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41 CLINICAL REPORT Comparison of the Treatment Outcome of Oral Tofacitinib with Other Conventional Therapies in Refractory Alopecia Totalis and Universalis: A Retrospective Study Jung-Won SHIN 1 , Chang-Hun HUH 1 , Min-Woo KIM 2 , Ji-Su LEE 2 , Ohsang KWON 2 , Soyun CHO 3 and Hyun-sun PARK 3 Departments of Dermatology: 1 Seoul National University Bundang Hospital, Gyeonggi, 2 Seoul National University Hospital, and 3 SMG-SNU Boramae Medical Center, Seoul, Korea Treatment of alopecia totalis and alopecia universalis is often challenging and unsatisfactory. Recently, Ja- nus kinase inhibitor has shown promising results. The aim of this study is to compare the efficacy and tole- rability of oral tofacitinib and conventional modalities for treating refractory alopecia totalis/universalis. A total of 74 patients (18 treated with tofacitinib, 26 treated with conventional oral treatment (steroid  ±  cy- closporine), and 30 treated with diphenylcyclopro- penone) were included in the study. The patients’ medical records were reviewed retrospectively. After 6 months, 44.4% of patients in the tofacitinib group, 37.5% in the conventional oral treatment group, and 11.1% in the diphenylcyclopropenone group achieved 50% improvements in the Severity of Alopecia Tool score. During treatment, 10% of patients in the to- facitinib group, 73.1% in the conventional oral treat- ment group, and 10% in the diphenylcyclopropenone group experienced adverse drug reactions. In conclu- sion, oral tofacitinib was more effective than diphe- nylcyclopropenone immunotherapy and more tolera- ble than conventional oral treatment after 6 months of treatment. Key words: alopecia totalis; alopecia universalis; cyclosporine; diphenylcyclopropenone; oral steroid; tofacitinib. Accepted Oct 3, 2018; Epub ahead of print Oct 3, 2018 Acta Derm Venereol 2019; 99: 41–46. Corr: Hyun-sun Park, SMG-SNU Boramae Medical Center, Boramae-ro- 5-gil, Dongjak-gu, Seoul 07061, Korea. E-mail: [email protected] A lopecia totalis (AT) and alopecia universalis (AU) are rare, extensive subtypes of alopecia areata (AA). AA is a common autoimmune dermatological disease characterized by non-scarring hairless patches on the scalp, often resulting in significant psychological distress for patients (1–3). The extent of alopecia may predict the degree of psychological distress experienced (2). Hence, given that AA can negatively influence the quality of life of patients, striving for a treatment with greater efficacy and safety is paramount. To date, despite numerous proposed treatment methods, there is no reliable and efficacious therapy for AA. Moreover, extensive forms of AA, such as AT and AU, are known to have a poorer treatment response with greater probability of developing chronic diseases (4). SIGNIFICANCE Treatment of severe alopecia areata is often challenging and unsatisfactory. However, oral tofacitinib, which is approved for treatment of rheumatoid arthritis, has had promising results. This is the first study to compare the treatment outcomes of tofacitinib with other conventional therapies in patients with refractory severe alopecia areata. After 6 months of treatment, patients on tofacitinib had a higher response rate and greater tolerability than those on topical immunotherapy and oral steroid  ±  immunosuppres- sant, respectively. This study will help clinicians to better determine treatment options for severe refractory alopecia areata. It has been shown recently that blocking the common signalling pathways downstream of cytokine receptors, particularly Janus kinase (JAK), may reverse AA in mice (5). Based on this information, JAK inhibitors have been proposed as a novel promising therapeutic option for AA. In several previous studies, tofacitinib, one of the JAK inhibitors, has been shown to have promising results in managing severe AA (6–8). However, lack of evidence, safety uncertainties, and high associated costs compared with other conventional methods have limited its app- lication in clinical practice. Thus far, to the best of our knowledge, there is no report comparing the treatment outcomes between tofacitinib and other conventional therapies. Hence, this study retrospectively compared the efficacy and tolerability of oral tofacitinib with con- ventional oral treatment and topical immunotherapy in treating refractory AT/AU. METHODS This study was approved by the Institutional Review Board of Seoul National University Bundang Hospital (B-1708/415-204) and SMG-SNU Boramae Medical Center (10-2017-24). Elec- tronic medical records and clinical photographs were reviewed for all patients with AT or AU who were treated in a tertiary or a secondary hospital with oral tofacitinib, conventional oral treat- ment (steroid  ±  cyclosporine), or diphenylcyclopropenone (DPCP) immunotherapy during a period between August 2005 and August 2017. Inclusion criteria were: (i) ≥ 18 years old; (ii) AT (> 80% scalp hair loss) and AU (> 80% scalp hair loss with total body hair loss); (iii) ≥1 year of current episode duration; (iv) previous treatment failure of at least one conventional oral treatment or im- munotherapy; and (v) at least 6-month follow-up period. Clinical This is an open access article under the CC BY-NC license. www.medicaljournals.se/acta Journal Compilation © 2019 Acta Dermato-Venereologica. doi: 10.2340/00015555-3057 Acta Derm Venereol 2019; 99: 41–46