Response to therapy in myasthenia gravis with anti-MuSK antibodies

Ann N Y Acad Sci. 2008:1132:76-83. doi: 10.1196/annals.1405.012.

Abstract

Myasthenia gravis (MG) with antibodies against the muscle-specific tyrosine kinase (MuSK abs) is often a severe disease requiring aggressive treatment. Various immunosuppressive (IS) regimens have been employed; the efficacy of plasma exchange is unanimously recognized, while the indication for thymectomy is controversial. We evaluated the response to therapy in 57 MuSK-positive patients (12 M/45 F) comparing our experience with other authors' results. Disease severity and response to treatment were graded according to MG Foundation of America; follow-up ranged from 0.5-29 years. Owing to both MG severity and the unsatisfactory response to cholinesterase inhibitors, most patients (54/57) needed IS treatment, and 35 received one or more courses of plasma exchange and intravenous immunoglobulin. At the end of follow-up, the rate of complete remission was 8.8%, and IS treatment had been withdrawn in only 10/54 patients. The extent of therapeutic response varied considerably. With conventional IS therapy (prednisone alone or in combination with azathioprine or cyclosporine), most patients achieved good control of their disease, but 30% of them were left with permanent facial and bulbar weakness. In patients with refractory disease, the use of mycophenolate mofetil and rituximab proved very effective, as also reported by other authors. In our and others' experience, MuSK-positive MG markedly improves with IS therapy, although, in comparison with the AChR-positive disease, it is characterized by a lower remission rate, as a higher proportion of patients remain dependent on treatment. Thymectomy is mostly considered scarcely effective; however, at present, no firm conclusions can be drawn on its role in the treatment of this form of MG.

Publication types

  • Clinical Trial

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antibodies / immunology*
  • Antibodies / therapeutic use*
  • Child
  • Cholinesterase Inhibitors / therapeutic use
  • Female
  • Follow-Up Studies
  • Humans
  • Immunotherapy
  • Male
  • Middle Aged
  • Myasthenia Gravis / enzymology
  • Myasthenia Gravis / immunology*
  • Myasthenia Gravis / pathology
  • Myasthenia Gravis / therapy*
  • Receptor Protein-Tyrosine Kinases / antagonists & inhibitors
  • Receptor Protein-Tyrosine Kinases / immunology*
  • Receptor Protein-Tyrosine Kinases / metabolism*
  • Receptors, Cholinergic / immunology*
  • Receptors, Cholinergic / metabolism*

Substances

  • Antibodies
  • Cholinesterase Inhibitors
  • Receptors, Cholinergic
  • MUSK protein, human
  • Receptor Protein-Tyrosine Kinases