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:
Toronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Univ Toronto, Univ Hlth Network, Toronto, ON, CanadaToronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Martinez-Harms, Rodrigo
Barnett, Carolina
论文数: 0引用数: 0
h-index: 0
机构:
Toronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Univ Toronto, Univ Hlth Network, Toronto, ON, CanadaToronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Barnett, Carolina
Alcantara, Monica
论文数: 0引用数: 0
h-index: 0
机构:
Toronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Univ Toronto, Univ Hlth Network, Toronto, ON, CanadaToronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
机构:
Univ Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Ctr Biomed Network Res Rare Dis CIBERER, Barcelona, SpainUniv Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Cortes-Vicente, Elena
Gallardo, Eduard
论文数: 0引用数: 0
h-index: 0
机构:
Univ Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Ctr Biomed Network Res Rare Dis CIBERER, Barcelona, SpainUniv Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Gallardo, Eduard
Angeles Martinez, Maria
论文数: 0引用数: 0
h-index: 0
机构:
Univ Autonoma Barcelona, Hosp Santa Creu & St Pau, Dept Immunol, Barcelona, SpainUniv Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Angeles Martinez, Maria
Diaz-Manera, Jordi
论文数: 0引用数: 0
h-index: 0
机构:
Univ Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Ctr Biomed Network Res Rare Dis CIBERER, Barcelona, SpainUniv Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Diaz-Manera, Jordi
Querol, Luis
论文数: 0引用数: 0
h-index: 0
机构:
Univ Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Ctr Biomed Network Res Rare Dis CIBERER, Barcelona, SpainUniv Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Querol, Luis
Rojas-Garcia, Ricard
论文数: 0引用数: 0
h-index: 0
机构:
Univ Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Ctr Biomed Network Res Rare Dis CIBERER, Barcelona, SpainUniv Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Rojas-Garcia, Ricard
Illa, Isabel
论文数: 0引用数: 0
h-index: 0
机构:
Univ Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
Ctr Biomed Network Res Rare Dis CIBERER, Barcelona, SpainUniv Autonoma Barcelona, Hosp Santa Cruz & St Pau, Dept Neurol, Neuromuscular Dis Unit, St Antoni Maria Claret 167, Barcelona 08025, Spain
机构:
Toronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Univ Toronto, Univ Hlth Network, Toronto, ON, CanadaToronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Martinez-Harms, Rodrigo
Barnett, Carolina
论文数: 0引用数: 0
h-index: 0
机构:
Toronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Univ Toronto, Univ Hlth Network, Toronto, ON, CanadaToronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Barnett, Carolina
Alcantara, Monica
论文数: 0引用数: 0
h-index: 0
机构:
Toronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
Univ Toronto, Univ Hlth Network, Toronto, ON, CanadaToronto Gen Hosp, Ellen & Martin Prosserman Ctr Neuromuscular Dis, Toronto, ON, Canada
机构:
Hosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, SpainHosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, Spain
Diaz Manera, J.
Rojas-Garcia, R.
论文数: 0引用数: 0
h-index: 0
机构:
Hosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, SpainHosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, Spain
Rojas-Garcia, R.
Pradas, J.
论文数: 0引用数: 0
h-index: 0
机构:
Hosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, SpainHosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, Spain
Pradas, J.
Juarez, C.
论文数: 0引用数: 0
h-index: 0
机构:
Hosp Santa Crue & St Pau, Dept Immunol, Barcelona, SpainHosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, Spain
Juarez, C.
Gallardo, E.
论文数: 0引用数: 0
h-index: 0
机构:
Hosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, SpainHosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, Spain
Gallardo, E.
Illa, I.
论文数: 0引用数: 0
h-index: 0
机构:
Hosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, SpainHosp Santa Crue & St Pau, Dept Neurol, Neuromuscular Dis Unit, Barcelona, Spain