Arthritis Medicine Tofacitinib May Be Effective in Treating Multiorgan Sarcoidosis, Study Reports
The arthritis medicine Xeljanz (tofacitinib) may be an effective therapeutic alternative to conventional medications for the treatment of multiorgan sarcoidosis.
This finding was described in the study, “Treatment of Multiorgan Sarcoidosis With Tofacitinib,” published in the journal ACR Open Rheumatology.
Sarcoidosis, an inflammatory condition that takes on different forms, can be difficult to treat. Typical treatment options often involve taking glucocorticoids, a type of corticosteroid hormone that can have several adverse effects.
A cell communication circuit called the JAK‐STAT pathway — for Janus kinase–signal transducer and activator of transcription — is constantly active in people with sarcoidosis, previous studies have demonstrated. The JAK-STAT pathway, present within immune cells, is involved in the production of inflammatory signals.
Xeljanz, marketed by Pfizer, is an inhibitor of the JAK‐STAT pathway commonly used to treat rheumatoid arthritis and other diseases. It also has been previously used to successfully treat three patients with cutaneous sarcoidosis.
In this case report, a team led by researchers at the Yale School of Medicine, in Connecticut, assessed the efficacy of Xeljanz in the treatment of a patient with long-term multiorgan sarcoidosis.
The patient, a 60‐year‐old woman, had a history of severe sarcoidosis involving the lungs, lymph nodes, bone, and skin for 21 years. She had attempted several commonly used therapies for sarcoidosis, including mycophenolate sodium, methotrexate, infliximab, prednisone, rituximab, and intravenous immunoglobulin (IVIG), but her condition still progressed.
When she first started on the study, she took 5 mg of Xeljanz twice daily while continuing to take her previous treatment regimen — specifically prednisone, rituximab, and IVIG.
After one month of this treatment plan, the patient had an improvement on her skin lesions. However, after two months, at the time she was due for a new dose of rituximab, she experienced worsening of her joint pain, and the team decided to increase her Xeljanz dose to 10 mg twice daily.
Between months two and four of the study, the patient took only Xeljanz, prednisone, and IVIG, and was able to achieve clinical resolution of her skin symptoms.
A skin biopsy taken at six months confirmed that she no longer had skin granulomas, a hallmark of sarcoidosis. The skin biopsy also showed that the presence of proteins linked to the JAK‐STAT pathway had decreased. Further, the levels of skin and blood biomarkers associated with inflammation were reduced.
Images from a PET‐CT scan after six months of treatment showed complete resolution of prior sarcoidosis in the skin, lungs, lymph nodes, and bones. The patient has currently stopped taking IVIG, and she is gradually reducing prednisone, while her health steadily improves on a tofacitinib dose of 10 mg in the morning and 5 mg at night.
Concerning Xeljanz’s safety, the patient tolerated the treatment well, and had only a urinary tract infection that was treated with antibiotics.
Taken together, “these data demonstrate remission of recalcitrant cutaneous and internal organ sarcoidosis with tofacitinib,” the researchers said.
“There is significant emerging evidence that JAK inhibitors are targeted treatment for sarcoidosis, an often‐morbid disease for which there is a paucity of safe, reliably effective therapies,” they said.
“Prospective studies of JAK inhibitors in sarcoidosis are underway (ClinicalTrials.gov identifiers NCT03910543 and NCT03793439),” the team added.
