Patterns and predictors of therapeutic response to efgartigimod in acetylcholine receptor-antibody generalized myasthenia gravis subtypes
Plain language summary Patterns and predictors of therapeutic response to efgartigimod in AChR generalized myasthenia gravis Myasthenia Gravis (MG) is an autoimmune disorder that can lead to potentially life-threatening complications. Efgartigimod is an approved biologic for generalized myasthenia g...
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Published in | Therapeutic advances in neurological disorders Vol. 18; p. 17562864251319656 |
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Main Authors | , , , , , , , , , , , , , , , , , , , , , , , , |
Format | Journal Article |
Language | English |
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SAGE Publications
01.01.2025
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ISSN | 1756-2864 1756-2856 1756-2864 |
DOI | 10.1177/17562864251319656 |
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Abstract | Plain language summary
Patterns and predictors of therapeutic response to efgartigimod in AChR generalized myasthenia gravis
Myasthenia Gravis (MG) is an autoimmune disorder that can lead to potentially life-threatening complications. Efgartigimod is an approved biologic for generalized myasthenia gravis (gMG). However, the patterns and predictors of therapeutic response to efgartigimod among the acetylcholine receptor-generalized MG (AChR-gMG) subtypes remain inconclusive. Our findings revealed patterns of treatment responses among AChR-gMG subtypes, with LOMG patients potentially presenting a more sustained response. |
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AbstractList | Efgartigimod is an approved biologic for generalized myasthenia gravis (gMG), which is an autoimmune disease and can potentially be life-threatening. However, the therapeutic response to efgartigimod among the acetylcholine receptor gMG (AChR-gMG) subtypes remains inconclusive.BackgroundEfgartigimod is an approved biologic for generalized myasthenia gravis (gMG), which is an autoimmune disease and can potentially be life-threatening. However, the therapeutic response to efgartigimod among the acetylcholine receptor gMG (AChR-gMG) subtypes remains inconclusive.To explore the patterns and predictors for the therapeutic response to efgartigimod among AChR-gMG subtypes.ObjectiveTo explore the patterns and predictors for the therapeutic response to efgartigimod among AChR-gMG subtypes.This prospective, observational study included AChR-gMG patients treated with efgartigimod at 15 centers in China with a follow-up for at least 20 weeks.DesignThis prospective, observational study included AChR-gMG patients treated with efgartigimod at 15 centers in China with a follow-up for at least 20 weeks.The primary outcome was the proportion of minimal symptom expression (MSE) responders, denoted by a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 0 or 1 within 4 weeks and maintained for ⩾4 weeks. AChR antibody-positive MG (AChR-MG) subtypes were classified into early onset myasthenia gravis (EOMG), late-onset myasthenia gravis (LOMG), and thymoma-associated myasthenia gravis (TAMG). The predictive factors for MSE responders were identified by univariate and multivariate logistic regression analysis.MethodsThe primary outcome was the proportion of minimal symptom expression (MSE) responders, denoted by a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 0 or 1 within 4 weeks and maintained for ⩾4 weeks. AChR antibody-positive MG (AChR-MG) subtypes were classified into early onset myasthenia gravis (EOMG), late-onset myasthenia gravis (LOMG), and thymoma-associated myasthenia gravis (TAMG). The predictive factors for MSE responders were identified by univariate and multivariate logistic regression analysis.One hundred sixteen patients were included with a median follow-up duration of 238 days (172.5-306.3). There were 50 (43.1%) patients with EOMG, 28 (24.1%) with LOMG and 38 (32.8%) with TAMG. After efgartigimod initiation, 35 (30.2%) patients were MSE responders, and the proportion of MSE responders was highest in the LOMG group (42.9%). The MG-ADL score reduction in the LOMG group was more significant than in the EOMG group by weeks 16 and 20 (both p = 0.022). Response patterns to efgartigimod among the AChR-MG subtypes differed as measured by the proportion of improved patients and MSE. LOMG presented sustained symptom control, while EOMG and TAMG showed more fluctuations. Eight TAMG patients (21.1%) switched to another biologic (p = 0.005). Baseline MG-ADL was an independent predictor for therapeutic response to efgartigimod (p < 0.001).ResultsOne hundred sixteen patients were included with a median follow-up duration of 238 days (172.5-306.3). There were 50 (43.1%) patients with EOMG, 28 (24.1%) with LOMG and 38 (32.8%) with TAMG. After efgartigimod initiation, 35 (30.2%) patients were MSE responders, and the proportion of MSE responders was highest in the LOMG group (42.9%). The MG-ADL score reduction in the LOMG group was more significant than in the EOMG group by weeks 16 and 20 (both p = 0.022). Response patterns to efgartigimod among the AChR-MG subtypes differed as measured by the proportion of improved patients and MSE. LOMG presented sustained symptom control, while EOMG and TAMG showed more fluctuations. Eight TAMG patients (21.1%) switched to another biologic (p = 0.005). Baseline MG-ADL was an independent predictor for therapeutic response to efgartigimod (p < 0.001).Our findings revealed patterns of treatment responses among AChR-gMG subtypes, with LOMG patients potentially presenting a more sustained response. These findings likely provide preliminary data for precision therapy in MG in the era of biologics.ConclusionOur findings revealed patterns of treatment responses among AChR-gMG subtypes, with LOMG patients potentially presenting a more sustained response. These findings likely provide preliminary data for precision therapy in MG in the era of biologics.NCT04535843.Trial registrationNCT04535843. Efgartigimod is an approved biologic for generalized myasthenia gravis (gMG), which is an autoimmune disease and can potentially be life-threatening. However, the therapeutic response to efgartigimod among the acetylcholine receptor gMG (AChR-gMG) subtypes remains inconclusive. To explore the patterns and predictors for the therapeutic response to efgartigimod among AChR-gMG subtypes. This prospective, observational study included AChR-gMG patients treated with efgartigimod at 15 centers in China with a follow-up for at least 20 weeks. The primary outcome was the proportion of minimal symptom expression (MSE) responders, denoted by a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 0 or 1 within 4 weeks and maintained for ⩾4 weeks. AChR antibody-positive MG (AChR-MG) subtypes were classified into early onset myasthenia gravis (EOMG), late-onset myasthenia gravis (LOMG), and thymoma-associated myasthenia gravis (TAMG). The predictive factors for MSE responders were identified by univariate and multivariate logistic regression analysis. One hundred sixteen patients were included with a median follow-up duration of 238 days (172.5-306.3). There were 50 (43.1%) patients with EOMG, 28 (24.1%) with LOMG and 38 (32.8%) with TAMG. After efgartigimod initiation, 35 (30.2%) patients were MSE responders, and the proportion of MSE responders was highest in the LOMG group (42.9%). The MG-ADL score reduction in the LOMG group was more significant than in the EOMG group by weeks 16 and 20 (both = 0.022). Response patterns to efgartigimod among the AChR-MG subtypes differed as measured by the proportion of improved patients and MSE. LOMG presented sustained symptom control, while EOMG and TAMG showed more fluctuations. Eight TAMG patients (21.1%) switched to another biologic ( = 0.005). Baseline MG-ADL was an independent predictor for therapeutic response to efgartigimod ( < 0.001). Our findings revealed patterns of treatment responses among AChR-gMG subtypes, with LOMG patients potentially presenting a more sustained response. These findings likely provide preliminary data for precision therapy in MG in the era of biologics. NCT04535843. Plain language summary Patterns and predictors of therapeutic response to efgartigimod in AChR generalized myasthenia gravis Myasthenia Gravis (MG) is an autoimmune disorder that can lead to potentially life-threatening complications. Efgartigimod is an approved biologic for generalized myasthenia gravis (gMG). However, the patterns and predictors of therapeutic response to efgartigimod among the acetylcholine receptor-generalized MG (AChR-gMG) subtypes remain inconclusive. Our findings revealed patterns of treatment responses among AChR-gMG subtypes, with LOMG patients potentially presenting a more sustained response. Patterns and predictors of therapeutic response to efgartigimod in AChR generalized myasthenia gravis Myasthenia Gravis (MG) is an autoimmune disorder that can lead to potentially life-threatening complications. Efgartigimod is an approved biologic for generalized myasthenia gravis (gMG). However, the patterns and predictors of therapeutic response to efgartigimod among the acetylcholine receptor-generalized MG (AChR-gMG) subtypes remain inconclusive. Our findings revealed patterns of treatment responses among AChR-gMG subtypes, with LOMG patients potentially presenting a more sustained response. Background: Efgartigimod is an approved biologic for generalized myasthenia gravis (gMG), which is an autoimmune disease and can potentially be life-threatening. However, the therapeutic response to efgartigimod among the acetylcholine receptor gMG (AChR-gMG) subtypes remains inconclusive. Objective: To explore the patterns and predictors for the therapeutic response to efgartigimod among AChR-gMG subtypes. Design: This prospective, observational study included AChR-gMG patients treated with efgartigimod at 15 centers in China with a follow-up for at least 20 weeks. Methods: The primary outcome was the proportion of minimal symptom expression (MSE) responders, denoted by a Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 0 or 1 within 4 weeks and maintained for ⩾4 weeks. AChR antibody-positive MG (AChR-MG) subtypes were classified into early onset myasthenia gravis (EOMG), late-onset myasthenia gravis (LOMG), and thymoma-associated myasthenia gravis (TAMG). The predictive factors for MSE responders were identified by univariate and multivariate logistic regression analysis. Results: One hundred sixteen patients were included with a median follow-up duration of 238 days (172.5–306.3). There were 50 (43.1%) patients with EOMG, 28 (24.1%) with LOMG and 38 (32.8%) with TAMG. After efgartigimod initiation, 35 (30.2%) patients were MSE responders, and the proportion of MSE responders was highest in the LOMG group (42.9%). The MG-ADL score reduction in the LOMG group was more significant than in the EOMG group by weeks 16 and 20 (both p = 0.022). Response patterns to efgartigimod among the AChR-MG subtypes differed as measured by the proportion of improved patients and MSE. LOMG presented sustained symptom control, while EOMG and TAMG showed more fluctuations. Eight TAMG patients (21.1%) switched to another biologic ( p = 0.005). Baseline MG-ADL was an independent predictor for therapeutic response to efgartigimod ( p < 0.001). Conclusion: Our findings revealed patterns of treatment responses among AChR-gMG subtypes, with LOMG patients potentially presenting a more sustained response. These findings likely provide preliminary data for precision therapy in MG in the era of biologics. Trial registration: NCT04535843. |
Author | Huang, Shixiong Chen, Ying Xi, Jianying Zhao, Zhongyan Shi, Jianquan Zhao, Chongbo Wang, Qinzhou Yan, Chong Zhang, Zhouao Wu, Zongtai Tan, Song Jin, Lei Du, Lei Hong, Daojun Song, Jie Yu, Yanyan Guo, Xiuming Yue, Yaoxian Jiang, Qilong Zeng, Quantao Zeng, Wenshuang Luo, Sushan Zou, Zhangyu Chen, Jing Zhang, Yong |
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University of Chinese Medicine, Guangzhou, China – sequence: 6 givenname: Jing surname: Chen fullname: Chen, Jing organization: Department of Neurology, The Second Affiliated Hospital of Soochow University, Suzhou, China – sequence: 7 givenname: Jianquan surname: Shi fullname: Shi, Jianquan organization: Department of Neurology, Nanjing First Hospital, Nanjing Medical University, Nanjing, China – sequence: 8 givenname: Yanyan surname: Yu fullname: Yu, Yanyan organization: Department of Neurology, The First Affiliated Hospital of Nanchang University, Nanchang, China – sequence: 9 givenname: Daojun surname: Hong fullname: Hong, Daojun organization: Department of Neurology, The First Affiliated Hospital of Nanchang University, Nanchang, China – sequence: 10 givenname: Quantao surname: Zeng fullname: Zeng, Quantao organization: Department of Neurology, Sichuan Provincial People’s Hospital, University of Electronic Science and Technology of China, Chengdu, China – sequence: 11 givenname: 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College, National Center for Neurological Disorders, Fudan University, No. 12 Wulumuqi Zhong Road, Jing’an District, Shanghai 200040, China – sequence: 25 givenname: Chongbo orcidid: 0000-0001-9481-1418 surname: Zhao fullname: Zhao, Chongbo organization: Huashan Rare Disease Center and Department of Neurology, Huashan Hospital, Shanghai Medical College, National Center for Neurological Disorders, Fudan University, No. 12 Wulumuqi Zhong Road, Jing’an District, Shanghai 200040, China |
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Keywords | early-onset thymoma late-onset efgartigimod therapeutic response generalized myasthenia gravis |
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Patterns and predictors of therapeutic response to efgartigimod in AChR generalized myasthenia gravis
Myasthenia Gravis (MG) is an... Efgartigimod is an approved biologic for generalized myasthenia gravis (gMG), which is an autoimmune disease and can potentially be life-threatening. However,... Patterns and predictors of therapeutic response to efgartigimod in AChR generalized myasthenia gravis Myasthenia Gravis (MG) is an autoimmune disorder that can... Background: Efgartigimod is an approved biologic for generalized myasthenia gravis (gMG), which is an autoimmune disease and can potentially be... |
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Title | Patterns and predictors of therapeutic response to efgartigimod in acetylcholine receptor-antibody generalized myasthenia gravis subtypes |
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