Therapy of myasthenia gravis: looking forward to the era of targeted immunotherapy / 中华神经科杂志

ABSTRACT

Myasthenia gravis is mainly acetylcholine receptor antibody-mediated, T cells-dependent and complement participated acquired autoimmune disease characterized by impairment of the neuromuscular transmission. The main clinical feature of the disease is the presence of fatigability or muscle weakness. Most patients can be successfully managed with nonspecific immunotherapies such as corticosteroid and non-steroidal immunosuppressants. However, the side effects caused by long-term corticosteroid therapy are still a hurdle in the treatment of myasthenia gravis (MG). Oral non-steroidal immunosuppressants, as add-on therapy, can greatly reduce the relapse of the disease, but some drugs have a slow onset of action and the potential for significant toxicity, and even increase the risk of infection and neoplasms with long-term treatment. Despite these therapies, a minority of patients can be refractory because of incompletely responding or not well tolerated to available therapies. Thus, the need to avoid the use of corticosteroids, or at least reduce their use as much as possible should concern all patients with MG. Targeted immunotherapy is a therapeutic monoclonal antibody or antibody fragment targeting immune cells, complement, neonatal Fc receptor and cytokines. Recently, targeted immunotherapy has completed phase Ⅱ and Ⅲ clinical trials in patients with MG, and some of them have been approved by Food and Drug Administration. These promising biologics showed efficacy in symptoms persistent improvement, steroids reduction and were well tolerated, now evolving into powerful tools changing the algorithm of MG. This paper summarizes the results of clinical trials of new biologics in MG and looks forward to the prospect of MG treatment.