Individuals with scleroderma are living significantly longer today, compared with 30 years ago, and the physicians who treat this rare disease of connective tissue hope the newer drugs now on the market may extend lives even further.
Scleroderma, a rare autoimmune disorder of unknown origin, results when collagen builds up in the body’s connective tissue and causes thickening of the skin. In the chronic and often progressive systemic form, scleroderma causes stiff joints and hardening of the internal organs and blood vessels.
In the July issue of the journal Annals of the Rheumatic Diseases, Georgetown University Medical Center professor Virginia Steen, M.D., studied 2,000 patients with scleroderma (also known as systemic sclerosis) treated between 1972 and 2001 at the University of Pittsburgh and found that 10-year survival steadily improved over those years by 12 percent-- from 54 percent to 66 percent.
“This is really good progress, which we hope to improve upon,” said Steen, a leading expert on the disorder who has helped make Georgetown University Hospital a national treatment center for scleroderma. In the 1980s, while at the University of Pittsburgh, she developed a natural history database of scleroderma patients and this database has continued to evolve into the largest in the United States.
The reason overall mortality has dropped, Steen says, is partly because a class of medications, angiotensin converting enzyme (ACE) inhibitors became available which dramatically improved the treatment of renal crisis. Renal crisis used to be almost always fatal and now it is a very treatable complication of scleroderma, she said.
Now pulmonary arterial hypertension and pulmonary fibrosis, a condition that is traditionally the second-most frequent cause of death, is responsible for most of the mortality, but these conditions may be increasingly treatable, she said. Patients may develop either disorder, or both.
“Before, it was kidney and then lung disorders that affected most of our patients, and now that we have successfully treated the kidneys, mortality due to lung disease has become the issue,” Steen said. “We are working on ways to use the newer medications to help treat these disorders, hoping to further improve overall survival.”
While all scleroderma-related disorders are believed to affect as many as 900,000 people in the United States, only between 40,000 and 165,000 are diagnosed with systemic scleroderma, according to the National Institutes of Health (NIH). Like all scleroderma disorders, systemic scleroderma affects more women than men, and varies in severity.
In this study, Steen collaborated with co-author Thomas Medsger, M.D., professor of medicine at the University of Pittsburgh to study patients with systemic scleroderma enrolled in the registry. They studied patients evaluated at the University of Pittsburgh between 1972 and 2001, dividing them into five-year time periods, depending on when they were seen.
They found survival improved in each of the five-year periods, and that the frequency of death due to renal crisis significantly decreased over the 30 years, from 42 percent to 6 percent. But the proportion of patients who died from pulmonary fibrosis increased from 6 percent to 33 percent, Steen said. The frequency of pulmonary hypertension also increased, but there was no change in gastrointestinal and heart-related deaths.
“Use of ACE inhibitors made all the difference in saving patients from renal failure, and now it is important that physicians use drugs now available to treat lung disorders and researchers continue to search for new therapies,” Steen said.
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