Sherman Matthew A, Nicolai Rebecca, Datyner Emily K, Rosina Silvia, Hamilton Angela, Ardalan Kaveh, Bader-Meunier Brigitte, Brown Amanda G, Jansen Marc H A, Kim Susan, Lang Bianca, Campanilho-Marques Raquel, McCann Liza J, Sanner Helga, Veldkamp Saskia R, Wilkinson Meredyth G Ll, Yi Belina Y, Kim Hanna, Tarvin Stacey E, Papadopoulou Charalampia
Division of Allergy, Immunology, and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA.
Division of Rheumatology, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Rheumatology (Oxford). 2025 Aug 1;64(8):4732-4737. doi: 10.1093/rheumatology/keaf086.
Janus kinase inhibition (JAKi) has been proposed as a treatment for idiopathic inflammatory myopathies to target increased interferon signalling. Predominantly retrospective reports have demonstrated effectiveness of JAKi in refractory JDM. However, JAKi remains an off-label treatment for JDM and there may be variation in use worldwide. An international survey was conducted to investigate approaches to JAKi for JDM.
The Childhood Arthritis and Rheumatology Research Alliance (CARRA) JDM Therapeutics workgroup and core members of the Paediatric Rheumatology European Society (PReS) JDM working party devised an electronic survey to assess the use of JAKi in JDM. CARRA and PReS members were invited by e-mail to complete the survey.
There were 229 respondents (18%), with 50% from the USA and 29% from Europe. One hundred and fifty had used JAKi for over 450 patients with JDM; among them, 77% noted clinical improvement in most or all patients and 17% reported side effects. The highest ranked perceived barriers to JAKi use were lack of clinical data and inability to obtain insurance approval. The highest ranked clinical indications for starting JAKi were refractory skin disease, refractory muscle disease, inability to wean steroids and intolerance to other steroid-sparing agents.
Paediatric rheumatologists use JAKi off-label for refractory JDM. Most providers noted clinical improvement in their patients. Barriers to JAKi use include lack of clinical data and insurance coverage. Clinical trials are needed to provide better data on the efficacy and safety of JAKi.
Janus激酶抑制（JAKi）已被提议作为治疗特发性炎性肌病的方法，以针对增加的干扰素信号传导。主要的回顾性报告已证明JAKi在难治性皮肌炎（JDM）中的有效性。然而，JAKi仍然是JDM的一种非标签治疗方法，并且在全球范围内的使用可能存在差异。进行了一项国际调查以研究JAKi治疗JDM的方法。
儿童关节炎和风湿病研究联盟（CARRA）的JDM治疗工作组以及欧洲儿科风湿病学会（PReS）JDM工作组的核心成员设计了一项电子调查，以评估JAKi在JDM中的使用情况。通过电子邮件邀请CARRA和PReS成员完成调查。
共有229名受访者（18%），其中50%来自美国，29%来自欧洲。150人曾将JAKi用于450多名JDM患者；其中，77%指出大多数或所有患者有临床改善，17%报告有副作用。使用JAKi的最高排名的感知障碍是缺乏临床数据和无法获得保险批准。开始使用JAKi的最高排名的临床指征是难治性皮肤病、难治性肌肉疾病、无法停用类固醇以及对其他类固醇替代药物不耐受。
儿科风湿病学家将JAKi用于难治性JDM的非标签治疗。大多数提供者指出他们的患者有临床改善。使用JAKi的障碍包括缺乏临床数据和保险覆盖。需要进行临床试验以提供关于JAKi疗效和安全性的更好数据。
