The clinical phenotypes of the juvenile idiopathic inflammatory myopathies.
The clinical phenotypes of the juvenile idiopathic inflammatory myopathies.
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DOI：
10.1097/md.0b013e31827f264d
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发表时间：
2013-01
期刊：
影响因子：
1.6
通讯作者：
with the Childhood Myositis Heterogeneity Collaborative Study Group
中科院分区：
文献类型：
--
作者：
Shah M;Mamyrova G;Targoff IN;Huber AM;Malley JD;Rice MM;Miller FW;Rider LG;with the Childhood Myositis Heterogeneity Collaborative Study Group
幼年特发性炎性肌病是一种全身性自身免疫性疾病，以骨骼肌无力、特征性皮疹和其他全身特征为特征。尽管JIM最常见的形式--幼年型皮肌炎（JDM）已得到很好的研究，但JIM的其他主要临床亚组，包括幼年型多发性肌炎（JPM）和与另一种自身免疫性或结缔组织疾病（JCTM）重叠的幼年型肌炎，尚未得到很好的表征，其与成人临床亚组的相似性尚不清楚。在一项全国性的登记研究中，我们招募了436例JIIM患者，包括354例JDM，33例JPM和49例JCTM。该研究的目的是比较人口统计学;临床特征;实验室指标，包括肌炎自身抗体;和这些临床亚组之间的结果，以及在一项单独的自然史研究中登记的特发性炎性肌病（IIM）成人患者的已发表数据。我们使用随机森林分类和逻辑回归模型比较临床亚组，单变量分析。JDM的特征是典型的皮疹，包括Gottron丘疹、日光性皮疹、颧骨皮疹、甲周毛细血管改变以及其他光敏性和血管性皮疹。JPM的特征是更严重的虚弱，更高的肌酸激酶水平，下降的发作，更频繁的心脏病。JCTM更常见间质性肺病、雷诺现象、关节痛和颧骨皮疹。自身抗体频率的差异也很明显，抗p155/140、抗MJ和抗Mi-2在JDM患者中更常见，抗信号识别颗粒和抗Jo-1在JPM中更常见，抗U1-RNP、PM-Scl和其他肌炎相关自身抗体在JCTM中更常见。JCTM患者的死亡率最高，而JPM患者的住院率和轮椅使用率最高。青少年和成人IIM亚组之间有一些共同的人口统计学和临床特征。然而，JDM和JPM患者的间质性肺病、雷诺现象、“机械手”和腕管综合征的发生率较低，死亡率也低于成年患者。我们的结论是，青少年肌炎是一组异质性疾病，不同的临床亚组，定义不同的临床和人口统计学特征，实验室特征和结果。
The juvenile idiopathic inflammatory myopathies (JIIM) are systemic autoimmune diseases characterized by skeletal muscle weakness, characteristic rashes, and other systemic features. Although juvenile dermatomyositis (JDM), the most common form of JIIM, has been well studied, the other major clinical subgroups of JIIM, including juvenile polymyositis (JPM) and juvenile myositis overlapping with another autoimmune or connective tissue disease (JCTM), have not been well characterized, and their similarity to the adult clinical subgroups is unknown. We enrolled 436 patients with JIIM, including 354 classified as JDM, 33 as JPM, and 49 as JCTM, in a nationwide registry study. The aim of the study was to compare demographics; clinical features; laboratory measures, including myositis autoantibodies; and outcomes among these clinical subgroups, as well as with published data on adult patients with idiopathic inflammatory myopathies (IIM) enrolled in a separate natural history study. We used random forest classification and logistic regression modeling to compare clinical subgroups, following univariate analysis. JDM was characterized by typical rashes, including Gottron papules, heliotrope rash, malar rash, periungual capillary changes, and other photosensitive and vasculopathic skin rashes. JPM was characterized by more severe weakness, higher creatine kinase levels, falling episodes, and more frequent cardiac disease. JCTM had more frequent interstitial lung disease, Raynaud phenomenon, arthralgia, and malar rash. Differences in autoantibody frequency were also evident, with anti-p155/140, anti-MJ, and anti-Mi-2 seen more frequently in patients with JDM, anti-signal recognition particle and anti-Jo-1 in JPM, and anti-U1-RNP, PM-Scl, and other myositis-associated autoantibodies more commonly present in JCTM. Mortality was highest in patients with JCTM, whereas hospitalizations and wheelchair use were highest in JPM patients. Several demographic and clinical features were shared between juvenile and adult IIM subgroups. However, JDM and JPM patients had a lower frequency of interstitial lung disease, Raynaud phenomenon, “mechanic’s hands” and carpal tunnel syndrome, and lower mortality than their adult counterparts. We conclude that juvenile myositis is a heterogeneous group of illnesses with distinct clinical subgroups, defined by varying clinical and demographic characteristics, laboratory features, and outcomes.