ABSTRACT
Systemic autoimmune rheumatic diseases are a clinically heterogeneous group of inflammatory disorders, making them ideal candidates diseases for a stratified approach to treatment. Progress in the field of biomarker discovery has been motivated by the need for newer approaches to optimize drug efficacy and to identify those patients who are most likely to derive clinical benefit.
The past two decades has witnessed a paradigm shift in the treatment of rheumatoid arthritis (RA), moving away from synthetic disease-modifying drugs toward targeted biological therapy. This expansion in the range of treatment options now drives biomarker discovery to aid therapeutic decision making and early use of the most suitable therapy for an individual. A number of early successes have given optimism for the many additional studies that are underway.
In contrast, drug discovery in connective tissues diseases (CTDs) has been disappointing, with only one new licensed agent in 60 years. Post hoc analysis of many “failed” trials has, however, demonstrated important differences in clinical response between subgroups of patients. Targeting specific immunopathogenic subsets is likely to improve the success of future trials and widen the choice of novel agents available for patients.
This section summarizes the breadth of current studies that are at the cornerstone of stratified medicine in RA and CTDs.
