Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Alopecia Areata
Treatment: Tofacitinib Citrate Powder Compound
The insurer denied the Tofacitinib Citrate Powder Compound
The denial is overturned. 

The patient is a female with alopecia areata, treated with minoxidil, anthralin and topical steroids.  The provider is requesting tofacitinib cream.

Yes, the proposed tofacitinib citrate powder compound is medically necessary.
Janus kinase (JAK) 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 Janus kinase (JAK) inhibitor, which blocks IL-15 signaling.  IL-15 (Interleukin 15) has a pivotal role in life and death of natural killer (NK) and CD8 memory T cells. IL-15 (Interleukin 15) signals through a heterotrimeric receptor involving the common gamma chain (c) shared with IL-2 (Interleukin 2), IL-4 (Interleukin 4), IL-7 (Interleukin 7), IL-9 (Interleukin 9), and IL-21 (Interleukin 21, IL-2/IL-15 (Interleukin 2/Interleukin15) receptor  (IL-15R) shared with IL-2 (Interleukin 2) and a private IL-15R (Interleukin 15 receptor) subunit. IFN (interferon)- or CD40 ligand-stimulated dendritic cells coordinately express IL-15 (Interleukin 15) and IL-15R (Interleukin 15 receptor). Cell surface IL-15R (Interleukin 15 receptor) presents IL-15 (Interleukin 15) in-trans to cells that express IL-2/IL-15R (Interleukin 2/Interleukin 15 receptor) and c. IL-15 (Interleukin 15) is being used to treat patients with metastatic malignancy. However, IL-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-15 action. Dysregulated IL-15 (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 Janus kinase (JAK) 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.

Given the age of the patient and vast literature support for use of Janus kinase (JAK) inhibitors in this extremely challenging condition, the request is medically necessary.