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.
Evoli Stampanoni-B, A., Alboini, P. E., Bisonni, A., Mastrorosa, A., Bartoccioni, E., Management challenges in muscle-specific tyrosine kinase myasthenia gravis., <<ANNALS OF THE NEW YORK ACADEMY OF SCIENCES>>, 2012; 2012 (N/A): 86-91. [doi:10.1111/j.1749-6632.2012.06781.x] [http://hdl.handle.net/10807/42540]
Management challenges in muscle-specific tyrosine kinase myasthenia gravis.
Evoli Stampanoni-B, Amelia;Alboini, Paolo Emilio;Bartoccioni, Emanuela
2012
Abstract
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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.