What is scleroderma?

Scleroderma, which means “hard skin,” is a relatively rare autoimmune disorder that causes specific tissues (skin, muscles, or blood vessels) in the body to become hard, tight, or thick.

The cause of scleroderma is unknown. What is known is that scleroderma causes an autoimmune response in the body (where the body’s immune system is causing inflammation) that causes an overproduction, or buildup, of collagen, a fibrous protein making up the connective tissues. In the case of scleroderma, the inflammation occurs in the connective tissue which can lead to scarring, thickening, or tightening of the skin. It can also cause other problems with organ function (lungs, heart, kidneys). Scleroderma affects women more often than men, and occurs most frequently between the ages of 30 to 50, though it’s been identified in the very young (pediatric scleroderma) and the elderly.

Rheumatologists are specialists in the diagnosis and treatment of scleroderma.

Types of scleroderma

Localized (or limited) scleroderma

Usually involving only a few patches of hardening or tightening in places on the skin and possibly muscle, and usually changes in only a few places on the skin or muscles and rarely spread elsewhere. Localized subtypes of scleroderma include:

  • Linear scleroderma
  • Morphea

Systemic scleroderma or systemic sclerosis (SSc)

About one third of patients have a more serious “systemic” (spread throughout the body) form of scleroderma which can affect the blood vessels, lungs (interstitial lung disease, pulmonary hypertension), heart, kidneys, or digestive tract (esophagus, intestines), as well as the skin. Subtypes of systemic scleroderma include:

  • Diffuse cutaneous systemic sclerosis
  • Limited cutaneous systemic sclerosis (including CREST syndrome)
  • Limited Systemic sclerosis

The effects of scleroderma can be mild, moderate, or serious (life-threatening). The seriousness depends on the part of the body that are affected and the extent to which they are affected.

Treatment

Different medications are used to treat the symptoms of scleroderma, however, there is no drug that has been proven to reverse, or stop, the disease. Mild to moderate scleroderma can be successfully treated for different symptoms including: Reynaud’s disease, heartburn, kidney disease, muscle pain or weakness, using a variety of medications. Cyclophosphamide, a chemotherapy, has also shown short-term benefits, as well as mycophenolate mofetil/cellcept, an immunosuppressant. However, for many patients, SSc remain a fatal disease.

Stem cell transplant for scleroderma (SSc)

Stem cell transplant can also help people with severe scleroderma. A phase 2 study of systemic sclerosis published in Blood 2007 showed a significant decrease in skin fibrosis after stem cell transplant. A more recent clinical phase 3 trial published in the New England Journal of Medicine showed that myeloablative (chemotherapy plus radiation plus immunosuppression) stem cell transplant was more effective treatment than cyclophosphamide therapy.

CBCI offers treatment with high-dose chemotherapy and autologous stem cell transplant to patients with systemic sclerosis who meet criteria for this treatment. If you think you meet the eligibility criteria, please contact us.

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