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

Dejaco, C.; Ramiro, S.; Touma, Z.; Bond, M.; Soowamber, M.; Sanchez-Alvarez, C.; Langford, C.A. (2023)

Article / Letter to editor

Glucocorticoids (GCs) are the gold standard for treatment of giant cell arteritis (GCA); however, there is a need for studies on GC-sparing agents, given that up to 85% of patients receiving GC only develop adverse events. Previous randomised controlled trials (RCTs) have applied different primary endpoints, limiting the comparison of treatment effects in meta-analyses and creating an undesired heterogeneity of outcomes. The harmonisation of response assessment is therefore an important unmet need in GCA research. In this viewpoint article, we discuss the challenges and opportunities with the development of new, internationally accepted response criteria. A change of disease activity is a fundamental component of response; however, it is debatable whether the ability to taper GC and/or the maintenance of a disease state for a specific time period, as applied in recent RCTs, should be part of response assessment. The role of imaging and novel laboratory biomarkers as possible objective markers of disease activity needs further investigation but might be a possibility when drugs directly or indirectly influence the levels of traditional acute-phase reactants such as erythrocyte sedimentation rate and C reactive protein. Futures response criteria might be constructed as a multidomain set, but the questions about which domains will be included and what their relative weights will be still need to be answered.

• Giant Cell Arteritis
• Outcome Assessment
• Health Care
• Patient Reported Outcome Measures
• Biological Therapy

• All authors: Dejaco, C.; Ramiro, S.; Touma, Z.; Bond, M.; Soowamber, M.; Sanchez-Alvarez, C.; Langford, C.A.
• Date: 2023-02-17
• Journal: Annals of the Rheumatic Diseases
• Volume: 82
• Issue: 7
• DOI: doi:10.1136/ard-2022-223751