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Update on outcome assessment in myositis
Lisa G Rider1, Rohit Aggarwal2, Pedro M Machado3
1Environmental Autoimmunity Group, National Institute of Environmental Health Sciences, US National Institutes of Health, Bethesda, MD, USA.
Nature Reviews. Rheumatology
|April 14, 2018
Summary
Idiopathic inflammatory myopathies (IIMs) are autoimmune diseases causing muscle weakness. New validated measures and criteria for assessing IIM disease activity and treatment response are improving clinical trials and therapy development.
Area of Science:
- Rheumatology
- Immunology
- Neurology
Background:
- Idiopathic inflammatory myopathies (IIMs) encompass adult and juvenile myositis syndromes, characterized by muscle weakness and inflammation.
- These systemic autoimmune diseases necessitate standardized outcome measures for clinical research.
Purpose of the Study:
- To review advances in outcome assessment for idiopathic inflammatory myopathies (IIMs).
- To highlight the development and validation of core set measures and response criteria for IIMs.
- To discuss emerging objective measures and future directions in IIM research.
Main Methods:
- Development and validation of core set measures for IIM disease activity assessment.
- Creation and validation of composite response criteria for adult and juvenile IIM patients.
- Ongoing validation of muscle strength/function measures and development of quality of life instruments.
Main Results:
- Validated core set measures and composite response criteria exist for adult and juvenile IIMs, with distinct improvement thresholds.
- New measures for muscle strength, function, and quality of life are under development.
- Disease state criteria for inactive disease and remission are utilized in clinical trials.
Conclusions:
- Advances in outcome assessment, including objective measures like MRI and biomarkers, are crucial for IIM treatment development.
- International collaborations are facilitating progress in understanding and treating IIMs.
- Improved assessment tools will accelerate the development of novel therapies for patients with IIMs.
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