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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Sarcoidosis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

Emily holds a Ph.D. in Biochemistry from the University of Iowa and is currently a postdoctoral scholar at the University of Wisconsin-Madison. She graduated with a Masters in Chemistry from the Georgia Institute of Technology and holds a Bachelors in Biology and Chemistry from the University of Central Arkansas. Emily is passionate about science communication, and, in her free time, writes and illustrates children’s stories.
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Özge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.
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Emily holds a Ph.D. in Biochemistry from the University of Iowa and is currently a postdoctoral scholar at the University of Wisconsin-Madison. She graduated with a Masters in Chemistry from the Georgia Institute of Technology and holds a Bachelors in Biology and Chemistry from the University of Central Arkansas. Emily is passionate about science communication, and, in her free time, writes and illustrates children’s stories.
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