Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Alopecia Areata
Treatment: Xeljanz 5mg (milligram) tablet
The insurer denied: Xeljanz 5mg (milligram) tablet
The denial is overturned.

The patient is a school aged male with alopecia universalis, a severe form of alopecia areata, for the past six years. He has tried and failed multiple topical steroids and topical and oral minoxidil. This is a request for Xeljanz (tofacitinib) 5 milligrams.

The health plan should cover the requested Xeljanz.

The current literature reflects Xeljanz as being more effective than other treatments. Alopecia areata (AA) is a relatively common disease, but no satisfactory treatment has yet been developed. Recently, research progress has been made in the pathogenesis of alopecia areata, revealing that autoreactive cytotoxic T cells are important and that the janus kinase (JAK) pathway is involved. Therefore, the potential of janus kinase inhibitors as therapeutic agents for alopecia areata is attracting attention. There have been a number of case reports and small clinical trials reporting promising outcomes of janus kinase inhibitors tofacitinib, ruxolitinib, and baricitinib for alopecia areata. The majority of the literature to date is based on small volume data, with a lack of definitive evidence or guidelines. A retrospective study of 90 adults with severe alopecia areata (at least 40% scalp hair loss, alopecia totalis, or alopecia universalis) who had stable or worsening disease for at least six months and received oral tofacitinib (5 to 10 milligrams twice daily) for at least four months (with or without adjuvant prednisone) supports benefit. Of the 65 patients with a duration of the current disease episode of 10 years or less, 77% had a clinical response (at least 6% improvement in the Severity of Alopecia Tool [SALT] score) and 58% achieved greater than 
50% improvement in the Severity of Alopecia Tool score over four to 18 months of treatment. Patients with a disease episode longer than 10 years were evidenced to be less likely to respond to treatment; the clinical response rate in this population was 32% (eight of 25 patients). No serious adverse effects occurred during treatment. There is promising evidence regarding the effectiveness of janus kinase inhibitors in alopecia areata. Future large-sized randomized studies are required to confirm findings. However, of all the treatments reported to work in alopecia areata, janus kinase inhibitors have the most promising results to date. Although the long-term safety of janus kinase inhibitors in children is still unknown, tofacitinib is undergoing clinical trials for juvenile idiopathic arthritis in children aged 2 to 18 years (NCT02592434 and NCT01500551). Reports from the experience of the authors suggest that tofacitinib may be a therapeutic option for children with severe alopecia areata who have failed conventional therapy. However, because of the potential for serious adverse effects with janus kinase inhibitors, including infection and malignancy, a thorough discussion of risks and benefits is necessary before the initiation of treatment. This is an accepted off-label use and dose for the treatment of alopecia areata based on the literature. Therefore, given the above, Xeljanz is the best treatment option for this patient at this time.