TY - JOUR
T1 - Management challenges in muscle-specific tyrosine kinase myasthenia gravis.
AU - Evoli Stampanoni-B, Amelia
AU - Alboini, Paolo Emilio
AU - Bisonni, Ana
AU - Mastrorosa, Alessia
AU - Bartoccioni, Emanuela
PY - 2012
Y1 - 2012
N2 - Myasthenia gravis with antibodies to muscle-specific tyrosine kinase (MuSK-MG) is generally considered a severe disease because of the associated weakness distribution with prevalent involvement of bulbar muscles and a rapidly progressive course and early respiratory crises. Its treatment can be unrewarding, owing to poor response to acetylcholinesterase inhibitors in most patients, disease relapses in spite of high-dose immunosuppression, and development of permanent bulbar weakness. High-dose prednisone plus plasma exchange is the recommended approach for treating rapidly progressive bulbar weakness. In the disease management, oral steroids proved effective, plasma exchange produced marked, albeit short-term, improvement, while conventional immunosuppressants were comparatively less effective. Rituximab is a promising treatment for refractory MuSK-MG; in uncontrolled studies, nearly all treated patients achieved significant improvement with substantial decrease of medication. It is yet to be clarified whether the early use of rituximab could prevent the permanent bulbar weakness, which constitutes a relevant disability in these patients.
AB - Myasthenia gravis with antibodies to muscle-specific tyrosine kinase (MuSK-MG) is generally considered a severe disease because of the associated weakness distribution with prevalent involvement of bulbar muscles and a rapidly progressive course and early respiratory crises. Its treatment can be unrewarding, owing to poor response to acetylcholinesterase inhibitors in most patients, disease relapses in spite of high-dose immunosuppression, and development of permanent bulbar weakness. High-dose prednisone plus plasma exchange is the recommended approach for treating rapidly progressive bulbar weakness. In the disease management, oral steroids proved effective, plasma exchange produced marked, albeit short-term, improvement, while conventional immunosuppressants were comparatively less effective. Rituximab is a promising treatment for refractory MuSK-MG; in uncontrolled studies, nearly all treated patients achieved significant improvement with substantial decrease of medication. It is yet to be clarified whether the early use of rituximab could prevent the permanent bulbar weakness, which constitutes a relevant disability in these patients.
KW - anti-MuSK antibodies
KW - myasthenia gravis
KW - anti-MuSK antibodies
KW - myasthenia gravis
UR - http://hdl.handle.net/10807/42540
U2 - 10.1111/j.1749-6632.2012.06781.x
DO - 10.1111/j.1749-6632.2012.06781.x
M3 - Article
SN - 0077-8923
VL - 2012
SP - 86
EP - 91
JO - Annals of the New York Academy of Sciences
JF - Annals of the New York Academy of Sciences
ER -