Objective: To elucidate the clinical features of patients with refractory juvenile dermatomyositis (JDM), and to explore the efficacy and safety of tofacitinib in the treatment of refractory JDM. Methods: A total of 75 JDM patients admitted to the Department of Rheumatology and Immunology in Shenzhen Children's Hospital from January 2012 to January 2021 were retrospectively analyzed, and to analyze the clinical manifestations, efficacy and safety of tofacitinib in the treatment of refractory JDM. Patients were divided into refractory group with using of glucocorticoids in combination with two or more anti-rheumatic drugs for treatment, and the presence of disease activity or steroid dependence after a one-year follow-up. The non-refractory group is defined as clinical symptoms disappeared, laboratory indicators were normal, and clinical remission was achieved after initial treatment, and the clinical manifestations and laboratory indexes of the two groups were compared. The Mann-Whitney U test, Fisher's precision probability test was used for intergroup comparison. Binary Logistic multivariate regression analysis was used to identify risk factors for refractory JDM. Results: Among the 75 children with JDM, 41 were males and 34 were females with a age of onset of 5.3 (2.3, 7.8) years. The refractory group consisted of 27 cases with a age of onset of 4.4 (1.5, 6.8) years, while the non-refractory group consisted of 48 cases with a age of onset of 5.9 (2.5, 8.0) years. Compared with 48 cases in the non-refractory group, the proportion of interstitial lesions and calcinosis in the refractory group was higher than that in the non-refractory group (6 cases (22%) vs. 2 cases (4%), 8 cases (30%) vs. 4 cases (8%), both P<0.05). Binary Logistic regression analysis showed that observation group were more likely to be associated with to interstitial lung disease (OR=6.57, 95%CI 1.22-35.31, P=0.028) and calcinosis (OR=4.63, 95%CI 1.24-17.25, P=0.022). Among the 27 patients in the refractory group, 22 cases were treated with tofacitinib, after treatment with tofacitinib, 15 of 19 cases (86%) children with rashes showed improvement, and 6 cases (27%) with myositis evaluation table score less than 48 score both were improved, 3 of 6 cases (27%) had calcinosis were relieved, and 2 cases (9%) had glucocorticoid-dependence children were successfully weaned off. During the tofacitinib treatment, there was no increase in recurrent infection, blood lipids, liver enzymes, and creatinine were all normal in the 22 cases. Conclusions: Children with JDM with calcinosis and interstitial lung disease are more likely to develop refractory JDM. Tofacitinib is safe and effective for refractory JDM.
目的： 分析难治性幼年皮肌炎患儿临床特征并总结托法替布治疗难治性幼年皮肌炎的有效性及安全性。 方法： 回顾性病例总结，收集2012年1月至2021年1月深圳市儿童医院风湿免疫科收治的75例幼年皮肌炎患儿的病例资料，分析托法替布治疗难治性幼年皮肌炎的临床表现及其效果及安全性。糖皮质激素联合两种或以上抗风湿药物治疗1年后仍存在疾病活动或激素依赖患儿为难治组；初始治疗后临床症状消失，实验室指标正常，达到临床缓解为非难治组，对比两组患儿的临床表现及实验室指标。组间比较采用Mann-Whitney U检验、Fisher确切概率检验。应用二元Logistic多因素回归分析难治组危险因素。 结果： 75例幼年皮肌炎患儿中男41例、女34例，发病年龄5.3（2.3，7.8）岁。难治组27例，发病年龄4.4（1.5，6.8）岁。非难治组48例，发病年龄5.9（2.5，8.0）岁。难治组合并间质性肺疾病和钙质沉着的比例均高于非难治组［6例（22%）比2例（4%），8例（30%）比4例（8%），均P<0.05］。二元Logistic回归分析示难治组更易发生间质性肺疾病（OR=6.57，95%CI 1.22~35.31，P=0.028）及钙质沉着（OR=4.63，95%CI 1.24~17.25，P=0.022）。27例难治组患儿中22例加用托法替布治疗，治疗后，19例（86%）患有皮疹的患儿中15例有改善，6例（27%）儿童肌炎评估表评分<48分的患儿均有改善，6例（27%）存在钙质沉着中的患儿中3例得到缓解，2例（9%）糖皮质激素依赖患儿均成功减量。22例患儿托法替布治疗中均未发现反复感染增加情况，血脂、肝酶、肌酐均正常。 结论： 合并间质性肺疾病及钙质沉着的幼年皮肌炎患儿发展成难治性幼年皮肌炎可能性高。托法替布治疗难治性幼年皮肌炎安全有效。.