University of Colorado, Department of Pediatrics, Division of Rheumatology, Denver, Colorado.
University of North Carolina at Chapel Hill, Department of Pediatrics, Division of Rheumatology.
Curr Opin Rheumatol. 2024 Sep 1;36(5):336-343. doi: 10.1097/BOR.0000000000001033. Epub 2024 Jul 10.
Antineutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV) is often organ- or life-threatening in children and impacts them during important periods of psychosocial and physical development. This review covers recent advances in the pathophysiology, diagnosis, management, and outcomes of AAV in children and highlights the ongoing need for funding and increased research collaboration.
Recent work has improved our understanding of AAV disease pathogenesis, potentially identifying new biomarkers and therapeutic targets. Collaborative clinical studies have also highlighted the variable manifestations in children and identified potential factors associated with poorer outcomes. Consensus-based treatment guidelines are also appearing, but clinical trials are still essential to better understanding treatment efficacy and safety in children affected by AAV. New, validated outcome measures, including those that are patient-reported, will facilitate these much-needed clinical trials in pediatric AAV.
There is a continued need for more rigorous study in pediatric AAV, however, there is certainly excitement with the increase in recent research relevant to the pediatric population.
抗中性粒细胞胞浆抗体（ANCA）相关性血管炎（AAV）在儿童中常具有器官和生命威胁性，并会对其心理社会和身体发育的重要时期产生影响。本文综述了儿童 AAV 的发病机制、诊断、治疗和结局的最新进展，并强调了对资金和增加研究合作的持续需求。
最近的研究工作提高了我们对 AAV 疾病发病机制的认识，可能确定了新的生物标志物和治疗靶点。协作性临床研究也突出了儿童中不同的表现，并确定了与较差结局相关的潜在因素。基于共识的治疗指南也在出现，但临床试验对于更好地了解儿童 AAV 的治疗效果和安全性仍然至关重要。新的、经过验证的结局衡量指标，包括患者报告的指标，将有助于在儿科 AAV 中进行这些急需的临床试验。
儿童 AAV 需要更严格的研究，但最近与儿科人群相关的研究增加确实令人兴奋。
