Appeal type: Medical necessity
Decision: Upheld

Diagnosis: Alopecia universalis

Treatment: Xeljanz

The insurer denied coverage for Xeljanz.

The denial is upheld.

This is a female patient with alopecia universalis. The patient has previously used topical steroids, minoxidil, and intralesional Kenalog with minimal clinical improvement. The provider recommended Xeljanz treatment for this patient.

According to the United States Department of Health & Human Services, National Institutes of Health (NIH) and Genetic & Rare Diseases Information Center (GARD) (2017), "No therapy has been found to work for everyone who has alopecia universalis (AU) which makes managing AU challenging. Although multiple treatments have been explored, no therapy is currently approved by the United States Food and Drug Administration (FDA). Some promising therapies include: diphenylcyclopropenone (DPCP), squaric acid dibutylester (SADBE), photodynamic therapy, steroids, cyclosporine in combination with methylprednisolone. There are several recent studies showing that a class of medication known as [Janus kinase] JAK inhibitors, which includes Tofacitinib and Ruxolitinib, are effective in alopecia areata, including AU.  However, JAK inhibitors have not yet been approved by the FDA for use in skin conditions."

Furthermore, according to Kassira et al (2017), "Management of [alopecia totalis (AT)] and [alopecia universalis (AU)] can be challenging, and although multiple treatment modalities have been explored, no therapy is currently FDA-approved." It further states, "Although certain treatments showed significant hair regrowth, no treatment was completely effective. The most promising therapies with the highest quality data include diphenylcyclopropenone, squaric acid dibutylester, photodynamic therapy, steroids, and cyclosporine in combination with methylprednisolone." 

As per Salman et al (2017) and Liu et al (2017), Xeljanz was studied and shown to have possible efficacy in treating patients with alopecia. However there is insufficient evidence by way of large randomized controlled cohort prospective trials showing the long-term safety and efficacy of Xeljanz in treating this patient population. As such, there are no clinical guidelines with regards to optimal dosing and duration of therapy. There is also a lack of consensus with regards to the optimal patient scenario. In addition, it has been well-documented in medical literature that Xeljanz is unable to maintain long-term efficacy for treatment of alopecia. It should also be noted that the FDA has not approved Xeljanz for alopecia areata/universalis. In addition, UpToDate notes "further study is necessary prior to conclusions about efficacy and safety." They noted that "Tofacitinib therapy is associated with increased risk for infection, including serious infections. The development of malignancy and laboratory abnormalities has also been reported in patients receiving tofacitinib therapy for other diseases." Lastly, this patient has Turner's syndrome in which alopecia areata is known to occur as part of the syndrome. This further raises issues regarding the role of Xeljanz in this patient. Thus, Xeljanz is not considered medically necessary for this patient.

The health care plan acted reasonably with sound medical judgment and in the best interest of the patient.                                                 

The carrier's denial of coverage for Xeljanz should be upheld. The medical necessity is not substantiated.