Appeal type: Medical necessity
Decision: Overturned

Diagnosis:  Severe Alopecia Areata.
Treatment:  Xeljanz 10MG (milligrams).

The insurer denied Xeljanz 10MG (milligrams).  The denial is overturned.

The patient is an adult male with alopecia areata, over 80 percent scalp hair loss prior to starting Xeljanz. Prior treatments include oral, topical and intralesional steroids. The provider is requesting continuation of Xeljanz which the patient has been well controlled for 2 years. The health plan denied the request as Xeljanz is not Food and Drug Administration (FDA) approved for alopecia areata.

The proposed Xeljanz is medically necessary.

A wide range of treatments have been used for the management of alopecia areata, most of which are not highly effective. Treatments include topical or intralesional corticosteroids, topical minoxidil (2 percent or 5 percent), topical anthralin, systemic corticosteroids, cyclosporine, and other immunosuppressants. A dependable and safe treatment for extensive disease (totalis or universalis) has yet to be found. Given the rarity of the disease, large randomized controlled trials are extremely difficult. Tofacitinib first demonstrated efficacy in alopecia areata in the article published in 2014. A patient with concomitant psoriasis and alopecia areata was treated with the medication and serendipitously his alopecia areata reversed. Since then, further case reports have emerged documenting its efficacy, which has led to the very recently published retrospective studies involving adults and adolescents, as well as others, summarized below:
- Published in 01/2017, the adult study involved 65 patients, defined as those with alopecia totalis or alopecia universalis. Seventy-seven percent achieved a clinical response, with 58 percent of patients achieving greater than 50 percent change in Severity of Alopecia Tool (SALT) score over 4 to 18 months of treatment. Patients with alopecia areata experienced a higher percent change in Severity of Alopecia Tool score than did patients with alopecia totalis or alopecia universalis (81.9 percent versus 59.0 percent). Tofacitinib was well tolerated, and there were no serious adverse events (3).
- The adolescent retrospective study, also published in 01/2017, involved 13 patients, aged 12 to 17 years. Nine patients experienced clinically significant hair regrowth. Median percent change in Severity of Alopecia Tool score was 93 percent (mean 61 percent; 1 percent-100 percent) at an average of 6.5 months of treatment. Adverse events were mild (4).
- Several case reports have also documented substantial hair regrowth leading to reestablishment of eyelashes and other functional hair reducing incidence of corneal abrasions, etc.
Alopecia areata and its variants is an extremely distressing disease. With further case reports of "off-label" uses of medications such as Xeljanz, more treatment options can become available. 
Biologic medications as a whole have many off label uses, leading to better outcomes in rare diseases for which large, randomized trials are not readily available.
Therefore, given the support in current literature and lack of effective therapies for alopecia areata, and this patient's favorable response to therapy, Xeljanz is medically necessary.