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Systemic sclerosis — or scleroderma that affects the skin and internal organs -, is one of the rarest autoimmune diseases, affecting roughly 100,000 people (primarily women) in the United States.,However, systemic sclerosis is devastating — it has the highest mortality rate among rheumatic diseases, according to Dinesh Khanna M.B.B.S., M.Sc., medicines for mental sickness director of the Michigan Medicine Scleroderma Program.
And with no licensed treatments available for this subset of scleroderma patients, rheumatology researchers are constantly searching for opportunities to use resources and technology that have proven beneficial in treating other autoimmune and rheumatic diseases.
One resource that clinicians at Michigan Medicine and the University of Pittsburgh recently explored in early-stage systemic sclerosis was an FDA-approved rheumatoid arthritis drug, tofacitinib. Their goals for the study included finding out if the drug was safe in patients and to understand how the drug would mechanically work at the cellular level in the disease.
“We wanted to understand first, if there was any clinical benefit of tofacitinib to patients, but we were also asking, what are the differences in the cells of healthy skin versus systemic sclerosis cells…how does the drug work?” said Khanna.
In their new study published in JCI Insight, researchers found that tofacitinib was well tolerated among patients with early systemic sclerosis, and discovered the drug primarily affected the protein, interferon, both in fibroblasts and keratinocytes cells.
The study sample size consisted of 15 patients with early diffuse cutaneous systemic sclerosis — patients with skin hardening and issues with organs. Of the total participants, 10 patients received 5 milligrams of tofacitinib twice a day, and the remaining received placebo in a double-blind randomized placebo-controlled trial.
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