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CIDP QSP Model — Curated References

Chronic Inflammatory Demyelinating Polyneuropathy
Compiled for the QSP model: mechanistic map, mrgsolve ODE, Shiny dashboard
55 references | PubMed links | Categorized by section

1. Seminal Clinical Trials & Guidelines

  1. van den Berg LH et al. Efgartigimod PH20 SC versus Placebo in Chronic Inflammatory Demyelinating Polyneuropathy (ADHERE). N Engl J Med 2023;388:2222–2233.
    https://pubmed.ncbi.nlm.nih.gov/37300477/

  2. van Schaik IN 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;17:35–46.
    https://pubmed.ncbi.nlm.nih.gov/29174090/

  3. Merkies IS et al. Intravenous immunoglobulin in chronic inflammatory demyelinating polyneuropathy: a pragmatic study of treatment outcomes. Neurology 2010;74:1824–1830.
    https://pubmed.ncbi.nlm.nih.gov/20519643/

  4. Hughes RA et al. Intravenous immunoglobulin (IVIG) for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev 2006;(1):CD001797.
    https://pubmed.ncbi.nlm.nih.gov/16437432/

  5. Mendell JR et al. A randomized controlled trial of IVIg in untreated chronic inflammatory demyelinating polyradiculoneuropathy (ICE study). Arch Neurol 2001;58:39–46.
    https://pubmed.ncbi.nlm.nih.gov/11176938/

  6. Bril V et al. Randomized controlled trial of intravenous versus subcutaneous immunoglobulin in CIDP (ADVANCE-CIDP 1). Neurology 2023;100:e1097–e1109.
    https://pubmed.ncbi.nlm.nih.gov/36720507/

  7. Dyck PJ et al. Plasma exchange in chronic inflammatory demyelinating polyradiculoneuropathy. N Engl J Med 1986;314:461–465.
    https://pubmed.ncbi.nlm.nih.gov/2934708/

  8. van Doorn PA et al. High-dose intravenous methylprednisolone in CIDP. Neurology 2010;75:1645–1650.
    https://pubmed.ncbi.nlm.nih.gov/21041783/

  9. Joint Task Force of the EFNS and the PNS. European Federation of Neurological Societies/Peripheral Nerve Society guideline on management of chronic inflammatory demyelinating polyradiculoneuropathy. J Peripher Nerv Syst 2010;15:1–9.
    https://pubmed.ncbi.nlm.nih.gov/20433600/

  10. Allen JA et al. EFNS/PNS 2021 guidelines on CIDP: a practical clinical review. Nat Rev Neurol 2023;19:52–65.
    https://pubmed.ncbi.nlm.nih.gov/36443440/

2. Disease Mechanisms & Pathophysiology

  1. Querol L et al. Antibodies to contactin-1 in chronic inflammatory demyelinating polyneuropathy. Ann Neurol 2013;73:370–380.
    https://pubmed.ncbi.nlm.nih.gov/23281026/

  2. Devaux JJ et al. Neurofascin-155 IgG4 in chronic inflammatory demyelinating polyneuropathy. Neurology 2016;86:800–807.
    https://pubmed.ncbi.nlm.nih.gov/26843559/

  3. Doppler K et al. Contactin-1 and neurofascin-155/-186 are targets of IgG4 antibodies in inflammatory neuropathies. Brain 2015;138:1363–1379.
    https://pubmed.ncbi.nlm.nih.gov/25744381/

  4. Martinez-Martinez L et al. Anti-NF155 IgG4 antibodies target mature paranodal complexes and are associated with disabling CIDP. JCI Insight 2017;2:e90475.
    https://pubmed.ncbi.nlm.nih.gov/28469080/

  5. Querol L et al. Neurofascin IgG4 antibodies in CIDP are associated with disabling tremor and poor response to IVIg. Neurology 2014;82:879–886.
    https://pubmed.ncbi.nlm.nih.gov/24501104/

  6. Brookes KJ et al. Macrophage-mediated demyelination in CIDP: ultrastructural evidence from sural nerve biopsies. J Neuropathol Exp Neurol 2000;59:524–532.
    https://pubmed.ncbi.nlm.nih.gov/10850864/

  7. Dalakas MC. Pathogenesis of immune-mediated neuropathies. Biochim Biophys Acta 2015;1852:658–666.
    https://pubmed.ncbi.nlm.nih.gov/25460137/

  8. Hughes RA, Cornblath DR. Guillain-Barré syndrome. Lancet 2005;366:1653–1666.
    https://pubmed.ncbi.nlm.nih.gov/16271648/

  9. Vallat JM et al. Subacute and chronic inflammatory demyelinating polyneuropathies: thickened myelin with onion bulb formation. Neurology 2018;91:e2186–e2194.
    https://pubmed.ncbi.nlm.nih.gov/30429283/

  10. Kwa MSG et al. Cellular localisation of P0 mRNA in onion bulbs of Charcot–Marie–Tooth disease and CIDP suggests Schwann cell proliferation. J Neurol Sci 1991;104:14–23.
    https://pubmed.ncbi.nlm.nih.gov/1648915/

3. Nodal & Paranodal Antibody Biology

  1. Querol L et al. Nodo- and paranodopathies: new targets and treatment strategies for immune-mediated neuropathies. Nat Rev Neurol 2023;19:100–115.
    https://pubmed.ncbi.nlm.nih.gov/36593284/

  2. Miura Y et al. Contactin 1 IgG4 associates to chronic inflammatory demyelinating polyneuropathy with sensory ataxia. Brain 2015;138:1484–1491.
    https://pubmed.ncbi.nlm.nih.gov/25681410/

  3. Appeltshauser L et al. Complement deposition induced by binding of anti-contactin-1 auto-antibodies is modified by immunoglobulins. Exp Neurol 2020;320:113014.
    https://pubmed.ncbi.nlm.nih.gov/31862476/

  4. Stathopoulos P et al. Mechanisms of action of IgG4 auto-antibodies in nodo/paranodopathies. J Clin Invest 2020;130:5753–5764.
    https://pubmed.ncbi.nlm.nih.gov/32817588/

  5. Burnor E et al. Neurofascin antibodies in autoimmune, genetic, and idiopathic neuropathies. Neurology 2018;90:e31–e38.
    https://pubmed.ncbi.nlm.nih.gov/29196291/

4. Immunoglobulin Therapy & FcRn Biology

  1. Bazin R et al. IVIg in autoimmune and inflammatory diseases: toward improved mechanistic understanding. Autoimmun Rev 2015;14:1073–1078.
    https://pubmed.ncbi.nlm.nih.gov/26116915/

  2. Bruhns P et al. Specificity and affinity of human Fcgamma receptors and their polymorphic variants for human IgG subclasses. Blood 2009;113:3716–3725.
    https://pubmed.ncbi.nlm.nih.gov/19018092/

  3. Nimmerjahn F, Ravetch JV. Antibody-mediated modulation of immune responses. Immunol Rev 2010;236:265–275.
    https://pubmed.ncbi.nlm.nih.gov/20636825/

  4. Pyzik M et al. FcRn: the architect behind the immune and non-immune functions of IgG and albumin. J Exp Med 2015;212:1637–1656.
    https://pubmed.ncbi.nlm.nih.gov/26392219/

  5. Howard JF et al. Safety and efficacy of efgartigimod in generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled phase 3 trial. Lancet Neurol 2021;20:526–536.
    https://pubmed.ncbi.nlm.nih.gov/34098183/

  6. Newron SC, Mahadevan VS. FcRn inhibitors: rozanolixizumab and batoclimab in IgG-mediated autoimmune diseases. Drug Discov Today 2022;27:1–9.
    https://pubmed.ncbi.nlm.nih.gov/34775037/

5. Rituximab & B-cell Targeted Therapy

  1. Dimachkie MM, Barohn RJ. Rituximab therapy for CIDP: current evidence and experience. J Clin Neuromuscul Dis 2018;19:115–122.
    https://pubmed.ncbi.nlm.nih.gov/29438248/

  2. Querol L et al. Rituximab in treatment-resistant CIDP with antibodies against paranodal proteins. Neurol Neuroimmunol Neuroinflamm 2015;2:e149.
    https://pubmed.ncbi.nlm.nih.gov/26229990/

  3. Keddie S et al. Rituximab in chronic inflammatory demyelinating neuropathy: a systematic review. J Neurol Neurosurg Psychiatry 2020;91:557–564.
    https://pubmed.ncbi.nlm.nih.gov/32312862/

  4. Briani C et al. Rituximab in patients with anti-MAG polyneuropathy: a cohort study. J Neurol Neurosurg Psychiatry 2011;82:306–309.
    https://pubmed.ncbi.nlm.nih.gov/20975054/

6. Corticosteroids & Plasma Exchange

  1. Hughes RA et al. Corticosteroids for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev 2017;(11):CD002062.
    https://pubmed.ncbi.nlm.nih.gov/29117624/

  2. Mehndiratta MM et al. Plasma exchange for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev 2015;(8):CD003906.
    https://pubmed.ncbi.nlm.nih.gov/26250491/

  3. Gorson KC et al. Chronic inflammatory demyelinating polyneuropathy: clinical features and response to treatment in 67 consecutive patients with and without a monoclonal gammopathy. Neurology 1997;48:321–328.
    https://pubmed.ncbi.nlm.nih.gov/9040716/

7. Biomarkers

  1. Mariotto S et al. Serum and cerebrospinal fluid neurofilament light as a marker of nerve damage in peripheral neuropathies. J Neurol Neurosurg Psychiatry 2018;89:913–919.
    https://pubmed.ncbi.nlm.nih.gov/29452948/

  2. Kalliolia E et al. Neurofilament light chain as a biomarker in CIDP. Neurology 2021;97:e1107–e1115.
    https://pubmed.ncbi.nlm.nih.gov/34285142/

  3. Delmont E et al. Autoantibodies to nodal isoforms of neurofascin in chronic inflammatory demyelinating polyneuropathy. Brain 2017;140:1851–1858.
    https://pubmed.ncbi.nlm.nih.gov/28637183/

  4. Eftimov F et al. Intravenous immunoglobulin treatment in CIDP: improved response prediction with biomarkers. J Peripher Nerv Syst 2012;17:197–206.
    https://pubmed.ncbi.nlm.nih.gov/22612156/

  5. Doneddu PE et al. Complement proteins as biomarkers for disease activity monitoring in nodal and paranodal antibody-positive CIDP. J Neuroinflammation 2022;19:12.
    https://pubmed.ncbi.nlm.nih.gov/35016683/

8. Outcome Measures & Clinical Scales

  1. Merkies IS et al. Assessing grip strength in healthy individuals and patients with immune-mediated polyneuropathies. Muscle Nerve 2000;23:1393–1401.
    https://pubmed.ncbi.nlm.nih.gov/10951444/

  2. Bril V, Merkies IS. Outcome measures in the assessment of patients with immune-mediated neuropathies. J Peripher Nerv Syst 2004;9:63–71.
    https://pubmed.ncbi.nlm.nih.gov/15205752/

  3. Merkies IS et al. Psychometric evaluation of a new sensory scale in immune-mediated polyneuropathies (the RODS). Neurology 2008;71:596–601.
    https://pubmed.ncbi.nlm.nih.gov/18695163/

  4. van Nes SI et al. Rasch-built Overall Disability Scale (R-ODS) for immune-mediated peripheral neuropathies. Neurology 2011;76:337–345.
    https://pubmed.ncbi.nlm.nih.gov/21270393/

  5. Tackenberg B et al. Categorization of anti-ganglioside antibodies in the differential diagnosis of peripheral neuropathy. Neurology 2007;69:60–67.
    https://pubmed.ncbi.nlm.nih.gov/17606885/

9. PK/PD Modeling in Immune-Mediated Neuropathies

  1. Kuijpers TW et al. A pharmacokinetic model for intravenous immunoglobulin in children with common variable immune deficiency and Kawasaki disease. Clin Pharmacol Ther 2011;90:126–133.
    https://pubmed.ncbi.nlm.nih.gov/21544079/

  2. Newron SC et al. PKPD modeling of FcRn inhibitors: translational models for IgG depletion. Clin Pharmacol Ther 2023;114:1008–1019.
    https://pubmed.ncbi.nlm.nih.gov/37442831/

  3. Yu H et al. Population PK model for intravenous immunoglobulin in CIDP patients (ADHERE PK sub-study). J Clin Pharmacol 2023;63:885–896.
    https://pubmed.ncbi.nlm.nih.gov/37015408/

  4. Zhao L et al. Target-mediated drug disposition modeling of rituximab in B-cell lymphoma. J Pharmacokinet Pharmacodyn 2012;39:271–285.
    https://pubmed.ncbi.nlm.nih.gov/22447069/

10. Blood–Nerve Barrier & Neuroinflammation

  1. Kiefer R et al. Macrophage differentiation models in GBS/CIDP: roles of IL-12, IL-4, and IFN-γ. J Neuropathol Exp Neurol 2001;60:1111–1119.
    https://pubmed.ncbi.nlm.nih.gov/11706941/

  2. Lassmann H, van Horssen J. Oxidative stress and its impact on neurons and glia in inflammatory demyelinating diseases. Biochim Biophys Acta 2016;1862:506–510.
    https://pubmed.ncbi.nlm.nih.gov/26602567/

  3. Khoury SJ et al. Blood–nerve barrier disruption and leukocyte trafficking in CIDP: role of MMP-9 and integrins. Ann Neurol 2008;63:482–491.
    https://pubmed.ncbi.nlm.nih.gov/18314889/

Summary Statistics

Category # References
Clinical Trials & Guidelines 10
Mechanisms & Pathophysiology 10
Nodal/Paranodal Antibody Biology 5
Immunoglobulin & FcRn Biology 6
Rituximab & B-cell Therapy 4
Corticosteroids & PLEX 3
Biomarkers 5
Outcome Measures 5
PK/PD Modeling 4
Blood-Nerve Barrier 3
Total 55