Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Alopecia Universalis
Treatment: Xeljanz
The insurer denied Xeljanz.
The determination is overturned.

This is a male patient with alopecia universalis, treated with topical steroids, topical minoxidil and topical tofacitinib. The provider is requesting Xeljanz. The health plan denied the request. At issue is whether the Xeljanz 5 milligram (mg) tablet is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.

The Xeljanz 5 mg tablet is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.
Alopecia universalis is a severe form of alopecia areata involving total body hair loss, including eyelashes, groin, etc. Regrowth of hair rarely occurs. Treatment studies for any form of alopecia areata are difficult to perform because the disease has an unpredictable course, and much investigation is still needed.
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%), 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.
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. Xeljanz is currently FDA-approved for Rheumatoid arthritis and undergoing approval trials for psoriasis and psoriatic arthritis. It is known to be safe and effective in the conditions for which it is Food and Drug Administration (FDA) approved. 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.
The 2019 systematic review (13) performed a literature search to identify clinical trials and case reports, and concluded that several clinical studies have shown very promising results in the treatment of AA with oral formulas of JAKs. These agents do need chronic administration to maintain response. Topical formulations did not show satisfactory responses. The safety profile of these agents appears to be favorable.
The meta-analysis (Yu et al) in 2021 identified a total of 1244 studies, of which only 12 studies met the inclusion criteria. Of the 346 patients in these 12 studies, 288 had received oral tofacitinib and 58 had received oral ruxolitinib. The overall Severity of Alopecia Tool 50 achievement rate was 66% (95% confidence interval, 54%-76%). Subgroup analysis revealed that drug choice, mean age, sex ratio and alopecia areata subtype ratio did not significantly affect the treatment response. Infections and laboratory abnormalities were the most common adverse events (98 and 65 cases of 319 patients, respectively).
Therefore given the support in current literature and lack of effective therapies for alopecia areata, Xeljanz is medically necessary. Xeljanz is approved for use in patients ages 2 and up for arthritis.