What is a response in randomised controlled trials in giant cell arteritis?

Area of Science:

• Rheumatology
• Clinical Trial Design

Background:

• Glucocorticoids (GCs) are standard for giant cell arteritis (GCA) but cause adverse events in up to 85% of patients.
• The need for GC-sparing agents in GCA is significant due to toxicity.
• Heterogeneity in primary endpoints across randomized controlled trials (RCTs) hinders meta-analyses and outcome comparisons in GCA.

Purpose of the Study:

• To discuss challenges and opportunities in developing internationally accepted response criteria for GCA.
• To explore the essential components of response assessment in GCA clinical trials.
• To identify potential objective markers for future GCA response criteria.

Main Methods:

• Viewpoint article discussing current unmet needs in GCA research.
• Analysis of existing RCT endpoints and their limitations.
• Consideration of novel assessment methods including imaging and biomarkers.

Main Results:

• Harmonization of response assessment is a critical unmet need in GCA research.
• Debate exists on including GC tapering and sustained remission in GCA response criteria.
• Imaging and novel biomarkers show potential as objective GCA disease activity markers.

Conclusions:

• Standardized, internationally accepted response criteria are essential for advancing GCA treatment research.
• Future GCA response criteria may incorporate a multidomain approach, but specific domains and weighting require further definition.
• Objective measures like imaging and biomarkers could enhance future GCA assessment, especially when influencing acute-phase reactants.