Scleroderma

Description

Alternative Names

Other Treatments

Interferons. Interferons include agents that are used in hepatitis. Such drugs have helped reduce liver scarring. Early research is suggesting interferon gamma (e.g., Actimmune) may reduce scarring in systemic sclerosis. In one early study, five year survival was 85% for patients with diffuse systemic sclerosis who took interferon gamma. In addition, skin softening improved in 40%.

Tumor-Necrosis Factor Modifiers. Tumor-necrosis factor (TNF) modifiers are major breakthroughs in the treatment of rheumatoid arthritis. They are genetically engineered to interfere with specific components of TNF, a powerful immune factor. Researchers believe they should be tested in other inflammatory conditions, including scleroderma. The current agents include infliximab (Remicade), etanercept (Enbrel), and adalimumab (Humira).

Halofuginone. Halofuginone, a drug that inhibits the synthesis of collagen, is showing some promise in preventing scarring. The agent blocks production of certain collagen types involved in cell proliferation.

Minocycline. Very small studies are reporting gradual and progressive improvement in some patients who take low doses of the minocycline. Although this drug is an antibiotic, in low doses it has anti-inflammatory characteristics that may help inhibit skin symptoms.

Investigative Procedures

Blood Exchange (Plasmapheresis). Plasmapheresis is a process in which the fluid part of the blood, called plasma, is removed from blood cells. The procedure involves first withdrawing blood from the patient. The plasma, which contains the immunologically active substances, is discarded and replaced with other fluids. The blood is then returned. In a small 2001 study, this procedure appeared to slow down the course of severe progressive systemic sclerosis. Other studies are underway.

Autologous Stem-Cell Transplantation. Researchers are investigating possible benefit using transplantation of the patient's own stem cells (called an autologous transplantation). (Patients with autoimmune diseases cannot be given cells from donors.) Stem cells are the early forms for all blood cells in the body (including red, white, and immune cells). The transplant procedures introduce normal white blood cells that replace the abnormal autoimmune cells. The procedure has improved or stabilized systemic scleroderma in some patients, with remissions lasting up to four years. Results of a major study evaluating stem-cell transplants in severe scleroderma are expected in 2005.

Of note, there are significant risks with the procedure. The procedure is also not at all foolproof:

• Transplantation does not work in all patients.
• It cannot reverse all damage that may have been done during the disease process.
• And, importantly the treatment carries significant dangers. The mortality rate from the procedure itself is 10% in scleroderma patients, which is higher than in other patient groups who are given transplants.

Because the procedure has serious side effects, experts suggest that the best candidates would be those at high risk for complications from scleroderma. In general, such patients would have diffuse scleroderma whose first symptoms occurred within the previous three years and who have evidence of at least mild abnormalities in the heart, lungs, or kidney. In general, patients with advanced scleroderma would not be the best candidates, because their condition is usually stable. In such cases, the risks of the procedure would outweigh the risks from the disease.

Alternative Treatments and Diet

Some patients avoid high-fiber diets (which include fruits and vegetables) and so their diets may lack sufficient nutrients. Supplements, then, may be needed, but patients would do best to consult someone experienced in dietary conditions associated with scleroderma. Because of reports that oxygen free radicals may play a role in the development of sclerosis, some researchers recommend taking antioxidant supplements (e.g., selenium, beta-carotene, vitamin C, vitamin E, and methionine). Studies have not reported much benefit from such supplements. In general, however, there have not been long-term trials and some may have been started to late in the course of the disease to have much effect.

Because of the difficult of the disease, many patients are tempted to try high-dose supplements or other alternative treatments. It is very important to note that this route is not without its hazards.