From the Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Hackensack Meridian School of Medicine at Seton Hall University, Nutley, New Jersey; UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania; Duke University School of Medicine, Durham, North Carolina; Boston Children's Hospital, Boston, Massachusetts; Children's Mercy Hospital, Kansas City, Missouri; University of Iowa Carver College of Medicine; University of Iowa Stead Family Children's Hospital, Iowa City, Iowa; Children's Medical Center of Dallas and UT Southwestern, Dallas, Texas; The Ohio State University, Columbus, Ohio; Mayo Clinic, Rochester, Minnesota; University of Colorado-Denver and Children's Hospital Colorado, Denver, Colorado; Rutgers University, School of Public Health, Newark, New Jersey, USA; University of Toronto and The Hospital for Sick Children, Toronto, Ontario, Canada.
S.C. Li, MD, PhD, Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center, Hackensack Meridian School of Medicine at Seton Hall University; K.S. Torok, MD, UPMC Children's Hospital of Pittsburgh; C.E. Rabinovich, MD, MPH, Duke University School of Medicine; F. Dedeoglu, MD, Boston Children's Hospital; M.L. Becker, MD, MSCE, Children's Mercy Hospital; P.J. Ferguson, MD, University of Iowa Carver College of Medicine; S.D. Hong, MD, University of Iowa Stead Family Children's Hospital; M.F. Ibarra, MD, Children's Mercy Hospital; K. Stewart, MD, Children's Medical Center of Dallas and UT Southwestern; E. Pope, MD, University of Toronto and The Hospital for Sick Children; G.C. Higgins, MD, PhD, The Ohio State University; R.M. Laxer, MD, University of Toronto and The Hospital for Sick Children; T. Mason II, MD, Mayo Clinic; R.C. Fuhlbrigge, MD, PhD, University of Colorado-Denver, and Children's Hospital Colorado; T. Andrews, MA, Rutgers University, School of Public Health.
J Rheumatol. 2020 Aug 1;47(8):1242-1252. doi: 10.3899/jrheum.190311. Epub 2019 Oct 15.
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.
在青少年局限性硬皮病（LS）中进行标准化治疗方案（共识治疗计划；CTP）的比较有效性可行性研究。
由儿童关节炎和风湿病研究联盟（CARRA）LS 工作组成员进行了一项前瞻性、多中心 1 年试点观察队列研究。患有活动性、中重度青少年 LS 的患者接受了 3 种 CTP 之一的治疗：单独使用甲氨蝶呤，或与静脉注射（30mg/kg/剂量，持续 3 个月）或口服皮质类固醇（2mg/kg/天，在 48 周内逐渐减少）联合使用。
共纳入 50 名具有青少年 LS 典型特征的患者，其中 44 名（88%）完成了研究。大多数患者有皮肤外受累。所有 3 种 CTP 患者均有改善，与基线相比，>75%的患者有较大或中度的改善。一些患者发生了损害。由于药物不耐受（n=6；14%）或治疗失败（n=11；25%），患者脱离了规定的治疗方案，所有 3 种 CTP 均发生了治疗失败。LS 皮肤评分和总体医生评估均显示出治疗的显著反应，在一些患者亚组中确定了反应水平的差异。反应差异与基线疾病活动水平、LS 亚型、皮肤疾病范围和皮肤外受累有关。
这项研究表明在青少年 LS 中进行比较有效性研究是可行的。CTP 被发现是安全、有效和可耐受的。我们的评估表现良好。由于即使活动得到有效控制，损害也很常见且可能进展，因此我们建议主要通过活动指标评估初始治疗效果。确定了一些潜在的反应混杂因素，值得进一步研究。
