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A pilot study of tofacitinib for refractory Behçet’s syndrome
  1. Jinjing Liu1,2,
  2. Yunxia Hou3,
  3. Luxi Sun1,2,
  4. Chaoran Li1,2,
  5. Lu Li1,2,
  6. Yan Zhao1,2,
  7. Xiaofeng Zeng1,2,
  8. Fengchun Zhang1,2,
  9. Wenjie Zheng1,2
  1. 1 Department of Rheumatology and Clinical Immunology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China
  2. 2 Key Laboratory of Rheumatology and Clinical Rheumatology, Ministry of Education, National Clinical Research Center for Dermatologic and Immunologic Diseases, Beijing, China
  3. 3 Department of Rheumatology, Affiliated Hospital of Inner Mongolia Medical University, Hohhot, China
  1. Correspondence to Professor Wenjie Zheng, Department of Rheumatology and Clinical Immunology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730, China; wenjzheng{at}gmail.com

http://dx.doi.org/10.1136/annrheumdis-2020-217307

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    Behçet’s syndrome (BS) is a chronic inflammatory disease characterised by recurrent oral and genital ulcers, frequently complicated with uveitis gastrointestinal, neurological, major vessel and cardiac involvement. For severe and (or) refractory BS, tumour necrosis factor alpha (TNF-α) inhibitors are indicated beyond glucocorticoids (GCs) and immunosuppressants,1 which are still responded inadequately in a small portion of refractory patients. Tofacitinib, a JAK1/3 inhibitor targeting T cell signalling, was approved for autoinflammatory diseases including rheumatoid arthritis, psoriatic arthritis and ulcerative colitis (UC).2 Given T cells are implicated in BS,3 tofacitinib could be a promising approach for BS.

    In this study, we explored the efficacy and safety of tofacitinib in refractory BS. We enrolled seven male and six female patients with BS fulfilling the 2013 International Criteria for BS,4 presented with active vascular/cardiac (n=5), gastrointestinal (n=6) and articular (n=2) involvements between May 2018 and January 2020 (table 1), with a mean age of 42.2±15.5 years and a median disease duration of 102 (IQR 72–240) months. At enrolment, all patients had active disease (Behçet's Disease Current …

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    Footnotes

    • Handling editor Josef S Smolen

    • Contributors All authors made substantial contributions to the conception and design of this study. LS, CL and LL acquired the data. JL and YH performed the data analysis and drafted the manuscript. YZ, XZ and FZ provided critical revisions to the manuscript. WZ designed the study, contributed to the discussion and edited the manuscript. All authors read and approved the final manuscript.

    • Funding This work was supported by the National Natural Science Foundation of China (grant number 81871299); CAMS Initiative for Innovative Medicine (grant numbers CIFMS2017-I2M-1-008 and CIFMS2016-I2M-1-013), National Key Research and Development Program: “Precise Medical Research” (grant number 2016YFC0906201).

    • Competing interests None declared.

    • Patient and public involvement Patients and/or the public were not involved in the design, or conduct, or reporting, or dissemination plans of this research.

    • Patient consent for publication Not required.

    • Ethics approval Institutional review board of Peking Union Medical College Hospital.

    • Provenance and peer review Not commissioned; externally peer reviewed.