Eleftheriou Despina, Batu Ezgi Deniz, Ozen Seza, Brogan Paul A
Paediatric and Adolescent Rheumatology, UCL Institute of Child Health, London, UK.
Paediatric Rheumatology Department, Hacettepe University, Ankara, Turkey.
Nephrol Dial Transplant. 2015 Apr;30 Suppl 1:i94-103. doi: 10.1093/ndt/gfu393. Epub 2014 Dec 30.
Primary systemic vasculitides of the young are relatively rare diseases, but are associated with significant morbidity and mortality, particularly if there is diagnostic delay. We provide an overview of paediatric vasculitides with emphasis on key differences in vasculitis presentation and management between children and adults. Significant advances in the field of paediatric vasculitis research include the development of classification criteria and disease outcome tools for paediatric disease; inclusion of paediatric patients in international multicentre randomized controlled trials of therapies in vasculitis; and development of rare disease trial designs for therapeutic trials of paediatric vasculitis. The continuation of unmet needs as well as the exploration of potential therapeutic avenues and considerations in the design of future trials are also discussed.
儿童原发性系统性血管炎是相对罕见的疾病，但与显著的发病率和死亡率相关，尤其是在诊断延迟的情况下。我们概述了儿童血管炎，重点介绍儿童和成人血管炎在临床表现和管理方面的关键差异。儿童血管炎研究领域的重大进展包括制定儿童疾病的分类标准和疾病结局工具；将儿童患者纳入血管炎治疗的国际多中心随机对照试验；以及开发用于儿童血管炎治疗试验的罕见病试验设计。还讨论了未满足需求的持续存在以及在未来试验设计中对潜在治疗途径的探索和考虑因素。
