Treatment

Because scleroderma is so variable, no single treatment or treatments can be applied to all patients. Each patient requires an individualized approach.

The first step is to determine what form the disease has taken:

• Is it local or systemic, and if systemic, is it limited or diffuse?
• If the disease is systemic, what organs, if any, are involved?

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Specific agents are used to help combat the various mechanisms and consequences of the disease.

• Some keep blood vessels open (prostacylins, ACE inhibitors and others) and are used to treat Raynaud's phenomenon, heart and kidney problems, and pulmonary hypertension.
• Others, notably cyclophosphamide, reduce inflammation and block the damaging immune factors and are proving to be helpful for improving skin thickness and reducing scarring, even in the lungs.
• Other agents are also used for specific complications, such as proton pump inhibitors for gastrointestinal problems or phototherapies for skin thickening.
• Promising investigative approaches include stem-cell transplantation.

The patient should receive treatments as early as possible in the course of the disease for specific complications to reduce progression before irreversible sclerosis occurs.

Problems in Developing Treatments for Scleroderma

There is no cure for scleroderma. Experimental work is on going to develop procedures or to find drugs that can treat the underlying mechanisms. Developing effective treatments for scleroderma is very problematic, however, for various reasons:

• Scleroderma has an extremely variable progression, making it is one of the most difficult rheumatic diseases to treat. It also makes drug development complicated.
• The disease, when advanced, affects many organs. Designing treatment strategies that will improve symptoms in some without affecting others is very difficult.
• Many drugs that are useful in other autoimmune inflammatory disorders have not proven to be very effective for scleroderma.
• Many studies do not take seasonal temperature changes into consideration, which are major factors in scleroderma and can distort results.
• The disease is so uncommon that there are few patients for clinical trials. Studies, then, are very small, sometimes having only four or five patients. It is very difficult then to design studies that can provide strong evidence on way or the other on treatment effects. Drugs that seem promising on small groups of patients often fail to come through on larger groups. For example, of great disappointment was the failure of relaxin (ConXn) to produce significant benefits. It had been the first drug approved specifically for scleroderma.

Treating the Whole Patient

The course of scleroderma is difficult to predict. The disease can evolve slowly over time with few symptoms, or progress rapidly and become very severe. The patient, then, must live with considerable uncertainty and emotional stress. Support associations, non-medical aids to help relieve symptoms, and other life-style measures can be extremely important and helpful.