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New treatment option investigated for difficult-to-treat muscle inflammation

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(Vienna, 10-07-2025) A research team at the Medical University of Vienna has systematically described the safety and efficacy of targeted immunotherapy in refractory idiopathic inflammatory myopathies (IIM) for the first time. The results of the retrospective observational study were published in the journal Rheumatology.

Idiopathic inflammatory myopathies are rare autoimmune diseases in which the immune system attacks the body's own muscle cells. The disease leads to persistent muscle weakness, limited physical capacity and can also affect internal organs such as the lungs or heart. Treatment usually involves anti-inflammatory drugs such as corticosteroids and other immunosuppressive substances. In some cases, however, the disease remains active despite these measures – this is referred to as a refractory course.

In the retrospective observational study now published by the MedUni Vienna research team led by Kastriot Kastrati and Helga Lechner-Radner (Division of Rheumatology, Department of Medicine III), the use of immune adsorption (IAS) was investigated in patients whose disease had not responded adequately to conventional therapies. Clinical data and treatment outcomes were analysed for a group of 23 patients who had received such therapy as part of their regular care between January 2000 and September 2021. Immunadsorption is a procedure in which harmful antibodies and inflammatory factors are specifically filtered out of the blood to reduce the excessive immune response. The evaluation was conducted retrospectively under everyday conditions and included clinical parameters such as muscle strength, laboratory values and information on tolerability.

This is the first systematic analysis of this form of treatment for therapy-resistant idiopathic inflammatory myopathies. The results show that the therapy was generally well tolerated. In some of the patients, signs of clinical improvement were observed, including on the basis of the IMACS response, an internationally used assessment system for recording disease activity in myositis. No serious side effects were documented, and mild to moderate side effects occurred in isolated cases. "Our study provides initial evidence of a potential therapeutic benefit in a patient group with very limited treatment options to date," the study authors say. The results form a basis for future controlled studies that could contribute to the further development of therapeutic approaches.

Idiopathic inflammatory myopathies are rare, with a prevalence of approximately 10 cases per 100,000 people in Europe. They mainly affect adults but can also occur in children. The disease is usually chronic and can become active in episodes. In severe cases, the lungs or heart may also be affected in addition to the muscles. Treatment aims to modulate the immune system and control inflammatory flare-ups. For patients who do not respond to established therapies, there is an urgent need for new, scientifically proven therapeutic approaches.

Publication: Rheumatology
Immunoadsorption as a novel therapy for refractory idiopathic inflammatory myopathies: a retrospective observational study.
Kastriot Kastrati, Thomas Karonitsch, Hanien Rajab, Roman Reindl-Schwaighofer, Farsad Eskandary, Sahra Pajenda, Daniel Mrak, Peter Maximilian Heil, Hans Peter Kiener, Michael Bonelli, Kurt Derfler , Daniel Aletaha, Josef S Smolen, Helga Radner.
https://doi.org/10.1093/rheumatology/keaf289