192例幼年皮肌炎临床特点及随访研究

Clinical characteristics and follow-up study of 192 cases of juvenile dermatomyositis

目的研究幼年皮肌炎患儿的治疗、随访及预后情况,探究影响疾病复发的因素。方法回顾性分析2014年6月至2019年6月于首都医科大学附属北京儿童医院风湿科诊治且随访至少6个月的幼年皮肌炎患儿的临床资料。采用Kaplan-Meier法绘制生存曲线,并通过Cox回归分析影响疾病复发的因素。结果192例幼年皮肌炎患儿中,男女比例为1∶1.46,中位起病月龄为65.5(43.5,98.8)个月。部分难治性患儿联合应用托法替布、沙利度胺、托珠单抗治疗后疾病好转。Kaplan-Meier生存曲线显示,患儿达到完全临床应答的中位时间为12个月。多因素Cox回归分析结果显示,起病至治疗时间长[RR(95%CI):1.042(1.001~1.084),P=0.044]是疾病复发的危险因素,起病时向阳疹阳性[RR(95%CI):0.208(0.049~0.894),P=0.035]是避免疾病复发的保护因素。结论多数幼年皮肌炎患儿经治疗可以达到完全临床应答,部分患儿存在疾病复发。沙利度胺、托法替布、托珠单抗的应用或可改善难治性幼年皮肌炎患儿的预后。起病至治疗时间长是疾病复发的危险因素。

Objective To study the treatment,follow-up and prognosis of pediatric patients with juvenile dermatomyositis(JDM),and to explore the factors affecting the recurrence of the disease.Methods The clinical data of pediatric patients with JDM who were diagnosed and treated in the Department of Rheumatology of Beijing Children′s Hospital,Capital Medical University from June 2014 to June 2019 and were followed up for at least 6 months were retrospectively analyzed.Kaplan-Meier method was used to plot the survival curve,and Cox regression was used to analyze the factors affecting the recurrence of the disease.Results Among the 192 pediatric patients with JDM,the ratio of males to females was 1∶1.46,and the median age of the patients with onset was 65.5(43.5,98.8)months.The combination treatment of tofacitinib,thalidomide,and tocilizumab improved the disease in some refractory pediatric patients with JDM.Kaplan-Meier survival curve showed that the median time of the pediatric patients to achieve complete clinical response was 12 months.The results of multivariate Cox regression analysis showed that long time from disease onset to treatment[RR(95%CI):1.042(1.001-1.084),P=0.044]was a risk factor of disease recurrence,and positive heliotrope rash[RR(95%CI):0.208(0.049-0.894),P=0.035]at the time of disease onset was a protective factor of avoiding disease recurrence.Conclusion Most pediatric patients with JDM can achieve complete clinical response after treatment,and some of them may experience the disease recurrence.The application of thalidomide,tofacitinib and tocilizumab may improve the prognosis of the pediatric patients with refractory JDM.The long time from disease onset to treatment is a risk factor of the disease recurrence.