Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Alopecia areata
Treatment: Xeljanz
The insurer denied Xeljanz.
The determination is upheld.

This is a male patient with severe alopecia areata universalis of the scalp, face, and body. The patient has tried and failed topical medications such as minoxidil and compounded Xeljanz, as well as intralesional steroids and oral minoxidil. Xeljanz resulted in dramatic improvement in only three months. After this regrowth occurred, then the oral minoxidil was discontinued and the hair regrowth continued (confirming that the Xeljanz, and not the minoxidil, was the medication that caused the hair regrowth).

On Xeljanz the patient's hair regrew all over his scalp, face, and body, so that his return to school after the coronavirus disease (COVID) pandemic was greatly facilitated with respect to the patient's mental and social/psychological health.

While understandable to consider Xeljanz (tofacitinib) for this patient's condition, the requested medication merits further study before being presumed acceptable for widespread clinical use. At this time, the long-term safety of this medication is not as confirmed as short-term efficacy in the use of Xeljanz for this condition.

Alopecia areata is notoriously difficult and unpredictable to treat, as well as unpredictable in its response to treatment. Oral immunosuppressants, light therapy, contact allergen treatment and related therapies have all resulted in variable and unsatisfactory outcomes, and self-resolution is often the only choice a patient has (along with hairpieces) for severe cases.

Xeljanz was denied Food and Drug Administration (FDA) approval for use in psoriasis, in large part for safety reasons, and is not without a significant risk of side effects that preclude an acceptable risk for use outside of the investigational setting for the condition of alopecia areata.

At this time, Xeljanz is only FDA-approved for rheumatoid arthritis patients who have failed methotrexate, for juvenile idiopathic arthritis, for psoriatic arthritis, for ankylosing spondylitis, and for ulcerative colitis--all five conditions have indications whereby the safety risks of not using treatment are significantly greater than treatment itself. Accordingly, the health plan policy [1] only covers Xeljanz for these four conditions. There is no significant history rheumatoid arthritis failing methotrexate to support medical necessity for Xeljanz for that indication. On a related note, Xeljanz has been recommended to be used with lower dosing in rheumatoid arthritis patients because of concerns about excessive blood clotting.

The same cannot be said for Xeljanz in the treatment of alopecia areata, where physical/functional impairment and overall health impacts are relatively minimal to nonexistent, despite the social/psychological impacts that can occur in some patients. The proper dosing, anticipated side effects and long-term outcomes are not known at this time for Xeljanz in the treatment of alopecia areata, as well as short-term and long-term safety risks of relevance to this patient's overall health. There are no large, randomized, controlled, prospective trials demonstrating and proving the safe and effective treatment of alopecia areata with a specific dosing of Xeljanz.

Therefore, as per the evidence-based literature, Xeljanz is not indicated for less-monitored outpatient clinical use. However, off-label use of Olumiant at the 4 milligram (mg) /day dosing is recommended as best consistent with standards of good medical practice to be pursued for this patient's benefit at this time.