幼年皮肌炎（Juvenile Dermatomyositis,JDM）是一种儿童期可累及全身的自身免疫性炎性肌病,主要累及皮肤和近端肌肉,还可以累及重要器官（如：肺、关节及胃肠道等）,常伴有特异性抗体的存在。目前关于JDM的治疗非常困难,一线药物是糖皮质激素和甲氨蝶呤,对于治疗效果不佳的患者,有些二线或三线药物起到一定的支持作用,在难治性患者中,静脉注射免疫球蛋白或环磷酰胺可能是有效的,同时生物制剂和小分子靶向药物也逐渐被应用于临床。而除糖皮质激素外的药物选择往往还需要综合临床症状、血清特异性抗体、病理结果及初步治疗的反应,在治疗过程中不断调整。这篇综述的目的是概述目前用于治疗JDM的药物、治疗效果观察和选择药物的策略,为临床治疗提供思路。

Juvenile dermatomyositis (JDM) is an autoimmune inflammatory myopathy in childhood that can affect the whole body, mainly the skin and proximal muscles. It can also affect vital organs (such as: lungs, joints, and gastrointestinal tract etc), often accompanied by the presence of specific antibodies. At present, the treatment of JDM is very difficult. The first-line drugs are glucocorticoids and methotrexate. For patients with poor therapeutic effects, some second-line or third-line drugs may play a supportive role. In refractory patients, intravenous injection of immunoglobulin or cyclophosphamide may be effective, while biologics and small molecule targeted drugs are gradually used in clinical practice. In addition to glucocorticoids, the choice of drugs often requires a combination of clinical symptoms, serum specific antibodies, pathological results and response to initial treatment, and continuous adjustments during the treatment process. The purpose of this review is to provide an overview the drugs currently used in the treatment of JDM, observation of therapeutic effects and strategies for drug selection, and to provide ideas for clinical treatment.