Scleroderma - Highlights

Highlights

Overview

• Scleroderma is a rare disease marked by the following:
  • Damage to the cells lining the walls of small arteries
  • An abnormal buildup of tough scar-like tissue in the skin

• To date, there is no cure and treatment aims to reduce symptoms.

• Treatment remains a challenge for systemic scleroderma. Localized scleroderma carries a good prognosis and normal lifespan.

• The cause and course of the disease is unclear, and more research is needed to assess treatment options.

Treatment

• Clinical trials are underway for Imatinib mesylate (Gleevec), an FDA approved cancer drug, for a first line treatment for scleroderma. It has shown promising results so far in treating the treatment of the scleroderma disease process, rather than the symptoms. This would be a major advancement in the treatment of scleroderma.

  Bosentan, an endothelin receptor antagonist, is currently under study in the US for systemic scleroderma. It is already approved for the treatment of pulmonary hypertension. The drug was approved in Europe in 2007 for the treatment of skin ulcers related to scleroderma.

• New therapies that are under study include stem cell transplants, immunosuppressive agents and other medications.

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Genetic Research

• Research has demonstrated that systemic sclerosis is an autoimmune disorder with a polygenic (involving more than one gene) basis.
• Several genes and gene-gene interactions have been identified as playing a role in systemic sclerosis. Many of these same genes are also linked to related diseases such as rheumatoid arthritis and systemic lupus erythematosus.

New Guidelines

• In 2009, the European League Against Rheumatism (EULAR) with experts from Europe, USA and Japan, developed 14 evidence-based recommendations for the treatment of Scleroderma. These guidelines will help guide rheumatologists in their treatment of patients with the disease.

Review Date: 01/04/2011
Reviewed By: Harvey Simon, MD, Editor-in-Chief, Associate Professor of Medicine, Harvard Medical School; Physician, Massachusetts General Hospital. Also reviewed by David Zieve, MD, MHA, Medical Director, A.D.A.M., Inc.

A.D.A.M., Inc. is accredited by URAC, also known as the American Accreditation HealthCare Commission (www.urac.org).