A definition of Juvenile Scleroderma in simple, easy to understand language

Treatment of Juvenile Scleroderma
Written by Balu Athreya, M.D.
duPont Hospital for Children
Wilmington, DE

Our current approach to the medical management of juvenile scleroderma is based on anecdotal reports and personal experience and not on solid scientific evidence.. This is because of several reasons. We do not have a good understanding of the basic mechanism of this group of diseases. There is no consensus on the classification of the various subtypes. It is difficult to measure the activity of the disease. Natural remission is very common particularly with the localized variety. In addition there are very few children with these diseases in any one pediatric rheumatology center and therefore it is difficult to perform controlled clinical trials. However several new developments in basic sciences give us hope for specific therapy. In addition, European Pediatric Rheumatology Society (PRES) has already conducted a world-wide study to collect data and efforts are being made to establish criteria for classification. Hopefully the next step will be multicenter clinical trials of potentially useful drugs.

Localized Scleroderma: This variety is also called morphea by some rheumatologists. There are several subtypes within this group, morphea en plaque, linear morphea or scleroderma, deep morphea and en coup de sabre. Decision to treat this type should be based on the specific subtype and the fact that majority of patients enter remission in about 3 to 5 years. Morphea, which is superficial is more of a cosmetic problem and it is preferable not to treat this condition with potentially toxic medications. Local ointments (such as Vitamin A and E) to soften the skin or local application of glucocorticoid creams are adequate. However, children with linear scleroderma, deep morphea and those whose lesions are active and spreading rapidly may benefit with the use of oral glucorticoids particularly during the early phase of the disease. This is used for only 2 to 3 months and tapered off. Other drugs used to treat localized scleroderma include d-Penicillamine, methotrexate, colchicine and hydroxychloroquine. One of these drugs is started early together with glucocorticoids and continued for 2 to 3 years at the least. At present, methotrexate seems to be the favorite although d-penicillamine has a longer track record..

Several other agents have been tried with limited or no success. These include phototherapy with UV A1 with and without psoralen which seems to be effective. However long term adverse effects are not known. Oral calcitrol therapy injection of interferon gamma into the skin lesion were found to be ineffective. Topical calcipotriene ( a synthetic form of Vitamin D) seems to effective but needs further testing.

As mentioned earlier local treatment with emollients may be helpful to keep the skin soft. The lesions may involve deeper tissues and cause atrophy of muscle and contractures. When skin lesions cross joints, contractures may develop. Therefore proper physical therapy program is necessary to stretch affected muscles and tissues, maintain position and maintain function.

Systemic Scleroderma (SS): There are two subtypes of this syndrome – diffuse cutaneous variety which is the more common type in children and the less common limited cutaneous variety. Both these types are much less common than in adults. Once again no drug has been shown to be effective in these subtypes based on controlled clinical trials. However, better management of the internal organ involvement has contributed to better quality of life and better survival as shown initially with the use of angiotensin-converting Enzyme (ACE) inhibitors to treat kidney disease of scleroderma.

Treatment of SS can be divided into two portions: 1.strategies aimed at modifying the basic disease process and 2. Strategies aimed at prevention and treatment of specific organ involvement.

Based on current knowledge, disease modifying therapy includes the use of drugs to suppress the immune system, to dilate blood vessels and increase blood flow and to prevent scarring (fibrosis).Several immunosuppressive drugs have been used including azathioprine, cytoxan, methotrexate and cyclosporin. There are no controlled trials to show any of these work, The only exception is the beneficial effect of intravenous cytoxan for the lung disease of scleroderma. Photapharesis and interferon gamma have not been found to be effective. Controlled trials of methotrexate in adults show conflicting results. However this is one of the commonly used drugs in systemic scleroderma. Finally autologous stem cell transplantation has been tried to treat severe scleroderma with limited success.

There are several exciting new developments in the treatment of Raynaud phenomenon and pulmonary hypertension – both caused by obstruction of blood flow. Nifedepine and amlodipine are the drugs of choice to treat Raynaud phenomenon. . Since occlusion of small vessels is the cause of ulcers of the finger tip and loss of finger tips, this should be treated aggressively with avoidance of cold exposure, use of nifidepine or amlodipine and small doses of aspirin. In patients with severe Raynaud’s phenomenon, intravenous or subcutaneous treatment with prostacyclin and its analogues is recommended. These treatment should be given under strict medical supervision. Some of the more recent developments include oral agents such as Bosentan and Viagra and nitrous oxide to treat pulmonary hypertension.

Among the antifibrotic drugs, d penicillaiine is the most studied. Although a large study in adults showed that it was not effective, some authors find fault with that study and still use this drug. In our own experience, this drug is as effective as any other drug proposed. The main concern is with its toxicity.

Newer understanding of the mechanism fibrosis has shown that a protein produced by the fibroblast called Transforming Growth Factor (TGF) is responsible for abnormal collagen production. Inhibition of this biological agent by antibodies has been shown to correct the abnormality in experimental models. Exciting new possibilities are being explored to control fibrosis using antibodies and chemicals that interfere with factors similar to TGF.

In treating individual organs affected in SS, the first step is early identification. For kidney involvement, angiotensin-converting enzyme inhibitors such as captopril and some of the newer agents that act on the receptors will help prevent progression of renal disease.

Patients with reflux in the esophagus should be treated with proper posture after meals, antacids and proton-pump inhibitors. Severe constipation may be treated with stool softeners and enema as needed.

Lung disease and increased pressure in the blood vessels of the lung are major problems for these patients. Some of the newer developments have made for better prognosis and longer survival. This includes the use of cytoxan and newer agents to treat pulmonary hypertension. In patients with interstitial lung disease, the treatment of choice is the use of intravenous cytoxan once a month for 6 months.

Arthritis and joint pain may be treated with nonsteroidal antiinflammatory agents such as ibuprofen.

General measures should include avoidance of cold exposure by keeping the temperature at home at a warmer level an the use of mittens and double socks when outdoors in winter. The child should be encouraged to lead as normal a life as possible. General skin care to keep it moist and supple should include emollient creams . Physical therapy measure such stretching exercises may be needed to maintain range of movement of joints and function. Splints may be needed to treat contractures. Fingertip ulceration should be prevented with good hygiene, aggressive treatment of Raynaud phenomenon and small dose of aspirin If they get infected, treatment with antibiotics is indicated.

Please keep in mind, this webpage is for your information only.
Please check with your child's physician for any treatments.

For more information on Juvenile Scleroderma, contact:

Juvenile Scleroderma Network, Inc.
1204 W. 13th Street, San Pedro, CA 90731

Tel: (310)519-9511 (Pacific Time)
24 Hour Support Line: 1-866-338-5892 (toll-free)

Speak to another JSD parent for emotional and logistical support provided by home-based JSD volunteers. For medical advice, please contact your child's physician.

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