While there is presently no cure for Scleroderma, there are several treatment options available that can enable those afflicted with the disease to alleviate some of the symptoms that it causes. Such treatments depend on the subtype of the disease, the stage, and the affected organs, since no two cases of Scleroderma are exactly alike. The current therapies focus on the four main complications of the disease, which include autoimmunity, inflammation, vascular disease, and tissue fibrosis.
In order to address the autoimmunity issue, immunosuppressive therapy is most often used. Researchers believe that the autoimmune process is causing inflammation, resulting in tissue damage and fibrosis. They think that the fibrosis is an "innocent bystander" that is being driven by the cytokines (chemical messengers) that are produced by the immune system. There have only been a few well designed studies into this type of therapy, which is one reason why a $1 million/year research budget is not nearly enough. However, several drugs are currently being used to suppress the immune system. These immunosuppressing medications include methotrexate, cyclosporine, antithymocyte globulin, mycophenolate mofetil and cyclophosphamide. A recent study suggested that methotrexate did not significantly alter the skin score (a measure of skin thickening) compared with placebo. Cyclosporine has not been completely studied because of reports of renal toxicity. The most promising drugs are mycophenolate mofetil or cyclophosphamide. Unfortunately, there is no placebo-controlled study to define their exact role in treating scleroderma, however, if used during the active inflammatory phase of the disease, they seem to be effective. In very severe cases of Scleroderma, the use of aggressive immunosuppressive therapy with very high doses of cyclophosphamide or with autologous bone marrow transplantation can be used, however, this treatment has potential risks.
The complication of inflamation can come in two forms in a patient with Scleroderma. The more conventional type can cause arthritis (inflammation in the joints), myositis (inflammation in the muscles), or serositis (inflammation in the lining of the heart or the lining of the lung). The most often used drug therapy for this symptom is a traditional anti-inflammatory drug, such as NSAIDs, like ibuprofen, or corticosteroids, like prednisone. The other type of inflammation relates to the skin and other tissue injury caused by the Scleroderma process. This type does not seem to respond to NSAIDs or corticosteroids. These medications also have serious potential side effects, including gastrointestinal disease, fluid retention, and renal disease. Prednisone is also known to cause bone weakening, which is why my mother's ribs collapsed onto her lungs just weeks before she passed.
The vascular disease caused by Scleroderma affects both the medium and small arteries. Low blood flow into the skin and tissues is believed not only to damage tissue by the lack of nutrition and oxygen but also 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). Calcium channel blockers, like nifedipine, a vasodilator (opens up the blood vessels) is effective in helping blood flow to the skin and the heart. Epoprostenol improves blood flow in the lungs. There is evidence that these vasodilators directly affect tissue fibrosis. Left untreated, scleroderma vascular disease can lead to thrombosis or advanced fibrosis, causing the occlusion of the vessels. To deter this, antiplatelet or anticoagulation therapy using low-dose aspirin is recommended.
For the fourth main complication of the disease, tissue fibrosis, there really is no good therapy that can be offered currently. In Scleroderma, excess collagen is produced in the skin and other organs. Several medications were being used, such as colchicine, para-aminobenzoic acid (PABA), dimethyl sulfoxide, and D-penicillamine, however, experts are not convinced of their effectiveness. The search for new drugs that alter the fibrotic reaction is currently 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.
Much more research is crucial to, not only discovering new therapies to combat the symptoms of the disease, but, ultimately to find a cure.
For more information, read
http://www.hopkinsscleroderma.org/patients/scleroderma-treatment-options/
http://www.hopkinsscleroderma.org/downloads/SystemicSclerosis_Chapter23.pdf
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