Over the last decade, there has been an increase in the use of targeted therapy using small molecules such as Janus kinase (JAK) inhibitors. Since the introduction of ruxolitinib, the first non-selective JAK inhibitor approved for use in myelofibrosis, many other JAK inhibitors have been tried in a wide spectrum of immune-mediated disorders. Although various trials have shown the promising efficacy of JAK inhibitors in immune-mediated inflammatory disorders (IMIDs), there is a growing concern over the major cardiovascular events and malignancies associated with the use of these molecules in older adults, particularly those over 65 years of age. In this review, we aim to discuss the immunology of the JAK-STAT pathway, the scope of use of JAK inhibitors, and their safety in paediatric practice. Here, we discuss high-quality evidence favouring the use of JAK inhibitors in children with juvenile idiopathic arthritis (JIA) who are refractory to one or more conventional/biological disease-modifying drugs, demonstrated in two randomised controlled trials (RCTs). In addition to JIA, there are reports favouring the role of JAK inhibitors in other IMIDs such as systemic-onset JIA and interferonopathies. Thus far, the existing literature suggests an acceptable safety profile for JAK inhibitors in children. With the expanding scope of JAK inhibitors in a wide range of IMIDs in children, there is a significant need for long-term close vigilance for any potential harm.

在过去的十年中，使用 Janus 激酶 (JAK) 抑制剂等小分子的靶向治疗的使用有所增加。自从鲁索替尼（第一个被批准用于治疗骨髓纤维化的非选择性 JAK 抑制剂）推出以来，许多其他 JAK 抑制剂已在广泛的免疫介导疾病中进行了尝试。尽管各种试验表明 JAK 抑制剂在免疫介导的炎症性疾病 (IMID) 中具有良好的疗效，但人们越来越关注与老年人（尤其是 65 岁以上老年人）使用这些分子相关的主要心血管事件和恶性肿瘤。年龄。在这篇综述中，我们旨在讨论 JAK-STAT 通路的免疫学、JAK 抑制剂的使用范围及其在儿科实践中的安全性。在这里，我们讨论了两项随机对照试验 (RCT) 中证实的高质量证据，这些证据支持对一种或多种常规/生物疾病缓解药物耐药的幼年特发性关节炎 (JIA) 儿童使用 JAK 抑制剂。除了 JIA 之外，还有报道支持 JAK 抑制剂在其他 IMID 中的作用，例如全身性 JIA 和干扰素病。迄今为止，现有文献表明 JAK 抑制剂在儿童中的安全性是可接受的。随着 JAK 抑制剂在儿童 IMID 中的应用范围不断扩大，非常需要长期密切警惕任何潜在危害。