Table of Contents
Scleroderma treatments vary depending on several variables:
- Is it local or systemic, and if systemic, is it limited or diffuse?
- If the disease is systemic, what organs, if any, are involved?
Although there is still no treatment for the underlying process of scleroderma, specific drugs and treatments help combat the various mechanisms and consequences of the disease.
- Some medications keep blood vessels open (prostacyclins, endothelin receptor antagonists, ACE inhibitors, phosphodiesterase 5 inhibitors, and others) and are used to treat Raynaud's phenomenon, heart and kidney problems, and pulmonary hypertension.
- Other drugs reduce inflammation and block damaging immune factors. These treatments, which include cyclophosphamide, penicillamine, and bone marrow transplantation, may be helpful for improving skin thickness and reducing scarring, even in the lungs.
- Doctors use other treatments for specific complications, such as proton pump inhibitors and pro-kinetic agents for gastrointestinal problems, or light treatments for skin thickening.
- Various investigative approaches exist, including stem-cell transplants.
Patients should receive treatments for specific complications as early as possible in the course of the disease, to reduce progression before irreversible hardening of tissues occurs.
There is no cure for scleroderma. Many drugs that are useful for other autoimmune inflammatory disorders have not proven to be very effective for scleroderma. Experimental work is ongoing to develop procedures or to find drugs that can treat the underlying processes that cause damage. Developing effective treatments for scleroderma is very problematic, however, for the following reasons:
- The course of scleroderma is hard to predict, making it 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 organs without affecting other organs is very difficult.
- The disease is so uncommon that there are few patients available for clinical trials. Studies, then, are very small, sometimes having only four or five patients. It is very difficult to design studies of this size that can provide strong evidence on treatment effects. Drugs that seem promising on small groups of patients often fail to show effectiveness on larger groups.
Treating the Whole Patient
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 (such as protection from trauma and cold), and other lifestyle measures can be extremely important and helpful.
Review Date: 01/04/2011
Reviewed By: Harvey Simon, MD, Editor-in-Chief, Associate Professor of Medicine, Harvard Medical School; Physician, Massachusetts General Hospital. Also reviewed by David Zieve, MD, MHA, Medical Director, A.D.A.M., Inc.