British Columbia's Children's Hospital and the University of British Columbia, Vancouver, British Columbia, Canada.
University of Chicago Medical Center, Chicago, Illinois.
Arthritis Care Res (Hoboken). 2022 Sep;74(9):1550-1558. doi: 10.1002/acr.24590. Epub 2022 Jun 1.
There is no standardized approach to the treatment of pediatric antineutrophil cytoplasmic antibody-associated vasculitis (AAV). Because of the rarity of pediatric AAV, randomized trials have not been feasible. The present study of the Childhood Arthritis and Rheumatology Research Alliance (CARRA) was undertaken to establish consensus treatment plans (CTPs) for severe pediatric AAV to enable the future study of comparative effectiveness and safety.
A workgroup of CARRA members (rheumatologists and nephrologists) formed the AAV Workgroup. This group performed a literature review on existing evidence-based treatments and guidelines for the management of AAV. They determined that the target population for CTP development was patients <18 years of age with new-onset granulomatosis with polyangiitis (GPA), microscopic polyangiitis, or renal-limited AAV (eosinophilic GPA was excluded), with presentation confined to those with severe disease (i.e., organ- or life-threatening). Face-to-face consensus conferences employed nominal group techniques to identify treatment strategies for remission induction and remission maintenance, data elements to be systematically collected, and outcomes to be measured over time.
The pediatric AAV Workgroup developed 2 CTPs for each of the remission induction and remission maintenance of severe AAV. A glucocorticoid-weaning regimen for induction and maintenance, a core data set, and outcome measures were also defined. A random sample of CARRA membership voted acceptance of the CTPs for remission induction and remission maintenance, with a 94% (75 of 80) and 98% (78 of 80) approval rate, respectively.
Consensus methodology established standardized CTPs for treating severe pediatric AAV. These CTPs were in principle accepted by CARRA-wide membership for the evaluation of pragmatic comparative effectiveness in a long-term registry.
目前尚无标准化的儿童抗中性粒细胞胞浆抗体相关性血管炎（AAV）治疗方法。由于儿科 AAV 较为罕见，因此无法进行随机试验。本项由儿童关节炎和风湿病研究联盟（CARRA）进行的研究旨在制定严重儿科 AAV 的共识治疗方案（CTP），以能够对比较疗效和安全性进行未来研究。
CARRA 成员（风湿病学家和肾脏病学家）工作组成立了 AAV 工作组。该小组对现有的循证治疗方法和 AAV 管理指南进行了文献回顾。他们确定，CTP 开发的目标人群是新诊断的肉芽肿性多血管炎（GPA）、显微镜下多血管炎或肾局限性 AAV（排除嗜酸性 GPA）的<18 岁患者，表现为严重疾病（即危及器官或危及生命）。通过面对面的共识会议，使用名义群体技术来确定缓解诱导和缓解维持的治疗策略、要系统收集的数据要素以及随时间推移测量的结果。
儿科 AAV 工作组为严重 AAV 的缓解诱导和缓解维持制定了 2 种 CTP。还定义了糖皮质激素撤药方案、核心数据集和结果衡量标准。随机抽取 CARRA 成员投票，接受缓解诱导和缓解维持的 CTP，分别有 94%（75/80）和 98%（78/80）的成员投票赞成。
共识方法为治疗严重儿科 AAV 制定了标准化的 CTP。这些 CTP 在原则上得到了 CARRA 全体成员的认可，可用于在长期注册研究中评估实用比较疗效。
