Small molecules in idiopathic inflammatory myopathies: a systematic review and a multicenter case series about Janus kinase inhibitors and apremilast

Reumatismo. 2025 Jan 13. doi: 10.4081/reumatismo.2025.1718. Online ahead of print.

Abstract

Objective: Idiopathic inflammatory myopathies (IIM) are rare autoimmune diseases that primarily affect striated muscles; skin, joints, and lungs may be involved with different degrees of severity. Traditional treatment relies on high-dose glucocorticoids and conventional synthetic disease-modifying antirheumatic drugs.

Methods: A growing amount of evidence is demonstrating the potential role of novel treatments in the management of IIM. We report our experience with Janus kinase inhibitors (JAKi) in these conditions and review the current evidence for the use of small molecules in real-life clinical practice.

Results: A total of 41 papers were retrieved from PubMed, 37 papers concerning IIM and JAKi, and 4 papers concerning IIM and apremilast.

Conclusions: An overall good efficacy was evidenced in IIM-associated skin lesions, including rash, ulcers, and calcinosis. If present, muscle and joint involvement demonstrated a good response to therapy, while it was not possible to draw any conclusion about dysphagia. No life-threatening adverse events were reported.