Department of Rheumatology, Beijing Children's Hospital, Capital Medical University, National Center for Children's Health, Beijing, 100045, China.
Department of Rheumatology, Children's Hospital of Fudan University, National Children's Medical Center, No. 399 Wanyuan Road, Shanghai, 201102, China.
Arthritis Res Ther. 2023 Oct 18;25(1):204. doi: 10.1186/s13075-023-03170-z.
Juvenile dermatomyositis (JDM) is a chronic autoimmune disease. Some patients remain in an active state even though they were administrated with a combination of corticosteroid and methotrexate. Existing research has suggested that interferon and Janus kinase played an important role in pathogenesis. Existing research has suggested the efficacy of JAK inhibitors (JAKi). Our retrospective study aimed to investigate the efficacy of tofacitinib in refractory JDM patients.
A total of eighty-eight patients in China who had been diagnosed with JDM and subjected to tofacitinib therapy for over 3 months were retrospectively analyzed. Skin and muscle manifestations were assessed using the Cutaneous Assessment Tool-binary method (CAT-BM), Childhood Myositis Assessment Scale (CMAS), and kinase. Pulmonary function was assessed using a high-resolution CT (computerized tomography) scan and pulmonary symptoms. All patients were subjected to regular follow-up, and core measures were assessed every 3 months after initiation. Furthermore, the data were analyzed using the Wilcoxon single test, Mann-Whitney U test, and chi-square test.
Compared with the baseline data, skin and muscle manifestations were found significantly improved during the respective follow-up visit. At the most recent follow-up, nearly 50% of patients achieved a clinical complete response and six patients received tofacitinib monotherapy. Sixty percent of patients suffering from interstitial lung disease well recovered on high-resolution CT. Seventy-five percent of patients showed a reduction in the size or number of calcinosis, and 25% of patients showed completely resolved calcinosis.
In this study, the result suggested that tofacitinib therapy exerted a certain effect on skin manifestations, muscle manifestations, interstitial lung disease (ILD), calcinosis, as well as downgrade of medication. In-depth research should be conducted to focus on the correlation between the pathogenesis of JDM and JAKi.
幼年型皮肌炎（JDM）是一种慢性自身免疫性疾病。尽管一些患者接受了皮质类固醇和甲氨蝶呤的联合治疗，但仍处于活动状态。现有研究表明，干扰素和 Janus 激酶在发病机制中发挥了重要作用。现有研究表明 JAK 抑制剂（JAKi）的疗效。我们的回顾性研究旨在探讨托法替尼在难治性 JDM 患者中的疗效。
回顾性分析在中国诊断为 JDM 并接受托法替尼治疗超过 3 个月的 88 例患者。采用皮肤评估工具-二进制法（CAT-BM）、儿童肌炎评估量表（CMAS）和激酶评估皮肤和肌肉表现。采用高分辨率 CT（计算机断层扫描）和肺症状评估肺功能。所有患者均进行定期随访，在起始后每 3 个月评估核心指标。此外，采用 Wilcoxon 单项检验、Mann-Whitney U 检验和卡方检验对数据进行分析。
与基线数据相比，在各自的随访中发现皮肤和肌肉表现明显改善。在最近的随访中，近 50%的患者达到临床完全缓解，6 例患者接受托法替尼单药治疗。60%的间质性肺病患者在高分辨率 CT 上恢复良好。75%的患者钙沉积的大小或数量减少，25%的患者钙沉积完全消退。
在这项研究中，结果表明托法替尼治疗对皮肤表现、肌肉表现、间质性肺病（ILD）、钙沉积以及药物降级有一定疗效。应深入研究 JDM 和 JAKi 发病机制之间的相关性。
