[New treatment options of myasthenia gravis, new approach to biological agents, reimbursement of new therapies in Hungary].

Abstract

Myasthenia gravis (MG) is an antibody-mediated disorder of the neuromuscular transmission, presenting with fatigable weakness that is either isolated to ocular muscles only or generalised (limb, bulbar and respiratory muscles can be affected). The disorder is estimated to affect around 1700 patients in our country. In some cases symptomatic treatment with acetylcholinesterase inhibitors may be sufficient, but most patients with MG require immunosuppressive drugs at some point for disease control, which is achieved in about 60-70% of patients. 10-20% of MG cases are refractory; they do not respond adequately to the traditional treatments or suffer from severe side effects. In the past few years, new biological agents against complement, the FcRn receptor, or B-cell antigens have been tested in clinical trials. These new therapies extend the possibilities for targeted immunotherapies and promise exciting new options with a relatively rapid mode of action. The goal of the treatment of MG is achieving remission, avoiding myasthenic crisis and eliminating symptoms which worsen the quality of life of our patients. The disease is rare, the clinical picture is variable, therefore the treatment of MG patients, especially of refractory MG should be conducted in neuroimmunological centres with adequate expertise in MG. For the adequate treatment of refractory MG we need the new drugs to be reimbursed in our country.