Treatment of Diffuse Cutaneous Systemic Sclerosis with Biologics, Small Molecules and Stem Cell Transplantation: What Is the Evidence to Date?

Abstract

Purpose of review: To give an overview of the current evidence for new therapies in diffuse cutaneous systemic sclerosis. Treatment with biologics, small molecules, and hemopoietic stem cell transplantation is reviewed. Recent findings: It is encouraging to see the amount of therapeutic studies in systemic sclerosis increasing. Although the effect of some therapeutic options is disappointing, in the near future, new trials will be published and several studies are underway. Summary: Systemic sclerosis is a serious life-threatening disease for which there is no cure available to date. Considering all forms of new therapies, autologous hematopoietic stem cell transplantation is the most effective therapy for well-selected patients with diffuse subtype despite the highest treatment related mortality. The effect of biologics in systemic sclerosis is disappointing, although data of several big trials are expected soon and could change this view. Tyrosine kinase inhibitors have not shown positive effects in the past and were hampered by side effects. The results of the thyrosine kinase inhibitor nitedanib in interstitial lung disease associated to systemic sclerosis are expected soon. Future research should focus on outcome measurements, combination therapy and treatment strategies in early disease.