Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Alopecia Areata.
Treatment: Tofacitinib Citrate POW (powder) Compound.
The insurer denied the Tofacitinib Citrate POW (powder) Compound.
The denial is overturned.

The patient is a male with alopecia areata, treated with oral dexamethasone and fexofenadine, as well as topical steroids, retinoids, and minoxidil. His provider is requesting topical Tofacitinib. The patient has most of the scalp affected.

The requested health service is medically necessary.
JAK (janus kinase) inhibitors are emerging as first line therapies for alopecia areata given their promising efficacy. 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.

Xeljanz is a Jak-kinase inhibitor, which blocks IL (interleukin)-15 signaling. IL (interleukin)-15 has a pivotal role in life and death of natural killer (NK) and CD8 (cluster of differentiation 8) memory T cells. IL (interleukin)-15 signals through a heterotrimeric receptor involving the common gamma chain (c) shared with IL (interleukin)-2, IL (interleukin)-4, IL (interleukin)-7, IL (interleukin)-9, and IL (interleukin)-21, IL (interleukin)-2/IL (interleukin)-15 receptor (IL-15R) shared with IL (interleukin)-2 and a private IL (interleukin)-15R subunit. IFN (interferon)- or CD40 (cluster of differentiation) ligand-stimulated dendritic cells coordinately express IL (interleukin)-15 and IL (interleukin)-15R. Cell surface IL (interleukin)-15R presents IL (interleukin)-15 in-trans to cells that express IL (interleukin)-2/IL (interleukin)-15R and c. IL (interleukin)-15 is being used to treat patients with metastatic malignancy. However, IL (interleukin)-15 is an inflammatory cytokine involved in immunological memory including that to self, thereby playing a role in autoimmune diseases. These insights provide the scientific basis for clinical strategies directed toward diminishing IL (interleukin)-15 action. Dysregulated IL (interleukin)-15 expression was demonstrated in patients with rheumatoid arthritis, inflammatory bowel disease, psoriasis, celiac disease, and alopecia areata.

Tofacitinib first demonstrated efficacy in alopecia areata in the article published several years ago. A patient with concomitant psoriasis and alopecia areata was treated with the medication and serendipitously his alopecia areata reversed. Since then further reports have emerged documenting its efficacy.

A phase I, 28 week prospective, placebo-controlled, double-blind study (Bokhari) in patients with alopecia universalis investigated hair regrowth with two topical JAK (janus kinase) inhibitors, 2% tofacitinib and 1% ruxolitinib. Topical clobetasol dipropionate 0.005% was the active comparator while vehicle was used as the placebo control. Sixteen patients were recruited for the study. Six patients demonstrated partial hair regrowth in areas treated with 2% tofacitinib ointment applied twice daily. Five patients demonstrated partial hair regrowth in the areas treated with 1% ruxolitinib ointment. Ten patients demonstrated partial hair regrowth in the areas treated with clobetasol dipropionate 0.05% ointment. No regrowth was observed in the placebo treated area.

This patient has already tried and failed topical steroids. Therefore given the benefit seen with topical agents, and the overall safety of the topical form in children, the request is medically necessary.

The health plan did not act reasonably, with sound medical judgment, and in the best interest of the patient.