Patient 7 presented with alopecia totalis (A) and attained 90% regrowth (B) after 3 months of treatment with oral tofacitinib.
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Ibrahim O, Bayart CB, Hogan S, Piliang M, Bergfeld WF. Treatment of Alopecia Areata With Tofacitinib. JAMA Dermatol. 2017;153(6):600–602. doi:10.1001/jamadermatol.2017.0001
Tofacitinib citrate is a Janus kinase 1/3 inhibitor approved for the treatment of rheumatoid arthritis, but it has recently been used to treat alopecia areata (AA).1-3 In this study, investigators searched the medical records of the Cleveland Clinic for any patients with confirmed AA who were treated with oral tofacitinib (Xeljanz; Pfizer) using a standardized, systematic treatment regimen.
A standard departmental treatment protocol was developed prior to this review. Treatment with tofacitinib citrate was initiated at 5 mg twice daily, and all other AA therapies were ceased. The daily dosage was increased, as allowable by insurance coverage, by 5 mg per month until the treating physician noted first signs of hair regrowth and then held the medication at that dose. The Cleveland Clinic Foundation Institutional Review Board approved this retrospective medical record review and waived the patient informed consent requirement. The review took place from May 1, 2016, to June 1, 2016.
Scalp hair loss was calculated from visit to visit by the same treating physician (M.P. or W.B.) using the validated Severity of Alopecia Tool (SALT)4 score, which can range from 0% to 100%; the higher the score, the greater the amount of scalp hair loss. In all, patients were treated by 2 different physicians (M.P. and W.B.) from January 10, 2015, to April 30, 2016.
We identified 13 patients with AA who were or are being treated with tofacitinib. The mean (SD) pretreatment scalp hair loss was 93% (11.5), with 2 patients having alopecia totalis (Figure, A) and 7 patients having alopecia universalis. Regrowth rate—(final SALT score − initial SALT score)/(initial SALT score) × 100—ranged from 2% to 90%, with a mean (SD) of 44.3% (31.9) and a median (range) of 50.5% (90 [0-90]) (Table). Seven patients (53.8%) achieved a regrowth of at least 50% (Figure, B). Response time—time from initiation of treatment to any sign of hair regrowth—ranged from 1 to 9 months, with a mean (SD) of 4.2 (2.6) months.
One patient developed a morbilliform eruption and peripheral edema that led to medication withdrawal. Two patients stopped therapy after 3 months because of loss of insurance and within 2 weeks experienced a shed that led back to baseline levels. The remaining 11 patients continued treatment. Of note, 2 patients demonstrated lipid and liver abnormalities that were resolved when the dose was reduced (Table).
Janus kinase inhibitors have been shown to attenuate the inflammatory cascade associated with AA.1 Our results indicate that tofacitinib is efficacious in the treatment of AA. Although small, our cohort achieved greater median improvement in SALT scores than reported in previously published studies (50.5% vs 21%).3 This outcome may be related to our higher doses, longer duration of therapy, and patients’ shorter duration of current disease episode. In addition, our results demonstrate lack of durability of effect after the discontinuation of therapy, a finding similar to that in other studies.3
Limitations of this study included the absence of a control group, which prevented the comparison of tofacitinib with a placebo, and the small sample size, which precluded subgroup analyses. In retrospect, the daily doses could have been lower and increased more gradually yet still achieved similar results given that patient response seemed to be both dose and time dependent.3 Dosages higher than 10 mg twice a day may be associated with higher risk for serious infections and malignant neoplasms, which have been reported at baseline dosages of 5 mg twice daily.5
Oral tofacitinib is a successful treatment for AA, but its efficacy varies widely. To truly assess efficacy, therapeutic trials should continue in the clinical setting for a minimum of 4 months and potentially up to 1 year. Patients should be informed of the lack of durability of effects after the treatment is discontinued. Future studies, including those6 using other Janus kinase inhibitors, such as ruxolitinib phosphate, are needed and should continue to elucidate these medications’ efficacy, safety, and durability in the treatment of AA.
Accepted for Publication: December 29, 2016.
Corresponding Author: Omer Ibrahim, MD, Department of Dermatology, Cleveland Clinic Foundation, 9500 Euclid Ave, A61, Cleveland, OH 44195 (email@example.com).
Published Online: March 29, 2017. doi:10.1001/jamadermatol.2017.0001
Author Contributions: Drs Ibrahim and Bergfeld had full access to all the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis.
Study concept and design: Ibrahim, Piliang, Bergfeld.
Acquisition, analysis, or interpretation of data: All authors.
Drafting of the manuscript: Ibrahim, Bergfeld.
Critical revision of the manuscript for important intellectual content: All authors.
Statistical analysis: Ibrahim, Bayart.
Administrative, technical, or material support: Bayart, Hogan, Piliang, Bergfeld.
Study supervision: Piliang, Bergfeld.
Conflict of Interest Disclosures: None reported.
Additional Contributions: We thank the patient depicted in the Figure for granting permission to publish this information.
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