Clinical endpoints in myositis: challenges and ways forward

Area of Science:

• Rheumatology
• Clinical Trials
• Outcome Measures

Background:

• Myositis clinical trials face challenges in comprehensively assessing disease activity.
• Traditional outcome measures like the Total Improvement Score (TIS) inadequately capture skin involvement, particularly in dermatomyositis (DM).

Purpose of the Study:

• To review advances in clinical endpoints for myositis, focusing on comprehensive assessment of muscle and skin disease activity.
• To highlight the implications of new outcome measures for clinical trial design and patient inclusivity.

Main Methods:

• Review of recent studies and clinical trial data.
• Analysis of traditional and novel outcome measures, including the Cutaneous Disease Area and Severity Index (CDASI) and Dermatomyositis Outcomes for Muscle and Skin (DMOMS).

Main Results:

• Traditional measures like TIS have limitations for skin-predominant DM.
• Skin-specific measures (CDASI) and composite measures (DMOMS) offer a more balanced assessment of disease activity.
• Clinical trial data show benefits using these novel measures in both classic and amyopathic DM.

Conclusions:

• Advancements in outcome measures are vital for inclusive and effective myositis clinical trials.
• Incorporating comprehensive tools like DMOMS improves assessment of muscle and skin disease activity.
• This shift is essential for addressing the needs of all idiopathic inflammatory myopathy patients, including those with skin-predominant DM.