This section of the Scleroderma FAQ is focused on standard treatment approaches that are designed either to target the overall disease process or to modify the disease in a way that can potentially improve more than one symptom, for example, bosentan (Tracleer) for skin ulcers and PAH. It is important to understand that no current conventional treatment is effective in stopping or reversing the overall course of systemic scleroderma. A number of medications have been demonstrated in well-designed scientific studies either to slow down the progression of specific existing symptoms or to reduce the development of new symptoms, at least in the short term.
However, while studies that have looked at changes in long-term survival rates for scleroderma patients over the past few decades show significant improvement over this time period, they do not directly demonstrate that any of these standard treatments are significantly improving long-term scleroderma patient survival. Instead, the improvements in patient longevity may be more a result of overall improvements in longevity in the general population, presumably as a result of improved health care and nutrition.
Immunosuppressant / Disease Modifying Medications
In addition to medications that are used to treat individual symptoms (thes are covered below), a number of different medications used in treating systemic scleroderma patients are designed to interrupt the disease process in a variety of ways. Since systemic scleroderma is considered an autoimmune disease, some of these drugs are designed to suppress the entire immune system, thereby (hopefully) reducing the disease level and slowing or stopping disease progression, for example, cyclophosphamide (Cytoxan). Others target specific aspects of the disease, such as the mechanisms involved in skin fibrosis. An example would be imatinib mesylate (Gleevec). A third category involves medications that are used to “regulate” the immune system, such as hydroxychloroquine (Plaquenil).
Potential Side Effects of Scleroderma Medications
It is very important for scleroderma patients who are exploring treatment options with their physicians to understand that many of these treatments are themselves toxic or have the potential of leading to serious side effects, either short-term or long-term. There is a clear trade-off about which patients need to be aware in order to make an informed decision as to whether or not to start a particular medication.
Also, even for patients with the same formal diagnosis, for example, anti-SCL70 positive diffuse scleroderma, there is wide variation in disease symptoms and progressions within that subset of patients. This means that it is critical for scleroderma patients to work with physicians who are knowledgeable about using these medications before starting treatment. At a minimum, many of these medications require close monitoring for potential side effects to prevent the development of problems that may be difficult to treat.
One issue that patients will discover if they review the current literature on standard scleroderma medications is that there is little consistency on how these medications are described and categorized. For example, in many articles, methotrexate is classified as an immunosuppressant drug. In other articles, it is put into a category called DMARD (disease-modifying anti-rheumatic drugs). This distinction is important for researchers but for patients it is best to understand what these medications are supposed to do, how this fits into the overall treatment plan that the patient and their physicians are using, and how to balance the potential gains from using these medications against the potential for (in some cases very significant) side effects and risks.
Table 3 below lists many of the currently used mainline medications for treating systemic scleroderma. The information presented varies widely in the literature and represents the author’s best effort to summarize current research literature.
Table 3: Immunosuppressant / Disease Modifying Medications
Generic Name |
Brand Names |
Targeted Symptoms |
Potential Side Effects |
Additional Notes |
|---|---|---|---|---|
| Azathioprine | Imuran Azasan |
ILD | Serious: increased susceptibility to infections and lymphoma. Patients need to be closely monitored. | Primary usage is to suppress the immune system to help prevent transplanted organ rejection. Considered less effective than cyclophosphamide, often combined with low doses of corticosteroids. |
| Cyclophosphamide | Cytoxan Neosar |
ILD | Severe: including hair loss, high blood pressure, kidney and liver problems, reduced ability to fight infections, increased risk of some forms of cancer. Patients need to be closely monitored. |
Anti-cancer drug, suppresses the immune system. Studies show modest improvement in lung functioning. |
| Cyclosporine | Neoral Sandimmune Restasis Gengraf |
Skin fibrosis | Severe: requires close monitoring for high blood pressure and potential major kidney problems | Immunosuppressant that is commonly used for treating rheumatoid arthritis. Limited effectiveness in Scleroderma. |
| D-penicillamine | Cuprimine Depen |
Skin fibrosis | Moderate: many drug interactions. Can cause serious birth defects if taken during pregnancy. Close monitoring is needed. | Classified as a disease modifying anti-rheumatic drug (DMARD) used primarily to treat patients with rheumatoid arthritis. Research suggests limited benefit. |
| Hydroxychloroquine | Plaquenil | Fatigue, joint pain | Mild: mostly GI symptoms, except for serious eye problems with chronic use at high dosages | Antimalarial drug, frequently used to treat lupus and rheumatoid arthritis. Limited research on specific effectiveness in scleroderma. |
| Imatinib mesylate | Gleevec | Skin fibrosis, pulmonary | Moderate | Anti-cancer drug. Research results are mixed. Recent well-controlled study failed to show any improvement in skin fibrosis for diffuse scleroderma patients. |
| IV immunoglobulin | Privigen Gammagard Gamunex Carimune |
Joint pain, skin fibrosis, pulmonary function | Mild | A well-designed study is now underway. |
| Methotrexate | Rheumatrex Trexall Amethopterin |
Joint stiffness, pain, and inflammation, skin fibrosis | Serious: patients should be closely monitored for potential liver damage. Can cause serious birth defects if taken during pregnancy. | Research suggests limited effectiveness in treatment of scleroderma. Commonly used to treat rheumatoid arthritis and lupus. Not for use by women able to get pregnant unless using two forms of contraception. |
| Mycophenolate mofetil | CellCept | Pulmonary fibrosis (ILD), skin fibrosis | Serious: increased susceptibility to infections and lymphoma. Patients need to be closely monitored. | Primary usage is to suppress the immune system to help prevent transplanted organ rejection. Considered less toxic than cyclophosphamide or azathioprine. |
| Prednisone | Coltran Orasone Deltasone Sterapred Rayos |
ILD | Severe: risk of kidney damage, pneumonia, cataracts, diabetes, and infections. | While glucocorticoids are generally useful with lupus and rheumatoid arthritis, they appear to have little benefit in most types of Scleroderma. |
| Rituximab | Rituxan | ILD | Severe: can have severe life threatening reactions when first administered. Also, for patients with certain (potentially undiagnosed) viral infections, rituximab can trigger life-threatening problems, including PML – a progressive brain infection. |
Suppresses B-cells, a form of white blood cells that generate antibodies that are assumed to trigger the development of scleroderma symptoms. This drug is normally used for treating non-Hodgkin’s lymphoma and other white blood cell related cancers |
| Bosentan | Tracleer | Skin ulcers, pulmonary artery hypertension (PAH) | Serious: including potential liver damage. Can cause serious birth defects if taken during pregnancy. | Not for use by women able to get pregnant unless using two forms of contraception. |