Scleroderma in children

Abstract

Juvenile systemic scleroderma (jSSc) is a rare childhood disease. In the review period covered within this article, there were few reports devoted exclusively to it. In the past year, there was no breakthrough regarding pathogenesis, classification, or treatment. In the pediatric field, the proposed classification system for jSSc shows significant progress toward a definition of the pediatric population. It should allow better evaluation of this patient group. In adult systemic scleroderma, European efforts to establish indices for disease activity have been published. They should help to assess the disease activity in a standardized way and therefore enable earlier adequate treatment for patients. Biologic markers and new assessment methods for particular organ involvement help to determine disease activity/severity as well. There is still no effective disease-modifying treatment for systemic scleroderma and jSSc. The summarized data of the phase I/II autologous stem cell transplantation trial have been published. The first guidelines for autologous stem cell transplantation for jSSc are proposed. In organ-specific treatment, the introduction of bosentan, a dual endothelin receptor antagonist, to control pulmonary hypertension is a real gain in the therapeutic options. New methods to assess prognosis are evolving and need to be validated in a larger patient population.