Treatment Overview

Guide

Scleroderma is not currently considered a curable disease. Most scleroderma treatments fall into two general categories:

  • Treatments for specific clinical symptoms such as heartburn, difficulty swallowing, Raynaud’s, digital ulceration, kidney problems, etc., can often be fairly effective, especially during the earlier stages of the disease. The specific treatments for most potential scleroderma-related clinical symptoms are covered in great detail in the Scleroderma FAQ in the section titled “Treatments: Specific Symptoms”.

  • Immune system regulation/suppression – Since scleroderma is considered an autoimmune disease where the symptoms ultimately develop as a result of the immune system creating antibodies that attack healthy cells, it is very common for physicians to try to reduce these systemic effects by using drugs that suppress the overall immune system. There are a number of different drugs that are used, including a number of different immunosuppressant drugs normally used for treating cancer. Some research studies show modest short-term improvements from using these drugs, including a reduction of some symptoms resulting in an improved quality of life for patients. However, there are no data that indicates immunosuppressant treatments increase overall longevity. The Scleroderma FAQ includes a list of commonly used immunosuppressant medications, including their major side effects, in the section titled “Treatments – General: Standard / Multi-Symptom”.

In addition, there are two other experimental / alternative treatment approaches that are discussed in some detail in the Scleroderma FAQ in the section titled “Treatments – General: Research-Based Experimental / Alternative”. These include:

  • Autologous Stem Cell Transplants (sometimes called hematopoietic stem cell transplants and abbreviated HSCT) – This is an experimental treatment that uses the patient’s own stem cells essentially to restart his/her immune system. Autologous stem cell transplant is a complex procedure, and there is definite risk associated with the procedure itself.  However, mortality rates are now much lower than in the initial studies as researchers have learned to better screen patients for this procedure.  One of the primary risk factors for treatment mortality is heart involvement, so patients who receive HSCT as part of these studies are screened carefully for potential existing cardiac problems before being accepted into the study.

    It is far too early to know how long HSCT therapy will last even if successful in the short-term.  However, the preliminary data suggest that this treatment approach may result in initial symptom improvement and improved five-year survival rates for patients with diffuse scleroderma.  This suggests that for some patients with early-stage, rapidly-progressing diffuse scleroderma, enrolling in an ongoing HSCT research study may be an appropriate option to consider.

  • Therapeutic Plasma Exchange – This treatment approach stems from a number of research studies that have consistently documented that scleroderma patients tend to have elevated blood viscosity (“thickness”), including excessive clumping of the red blood cells.  Some researchers have speculated that all scleroderma symptoms occur as a result of repeated damage to the cells that line the smallest blood vessels (microcapillaries), which could theoretically be caused by these clumped red blood cells. A number of pilot research studies have demonstrated that a procedure called therapeutic plasma exchange (TPE; sometimes called plasmapheresis) eliminates red blood cell clumping for a number of months and also leads to significant symptom improvement.  While TPE is fairly expensive (comparable to biologic drugs commonly used to treat patients with rheumatoid arthritis or lupus), it does not have the risks that are inherent in treatments that suppress the immune system for a long period of time.  Medicare does cover TPE treatments for treating systemic scleroderma if the disease is life threatening and the patient is not responding to other treatments.  Some insurance companies follow Medicare guidelines and are likely to cover TPE as well.

    There is a detailed discussion of therapeutic plasma exchange in the Research section of the Scleroderma Education Project website.

Baseline Testing
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Baseline Testing
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