Tofacitinib shows promise in scleroderma patients, researchers are optimistic for next phase of study
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.
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.
Over the course of the 24-week trial period, researchers found no patients who exhibited severe adverse effects at or before the trial ended. Additionally, to measure tofacitinib’s effectiveness, the modified Rodnan skin score (mRSS) and other measures were usedbwith patients to measure improvement throughout the trial.
These results showed that the average mRSS score and other measures improved over the course of the trial. In addition, patients on placebo went on open label tofacitinib after 24 weeks and there was ongoing improvement during the next 24 weeks, indicating improvement in the measure.
