Kobayashi Ichiro
Center for Pediatric Allergy and Rheumatology, KKR Sapporo Medical Center, 3-40 Hiragishi 1-6, Toyohira-ku, Sapporo 060-0931, Japan.
Children (Basel). 2024 Aug 27;11(9):1046. doi: 10.3390/children11091046.
Juvenile idiopathic inflammatory myopathy (JIIM) is a rare systemic autoimmune disease characterized by skeletal muscle weakness with or without a skin rash. Juvenile dermatomyositis (JDM) is the most common subtype of JIIM, accounting for 80% of JIIM. Recent studies identified several myositis-specific autoantibodies (MSAs) and myositis-associated autoantibodies (MAAs). Each MSA or MAA is associated with distinct clinical features and outcomes, although there are several differences in the prevalence of MSA/MAA and autoantibody-phenotype relationships between age and ethnic groups. Histopathological studies have revealed critical roles of type I interferons and vasculopathy in the development of JDM. Serological classification mostly corresponds to clinicopathological classification. Novel therapeutic agents, such as biologics and Janus kinase inhibitors (JAKi), have been developed; however, to date, there is a lack of high-level evidence. As advances in treatment have reduced the mortality rate of JIIM, recent studies have focused on medium- and long-term outcomes. However, rapidly progressive interstitial lung disease (RP-ILD) remains a major cause of death in anti-melanoma differentiation gene 5 autoantibody-positive JDM. Early diagnosis and intervention using a multi-drug regimen is critical for the treatment of RP-ILD. Rituximab and JAKi may reduce mortality in patients with JDM-associated RP-ILD refractory to conventional therapy.
青少年特发性炎性肌病(JIIM)是一种罕见的系统性自身免疫性疾病,其特征为骨骼肌无力,可伴有或不伴有皮疹。青少年皮肌炎(JDM)是JIIM最常见的亚型,占JIIM的80%。最近的研究发现了几种肌炎特异性自身抗体(MSA)和肌炎相关自身抗体(MAA)。尽管不同年龄和种族群体之间MSA/MAA的患病率以及自身抗体与表型的关系存在一些差异,但每种MSA或MAA都与独特的临床特征和预后相关。组织病理学研究揭示了I型干扰素和血管病变在JDM发病过程中的关键作用。血清学分类大多与临床病理分类相对应。已经开发了新型治疗药物,如生物制剂和Janus激酶抑制剂(JAKi);然而,迄今为止,缺乏高水平的证据。随着治疗进展降低了JIIM的死亡率,最近的研究集中在中长期预后。然而,快速进展性间质性肺病(RP-ILD)仍然是抗黑色素瘤分化基因5自身抗体阳性JDM的主要死亡原因。采用多药方案进行早期诊断和干预对于RP-ILD的治疗至关重要。利妥昔单抗和JAKi可能会降低对传统治疗难治的JDM相关RP-ILD患者的死亡率。