Scleroderma is a rare autoimmune disease where the body’s immune system targets healthy connective tissue. This triggers a damage response that leads to the overproduction of the protein collagen, a major component of scar tissue. This results in the characteristic patches of tight and hard scar-like skin seen in scleroderma patients.
In more severe cases, scleroderma can cause scarring in internal organs, preventing their normal function and leading to a wide range of symptoms.
What are immunosuppressants?
There is currently no cure for scleroderma. However, there are therapies available to help manage the symptoms. Immunosuppressants are a type of medication that help reduce the symptoms of scleroderma. They act to reduce or prevent the immune response from occurring.
Types of immunosuppressants
Most immunosuppressants are nonselective, meaning that they do not target a specific part of the immune response. Instead, they act to either trigger the death of immune cells or prevent the production of more immune cells.
There are also therapies that can act to alter the immune response in a targeted manner. For example, rituximab acts to reduce levels of B-cells, a type of immune cell that can be involved in systemic sclerosis, by targeting a specific protein expressed by the cells.
Immunosuppressants are not specifically approved to treat scleroderma, but they are often prescribed off-label. Examples of immunosuppressants used to treat scleroderma include methotrexate, CellCept (mycophenolate mofetil), Cytoxan (cyclophosphamide), Imuran (azathioprine), cyclosporine, and Thymoglobulin (anti-thymocyte globulin).
Clinical trials
Currently, there are few large randomized controlled clinical trials to confirm the effectiveness of immunosuppressants for scleroderma. However, evidence from observational studies suggests that they may have clinical benefits.
For example, CellCept may be prescribed off-label as observational studies suggest it may improve skin symptoms and lung function in scleroderma patients.
Results from a large-scale scale observational study, published in the Annals of Rheumatic Diseases, suggest that immunosuppressants may be effective in early systemic scleroderma patients, but the benefit is reduced after 12 years of treatment.
Other information
Immunosuppressants may require a period of time before improvements are seen.
Because immunosuppressants prevent the normal function of the immune response, patients have an increased risk of infections. Side effects can vary between medications, but common ones can include stomach pain, diarrhea, nausea or vomiting, hair loss, and low blood cell counts.
Use of some immunosuppressants may increase the risk of developing some cancers.
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Scleroderma News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.