Safety and outcomes of eculizumab for acetylcholine receptor‐positive generalized myasthenia gravis in clinical practice

Suh, Joome; Clarke, Virginia; Amato, Anthony A.; Guidon, Amanda C.

doi:10.1002/mus.27656

Muscle and Nerve

2022

DOI: 10.1002/mus.27656

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Safety and outcomes of eculizumab for acetylcholine receptor‐positive generalized myasthenia gravis in clinical practice

Joome Suh

Virginia Clarke

Anthony A. Amato

et al.

Abstract: Introduction/Aims: Safety and outcomes data on eculizumab for generalized myasthenia gravis (gMG) in clinical practice remain limited. Outcomes and concomitant medication use may differ in practice compared with clinical trials. We analyzed the clinical and safety outcomes of patients who received eculizumab at our institutions.Methods: Patients with acetylcholine receptor antibody positive (AChR+) gMG, who

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2024

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Cited by 3 publications

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United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use

Habib,

Klink,

Muppidi

et al. 2024

J Neurol

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Background/objectives The phase 3 REGAIN study and its open-label extension demonstrated the efficacy of the complement C5 inhibitor eculizumab in patients with treatment-refractory, acetylcholine receptor antibody–positive generalized myasthenia gravis (gMG). The aim of the ELEVATE study was to assess the effectiveness of eculizumab in clinical practice in adults with MG in the United States. Methods A retrospective chart review was conducted in adults with MG who initiated eculizumab treatment between October 23, 2017 and December 31, 2019. Outcomes assessed before and during eculizumab treatment using a pre- versus post-treatment study design included Myasthenia Gravis–Activities of Daily Living (MG-ADL) total scores; minimal symptom expression (MSE); physician impression of clinical change; minimal manifestation status (MMS); and concomitant medication use. Results In total, 119 patients were included in the study. A significant reduction was observed in mean MG-ADL total score, from 8.0 before eculizumab initiation to 5.4 at 3 months and to 4.7 at 24 months after eculizumab initiation (both p < 0.001). At 24 months after eculizumab initiation, MSE was achieved by 19% of patients. MMS or better was achieved by 30% of patients at 24 months. Additionally, 64% of patients receiving prednisone at eculizumab initiation had their prednisone dosage reduced during eculizumab treatment and 13% discontinued prednisone; 32% were able to discontinue nonsteroidal immunosuppressant therapy. Discussion Eculizumab treatment was associated with sustained improvements in MG-ADL total scores through 24 months in adults with MG. Prednisone dosage was reduced in approximately two-thirds of patients, suggesting a steroid-sparing effect for eculizumab.

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United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use

Habib,

Klink,

Muppidi

et al. 2024

J Neurol

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Eculizumab in refractory myasthenia gravis: a real-world single-center experience

Ricciardi,

Erra,

Tuccillo

et al. 2024

Neurol Sci

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Eculizumab Versus Rituximab for Refractory Antiacetylcholine Receptor Antibody‐Positive Generalized Myasthenia Gravis: A Single‐Center Experience

Durmus,

Çakar,

Parman

2024

Acta Neurologica Scandinavica

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Background: Rituximab (RTX) and eculizumab (ECU) are treatment options for refractory myasthenia gravis (MG), but comparative clinical data derived from real‐world experience are limited. Here, we describe the baseline characteristics, treatment, and safety outcomes of patients with antiacetylcholine receptor antibody‐positive (AChR+) generalized myasthenia gravis (gMG) treated with ECU and/or RTX in our clinic.Methods: Patients with refractory AChR+ gMG who received ECU or/and RTX treatment for more than 1 year at the Department of Neurology, Istanbul Faculty of Medicine were included in this observational study. After obtaining written patient consent, data were collected retrospectively from medical records.Results: Twelve patients treated with ECU and 25 patients treated with RTX were included in the analysis. Groups were comparable with regard to demographic and clinical characteristics, including age at onset of MG, disease duration, and history of thymoma. ECU was associated with significantly better outcomes compared with RTX, as measured by decreases in the mean MG activities of daily living score at 1 (p = 0.024), 3 (p < 0.001), 6 (p < 0.001), and 12 (p < 0.001) months of treatment; steroid‐sparing effect after 1 year of treatment (decrease in mean [standard deviation] daily prednisolone dose of −21.8 mg [13.5] vs. −6.6 mg [9.4] with RTX; p < 0.001); and need for rescue treatment and number of myasthenic crisis episodes during treatment (p < 0.001). No new safety signals were observed with either treatment.Conclusion: Our data provide real‐world evidence supporting ECU over RTX to treat patients with refractory AChR+ gMG.

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Safety and outcomes of eculizumab for acetylcholine receptor‐positive generalized myasthenia gravis in clinical practice

Cited by 3 publications

References 17 publications

United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use

United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use

Eculizumab in refractory myasthenia gravis: a real-world single-center experience

Eculizumab Versus Rituximab for Refractory Antiacetylcholine Receptor Antibody‐Positive Generalized Myasthenia Gravis: A Single‐Center Experience

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