Xeljanz (Tofacitinib)

Xeljanz (tofacitinib) is an oral treatment originally developed at the National Institutes of Health through collaboration with Pfizer.

The U.S. Food and Drug Administration approved Xeljanz as a treatment for rheumatoid arthritis, psoriatic arthritis, and ulcerative colitis. It has also been used off-label to treat cutaneous sarcoidosis.

How does Xeljanz work?

Sarcoidosis is an autoimmune disease in which granulomas (clumps of immune cells) form in different tissues and organs. These clumps can lead to scarring and interfere with function of the affected organ. Cutaneous sarcoidosis refers to the formation of these granulomas in the skin, leading to painful lesions and scarring.

When the immune system is active, different types of immune cells communicate with one another via small molecules called cytokines. Pro-inflammatory cytokines signal to the immune system to increase inflammation, calling immune cells to infiltrate a certain area in response to a threat such as an infection or an injury.

However, in autoimmune diseases, the immune system responds to things that are not threats, such as the body’s own cells and tissues, and immune cells congregate where they should not.

Xeljanz is a partial and reversible inhibitor of an enzyme called Janus kinase (JAK) that is involved in passing on cytokine-mediated signals between cells. Blocking the function of JAK means that downstream proteins known as  STATs are not activated. As a result, immune cells divide much more slowly and do not become pro-inflammatory.

Xeljanz in clinical trials

Several studies have reported individual cases of cutaneous sarcoidosis patients treated successfully with Xeljanz.

In one small study, a sarcoidosis patient with granulomas present in the lungs, lymph nodes, bone, and skin was treated with Xeljanz. The patient had not responded to other sarcoidosis treatments. Researchers used multiple biopsies showing granulomas to diagnose sarcoidosis and a positron emission tomography-computed tomography (PET-CT) image to assess the presence of lesions throughout the patient’s body. The scans were repeated at six months and nine months and showed complete remission of internal sarcoidosis lesions.

An open-label Phase 1 clinical trial (NCT03910543) testing the efficacy of Xeljanz is currently recruiting up to 15 cutaneous sarcoidosis patients at the Yale School of Medicine in Connecticut. Patients will receive the treatment twice daily for six to 12 months. Primary outcomes are an improvement in cutaneous sarcoidosis activity and morphology instrument and granuloma activity scoring index after six months of treatment. Secondary outcomes include improvement in internal organ sarcoidosis, such as that in the lungs or heart, and skin-related quality of life. PET-CT scans are taken before and after the treatment.

Another open-label Phase 1 trial (NCT03793439) is recruiting up to five patients with pulmonary sarcoidosis at the Oregon Health & Science University to determine if further studies on Xeljanz treatment are warranted. Patients are given Xeljanz twice daily for 16 weeks, with diagnostic assessments at weeks two, four, eight, 12, and 16. The primary outcome measure is a 50% or better reduction in corticosteroid use among treated patients without a decline in lung function.

Other information

Xeljanz can cause side effects, including diarrhea, headache, and stomach pain, and may increase a patient’s susceptibility to infection.


Last updated: Jan. 24, 2020


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