Rheumatology Unit, Department of Medicine, Solna, Karolinska Institutet, Center for Molecular Medicine, Karolinska University Hospital, Stockholm, Sweden.
Curr Opin Rheumatol. 2017 Nov;29(6):652-659. doi: 10.1097/BOR.0000000000000430.
Idiopathic inflammatory myopathies (IIMs) are complex multisystemic autoimmune diseases. Glucocorticoids remain the cornerstone of treatment in IIM, and the benefit of additional immunosuppressors is still debated. A limited number of controlled clinical trials have been available to support treatment guidelines, but in the last year, several clinical trials have been published. In this review, the highlights of recently published and on-going clinical trials in IIM will be summarized and discussed.
Post hoc analyses of a large randomized controlled trial (RCT) suggested new predictive factors of response to rituximab in refractory IIM individuals. An international collaboration enabled the completion of a large RCT in early juvenile dermatomyositis that will orient first-line treatment in that population. New approaches are showing encouraging results in inclusion body myositis.
Recent advances in molecular mechanisms underlying IIM pathogenesis and the development of novel targeted therapies have influenced recent and on-going clinical research.
特发性炎性肌病(IIM)是一种复杂的多系统自身免疫性疾病。糖皮质激素仍然是 IIM 治疗的基石,而额外使用免疫抑制剂的益处仍存在争议。虽然有少数对照临床试验可以支持治疗指南,但在过去的一年中,已经发表了多项临床试验。在这篇综述中,我们将总结和讨论最近发表的和正在进行的 IIM 临床试验的重点。
一项大型随机对照试验(RCT)的事后分析提示了利妥昔单抗治疗难治性 IIM 患者的新的预测反应的因素。一项国际合作使早期儿童皮肌炎的大型 RCT 得以完成,这将为该人群的一线治疗提供指导。新的方法在包涵体肌炎中显示出令人鼓舞的结果。
IIM 发病机制的分子机制和新型靶向治疗的发展方面的最新进展影响了最近和正在进行的临床研究。