Clinical pearls and promising therapies in myositis

ABSTRACT

Introduction

Idiopathic inflammatory myopathies (IIMs) represent a diverse group of systemic autoimmune disorders with variable clinical manifestations and disease course. Currently, the challenges of IIMs are multifold, including difficulties in timely diagnosis owing to clinical heterogeneity, limited insights into disease pathogenesis, as well as a restricted number of available therapies. However, advances utilizing myositis-specific autoantibodies have facilitated the definition of subgroups as well as the prediction of clinical phenotypes, disease course, and response to treatment.

Areas covered

Herein we provide an overview of the clinical presentations of dermatomyositis, anti-synthetase syndrome, immune-mediated necrotizing myopathy, and inclusion body myositis. We then provide an updated review of available and promising therapies for each of these disease groups. We synthesize current treatment recommendations in the context of case-based construct to facilitate application to patient care. Finally, we provide high-yield, clinical pearls relevant to each of the subgroups that can be incorporated into clinical reasoning.

Expert opinion

There are many exciting developments on the horizon for IIM. As insights into pathogenesis evolve, the therapeutic armamentarium is expanding with many novel therapies in development, holding promise for more targeted treatment approaches.

KEYWORDS:

• Anti-synthetase syndrome
• clinical challenges
• clinical pearls
• dermatomyositis
• idiopathic inflammatory myopathies
• immune-mediated necrotizing myopathies
• inclusion body myositis
• promising therapies

Article highlights

• Highlight the key clinical features DM, ASyS, IMNM, and IBM.

• Guidance on IIM management strategies (Figure 1).

• Up-to-date summary of promising therapies in clinical trial development.

• Case-based approach to synthesize clinical features and treatment approaches to challenging cases of DM, ASyS, IMNM, and IBM.

• Clinical pearls relevant to each of the four cases (Figure 4).

Declaration of interest

J Paik reports consultant fees from Alexion, Riovant, ArgenX, EMD-Serono, Pfizer, Kezar, and Guidepoint; clinical trial research support from Alexion, Pfizer, and Kezar. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This work was supported by the Jerome L. Greene Foundation Discovery Fund (Connolly, Paik) and grant number K23AR073927 (Paik) from the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS).