Environmental Autoimmunity Group, Clinical Research Branch, National Institute of Environmental Health Sciences, National Institutes of Health, Building 10, Rm 4-2352, MSC 1301, 10 Center Drive, Bethesda, MD 20892-1301.
National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD.
Semin Arthritis Rheum. 2018 Dec;48(3):513-522. doi: 10.1016/j.semarthrit.2018.03.016. Epub 2018 Mar 28.
Few controlled studies are available to guide treatment decisions in juvenile dermatomyositis (JDM). This study evaluated therapies received, changes of treatment over time, and factors associated with medication choices in JDM.
We performed a retrospective analysis of the number and type of therapies and duration of treatment for 320 patients with JDM enrolled in a North American registry. Kaplan-Meier and logistic regression analysis were used to assess the association of demographic and clinical features and autoantibodies with medication usage.
High-dose oral prednisone was the primary therapy given to 99% of patients. 1997 was determined to be a threshold year for increasing usage of medications other than prednisone. The median time to half the initial oral prednisone dose was shorter in patients diagnosed after vs. before 1997 (10 vs. 19 months, P < 0.01). Patients received intravenous methylprednisolone (IVMP), methotrexate, intravenous immunoglobulin, antimalarial drugs, and combination therapy more frequently when diagnosed after 1997. IVMP was frequently received by patients with severe illness onset, anti-p155/140 (anti-TIF1) and anti-MJ (anti-NXP2) autoantibodies. Treatment with methotrexate was associated with older age at diagnosis and anti-MJ autoantibodies, while antimalarial therapy was associated with anti-p155/140 autoantibodies and mild onset severity.
Oral prednisone has been the mainstay of therapy in JDM, and prednisone was reduced faster in patients diagnosed after 1997 when there was also an increase in other medications. Specific medications received by patients with JDM correlated with year and age at diagnosis, myositis autoantibodies, onset severity, and illness features.
关于幼年特发性皮肌炎（JDM）的治疗决策，目前仅有少数对照研究可供参考。本研究旨在评估 JDM 患者的治疗方法、随时间变化的治疗方案改变，以及与药物选择相关的因素。
我们对纳入北美登记处的 320 例 JDM 患者的治疗方案数量和类型以及治疗持续时间进行了回顾性分析。采用 Kaplan-Meier 和逻辑回归分析评估人口统计学和临床特征以及自身抗体与药物使用之间的关系。
99%的患者接受了大剂量口服泼尼松治疗。1997 年被确定为泼尼松以外药物使用增加的阈值年。与 1997 年前相比，1997 年后诊断的患者初始口服泼尼松剂量减半的中位时间更短（10 个月 vs. 19 个月，P < 0.01）。1997 年后诊断的患者更频繁地接受静脉注射甲基泼尼松龙（IVMP）、甲氨蝶呤、静脉注射免疫球蛋白、抗疟药物和联合治疗。病情严重、抗 p155/140（抗 TIF1）和抗 MJ（抗 NXP2）自身抗体阳性的患者更常接受 IVMP 治疗。接受甲氨蝶呤治疗与诊断时年龄较大和抗 MJ 自身抗体有关，而接受抗疟治疗与抗 p155/140 自身抗体和疾病较轻的发病严重程度有关。
口服泼尼松一直是 JDM 的主要治疗方法，1997 年后诊断的患者中，泼尼松的剂量减少得更快，同时其他药物的使用也有所增加。JDM 患者接受的具体药物与诊断年份和年龄、肌炎自身抗体、发病严重程度和疾病特征有关。
