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, treated with topical steroid and topical and oral minoxidil. The provider is requesting Xeljanz (tofacitinib). The health plan denied coverage, as Xeljanz is not United States Food and Drug Administration (FDA) approved for alopecia areata/universalis. This denial is being appealed.

The health plan should cover the requested Xeljanz.

Xeljanz is approved in adults for rheumatoid arthritis (RA), psoriatic arthritis, ankylosing spondylitis, ulcerative colitis, and COVID19, and for children as young as two for juvenile arthritis.
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% 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. Tofacitinib (Xeljanz) first demonstrated efficacy in alopecia areata in an 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 January 2017, an adult study involved 65 patients, defined as those with alopecia totalis or alopecia universalis. Of those, 77% achieved a clinical response, with 58% of patients achieving greater than 50% change in Severity of Alopecia Tool (SALT) score over four 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% versus 59.0%). Tofacitinib was well tolerated, and there were no serious adverse events (3).
An adolescent retrospective study, also published in January 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% (mean 61%; 1%-100%) at an average of 6.5 months of treatment. Adverse events were mild (4).
Another study published in September 2016 was a two-center, open-label, single-arm trial of 66 patients with alopecia areata with greater than 50% scalp hair loss, alopecia totalis (AT), and 
alopecia universalis (AU). Tofacitinib (5 milligrams) was given twice daily for three months. Endpoints included regrowth of scalp hair, as assessed by the Severity of Alopecia Tool, duration of hair growth after completion of therapy, and disease transcriptome. In this study, 32% experienced 50% or greater improvement in Severity of Alopecia Tool score with no significant adverse events (5).
Another recent case report outlined eight adolescent patients ages 12-19 with alopecia universalis. All patient experienced substantial regrowth of hair including body, eyebrow, and 
eyelash hair and are still on tofacitinib, with no adverse effects (7). Alopecia areata and its variants are extremely distressing diseases. With further case reports of off-label uses of medications such as Xeljanz, more treatment options can become available. Xeljanz is currently United States Food and Drug Administration (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 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. Therefore, given the support in current literature and lack of effective therapies for alopecia areata, and approval of Xeljanz for children as young as 2 for other conditions, Xeljanz is medically appropriate in this case.