Article Text

Download PDFPDF
Efficiency of tofacitinib in refractory interstitial lung disease among anti-MDA5 positive juvenile dermatomyositis patients
  1. Yuan Xue1,
  2. Junmei Zhang1,2,3,
  3. Jianghong Deng1,
  4. Weiying Kuang1,
  5. Jiang Wang1,
  6. Xiaohua Tan1,
  7. Chao Li1,
  8. Shipeng Li1,
  9. Caifeng Li1
  1. 1 Department of Rheumatology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, China
  2. 2 Department of Nephrology and Rheumatology, Children's Hospital Affiliated to Zhengzhou University, Zhengzhou, China
  3. 3 Department of Nephrology and Rheumatology, Henan Children's Hospital Zhengzhou Children's Hospital, Zhengzhou, China
  1. Correspondence to Professor Caifeng Li, Department of Rheumatology, Beijing Children's Hospital, Capital Medical University, National Centre for Children’s Health, Beijing, 100045, China; caifeng_li{at}yeah.net

Statistics from Altmetric.com

Request Permissions

If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.

As one of the most prevalent inflammatory myopathy disease in childhood, the patients with juvenile dermatomyositis (JDM) suffer from a great heterogeneity in clinical manifestations.1 Among them, anti-MDA5 presented with a a greater risk with interstitial lung disease (ILD), which may lead to poor prognosis. In clinical practice, some patients are refractory to immunosuppressive treatments. JAK inhibitors (JAKi) can regulate JAK-STAT pathway and decrease the interferon-induced STAT1 phosphorylation, and has been applied into both adult DM and patients with JDM.2–5 Here, we share the experience of JAKi therapy in nine anti-MDA5 positive refractory JDM cases and tried to identify the risk factors for poor response. The nine patients were diagnosed and classified according to Bohan and Peter’s criteria and received treatment between July 2017 and November 2022. We obtained written informed consent from the guardians of all patients before initiating JAKi therapy.

Among the nine patients, 33% (3/9) patients were female, the mean age of onset was 5.1±3.6 years, the mean age to start add-on JAKi therapy was 8.3±3.5 years. Three cases of them progressed rapidly in ILD. All the nine cases suffered from refractory ILD with poor …

View Full Text

Footnotes

  • Handling editor Josef S Smolen

  • YX and JZ contributed equally.

  • Contributors YX and JZ contribute the same in this study.YX,JZ and CFL designed the research study; YX wrotethe manuscript; JZ,JD, and WK collected the clinical data.SL and XT revised themanuscript. All authors read and approved the final manuscript.

    YX,JZ and CFL designed the research study; YX wrotethe manuscript; JZ,JD, and WK collected the clinical data.SL and XT revised themanuscript. All authors read and approved the final manuscript.

  • Funding This study was supported by the Capital's Fund's for Health Improvement and Research(CFH2022-2-2096) and National Regional Medical Center Opening Project(NRMC0110).

  • 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.

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

  • Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.