Efgartigimod is a neonatal Fc receptor (FcRn) inhibitor that primarily diminishes antibody levels by inhibiting antibody (IgG) reabsorption and is noted for its rapid onset of action and low immunogenicity. Currently, efgartigimod is approved for the treatment of anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG), yet there are no relevant clinical trials for myasthenic crisis (MC). Consequently, we performed a systematic review of the available literature to evaluate the clinical efficacy of efgartigimod for the treatment of MC. We conducted a search of the PubMed, Web of science, Embase, and Scopus databases for all observational studies published until September 30, 2024. Risk of bias was assessed using the Joanna Briggs Institute (JBI) Case Report and Series Critical Assessment Checklist for quality assessment of included studies. A total of nine case reports/series, all observational studies, were included. Twenty MC patients were included, all of whom exhibited clinically significant improvement after treatment with Efgartigimod, with a sustained decrease in AChR antibody serum titers and a significant decrease in IgG levels throughout the treatment cycle. Efgartigimod demonstrates favorable clinical efficacy. Efgartigimod may serve as an effective treatment for patients with MC. However, further studies are required to clarify the efficacy of Efgartigimod alone. PROSPERO registration number: CRD42023430032.
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A systematic review of efgartigimod as an effective treatment for myasthenic crisis.
Naunyn Schmiedebergs Arch Pharmacol
February 2025
Department of Neurology, First Affiliated Hospital, Chongqing Medical University, Chongqing, China.
Efgartigimod is a neonatal Fc receptor (FcRn) inhibitor that primarily diminishes antibody levels by inhibiting antibody (IgG) reabsorption and is noted for its rapid onset of action and low immunogenicity. Currently, efgartigimod is approved for the treatment of anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG), yet there are no relevant clinical trials for myasthenic crisis (MC). Consequently, we performed a systematic review of the available literature to evaluate the clinical efficacy of efgartigimod for the treatment of MC.
View Article and Find Full Text PDFSequential administration of efgartigimod shortened the course of Guillain-Barré syndrome: a case series.
Ther Adv Neurol Disord
February 2025
Department of Neurology, West China Hospital, Sichuan University, No. 37 Guoxue Road, Chengdu, Sichuan 610041, China.
Guillain-Barré syndrome (GBS) is a serious neurological condition with limited treatment options. A recent report demonstrated successful treatment with efgartigimod alone in two patients with GBS, although it did not significantly shorten the disease duration. This case series investigates the effects of sequential efgartigimod administration in patients with different GBS phenotypes and varying levels of disease severity.
View Article and Find Full Text PDFFast-acting treatment of myasthenic crisis with efgartigimod from the perspective of the neonatal intensive care unit.
BMC Neurol
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Department of Neurology, The First Affiliated Hospital, Sun Yat-sen University, Guangdong Provincial Key Laboratory of Diagnosis and Treatment of Major Neurological Diseases, National Key Clinical Department and Key Discipline of Neurology, No. 58 Zhongshan Road 2, Guangzhou, 510080, China.
Background: Myasthenic crisis (MC) refers to rapid deterioration of myasthenia gravis (MG), affecting lung and bulbar muscles and causing breathing difficulties. Currently, efgartigimod has shown good therapeutic effects in patients with generalized myasthenia gravis (GMG). This retrospective real-world study explored the effectiveness of efgartigimod in patients with MC.
View Article and Find Full Text PDFResponse of refractory residual ocular symptoms to efgartigimod in generalized myasthenia gravis: A real-world case series.
J Neuroimmunol
February 2025
Department of Neurology, Huashan Hospital, Fudan University, Shanghai, China; Huashan Rare Disease Center, Huashan Hospital, Fudan University, Shanghai, China; National Center for Neurological Diseases, Shanghai, China. Electronic address:
Objective: To evaluate the efficacy of efgartigimod (EFG) in treating residual ocular symptoms in myasthenia gravis (MG) patients with acetylcholine receptor antibodies (AChR-Ab).
Methods: Five MG patients with refractory residual ocular symptoms treated with EFG at Huashan Hospital were included. The demographic and clinical information was collected, and MG Activities of Daily Living (MG-ADL) scores and Quantitative Myasthenia Gravis (QMG) scores was elevated weekly during the 8-week follow up period.
The efficacy and safety between efgartigimod and intravenous immunoglobulin in elderly generalized myasthenia gravis patients.
Clin Immunol
February 2025
The First Affiliated Hospital of Guangzhou University of Chinese Medicine, Guangzhou, Guangdong, China. Electronic address:
The study examines efgartigimod and intravenous immunoglobulin (IVIg) in elderly patients with generalized myasthenia gravis (GMG), focusing on changes in Myasthenia Gravis Activities of Daily Living (MG-ADL) scores, pyridostigmine dosage, and minimal symptom expression (MSE) over an 8-week period. Among 74 enrolled patients, efgartigimod showed greater reduction in MG-ADL scores compared to IVIg at weeks 4 and 8, with no serious adverse events, suggesting its superior efficacy and safety in elderly Chinese patients with acetylcholine receptor antibody-positive (AChR-Ab(+)) GMG.
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