Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Alopecia
Treatment:  Xeljanz 10 mg (milligrams) tablets, pre-service
The insurer denied the Xeljanz 10 mg tablets, pre-service.
The denial is overturned.

The patient is a woman with alopecia areata (AA) affecting more than 50% (percent) of the scalp. She has tried and failed topical, intralesional, and systemic steroids. She is allergic to hydroxychloroquine. The disease has greatly affected her quality of life, induced depression, and societal withdrawal. This is a request for Xeljanz 10 mg (milligrams) BID (twice a day).

Yes, the health plan should cover the proposed treatment.

The current literature reflects Xeljanz as being more effective than other treatments.

AA is a relatively common disease, but no satisfactory treatment has yet been developed. Recently, research progress has been made in the pathogenesis of AA, 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 AA 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 AA. 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 AA (at least 40% 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 mg BID) 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 10 years or less, 77% had a clinical response (at least 6% improvement in the Severity of Alopecia Tool [SALT] score) and 58% achieved greater than 50% improvement in the SALT score over 4 to 18 months of treatment. Patients with a disease episode longer than 10 years appeared less likely to respond to treatment; the clinical response rate in this population was 32% (8 of 25 patients). No serious adverse effects occurred during treatment.

There is promising low-quality evidence regarding the effectiveness of JAK inhibitors in AA. Future large-sized randomized studies are required to confirm findings. However, of all the treatments reported to work in AA, JAK inhibitors have the most promising results to date. Because of the potential for serious adverse effects with areata, janus 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 AA based on the literature.

Therefore, given the above, Xeljanz is the best treatment option for this patient at this time.

Yes, the current literature reflects tofacitinib (Xeljanz) as being more effective than other
treatments. AA is a complex autoimmune disease with a multi-factorial etiology that has severe negative impact on a patient's psychoemotional well-being as well as their quality of life, and accounts for a significant global disease burden. With greater knowledge and a better understanding into the pathomechanism of AA, the emergence of JAK inhibitor use for AA, treatment is in fact very promising and serves as an effective and viable strategy in treating this debilitating disease. Oral and topical formulation of JAK inhibitors tofacitinib, ruxolitinib, and baricitinib were studied in the treatment of AA and its variants. A growing body of scientific publications, mostly in the form of case reports, has demonstrated exceptional efficacy of oral formulations of JAK inhibitors,
particularly with tofacitinib and ruxolitinib in treating moderate to severe, as well as refractory recalcitrant cases of AA. Moreover, it was demonstrated that these patients would need to maintain JAK inhibitor treatment in order to maintain hair regrowth. In addition to these, reports also identified adverse effects that were not severe, but mostly mild and transient.