Internal Medicine Residency Program, University of Connecticut , Farmington, CT, USA.
Department of Medicine, Rheumatology and Clinical Immunology, University of Pittsburgh , Pittsburgh, PA, USA.
Expert Opin Emerg Drugs. 2020 Sep;25(3):213-228. doi: 10.1080/14728214.2020.1787985. Epub 2020 Jul 10.
Currently, there are no proven drugs that are FDA approved for the treatment of dermatomyositis (DM), even though multiple clinical trials are ongoing to evaluate safety and efficacy of novel therapeutics in DM. The purpose of this review is to highlight the biological plausibility, existing clinical evidence as well as completed and ongoing clinical trials for various drugs in pipeline for development for use in dermatomyositis.
The drugs with the strongest evidence have been included in this review with a focus on the mechanism of their action pertaining to the disease process, clinical studies including completed and ongoing trials. With better understanding of the underlying pathophysiologic process, there are new molecular targets that have been identified that can be targeted by these novel drugs, predominantly biologic drugs.
There are various drugs being evaluated in phase II/III clinical trials that hold promise in DM. At the forefront of these are immunoglobulin, Lenabasum, and Abatacept for which phase III clinical trials are ongoing. In addition, promising clinical studies are ongoing or reported for KZR-616, anti-B cell therapy, anti-interferon drugs, and Repository Corticotrophin Injection (RCI).
目前，尚无经美国食品药品监督管理局批准的治疗皮肌炎（DM）的药物，尽管正在进行多项临床试验以评估新型治疗药物在 DM 中的安全性和疗效。本文旨在强调各种处于研发管道中的药物在皮肌炎中的生物学合理性、现有临床证据以及已完成和正在进行的临床试验。
本文纳入了具有最强证据的药物，重点介绍了其作用机制与疾病过程的相关性，包括已完成和正在进行的临床试验。随着对潜在病理生理过程的更好理解，已经确定了可以通过这些新型药物靶向的新分子靶点，主要是生物药物。
有各种药物正在进行 II/III 期临床试验，这些药物在 DM 中具有广阔的应用前景。处于这一领域前沿的是免疫球蛋白、Lenabasum 和 Abatacept，目前正在进行 III 期临床试验。此外，正在进行或已报告 KZR-616、抗 B 细胞治疗、抗干扰素药物和促肾上腺皮质激素释放因子注射剂（RCI）的有前途的临床研究。
