Outcome Measures in Inflammatory Myopathies

Challenges in Developing Outcome Measures for IIM

  1. Lack of universally standardized outcome measures that can accurately capture the full range of disease manifestations. Muscle
    weakness, extramuscular involvement (such as skin, lung, and joint involvement), and patient-reported outcomes all need to be considered.
  2. The varied ways in which IIM manifests and progresses in different patients make it challenging to develop outcome measures that are both precise and responsive.
  3. The rarity of the disease makes it difficult to collect sufficient data for validation, especially across different clinical and research settings.

Aims of the Outcome Measures Working Group:

  1. Developing Comprehensive Outcome Measures:
    Create and validate reliable, meaningful and responsive outcome measures that encompass both muscle, skin and extra-muscular
    manifestations, ensuring that the full spectrum of IIM is captured in clinical assessments and research trials.
  2. Harmonizing Outcome Measures Across Studies:
    Standardize outcome measures across clinical trials and observational studies to ensure comparability of results, facilitating more robust meta-analyses and evidence synthesis.
  3. Incorporating Patient-Reported or Centered Outcomes:
    Develop and promote the use of patient-reported or patient centered outcome measures (PRO/PCOs) to capture the impact of IIM on quality of life, fatigue, and physical function from the patient’s perspective.
  4. Promoting International Collaboration:
    Encourage collaboration between international research groups to establish large datasets for validating outcome measures and ensuring they are applicable to diverse populations.
  5. Refining Tools for Clinical Practice:
    Work on translating validated outcome measures into tools that can be easily used in routine clinical practice, helping physicians to better track disease activity and response to treatment in real-time.
  6. Ensuring Responsiveness:
    Focus on developing outcome measures that are sensitive enough to detect subtle changes in disease activity, allowing for more timely and appropriate adjustments to treatment plans.