Localized Scleroderma: Overview and Treatment Approach
Written by Ronald Laxer, M.D.
Chief, Pediatric Rheumatologist
It is unfortunate that the word “scleroderma” is used to describe both the localized and systemic forms of a disease whose major feature is hardening of the skin. The localized form, which is by far the most common in children, does not progress to the systemic form and therefore is not associated with internal organ disease that is so common in systemic scleroderma.
Localized scleroderma can occur in several different forms. In children, linear scleroderma is the most common type, and is typified by a band of hard skin (and tissues under the skin) that usally involves a limb and frequently goes across joints. Morphea is typified by oval shaped, ivory color hard lesions that are most common over the abdomen and back. “En coupe de sabre” lesions are a form of linear scleroderma that occurs on the forehead. They often extend into the scalp and may cross the inner aspect of an eye and involve the nose. In Parry-Romberg syndrome, the skin does not harden but there is a disappearance of the tissues under the skin resulting in atrophy of one side of the face, below the forehead.
There is no known cause for the appearance of localized scleroderma. Theories that have been put forward but remain unproven include infection and previous injury. There are a number of elevated immunologic proteins in the blood of some patients, suggesting that there is overactivity of the immune system.
Over the years, many different treatments have been tried for localized scleroderma. To date, none have been proven to be effective using the ideal research method of comparing two identical groups, in whom one received treatment and the other didn’t. Drug studies are difficult to do in patients with localized scleroderma for several reasons: 1) The disease is generally uncommon and therefore it is difficult to recruit adequate numbers of patients for studies. 2) It is difficult to be sure if a lesion is “active” and therefore able to improve with treatment. 3) The disease has a natural tendency to remit on its own. This means that even without treatment, the process eventually stops. The average time for this to occur is between 3-5 years. 4) The lesions tend to grow slowly, and so if is difficult to tell in a short period of time if a treatment is effective or not. Agents which have been suggested to be effective in the treatment of localized scleroderma include penicillamine, hydroxychloroquine, corticosteroids, vitamin D (both on the skin or by mouth) and ultraviolet light treatment.
Recently, we used a combination of corticosteriods and methotrexate to treat 10 children with localized scleroderma. The indications for treatment included significant facial deformity, rapidly progressive lesions that were crossing joint lines with a risk of reduced joint function if the area over the joint hardened significantly. Methotrexate was chosen as maintenance treatment for several reasons. It is active against some components of the immune system which are abnormal in localized scleroderma. There were some suggestions that it had been effective in systemic scleroderma; individual physicians had reported success with methotrexate; and the drug has proven to be safe and effective over 15 years of use in children with juvenile rheumatoid arthritis. The corticosteriods were prescribed to get a rapid onset of potent anti-inflammatory treatment. The route of administering the corticosteriod treatment was the intravenous one for the majority patients, who received treatment daily for 3 days, every 4 weeks, for 3 seperate treatments. This was given on an outpatient basis, and no complications were observed. One boy with a rapidly progressing lesion was also give prednisone by mouth on a daily basis, and one girl had previously received prednisone and her parents preferred to have her receive methotrexate alone. In general, the results were quite successful and we believe that he combination is currently a good method of treatment for patients with localized scleroderma whose physicians feel that treatment is indicated.
Methotrexate used in the doses prescribed for juvenile arthritis or localized scleroderma is very safe. It is given once a week either by mouth or via injection into the skin (like insulin for diabetes). Common side effects include canker sores and nausea; these can usually be prevented with the use of the vitamin folic acid given daily. Liver function tests and complete blood counts are monitored monthly and the doses adjusted accordingly. Patients most “at-risk” of liver abnormalities are those with diabetes, history of family hepatitis, who are obese, or who consume alcohol. There may be an increased risk from infection, especially chicken pox, and patients should be vaccinated against the chicken pox prior to starting methotrexate treatment. Some children complain of fatigue and headache for 24 hours after the methotrexate dose. Rare side effects include “allergy” in the lungs, mild hair loss and diarrhea. In adults, a form of lymph node cancer that is also seen in patients who receive organ transplants may occur with methotrexate treatment, many of these disappear when the methotrexate is stopped. There is no risk of later infertility.
In summary, there do appear to be some treatments which have the potential to halt the progression of the lesions of localized scleroderma. More research is needed to study the mechanisms of disease and response to treatment.
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For more information on Juvenile Scleroderma, contact:
Juvenile Scleroderma Network, Inc.
1204 W. 13th Street, San Pedro, CA 90731
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