Therapy of autoinflammatory syndromes.
The therapy of autoinflammatory syndromes is an excellent example of the power of translational research. Recent advances in our understanding of the molecular and immunologic basis of this newly identified classification of disease have allowed for the application of novel, effective, targeted treatments with life-changing effects on patients. Although colchicine and TNF-alpha inhibitors are important therapies for specific autoinflammatory syndromes, the novel IL-1-targeted drugs are particularly effective for many of these diseases. Recently, the pharmaceutical industry has adopted a strategy of confirming the efficacy of new targeted drugs in often-ignored patients with orphan diseases, and US Food and Drug Administration policies have allowed for accelerated approval of these drugs, creating a win-win situation for patients and industry. This article reviews the general approach to the therapy of autoinflammatory diseases, focusing on current approved therapies and novel approaches that might be used in the future.
Hoffman
Hal M
HM
Department of Pediatrics and Medicine, Division of Allergy, Immunology, and Rheumatology, University of California at San Diego, School of Medicine, 9500 Gilman Dr, La Jolla, CA 92093-0635, USA. hahoffman@ucsd.edu
D018501:Antirheumatic Agents / Q000627:therapeutic use; D001323:Autoantibodies / Q000097:blood; D001327:Autoimmune Diseases / Q000188:drug therapy* / Q000276:immunology; D015551:Autoimmunity; D002986:Clinical Trials as Topic; D003078:Colchicine / Q000627:therapeutic use; D056660:Hereditary Autoinflammatory Diseases / Q000188:drug therapy* / Q000378:metabolism; D006801:Humans; D053590:Interleukin 1 Receptor Antagonist Protein / Q000627:therapeutic use; D053583:Interleukin-1beta / Q000276:immunology / Q000378:metabolism*; D013577:Syndrome; D050257:Tubulin Modulators / Q000627:therapeutic use; D014409:Tumor Necrosis Factor-alpha / Q000276:immunology / Q000378:metabolism*
D018501:Antirheumatic Agents; D001323:Autoantibodies; D053590:Interleukin 1 Receptor Antagonist Protein; D053583:Interleukin-1beta; D050257:Tubulin Modulators; D014409:Tumor Necrosis Factor-alpha; D003078:Colchicine