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. He has a medical history of alopecia universalis. The appeals letter endorses a history of failure to topical steroids, oral minoxidil, and topical minoxidil. This request for coverage of Xeljanz (tofacitinib) is under review.
the health plan should cover the requested Xeljanz. Alopecia universalis in pediatric patients presents a significant therapeutic challenge due to the absence of United States Food and Drug Administration (FDA)-approved treatments and limited efficacy of conventional therapies such as topical steroids and minoxidil. Recent research has shed light on the potential role of Janus kinase (JAK) inhibitors, such as Xeljanz, in addressing this autoimmune condition. Janus kinase, signal transducer and activator of transcription protein signaling has been implicated in the pathogenesis of alopecia areata, with dysregulated cytokine expression contributing to hair follicle damage. Several case reports and small-scale studies have demonstrated promising outcomes with janus kinase inhibitors, including tofacitinib, in 
promoting hair regrowth in both adults and children with alopecia areata. By inhibiting the janus kinase pathway, tofacitinib modulates immune responses and may help restore hair follicle function. Its safety profile in pediatric populations has been well studied in the context of other autoimmune conditions, providing valuable insights into its potential use for alopecia universalis. Considering the refractory nature of the patient's condition and the limited treatment options available, the use of tofacitinib under medical supervision is medically appropriate. A literature review notes, "Various first-generation JAK [janus kinase] inhibitors, which include ruxolitinib, tofacitinib and baricitinib, have been found to be highly efficacious and well tolerated in patients with psoriasis, atopic dermatitis, vitiligo, and AA [alopecia areata] among others. Studies of AA [alopecia areata] mouse models revealed the importance of JAK-STAT [janus kinase, signal transducer and activator of transcription protein] signaling for development of autoreactive cluster of differentiation 8 (CD8+) T-cell targeting of the hair follicle and confirmed that JAK [janus kinase] inhibitors promoted hair regrowth in affected animals. The first patient with AA [alopecia areata] treated with tofacitinib was reported in 2014, and since then, a number of case series and a few small clinical trials have demonstrated hair regrowth in adult patients with moderate to severe AA [alopecia areata] treated with JAK [janus kinase] inhibitors. (1)"
A case series notes, "Although presently there are no long-term safety data in the pediatric population, tofacitinib is undergoing clinical trials for juvenile idiopathic arthritis in children aged 2 to 18 years (NCT02592434 and NCT01500551). On the basis of their weights, the dose of 5 mg [milligrams] twice daily that we used in our patients is consistent with the dosing in these clinical trials, except in the case of patient 1, who would have received 4 mg [milligrams] twice daily. Although ours is a small series, the results presented here are favorable and corroborate those of prior larger studies in adolescents and adults. AA [alopecia areata] can be devastating at any age, but in our experience, childhood and adolescence represent particularly vulnerable periods for patients. We suggest that after proper counseling regarding the risks, including severe infection and malignancy, the use of tofacitinib may be considered for preadolescent children with AA [alopecia areata] who are experiencing psychosocial impairment. (2)"