Casal-Dominguez Maria, Pinal-Fernández Iago, Mammen Andrew L
Muscle Disease Unit, Laboratory of Muscle Stem Cells and Gene Regulation, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, 50 South Drive, Room 1141, Building 50, MSC 8024, Bethesda, MD 20892, USA.
Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
Curr Treatm Opt Rheumatol. 2022 Dec;8(4):105-116. doi: 10.1007/s40674-022-00198-1. Epub 2022 Sep 28.
Autoimmune myopathies (IMs) are a group of diseases characterized by muscle weakness and inflammatory infiltrates on the muscle biopsy. 'However, muscle involvement is not always present and other organs and tissues (lung, skin, and joints) are commonly affected. The most accepted classification for IM includes dermatomyositis, sporadic inclusion body myositis, immune-mediated necrotizing myopathy, polymyositis, and overlap myositis. Seventy percent of the autoantibodies that IM patients have are myositis-specific autoantibodies (MSAs). Importantly, MSAs are associated with specific clinical phenotypes in IM patients. The therapy of IMs consists of glucocorticoids, immunosuppressants, biologic, and immunomodulatory drugs, but there is recent evidence that patients with some types of MSAs respond better to certain treatments. The purpose of this review is to summarize the IM treatment from an autoantibody perspective.
Each MSA is maybe associated with the activation of specific pathogenic pathways which can be effectively targeted by specific drugs. The last few years have shown multiple examples of successful treatments for each MSA group of patients as we will describe in the following paragraphs.
The adverse effects of the IM therapeutic agents and some patients' refractoriness call for a continued search for better and more targeted therapies. MSA groups should be considered for a treatment decision.
自身免疫性肌病(IMs)是一组以肌肉活检显示肌肉无力和炎性浸润为特征的疾病。然而,并非总是出现肌肉受累情况,其他器官和组织(肺、皮肤和关节)也常受到影响。IM最常用的分类包括皮肌炎、散发性包涵体肌炎、免疫介导的坏死性肌病、多发性肌炎和重叠性肌炎。IM患者中70%的自身抗体是肌炎特异性自身抗体(MSAs)。重要的是,MSAs与IM患者的特定临床表型相关。IMs的治疗包括糖皮质激素、免疫抑制剂、生物制剂和免疫调节药物,但最近有证据表明,某些类型MSAs的患者对特定治疗反应更好。本综述的目的是从自身抗体角度总结IM的治疗。
每种MSA可能与特定致病途径的激活有关,而这些途径可被特定药物有效靶向。过去几年中,针对每组MSA患者都有多个成功治疗的例子,如下文所述。
IM治疗药物的不良反应以及一些患者的难治性,都需要继续寻找更好、更有针对性的治疗方法。治疗决策应考虑MSA分组。