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Abstract：
To perform a comparative effectiveness feasibility study in juvenile localized scleroderma (LS), using standardized treatment regimens (consensus treatment plans; CTP).
A prospective, multicenter 1-year pilot observational cohort study was performed by Childhood Arthritis and Rheumatology Research Alliance (CARRA) LS workgroup members. Patients with active, moderate to severe juvenile LS were treated with one of 3 CTP: methotrexate alone, or in combination with intravenous (30 mg/kg/dose for 3 mos) or oral corticosteroids (2 mg/kg/day tapered by 48 weeks).
Fifty patients, with demographics typical for juvenile LS, were enrolled, and 44 (88%) completed the study. Most had extracutaneous involvement. Patients improved in all 3 CTP, with > 75% having a major or moderate level of improvement compared to baseline. Damage accrued in some patients. Major deviations from prescribed regimen resulted from medication intolerance (n = 6; 14%) or treatment failure (n = 11; 25%); failures occurred in all 3 CTP. Significant responses to treatment were demonstrated by LS skin scoring measures and overall physician assessments, with differences in response level identified in some patient subsets. Response differences were associated with baseline disease activity level, LS subtype, skin disease extent, and extracutaneous involvement.
This study demonstrates the feasibility of conducting juvenile LS comparative effectiveness studies. The CTP were found to be safe, effective, and tolerable. Our assessments performed well. Because damage is common and may progress despite effective control of activity, we recommend initial treatment efficacy be evaluated primarily by activity measures. Potential confounders for response were identified that warrant further study.
A prospective, multicenter 1-year pilot observational cohort study was performed by Childhood Arthritis and Rheumatology Research Alliance (CARRA) LS workgroup members. Patients with active, moderate to severe juvenile LS were treated with one of 3 CTP: methotrexate alone, or in combination with intravenous (30 mg/kg/dose for 3 mos) or oral corticosteroids (2 mg/kg/day tapered by 48 weeks).
Fifty patients, with demographics typical for juvenile LS, were enrolled, and 44 (88%) completed the study. Most had extracutaneous involvement. Patients improved in all 3 CTP, with > 75% having a major or moderate level of improvement compared to baseline. Damage accrued in some patients. Major deviations from prescribed regimen resulted from medication intolerance (n = 6; 14%) or treatment failure (n = 11; 25%); failures occurred in all 3 CTP. Significant responses to treatment were demonstrated by LS skin scoring measures and overall physician assessments, with differences in response level identified in some patient subsets. Response differences were associated with baseline disease activity level, LS subtype, skin disease extent, and extracutaneous involvement.
This study demonstrates the feasibility of conducting juvenile LS comparative effectiveness studies. The CTP were found to be safe, effective, and tolerable. Our assessments performed well. Because damage is common and may progress despite effective control of activity, we recommend initial treatment efficacy be evaluated primarily by activity measures. Potential confounders for response were identified that warrant further study.
摘要：
在青少年局限性硬皮病（LS）中进行比较有效性可行性研究，使用标准化治疗方案（共识治疗计划；CTP）。
有前景的,由儿童关节炎和风湿病研究联盟(CARRA)LS工作组成员进行多中心1年先导观察性队列研究.活跃的患者，中度至重度青少年LS采用3种CTP中的一种：甲氨蝶呤单独治疗，或与静脉内（30mg/kg/剂量3个月）或口服皮质类固醇（2mg/kg/天逐渐减少48周）联合使用。
五十个病人,青少年LS的典型人口统计，被注册，44人（88%）完成了研究。大多数有皮外受累。患者在所有3个CTP中得到改善，与基线相比，>75%的患者有主要或中等水平的改善。一些患者受到损害。由于药物不耐受（n=6;14％）或治疗失败（n=11;25％）导致处方方案的主要偏差；所有3个CTP均发生失败。通过LS皮肤评分措施和总体医生评估证明了对治疗的显着反应。在一些患者亚组中识别出的应答水平存在差异。反应差异与基线疾病活动水平相关，LS亚型,皮肤病程度，和皮外受累。
这项研究证明了进行青少年LS比较有效性研究的可行性。CTP被发现是安全的,有效,和可容忍的。我们的评估表现良好。因为损害是常见的，尽管有效控制了活动，但损害可能会进展，我们建议主要通过活动措施评估初始治疗疗效.确定了潜在的反应混杂因素，需要进一步研究。
有前景的,由儿童关节炎和风湿病研究联盟(CARRA)LS工作组成员进行多中心1年先导观察性队列研究.活跃的患者，中度至重度青少年LS采用3种CTP中的一种：甲氨蝶呤单独治疗，或与静脉内（30mg/kg/剂量3个月）或口服皮质类固醇（2mg/kg/天逐渐减少48周）联合使用。
五十个病人,青少年LS的典型人口统计，被注册，44人（88%）完成了研究。大多数有皮外受累。患者在所有3个CTP中得到改善，与基线相比，>75%的患者有主要或中等水平的改善。一些患者受到损害。由于药物不耐受（n=6;14％）或治疗失败（n=11;25％）导致处方方案的主要偏差；所有3个CTP均发生失败。通过LS皮肤评分措施和总体医生评估证明了对治疗的显着反应。在一些患者亚组中识别出的应答水平存在差异。反应差异与基线疾病活动水平相关，LS亚型,皮肤病程度，和皮外受累。
这项研究证明了进行青少年LS比较有效性研究的可行性。CTP被发现是安全的,有效,和可容忍的。我们的评估表现良好。因为损害是常见的，尽管有效控制了活动，但损害可能会进展，我们建议主要通过活动措施评估初始治疗疗效.确定了潜在的反应混杂因素，需要进一步研究。
