Biomarkers in rheumatology: promise and pitfalls

摘要

Recent advances in the development of novelrnimmunomodulatory therapies for various autoimmunernconditions have revolutionized both clinicalrnpractice and research in rheumatology. Thernimproved efficacy achieved, for example, withrnmacromolecule TNF inhibitors in rheumatoidrnarthritis (RA), has certainly increased our expectationsrnas regards the outcomes of therapy. Diseasernremission for RA patients is not only desirable, butrnis considered attainable. With ever-expandingrnnumbers of targeted therapies approved for thernclinic and more in development, key unmet needsrnincreasingly arise. The success of biologic agents inrnrheumatology has spawned considerable researchrninto additional potential targets and agents. Forrnnew drugs in early phases of development, is thererna way to define whether they have reasonable efficacy,rnacceptable tolerability, and are worthwhile ofrnfurther study without large and long clinical trials?rnWith the surfeit of putative therapies in the pipeline,rnthere are neither the resources nor thernpatients to test each one. Invariably, solutions tornsuch questions include discussions of biomarkers.rnFor example, is it possible to know which specificrnpatients are the most appropriate candidates forrnwhich particular agents? The newer agents havernpresumably distinct mechanisms of action, andrnwhile some patients have tremendous responses torntreatment, others fail to respond. Being able torndefine the subset of patients most likely to achievernbenefit and least likely to experience toxicity to arngiven type of agent a priori would be a tremendous