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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 …
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.
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