Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Alopecia totalis and arthritis. 
Treatment: Xeljanz. 

The insurer denied the Xeljanz. 
The denial is overturned. 

The patient is a female with alopecia totalis and arthritis for whom Xeljanz is requested. She has tried and failed topical and intralesional steroid, systemic steroids, cyclosporine, topical anthralin, and topical tacrolimus. 

Yes, the proposed treatment with Xeljanz is medically necessary. 

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 JAK 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 JAK 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 percent 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 [mg] 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 ten years or less, 77 percent had a clinical response (at least six percent improvement in the Severity of Alopecia Tool [SALT] score) and 58 percent achieved greater than 50 percent improvement in the SALT score over four to 18 months of treatment. Patients with a disease episode longer than ten years appeared less likely to respond to treatment; the clinical response rate in this population was 32 percent (eight of 25 patients). No serious adverse effects occurred during treatment. 

Of all the treatments reported to work in alopecia areata, JAK inhibitors have the most promising results to date. Although the long-term safety of JAK inhibitors in children is still unknown, tofacitinib is undergoing clinical trials for juvenile idiopathic arthritis in children aged two to 18 years. Reports from the experience of 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 JAK 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. Given this patient's diagnosis and history, treatment with Xeljanz would be considered to be reasonable and consistent with the current standard of care in the dermatology community.