What Is in the Neuromuscular Junction Literature?
Journal
Journal of clinical neuromuscular disease
ISSN: 1537-1611
Titre abrégé: J Clin Neuromuscul Dis
Pays: United States
ID NLM: 100887391
Informations de publication
Date de publication:
01 Dec 2023
01 Dec 2023
Historique:
medline:
15
11
2023
pubmed:
14
11
2023
entrez:
14
11
2023
Statut:
ppublish
Résumé
This update covers several articles on diagnosis and misdiagnosis of myasthenia gravis (MG), the role of complement in MG, and then an impressive number of recent treatment trials. There is a negative study on any corticosteroid-sparing effect of intravenous immunoglobulin. A number of positive studies are reviewed. Open-label extension studies of phase 3 trials showed benefit regarding quality of life with efgartigimod and in functional measures with ravulizumab. The phase 3 RAISE trial of zilucoplan, a self-administered complement C5 inhibitor, is covered as well as the MyCarinG trial of rozanolixizumab. The notion of using fast-acting therapies early in the course of MG is addressed. The last sections center on MG and Lambert-Eaton myasthenic syndrome as a consequence of immune checkpoint inhibitor therapy.
Identifiants
pubmed: 37962198
doi: 10.1097/CND.0000000000000456
pii: 00131402-202312000-00008
doi:
Substances chimiques
Complement Inactivating Agents
0
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
107-114Informations de copyright
Copyright © 2023 Wolters Kluwer Health, Inc. All rights reserved.
Déclaration de conflit d'intérêts
The author reports no conflicts of interest.
Références
Andrapalliyal N, Claytor B, Li Y. Incidence and causes of overdiagnosis of myasthenia gravis. Muscle Nerve. 2023;67:464–468.
Silvestri NJ, Narayanaswami P. The diagnosis of myasthenia gravis: the sensitive issue of specificity. Muscle Nerve. 2023;67:436–438.
Harrison P, Barton J, Winkel A. Chronic mimics of myasthenia gravis: a retrospective case series. Neuromuscul Disord. 2023;33:250–256.
Obaid AH, Zografou C, Vadysirisack DD, et al. Heterogeneity of acetylcholine receptor autoantibody-mediated complement activity in patients with myasthenia gravis. Neurol Neuroimmunol Neuroinflamm. 2022;9:e1169.
Stascheit F, Chuquisana O, Keller CW, et al. Complement activation profiles in anti-acetylcholine receptor positive myasthenia gravis. Eur J Neurol. 2023;30:1409–1416.
Bril V, Szczudlik A, Vaitkus A, et al. Randomized double-blind placebo-controlled trial of the corticosteroid-sparing effects of immunoglobulin in myasthenia gravis. Neurology. 2023;100:e671–e682.
Howard JF Jr, Bril V, Vu T, et al. Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021;20:526–536.
Saccà F, Barnett C, Vu T, et al. Efgartigimod improved health-related quality of life in generalized myasthenia gravis: results from a randomized, double-blind, placebo-controlled, phase 3 study (ADAPT). J Neurol. 2023;270:2096–2105.
Vu T, Meisel A, Mantegazza R, et al. Terminal complement inhibitor ravulizumab in generalized myasthenia gravis. NEJM Evid. Published online April 26, 2022. 10.1056/EVIDoa21000066
doi: 10.1056/EVIDoa21000066
Meisel A, Annane D, Vu T, et al. Long-term efficacy and safety of ravulizumab in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: results from the phase 3 CHAMPION MG open-label extension. J Neurol. 2023:1–14.
Howard JF Jr, Bresch S, Genge A, et al. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023;22:395–406.
Bril V, Benatar M, Andersen H, et al. Efficacy and safety of rozanolixizumab in moderate to severe generalized myasthenia gravis: a phase 2 randomized control trial. JAMA Neurol. 2020;77:582–592.
Bril V, Drużdż A, Grosskreutz J, et al. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22:383–394.
Verschuuren J. New therapies for autoimmune myasthenia gravis. Lancet Neurol. 2023;22:368–369.
Smith AG. Looming crisis of financial toxicity in neurologic drug pricing: from out-of-pocket to out-of-community costs. Neurology. 2023;100:401–402.
Piehl F, Eriksson-Dufva A, Budzianowska A, et al. Efficacy and safety of rituximab for new-onset generalized myasthenia gravis: the RINOMAX randomized clinical trial. JAMA Neurol. 2022;79:1105–1112.
Uzawa A, Suzuki S, Kuwabara S, et al. Effectiveness of early cycles of fast-acting treatment in generalised myasthenia gravis. J Neurol Neurosurg Psychiatry. 2023;94:467–473.
Mikami T, Liaw B, Asada M, et al. Neuroimmunological adverse events associated with immune checkpoint inhibitor: a retrospective, pharmacovigilance study using FAERS database. J Neurooncol. 2021;152:135–144.
Marini A, Bernardini A, Gigli GL, et al. Neurologic adverse events of immune checkpoint inhibitors: a systematic review. Neurology. 2021;96:754–766.
Zhao S, Zhou Y, Sun W, et al. Clinical features, diagnosis, and management of pembrolizumab-induced myasthenia gravis. Clin Exp Immunol. 2023;211:85–92.
Masi G, Pham MC, Karatz T, et al. Clinicoserological insights into patients with immune checkpoint inhibitor-induced myasthenia gravis. Ann Clin Transl Neurol. 2023;10:825–831.
Müller-Jensen L, Knauss S, Ginesta Roque L, et al. Autoantibody profiles in patients with immune checkpoint inhibitor-induced neurological immune related adverse events. Front Immunol. 2023;14:1108116.
Nakatani Y, Tanaka N, Enami T, et al. Lambert-Eaton myasthenic syndrome caused by nivolumab in a patient with squamous cell lung cancer. Case Rep Neurol. 2018;10:346–352.
Agrawal K, Agrawal N. Lambert-Eaton myasthenic syndrome secondary to nivolumab and ipilimumab in a patient with small-cell lung cancer. Case Rep Neurol Med. 2019; 2019;1–4.
Gill AJ, Gandhy S, Lancaster E. Nivolumab-associated Lambert-Eaton myasthenic syndrome and cerebellar dysfunction in a patient with a neuroendocrine tumor. Muscle Nerve. 2021;63:E18–E21.
Kunii E, Owaki S, Yamada K, et al. Lambert-Eaton myasthenic syndrome caused by atezolizumab in a patient with small-cell lung cancer. Intern Med. 2022;61:1739–1742.
Imai S, Mitsuboshi S, Hori S, et al. Increased risk of Lambert-Eaton myasthenic syndrome (LEMS) in small-cell lung cancer patients treated with immune checkpoint inhibitor. Eur J Cancer. 2023;180:1–3.
Takigawa Y, Watanabe H, Omote Y, et al. Lambert-eaton myasthenic syndrome recurrence induced by pembrolizumab in a patient with non-small-cell lung cancer. Intern Med. 2023;62:1055–1058.