferon beta-1a in chronic inflammatory demyelinating polyradiculoneuropathy. Neurology. 2010;74(8):651–657.
   PubMed Web of Science ®Google Scholar
• Eftimov F, Winer JB, Vermeulen M, et al. Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev. 2013;12:CD001797.
   Google Scholar
• Hughes R, Dalakas MC, Merkies I, et al. Oral fingolimod for chronic inflammatory demyelinating polyradiculoneuropathy (FORCIDP Trial): a double-blind, multicentre, randomised controlled trial. Lancet Neurol. 2018;17(8):689–698.
   PubMed Web of Science ®Google Scholar
• van Schaik IN, Bril V, van Geloven N, et al. Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (PATH): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2018;17(1):35–46.
   PubMed Web of Science ®Google Scholar
• Lewis RA, Cornblath DR, Hartung HP, et al. Placebo effect in chronic inflammatory demyelinating polyneuropathy: the PATH study and a systematic review. J Peripher Nerv Syst. 2020;25(3):230–237.
   PubMed Web of Science ®Google Scholar
• Gorson KC, van Schaik IN, Merkies IS, et al. Chronic inflammatory demyelinating polyneuropathy disease activity status: recommendations for clinical research standards and use in clinical practice. J Peripher Nerv Syst. 2010;15(4):326–333.
   PubMed Web of Science ®Google Scholar
• Vanhoutte EK, Faber CG, Merkies IS, et al. 196th ENMC international workshop: outcome measures in inflammatory peripheral neuropathies 8–10 February 2013, Naarden, The Netherlands. Neuromuscul Disord. 2013;23(11):924–933.
   PubMed Web of Science ®Google Scholar
• Gwathmey KG, Conaway MR, Sadjadi R, et al. Construction and validation of the chronic acquired polyneuropathy patient-reported index (CAP-PRI): a disease-specific, health-related quality-of-life instrument. Muscle Nerve. 2016;54(1):9–17.
   PubMed Web of Science ®Google Scholar
• Rajabally YA, Fatehi F. Outcome measures for chronic inflammatory demyelinating polyneuropathy in research: relevance and applicability to clinical practice. Neurodegener Dis Manag. 2019;9(5):259–266.
   PubMed Web of Science ®Google Scholar
• Allen JA, Merkies ISJ, Lewis RA. Monitoring clinical course and treatment response in chronic inflammatory demyelinating polyneuropathy during routine care: a review of clinical and laboratory assessment measures. JAMA Neurol. 2020;77(9):1159–1166.
   PubMed Web of Science ®Google Scholar
• van Nes SI, Vanhoutte EK, Van Doorn PA, et al. Rasch-built overall disability scale (R-ODS) for immune-mediated peripheral neuropathies. Neurology. 2011;76(4):337–345.
   PubMed Web of Science ®Google Scholar
• van Schaik IN, Eftimov F, Van Doorn PA, et al. Pulsed high-dose dexamethasone versus standard prednisolone treatment for chronic inflammatory demyelinating polyradiculoneuropathy (PREDICT study): a double-blind, randomised, controlled trial. Lancet Neurol. 2010;9(3):245–253.
   PubMed Web of Science ®Google Scholar
• Nobile-Orazio E, Cocito D, Jann S, et al. Intravenous immunoglobulin versus intravenous methylprednisolone for chronic inflammatory demyelinating polyradiculoneuropathy: a randomised controlled trial. Lancet Neurol. 2012;11(6):493–502.
   PubMed Web of Science ®Google Scholar
• Kuitwaard K, Brusse E, Jacobs BC, et al. Randomized trial of intravenous immunoglobulin maintenance treatment regimens in chronic inflammatory demyelinating polyradiculoneuropathy. Eur J Neurol. 2021;28(1):286–296.
   PubMed Web of Science ®Google Scholar
• Cornblath DR, Hartung HP, Katzberg HD, et al. A randomised, multi-centre phase III study of 3 different doses of intravenous immunoglobulin 10% in patients with chronic inflammatory demyelinating polyradiculoneuropathy (ProCID trial): study design and protocol. J Peripher Nerv Syst. 2018;23(2):108–114.
   PubMed Web of Science ®Google Scholar
• ISRCTN Registry. Intravenous immunoglobulin overtreatment in chronic inflammatory demyelinating polyneuropathy. 2020. [cited 2021 Jun 23]. Available from: http://www.isrctn.com/ISRCTN13637698
   Google Scholar
• Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials. 1989;10(4):407–415.
   PubMedGoogle Scholar
• Draak THP, De Greef BTA, Faber CG, et al. The minimum clinically important difference: which direction to take. Eur J Neurol. 2019;26(6):850–855.
   PubMed Web of Science ®Google Scholar
• FDA-NIH Biomarker Working Group. BEST (Biomarkers, EndpointS, and other tools) resource [Internet]. Silver Spring (MD): Food and Drug Administration (US); Bethesda (MD): National Institutes of Health (US); 2016. [cited 2021 Jun 23]. Available from: https://www.ncbi.nlm.nih.gov/books/NBK326791/
   Google Scholar
• Amur S. Biomarker terminology: speaking the same language. 2017. [cited 2021 Jun 23]. Available from: https://www.fda.gov/files/BIOMARKER-TERMINOLOGY--SPEAKING-THE-SAME-LANGUAGE.pdf
   Google Scholar
• Härtig F, Ross M, Dammeier NM, et al. Nerve ultrasound predicts treatment response in chronic inflammatory demyelinating polyradiculoneuropathy -a prospective follow-up. Neurotherapeutics. 2018;15(2):439–451.
   PubMed Web of Science ®Google Scholar
• Zaidman CM, Pestronk A. Nerve size in chronic inflammatory demyelinating neuropathy varies with disease activity and therapy response over time: a retrospective ultrasound study. Muscle Nerve. 2014;50(5):733–738.
   PubMed Web of Science ®Google Scholar
• Doppler K, Werner C, Sommer C. Disruption of nodal architecture in skin biopsies of patients with demyelinating neuropathies. J Peripher Nerv Syst. 2013;18(2):168–176.
   PubMed Web of Science ®Google Scholar
• Doppler K, Appeltshauser L, Wilhelmi K, et al. Destruction of paranodal architecture in inflammatory neuropathy with anti-contactin-1 autoantibodies. J Neurol Neurosurg Psychiatry. 2015;86(7):720–728.
   PubMed Web of Science ®Google Scholar
• van Lieverloo GGA, Wieske L, Verhamme C, et al. Serum neurofilament light chain in chronic inflammatory demyelinating polyneuropathy. J Peripher Nerv Syst. 2019;24(2):187–194.
   PubMed Web of Science ®Google Scholar
• Wang H, Davison M, Wang K, et al. Transmembrane protease serine 5: a novel Schwann cell plasma marker for CMT1A. Ann Clin Transl Neurol. 2020;7(1):69–82.
   PubMed Web of Science ®Google Scholar
• Capodivento G, De Michelis C, Carpo M, et al. Nobbio LCSF sphingomyelin: a new biomarker of demyelination in the diagnosis and management of CIDP and GBS. J Neurol Neurosurg Psychiatry. 2021 Mar;92(3):303–310.
   PubMed Web of Science ®Google Scholar
• Illes Z, Blaabjerg M. Cerebrospinal fluid findings in Guillain-Barré syndrome and chronic inflammatory demyelinating polyneuropathies. Handb Clin Neurol. 2017;146:125–138.
   PubMedGoogle Scholar
• Vural A, Doppler K, Meinl E. Autoantibodies against the node of Ranvier in seropositive chronic inflammatory demyelinating polyneuropathy: diagnostic, pathogenic, and therapeutic relevance. Front Immunol. 2018;9:1029.
   PubMed Web of Science ®Google Scholar
• Pascual-Goñi E, Martín-Aguilar L, Luis Querol L. Autoantibodies in chronic inflammatory demyelinating polyradiculoneuropathy. Curr Opin Neurol. 2019;32(5):651–657.
   PubMed Web of Science ®Google Scholar
• Querol L, Rojas-García R, Diaz-Manera J, et al. Rituximab in treatment-resistant CIDP with antibodies against paranodal proteins. Neurol Neuroimmunol Neuroinflamm. 2015 3;2(5):e149.
   PubMed Web of Science ®Google Scholar
• Moritz CP, Tholance Y, Stoevesandt O, et al. CIDP antibodies target junction proteins and identify patient subgroups: an autoantigenomic approach. Neurol Neuroimmunol Neuroinflamm. 2021 Jan 6;8(2):e944.
   PubMed Web of Science ®Google Scholar
• Mausberg AK, Dorok M, Stettnet M, et al. Recovery of the T-cell repertoire in CIDP by IV immunoglobulins. Neurology. 2013;80(3):296–303.
   PubMed Web of Science ®Google Scholar
• Bril V, Banach M, Dalakas MC, et al. Electrophysiologic correlations with clinical outcomes in CIDP. Muscle Nerve. 2010;42(4):492–497.
   PubMed Web of Science ®Google Scholar
• Chin RL, Deng C, Bril V, et al. Follow-up nerve conduction studies in CIDP after treatment with IGIV-C: comparison of patients with and without subsequent relapse. Muscle Nerve. 2015;52(4):498–502.
   PubMed Web of Science ®Google Scholar
• Rabin M, Mutlu G, Stojkovic T, et al. Chronic inflammatory demyelinating polyradiculoneuropathy: search for factors associated with treatment dependence or successful withdrawal. J Neurol Neurosurg Psychiatry. 2014;85(8):901–906.
   PubMed Web of Science ®Google Scholar
• Kerasnoudis A, Pitarokoili K, Gold R, et al. Nerve ultrasound and electrophysiology for therapy monitoring in chronic inflammatory demyelinating polyneuropathy. J Neuroimaging. 2015;25(6):931–939.
   PubMed Web of Science ®Google Scholar
• Delmont E, Benvenutto A, Grimaldi S, et al. Motor unit number index (MUNIX): is it relevant in chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)? Clin Neurophysiol. 2016;127(3):1891–1894.
   PubMed Web of Science ®Google Scholar
• Lawley A, Seri S, Rajabally YA. Motor unit number index (MUNIX) in chronic inflammatory demyelinating polyneuropathy: a potential role in monitoring response to intravenous immunoglobulins. Clin Neurophysiol. 2019;130(10):1743–1749.
   PubMed Web of Science ®Google Scholar
• Korporal-Kuhnke M, Jürgen Haas J, Schwarz A, et al. Plasmacytosis is a common immune signature in patients with MMN and CIDP and responds to treatment with IVIg. J Neuroimmunol. 2015;278:60–68.
   PubMed Web of Science ®Google Scholar
• Trouvin AP, Jacquot S, Grigioni S, et al. Usefulness of monitoring of B cell depletion in rituximab-treated rheumatoid arthritis patients in order to predict clinical relapse: a prospective observational study. Clin Exp Immunol. 2015;180(1):11–18.
   PubMed Web of Science ®Google Scholar
• Wijetilleka S, Mukhtyar C, Jayne D, et al. Immunoglobulin replacement for secondary immunodeficiency after B-cell targeted therapies in autoimmune rheumatic disease: systematic literature review. Autoimmun Rev. 2019;18(5):535–541.
   PubMed Web of Science ®Google Scholar
• Kuitwaard K, Van Doom PA, Vermeulen M, et al. Serum IgG levels in IV immunoglobulin treated chronic inflammatory demyelinating polyneuropathy. J Neurol Neurosurg Psychiatry. 2013;84(8):859–861.
   PubMed Web of Science ®Google Scholar
• Allen JA, Pasnoor M, Dimachkie MM, et al. Quantifying treatment-related fluctuations in CIDP: results of the GRIPPER study. Neurology. 2021;96(14):e1876–e1886.
   PubMed Web of Science ®Google Scholar