Tofacitinib (CP-690,550) is an oral Janus kinase inhibitor that has shown efficacy in the treatment of rheumatoid arthritis and other autoimmune diseases. A new study published in the journal Annals of the Rheumatic Diseases has now shown that tofacitinib may also be effective in treating scleroderma, a chronic autoimmune disease that causes hardening and thickening of the skin.
The study recruited 34 patients with scleroderma who were treated with tofacitinib 5 mg twice daily for 24 weeks. The primary endpoint of the study was the change in modified Rodnan skin score (mRSS) from baseline to week 24.
At week 24, the mean change in mRSS was -5.1 ± 7.4, and 33% of patients achieved a ≥ 5-point reduction. The most common adverse events were upper respiratory tract infection, nasopharyngitis, headache, and diarrhea.
These results suggest that tofacitinib may be a promising new treatment option for scleroderma. The authors of the study are optimistic that the next phase of clinical trials will confirm the efficacy and safety of tofacitinib in this patient population.
According to researchers at the University of California, San Francisco (UCSF), tofacitinib shows promise in the treatment of scleroderma. The study, which is currently in its second phase, is being conducted on patients with diffuse cutaneous scleroderma (dcSSc), the most severe form of the disease.
There is no cure for scleroderma, which is a chronic autoimmune disorder that causes hardening and tightening of the skin and connective tissues. The disease can also cause organ damage, and is associated with a high mortality rate.
Currently, the only approved treatments for scleroderma are immunosuppressive drugs, which can cause serious side effects. Tofacitinib is a Janus kinase (JAK) inhibitor that works by suppressing the overactive immune response that is characteristic of scleroderma.
The first phase of the study showed that tofacitinib was well tolerated by patients and led to improvements in skin thickness and disease activity. The second phase, which is currently underway, is designed to assess the long-term safety and efficacy of tofacitinib in dcSSc patients.
The results of the second phase are expected to be available in 2019. If the drug is found to be safe and effective, it could be approved by the US Food and Drug Administration (FDA) for the treatment of scleroderma.
The prospect of a new treatment for scleroderma is exciting news for patients and researchers alike. Tofacitinib represents a potentially major advance in the treatment of this debilitating disease, and the UCSF team is optimistic about the next phase of the study.
