From the Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Department of Pediatrics, Hackensack; Stevens Institute of Technology, Hoboken, New Jersey, USA; Hospital for Sick Children, Toronto, Ontario, Canada; Texas Scottish Rite Hospital, University of Texas (UT) Southwestern, Dallas, Texas; The Ohio State University and Nationwide Children's Hospital, Columbus, Ohio; Duke University, Durham, North Carolina; Riley Hospital for Children at Indiana University Health, Indianapolis, Indiana; Rockefeller University, New York, New York, USA; Hamburger Zentrum für Kinder- und Jugendrheumatologie, Hamburg, Germany; Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA.
S.C. Li, MD, PhD, Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Department of Pediatrics; X. Li, PhD, Stevens Institute of Technology; E. Pope, MD, Hospital for Sick Children; K. Stewart, MD, Texas Scottish Rite Hospital, UT Southwestern; G.C. Higgins, MD, PhD, The Ohio State University and Nationwide Children's Hospital; C.E. Rabinovich, MD, MPH, Duke University; K.M. O'Neil, MD, Riley Hospital for Children at Indiana University Health; K.A. Haines, MD, Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Department of Pediatrics; R.M. Laxer, MD, Hospital for Sick Children; M. Punaro, MD, Texas Scottish Rite Hospital, UT Southwestern; H. Jacobe, MD, UT Southwestern; T. Andrews, MA, Hackensack University Medical Center; K. Wittkowski, PhD, Rockefeller University; T. Nyirenda, PhD, Hackensack University Medical Center; I. Foeldvari, MD, Hamburger Zentrum für Kinder- und Jugendrheumatologie; K.S. Torok, MD, Children's Hospital of Pittsburgh.
J Rheumatol. 2018 Dec;45(12):1680-1688. doi: 10.3899/jrheum.171381. Epub 2018 Sep 15.
To identify clinical features that define disease activity in pediatric localized scleroderma (LS), and determine their specificity and importance.
We conducted a multicenter prospective study of patients with active and inactive LS skin lesions. A standardized evaluation of a single designated study lesion per subject was performed at 3 visits. We evaluated the pattern and correlation between assessed features and physician's global assessments of activity (PGA-A).
Ninety of 103 subjects had evaluable data; 66 had active and 24 inactive disease. Subjects had similar age of onset, sex, and disease patterns. Linear scleroderma was the most common subtype. Features specific for active disease included erythema, violaceous color, tactile warmth, abnormal skin texture, and disease extension. Scores for these variables changed over time and correlated with PGA-A of the lesion. Active and inactive lesions could not be distinguished by the presence or level of skin thickening, either of lesion edge or center. However, in active lesions, skin thickening scores did correlate with PGA-A scores. Regression analysis identified the combination of erythema, disease extension, violaceous color, skin thickening, and abnormal texture as predictive of PGA-A at study entry. Damage features were common irrespective of activity status.
We identified variables strongly associated with disease activity, expanding upon those used in current measures, and determined their relative importance in physician activity scoring. Skin thickening was found to lack specificity for disease activity. These results will help guide development of a sensitive, responsive activity tool to improve care of patients with LS.
确定定义儿童局限性硬皮病（LS）疾病活动的临床特征，并确定其特异性和重要性。
我们对活动性和非活动性 LS 皮肤损伤的患者进行了多中心前瞻性研究。对每个患者的单个指定研究病变进行了 3 次就诊的标准化评估。我们评估了评估特征与医生整体活动评估（PGA-A）之间的模式和相关性。
90 名 103 名受试者中有可评估的数据；66 名患有活动性疾病，24 名患有非活动性疾病。受试者的发病年龄、性别和疾病模式相似。线性硬皮病是最常见的亚型。活动性疾病的特征包括红斑、紫红色、触觉温暖、异常皮肤纹理和疾病扩展。这些变量的评分随时间而变化，并与病变的 PGA-A 相关。活跃和非活跃的病变不能通过皮肤增厚的存在或水平来区分，无论是病变边缘还是中心。然而，在活跃的病变中，皮肤增厚评分与 PGA-A 评分相关。回归分析确定了红斑、疾病扩展、紫红色、皮肤增厚和异常纹理的组合作为研究开始时预测 PGA-A 的预测因子。损伤特征无论活动状态如何都很常见。
我们确定了与疾病活动密切相关的变量，扩展了目前使用的测量方法，并确定了它们在医生活动评分中的相对重要性。皮肤增厚被发现缺乏对疾病活动的特异性。这些结果将有助于指导开发敏感、响应性的活动工具，以改善 LS 患者的护理。
