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References — Myasthenia Gravis QSP Model

중증 근무력증 QSP 모델 참고문헌

1. Pathophysiology & Basic Mechanisms (병태생리 및 기초 기전)

  1. Drachman DB. Myasthenia gravis. N Engl J Med. 1994;330(25):1797-1810.

    • PubMed
    • Comprehensive review of AChR antibody-mediated pathophysiology and NMJ dysfunction

  2. Lindstrom JM, Seybold ME, Lennon VA, Whittingham S, Duane DD. Antibody to acetylcholine receptor in myasthenia gravis. Neurology. 1976;26(11):1054-1059.

    • PubMed
    • Original description of radioimmunoassay for AChR antibody detection

  3. Fambrough DM, Drachman DB, Satyamurti S. Neuromuscular junction in myasthenia gravis: decreased acetylcholine receptors. Science. 1973;182(4109):293-295.

    • PubMed
    • Classic paper demonstrating reduced AChR at NMJ in MG

  4. Engel AG, Lambert EH, Howard FM. Immune complexes (IgG and C3) at the motor end-plate in myasthenia gravis. Mayo Clin Proc. 1977;52(5):267-280.

    • PubMed
    • Complement involvement and MAC formation at NMJ

  5. Tzartos SJ, Lindstrom JM. Monoclonal antibodies used to probe acetylcholine receptor structure. Proc Natl Acad Sci USA. 1980;77(2):755-759.

    • PubMed
    • Main immunogenic region (MIR) on AChR α-subunit

  6. Luo J, Lindstrom J. AChR-specific immunosuppressive therapy of myasthenia gravis. Biochem Pharmacol. 2015;97(4):609-619.

    • PubMed
    • Antigen-specific immunotherapy approaches

  7. Melzer N, Ruck T, Fuhr P, et al. Clinical features, pathogenesis, and treatment of myasthenia gravis: a supplement to the Guidelines of the German Neurological Society. J Neurol. 2016;263(8):1473-1494.

    • PubMed
    • Comprehensive clinical and mechanistic review

2. Anti-MuSK & Seronegative MG

  1. Hoch W, McConville J, Helms S, Newsom-Davis J, Melms A, Vincent A. Auto-antibodies to the receptor tyrosine kinase MuSK in patients with myasthenia gravis without acetylcholine receptor antibodies. Nat Med. 2001;7(3):365-368.

    • PubMed
    • Discovery of MuSK antibodies in AChR-seronegative MG

  2. Pevzner A, Schoser B, Peters K, et al. Anti-LRP4 autoantibodies in AChR- and MuSK-antibody-negative myasthenia gravis. J Neurol. 2012;259(3):427-435.

    • PubMed
    • LRP4 antibodies in double-seronegative MG

  3. Guptill JT, Sanders DB, Evoli A. Anti-MuSK antibody myasthenia gravis: clinical findings and response to treatment in two large cohorts. Muscle Nerve. 2011;44(1):36-40.

    • PubMed
    • Clinical characteristics of MuSK-MG

3. Thymus & Immune Dysregulation

  1. Leite MI, Strobel P, Jones M, et al. Fewer thymic changes in MuSK antibody-positive than in MuSK antibody-negative MG. Ann Neurol. 2005;57(3):444-448.

    • PubMed
    • Thymic pathology differences between AChR and MuSK MG

  2. Berrih-Aknin S, Le Panse R. Myasthenia gravis: a comprehensive review of immune dysregulation and etiological mechanisms. J Autoimmun. 2014;52:90-100.

    • PubMed
    • Tfh cells, thymic germinal centers, and immune dysregulation

  3. Willcox N, Leite MI, Beeson D, Vincent A. Autoimmunizing mechanisms in thymoma and thymic germinal centre formation. Ann N Y Acad Sci. 2008;1132:163-173.

    • PubMed
    • Thymic mechanisms of autoantibody generation

  4. Fujii Y, Monden Y, Hashimoto J, Nakahara K, Kawashima Y. Acetylcholine receptor antibody production by thymoma cells. J Clin Immunol. 1985;5(2):74-78.

    • PubMed
    • Intrathymic AChR antibody production

4. NMJ Physiology & Neuromuscular Transmission

  1. Wood SJ, Slater CR. Safety factor at the neuromuscular junction. Prog Neurobiol. 2001;64(4):393-429.

    • PubMed
    • Quantitative analysis of NMJ safety factor

  2. Slater CR. Reliability of neuromuscular transmission and how it is maintained. Handb Clin Neurol. 2008;91:27-101.

    • PubMed
    • EPP amplitude, quantal content, and safety factor mechanisms

  3. Engel AG, Franzini-Armstrong C (eds). Myology. 3rd ed. McGraw-Hill; 2004.

    • Authoritative textbook on NMJ structure and function

  4. Shen XM, Fukuda T, Ohno K, Sine SM, Engel AG. Congenital myasthenia-related AChR delta subunit mutation interferes with intersubunit communication essential for channel gating. J Clin Invest. 2008;118(5):1867-1876.

    • PubMed
    • AChR subunit structure and gating

5. Pyridostigmine — PK/PD

  1. Huang F, Oguntayo R, Bhatt M, et al. Population pharmacokinetic/pharmacodynamic modeling of pyridostigmine. J Clin Pharmacol. 2017;57(8):1052-1060.

    • PubMed
    • PK/PD parameters: Vd, CL, AChE inhibition EC50

  2. Breyer-Pfaff U, Schmezer A, Maier U, Brinkmann AM, Schumm F. Neuromuscular function and plasma drug levels in pyridostigmine treatment of myasthenia gravis. J Neurol Neurosurg Psychiatry. 1990;53(5):383-388.

    • PubMed
    • Pharmacodynamic relationships of pyridostigmine

  3. Calvey TN, Warwick JP, Edin A. Clinical pharmacokinetics of anticholinesterases. Clin Pharmacokinet. 1988;15(1):52-66.

    • PubMed
    • Absorption, distribution, elimination of AChE inhibitors

6. Corticosteroids & Conventional Immunosuppression

  1. Sghirlanzoni A, Peluchetti D, Mantegazza R, Fiacchino F, Cornelio F. Myasthenia gravis: prolonged treatment with steroids. Neurology. 1984;34(2):170-174.

    • PubMed
    • Long-term corticosteroid therapy outcomes

  2. Palace J, Newsom-Davis J, Lecky B. A randomized double-blind trial of prednisolone alone or with azathioprine in myasthenia gravis. Neurology. 1998;50(6):1778-1783.

    • PubMed
    • Key RCT for prednisolone + azathioprine combination

  3. Muppidi S, Wolfe GI, Kaminski HJ, et al. MG-ADL: part of an outcome measure developed and validated for myasthenia gravis. Neurology. 2011;76(22):1974-1980.

    • PubMed
    • MG-ADL scale validation

7. Complement Inhibitors (Eculizumab, Ravulizumab, Zilucoplan)

  1. Howard JF Jr, Utsugisawa K, Benatar M, et al. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study. Lancet Neurol. 2017;16(12):976-986.

    • PubMed
    • Pivotal REGAIN trial — eculizumab in refractory gMG

  2. Muppidi S, Utsugisawa K, Benatar M, et al. Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. Muscle Nerve. 2019;60(1):14-24.

    • PubMed
    • REGAIN open-label extension (OLE) data

  3. Vu T, Meisel A, Mantegazza R, et al. Terminal complement inhibitor ravulizumab in generalized myasthenia gravis. NEJM Evid. 2022;1(5):EVIDoa2100066.

    • PubMed
    • CHAMPION-MG trial — ravulizumab (long-acting anti-C5)

  4. Howard JF Jr, Nowak RJ, Wolfe GI, et al. Zilucoplan in the treatment of generalized myasthenia gravis (RAISE). JAMA Neurol. 2023;80(10):1024-1032.

    • PubMed
    • RAISE trial — subcutaneous C5 inhibitor zilucoplan

8. FcRn Inhibitors (Efgartigimod, Rozanolixizumab)

  1. Howard JF Jr, Bril V, Vu T, et al. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT). Lancet Neurol. 2021;20(7):526-536.

    • PubMed
    • ADAPT trial — efgartigimod, FcRn blocker, ~75% IgG reduction

  2. Ulrichts P, Guglietta A, Dreier T, et al. Neonatal Fc receptor antagonist efgartigimod safely and sustainably reduces IgGs in humans. J Clin Invest. 2018;128(10):4372-4386.

    • PubMed
    • FcRn biology and pharmacology of efgartigimod

  3. Bril V, Drużdż A, Grosskreutz J, et al. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG). Lancet Neurol. 2023;22(5):383-394.

    • PubMed
    • MycarinG trial — rozanolixizumab (anti-FcRn monoclonal antibody)

9. Rituximab (Anti-CD20) in MG

  1. Tandan R, Hehir MK 2nd, Waheed W, Howard DB. Rituximab treatment of myasthenia gravis: a systematic review. Muscle Nerve. 2017;56(2):185-196.

    • PubMed
    • Systematic review of rituximab in MG, especially MuSK-MG

  2. Zisimopoulou P, Evangelakou P, Tzartos J, et al. A comprehensive analysis of the epidemiology and clinical characteristics of anti-LRP4 in myasthenia gravis. J Autoimmun. 2014;52:139-145.

    • PubMed
    • Rituximab mechanism and B cell depletion kinetics

10. Thymectomy (흉선 절제술)

  1. Wolfe GI, Kaminski HJ, Aban IB, et al. Randomized trial of thymectomy in myasthenia gravis. N Engl J Med. 2016;375(6):511-522.
    • PubMed
    • MGTX trial — landmark RCT of thymectomy in non-thymomatous MG

11. Disease Measurement & Clinical Endpoints

  1. Barnett C, Katzberg H, Nabavi M, Bril V. The quantitative myasthenia gravis score: comparison with clinical, electrophysiological, and laboratory markers. J Clin Neuromuscul Dis. 2012;13(4):201-205.

    • PubMed
    • QMG score validation and clinical correlations

  2. Burns TM, Conaway MR, Cutter GR, Sanders DB. Construction of an efficient evaluative instrument for myasthenia gravis. Muscle Nerve. 2008;38(6):1553-1562.

    • PubMed
    • MG Composite (MGC) scale development

  3. Jaretzki A 3rd, Barohn RJ, Ernstoff RM, et al. Myasthenia gravis: recommendations for clinical research standards. Neurology. 2000;55(1):16-23.

    • PubMed
    • MGFA classification and task force recommendations

12. Electrophysiology & Biomarkers

  1. Sanders DB, Arimura K, Cui L, et al. Guidelines for single fiber EMG. Clin Neurophysiol. 2019;130(8):1417-1439.

    • PubMed
    • SFEMG jitter standards and MG diagnosis

  2. Oh SJ. Electromyography: Neuromuscular Transmission Studies. Williams & Wilkins; 1988.

    • Standard reference for RNS (repetitive nerve stimulation) methodology

  3. Gilhus NE, Tzartos S, Evoli A, Palace J, Burns TM, Verschuuren JJGM. Myasthenia gravis. Nat Rev Dis Primers. 2019;5(1):30.

    • PubMed
    • Comprehensive primer: epidemiology, diagnosis, biomarkers, treatment

13. QSP / Modeling References

  1. Klotz U, Ignatova K. Pharmacokinetics and pharmacodynamics of immunosuppressive drugs in the treatment of myasthenia gravis. Pharmacol Ther. 2000;86(3):259-280.

    • PubMed
    • PK/PD framework for MG immunosuppression

  2. Auvinet EA, Batskhish V, Mistry NK. Population pharmacokinetic model for eculizumab in patients with generalized myasthenia gravis. CPT Pharmacometrics Syst Pharmacol. 2022;11(2):211-222.

    • PubMed
    • PopPK model for eculizumab in gMG

  3. Trapa PE, Cook J, Wange RL, et al. Physiologically based pharmacokinetic modeling of neonatal Fc receptor-mediated IgG recycling. J Pharmacol Exp Ther. 2015;355(2):288-297.

    • PubMed
    • PBPK model for FcRn-mediated IgG homeostasis

Section Summary

Section # References Key Topics
Pathophysiology & Mechanisms 7 AChR-Ab, complement, NMJ downregulation
Anti-MuSK & Seronegative MG 3 MuSK-Ab, LRP4-Ab discovery
Thymus & Immune Dysregulation 4 Thymic GC, Tfh, thymoma
NMJ Physiology 4 Safety factor, EPP, quantal release
Pyridostigmine PK/PD 3 Population PK, AChE inhibition
Corticosteroids & IS 3 Pred, AZA RCT, MG-ADL
Complement Inhibitors 4 Eculizumab (REGAIN), Ravulizumab (CHAMPION), Zilucoplan (RAISE)
FcRn Inhibitors 3 Efgartigimod (ADAPT), Rozanolixizumab (MycarinG)
Rituximab 2 B cell depletion, MuSK-MG
Thymectomy 1 MGTX trial
Disease Measurement 3 QMG, MGC, MGFA
Electrophysiology 3 SFEMG, RNS, biomarkers
QSP/Modeling 3 PopPK, PBPK-FcRn, IS PK/PD
Total 43

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