Diagnosis and treatment of systemic and localized scleroderma

Abstract

Scleroderma or progressive systemic sclerosis is diagnosed clinically by typical features of skin thickening, Raynaud’s phenomenon and visceral organ involvement, and serologically by distinct autoantibody subsets. These differentiate the disease into the ‘limited’ and ‘diffuse’ variants. In addition, a distinct form of scleroderma, termed ‘localized’ scleroderma, is characterized by skin thickening in the absence of visceral involvement. Treatment of scleroderma in the past was largely symptomatic and with immunosuppressives, acting against the organ system involved and the aberrant immune system. Recently, with newer insights into disease pathogenesis, drug therapies targeting the pathogenetic mechanisms of fibrosis, vasculopathy and autoimmunity are being evolved. Some of these newer therapies are the endothelin receptor blockers, phosphodiesterase inhibitors, tyrosine kinase inhibitors and autologous stem cell transplant, while others are still evolving. They may hold the key to improved future outcome of this disease, which was once thought to be potentially incurable.