Myasthenia gravis;
Neonatal Fc receptor;
Activities of daily;
Living;
Corticosteroids;
Efgartigimod;
FcRn antagonist;
DOUBLE-BLIND;
D O I:
10.1016/j.nmd.2024.04.005
中图分类号:
R74 [神经病学与精神病学];
学科分类号:
摘要:
The effect of treatment with efgartigimod in seronegative myasthenia gravis (MG) remains unclear. This retrospective study aimed to evaluate symptomatic changes and safety of treatment with efgartigimod in patients with generalized MG (gMG) double-seronegative for acetylcholine receptor antibody and muscle-specific kinase antibody. We reviewed the medical records of double-seronegative gMG treated with 10 mg/kg efgartigimod once/week per cycle (4 weeks) from June 2022 to June 2023. A total of 16 patients were included. MG-activities of daily living (ADL) scores improved from 9.2 to 7.4. Mean prednisolone dose was reduced from 5.4 to 4.1 mg/day. The duration before MG-ADL deterioration after the end of a cycle was 6.1 weeks. Five patients had mild adverse events. This retrospective study revealed no significant treatment benefit in the outcomes of patients with double-seronegative gMG treated with efgartigimod. (c) 2024 Elsevier B.V. All rights reserved.
机构:
Fdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, ItalyFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Mantegazza, Renato
Bril, Vera
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机构:
Univ Toronto, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, CanadaFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Bril, Vera
Vu, Tuan
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机构:
Univ S Florida, Morsani Coll Med, Neurol, Tampa, FL 33620 USAFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Vu, Tuan
Karam, Chafic
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机构:
Univ Penn, Sch Med, Dept Neurol, Philadelphia, PA 19104 USAFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Karam, Chafic
Peric, Stojan
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机构:
Univ Clin Ctr Serbia, Neurol Clin, Belgrade, SerbiaFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Peric, Stojan
Margania, Temur
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机构:
New Hosp, Dept Neurol & Neurorehabil, Tbilisi, GeorgiaFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Margania, Temur
Murai, Hiroyuki
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机构:
Int Univ Hlth & Welf, Neurol, Tokyo, JapanFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Murai, Hiroyuki
Bilinska, Malgorzata
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机构:
Wroclaw Med Univ, Dept & Clin Neurol, Wroclaw, PolandFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Bilinska, Malgorzata
Shakarishvili, Roman
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Sarajishvili Inst Neurol & Neurosurg, Neurol, Tbilisi, PolandFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Shakarishvili, Roman
Smilowski, Marek
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Med Univ Silesia, Dept Hematol & Bone Marrow Transplantat, Katowice, PolandFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Smilowski, Marek
Guglietta, Antonio
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Argenx, Clin Dev, Ghent, BelgiumFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Guglietta, Antonio
Ulrichts, Peter
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Argenx, Clin Dev, Ghent, BelgiumFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Ulrichts, Peter
Vangeneugden, Tony
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Argenx, Clin Dev, Ghent, BelgiumFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Vangeneugden, Tony
Utsugisawa, Kimiaki
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Hanamaki Gen Hosp, Neurol, Hanamaki, JapanFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Utsugisawa, Kimiaki
Verschuuren, Jan
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Leiden Univ, Med Ctr, Neurol, Leiden, NetherlandsFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
Verschuuren, Jan
Howard, James
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机构:
Univ N Carolina, Neurol, Chapel Hill, NC 27515 USAFdn IRCCS Ist Neurol Carlo Besta, Neurol Iv Neuroimmunol & Neuromuscular Dis Unit, Milan, Italy
机构:
Hainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Wang, Pan
Zhang, Bo
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机构:
Hainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Zhang, Bo
Yin, Jian
论文数: 0引用数: 0
h-index: 0
机构:
Beijing Anzhen Hosp, Dept Neurol, Beijing, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Yin, Jian
Xi, Jianying
论文数: 0引用数: 0
h-index: 0
机构:
Fudan Univ, Huashan Hosp, Dept Neurol, Shanghai, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Xi, Jianying
Tan, Ying
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机构:
Peking Union Med Coll Hosp, Dept Neurol, Beijing, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Tan, Ying
Gao, Feng
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h-index: 0
机构:
Peking Univ First Hosp, Dept Neurol, Beijing, Peoples R China
Army Med Univ, Daping Hosp, Ctr Clin Neurosci, Chongqing, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Gao, Feng
Zeng, Fan
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机构:
Army Med Univ, Daping Hosp, Dept Neurol, Chongqing, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Zeng, Fan
Chang, Ting
论文数: 0引用数: 0
h-index: 0
机构:
Fourth Mil Med Univ, Tangdu Hosp, Dept Neurol, Xian, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Chang, Ting
Zhou, Hao
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h-index: 0
机构:
Cent South Univ, Xiangya Hosp, Dept Neurol, Changsha, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Zhou, Hao
Liang, Hui
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机构:
Hainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Liang, Hui
Zhao, Zhongyan
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h-index: 0
机构:
Hainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Zhao, Zhongyan
Yang, Huan
论文数: 0引用数: 0
h-index: 0
机构:
Cent South Univ, Xiangya Hosp, Dept Neurol, Changsha, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Yang, Huan
Zhao, Chongbo
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机构:
Fudan Univ, Huashan Hosp, Dept Neurol, Shanghai, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China
Zhao, Chongbo
Huang, Shixiong
论文数: 0引用数: 0
h-index: 0
机构:
Hainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R ChinaHainan Med Univ, Hainan Gen Hosp, Dept Neurol, Hainan Affiliated Hosp, Haikou, Peoples R China