Promising and Upcoming Treatments in Myositis
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INFLAMMATORY MUSCLE DISEASE (L DIEDERICHSEN AND H CHINOY, SECTION EDITORS)
Promising and Upcoming Treatments in Myositis Lauren N. Smith 1 & Julie J. Paik 2
# Springer Science+Business Media, LLC, part of Springer Nature 2020
Abstract Purpose of Review To highlight new and emerging treatment targets in myositis. Recent Findings The landscape of novel therapeutics in myositis has vastly changed in the past 5 years. This is largely due to a greater understanding of the pathogenesis of myositis and validation of more robust outcome measures that standardize the ability to assess treatment response. Clinical trials in dermatomyositis are leading the way with ongoing multicenter, international phase 3 clinical trials. Proof-of-concept studies targeting the JAK/STAT pathway have also showed early promise in treating refractory dermatomyositis in adults and children. Summary This review highlights that the future armamentarium of therapeutic drugs will likely be larger and more selective in treating different subgroups of myositis. Keywords Myositis . Dermatomyositis . Immune-mediated necrotizing myopathy . Novel treatments
Introduction Idiopathic inflammatory myopathies (IIMs) are a heterogeneous group of autoimmune disorders characterized by immune-mediated muscle injury that results in muscle inflammation and skeletal muscle weakness. Myositis can be challenging to manage because it commonly encompasses extramuscular manifestations such as skin, lung, and joint involvement. The four main subgroups of myositis that have been identified are dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), polymyositis (PM), and sporadic inclusion body myositis (sIBM) [1, 2]. Overlap myositis including antisynthetase syndrome are increasingly being recognized as a separate entity [3]. While the classification criteria of myositis have evolved over the years, new treatments have lagged behind. Our current treatment modalities are based mostly on retrospective case series and case reports This article is part of the Topical Collection on Inflammatory Muscle Disease * Julie J. Paik [email protected] 1
Johns Hopkins University School of Medicine, 5200 Eastern Ave, MFL Building, Center Tower Suite 4100, Baltimore, MD, USA
2
Johns Hopkins University School of Medicine, 5200 Eastern Avenue, MFL Building, Center Tower Suite 4500, Baltimore, MD 21224, USA
with a paucity of randomized controlled clinical trials. However, with the recently developed and validated ACR/ EULAR Myositis Response Criteria [4] to assess treatment response coupled with advances in understanding the pathogenesis of myositis, there have been multiple promising treatments that we will highlight in this review. Before discussing new and emerging therapies, we will briefly discuss current treatments commonly used in clinical practice and the studies that have demonstrated their efficacy.
Current Treatments in Myositis While glucocorticoids are used in the acute management of myositis, steroid sparing agents are instituted simultaneously or early on the
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