Treatment Overview

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”.
  • 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 three 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 – this is an experimental treatment that uses the patient’s own stem cells to essentially restart their 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 HSCT. One of the primary risk factors for treatment mortality is heart involvement, so patients that 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.
  • Blood Hyperviscosity / Plasmapheresis – this treatment approach stems from a number of research studies that have consistently documented that Scleroderma patients tend to have elevated blood viscosity (“thickness”) arising from 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 plasmapheresis eliminates red blood cell clumping for a number of months and also leads to significant symptom improvement. While plasmapheresis is expensive (but is covered by Medicare and many insurance companies), it does not have the risks that are inherent in treatments that suppress the immune system for a long period of time. The website includes a lengthy article on the topic of Scleroderma and blood hyperviscosity that explores the research on this topic in depth. This article is titled Scleroderma Blood Hyperviscosity – Implications for Research and Treatment and is located in the Technical Articles section of the website.

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