A study investigating patients diagnosed with, or at risk for, early diffuse systemic sclerosis indicates that standard scleroderma treatment seems to have only minimal effects on preventing increased organ damage and disease progression in this cohort.
The study evaluated more than 300 participants treated at 12 scleroderma centres in the U.S. between 2011 and 2020. Nearly two-thirds of the subjects were treated with the immunosuppressive therapy mycophenolate mofetil. (Although not approved for this indication in the U.S., the study reports it is commonly used to manage this rare form of scleroderma. Two therapies have been approved in the U.S. for scleroderma—nintedanib and tocilizumab. Both target lung scarring and lung function.)
The article was published in Arthritis Research & Therapy. DOI: 10.1186/s13075-021-02548-1
In a press release from Michigan Medicine, Dinesh Khanna, MBBS, said: “Despite using the standard of care therapies, patients continue to have worse thickening of their skin and internal organ involvement, including lung scarring that has an impact on their disability, function and survival. This leads to significant damage to the internal organs and an increased likelihood of death due to heart, gut and lung involvement.”
He is the corresponding author of the paper, and a professor of rheumatology at Michigan Medicine and director of the University of Michigan’s Scleroderma Program.
“This form of scleroderma has the highest fatality rate of any rheumatic disease,” he said in the release. “This is a clear opportunity to find new therapies that would improve patients’ quality of life, decrease the internal organ involvement and, therefore, improve survival rates.”