Argüelles Balas Delia, Barral Mena Estefanía, Martín Díaz M Ángeles, Calvo-Aranda Enrique
Paediatric Department, Hospital Universitario Infanta Leonor, Madrid, Spain
Paediatric Department, Hospital Universitario Infanta Leonor, Madrid, Spain.
RMD Open. 2025 May 21;11(2):e005651. doi: 10.1136/rmdopen-2025-005651.
Juvenile dermatomyositis (JDM) is a multisystem autoimmune disease associated with considerable morbidity and mortality, having a significant impact on patient health. Early diagnosis and appropriate treatment play a key role in optimising outcomes. JDM management remains challenging, particularly in severe or refractory cases, due to the limited evidence regarding therapeutic options. Given these challenges, Janus kinase inhibitors, including tofacitinib, have emerged as promising alternatives, yet evidence in paediatric populations is still scarce. We present the case of a child with JDM who achieved sustained remission with tofacitinib monotherapy following the failure of previous therapies. While its use is currently restricted to refractory cases, these findings suggest its potential for earlier integration into treatment algorithms. This work aims to provide valuable insights to guide therapeutic strategies in JDM.
幼年皮肌炎（JDM）是一种多系统自身免疫性疾病，伴有相当高的发病率和死亡率，对患者健康有重大影响。早期诊断和适当治疗在优化治疗结果方面起着关键作用。JDM的管理仍然具有挑战性，特别是在重症或难治性病例中，因为关于治疗选择的证据有限。鉴于这些挑战，包括托法替布在内的Janus激酶抑制剂已成为有前景的替代药物，但儿科人群中的证据仍然很少。我们报告了一例JDM患儿，在先前治疗失败后，托法替布单药治疗使其获得持续缓解。虽然目前其使用仅限于难治性病例，但这些发现表明它有可能更早地纳入治疗方案。这项工作旨在提供有价值的见解，以指导JDM的治疗策略。
