Disease-modifying treatment in systemic sclerosis: current status.
OBJECTIVE
To review evidence and best practice for current disease-modifying therapies for the treatment of systemic sclerosis.


RESULTS
Cyclophosphamide remains the treatment of choice for lung disease and severe skin disease associated with systemic sclerosis. Methotrexate is the treatment of choice for scleroderma overlap syndromes, whereas mycophenolate and azathioprine are also used for both skin and lung disease, alone or for maintenance therapy after cyclophosphamide induction. Haematopoietic stem cell transplantation and imatinib look promising, but trial results are awaited. Relaxin is contraindicated due to inefficacy and severe renal side effects on discontinuation of the drug. Tolerance to type I collagen may be a useful treatment in a carefully selected group of patients. Further trials are needed for biological agents such as infliximab, rituximab and intravenous immunoglobulin.


CONCLUSIONS
Although there is still no treatment that is well tolerated and unequivocally effective currently for systemic sclerosis, we have come a long way in the past number of years with respect to identifying possible treatments and new therapeutic targets. A number of novel agents including antiinterleukin-6, transforming growth factor-beta-directed therapies and other novel biological agents such as hyperimmune caprine serum are being developed based on new insights into the pathophysiology of disease.
Quillinan
Niamh P
NP
Centre for Rheumatology and Connective Tissue Diseases, Royal Free Hospital, London, UK.
Denton
Christopher P
CP
D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D058846:Antibodies, Monoclonal, Murine-Derived; D001324:Autoantigens / Q000008:administration & dosage; D001379:Azathioprine / Q000627:therapeutic use; D024042:Collagen Type I / Q000276:immunology; D055513:Connective Tissue Growth Factor / Q000037:antagonists & inhibitors; D003520:Cyclophosphamide / Q000627:therapeutic use; D055317:Evidence-Based Practice; D005355:Fibrosis / Q000188:drug therapy; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000627:therapeutic use; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008727:Methotrexate / Q000627:therapeutic use; D009173:Mycophenolic Acid / Q000031:analogs & derivatives / Q000627:therapeutic use; D010956:Plasmapheresis; D000069283:Rituximab; D012595:Scleroderma, Systemic / Q000276:immunology / Q000628:therapy*; D016212:Transforming Growth Factor beta / Q000037:antagonists & inhibitors; D014182:Transplantation, Autologous; D014409:Tumor Necrosis Factor-alpha / Q000037:antagonists & inhibitors
D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D001324:Autoantigens; C526317:CCN2 protein, human; D024042:Collagen Type I; D016756:Immunoglobulins, Intravenous; D007166:Immunosuppressive Agents; D016212:Transforming Growth Factor beta; D014409:Tumor Necrosis Factor-alpha; D055513:Connective Tissue Growth Factor; D000069283:Rituximab; D003520:Cyclophosphamide; D009173:Mycophenolic Acid; D001379:Azathioprine; D008727:Methotrexate