Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Alopecia Universalis
Treatment: Xeljanz 10mg Tablet
The insurer denied the Xeljanz 10mg Tablet.
The denial is overturned. 

The patient is a female with severe, diffuse hair loss (alopecia universalis) all over her body since the age of seven. Due to lack of eyelashes and eyebrows, she experiences severe eye irritation. She has tried and failed to topical and intralesional steroid, as well as a trial of Dupixent. This is a request for tofacitinib (Xeljanz).

Yes, the treatment with Xeljanz is medically necessary, and it s likely to be more beneficial than any other therapy.

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

Alopecia areata universalis (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 (alopecia areata), revealing that autoreactive cytotoxic T cells are important and that the Janus kinase (JAK) pathway is involved. Therefore, the potential of JAK (Janus kinase) inhibitors as therapeutic agents for AA (alopecia areata) is attracting attention. There have been a number of case reports and small clinical trials reporting promising outcomes of JAK (Janus kinase) inhibitors tofacitinib, ruxolitinib and baricitinib for AA (alopecia areata). The majority of the literature to date is based on small volume data, with a lack of definitive evidence or guidelines.

To determine the expected response of AA (alopecia areata) to JAK (Janus kinase) inhibitor therapy and factors which influence response and recurrence rates, Phan et al. performed a systematic review and meta-analysis according to PRISMA (Preferred Reporting Items for Systematic Reviews and Meta-Analyses) guidelines. From 30 studies and 289 cases, there were 72.4% responders, good responders 45.7% and partial responders 21.4%. Mean time to initial hair growth was 2.2 - 6.7 months, and time to complete hair regrowth was 6.7 - 2.2 months. All 37 recurrences occurred when treatment was ceased after 2.7 months. Oral route was significantly associated with response to treatment compared to topical therapy. No difference was found between pediatric and adult cases in proportion of responses.

There is promising low-quality evidence regarding the effectiveness of JAK (Janus kinase) inhibitors in AA (alopecia areata). Future large-sized randomized studies are required to confirm findings. However, of all the treatments reported to work in alopecia areata, JAK (Janus kinase) inhibitors have the most promising results to date. Although the long-term safety of JAK (Janus kinase) inhibitors in children is still unknown, tofacitinib is undergoing clinical trials for juvenile idiopathic arthritis in children aged two to 18 years. Reports from the experience of authors suggest that tofacitinib may be a therapeutic option for children with severe AA (alopecia areata) who have failed conventional therapy. However, because of the potential for serious adverse effects with JAK (Janus kinase) inhibitors, including infection and malignancy, a thorough discussion of risks and benefits is necessary before the initiation of treatment.

Therefore, given the above, the treatment with Xeljanz (tofacitinib) is the best treatment option for this patient at this time, and as such, it is medically necessary.

The patient has failed standard therapy, and Xeljanz (tofacitinib) is likely to be more beneficial than any other therapy.

Given this patient's diagnosis and history, treatment with Xeljanz would be considered to be reasonable and consistent with the current standard of care in the dermatology community.