TREATMENT & MEDICATIONS

Because everyone reacts to Scleroderma differently, identifying your disease subtype, stage, and involved organs is very important in determining the best course of action for treatment. Typically medications recommended for Scleroderma treatment focus on the four main features of the disease: inflammation, autoimmunity, vascular disease, and tissue fibrosis. While no two cases are identical, below lists some common treatment options:

  • Anti-Inflammatory Medications: there are two major types of inflammation that are related to the disease process. The first is a more conventional type that can cause arthritis (inflammation in the joints), myositis (inflammation in the muscles), or serositis [inflammation in the lining of the heart (pericarditis) or lining of the lung (pleuritis)]. This type of inflammation responds to traditional antiinflammatory drugs: NSAIDs (e.g. ibuprofen) or corticosteroids (e.g. prednisone). The other type of inflammation relates to the skin and other tissue injury caused by the scleroderma process. This phase of the disease does not appear to respond to NSAIDs or corticosteroids, although the exact role of corticosteroids is not fully studied. There are risks associated with the use of these agents, including gastrointestinal disease, fluid retention, and renal toxicity. 
  • Immunosuppressive Therapy: one of the most popular approach to controlling the inflammatory phase of scleroderma. This treatment focuses on the associated tissue damage and fibrosis caused by Scleroderma. There are several drugs that are being used, but only a few well designed studies have been performed. These immunosuppressing drugs include methotrexate, cyclosporine, antithymocyte globulin, mycophenolate mofetil and cyclophosphamide. A major area of current research is the use of aggressive immunosuppressive therapy either with very-high-dose cyclophosphamide or with autologous bone marrow transplantation.
  • Vascular Disease Drug Therapy: the vascular affects of scleroderma are widespread and affect medium and small arteries. The main result of this vascular affect is Raynaud's Disease, and there is evidence that repeated episodes of ischemia (low-oxygen state) occur in other tissues. Low blood flow into the skin and tissues is thought not only to damage tissue by the lack of nutrition and oxygen but to activate fibroblasts and promote tissue fibrosis. Therefore, treatment of the vascular disease is now considered crucial to controlling the disease as a whole as well as preventing specific organ damage. There are three major features of the vascular disease that potentially need treatment: vasospasm (spasm of blood vessels), a proliferative vasculopathy (thickening of blood vessels), and thrombosis (blood clots) or structural occlusion of the vessel lumen (blockage of blood vessels). Vasospasm is best treated with vasodilator therapy (drugs that open blood vessels). The most effective and popular vasodilator therapy continues to be the calcium channel blockers (e.g., nifedipine).
  • Anti-Fibrotic Agents: several drugs are used that have in vitro (in the tissue culture) ability to reduce collagen production or to destabilize tissue collagen. The older medications in this category include colchicine, para-aminobenzoic acid (PABA), dimethyl sulfoxide, and D-penicillamine. The search for new drugs that alter the fibrotic reaction is probably one of the most active areas of scleroderma research. Strategies include directly suppressing the fibroblast and its ability to make collagen, inhibiting the cytokines that activate the fibroblast, and the use of agents that might break down collagen faster and promote tissue remodeling.

Learn more at Johns Hopkins Scleroderma Center