Disease-modifying treatment in systemic sclerosis: current status

To review evidence and best practice for current disease-modifying therapies for the treatment of systemic sclerosis.

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.

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.