Department of Women's and Children's Health, Institute of Life Course and Medical Sciences, University of Liverpool, UK; Department of Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust Hospital, Liverpool, UK.
Department of Women's and Children's Health, Institute of Life Course and Medical Sciences, University of Liverpool, UK; Department of Paediatric Rheumatology, Alder Hey Children's NHS Foundation Trust Hospital, Liverpool, UK.
Clin Immunol. 2023 Jun;251:109344. doi: 10.1016/j.clim.2023.109344. Epub 2023 Apr 23.
Chronic nonbacterial osteomyelitis (CNO) is an autoinflammatory bone disease that primarily affects children and adolescents. CNO is associated with pain, bone swelling, deformity, and fractures. Its pathophysiology is characterized by increased inflammasome assembly and imbalanced expression of cytokines. Treatment is currently based on personal experience, case series and resulting expert recommendations. Randomized controlled trials (RCTs) have not been initiated because of the rarity of CNO, expired patent protection of some medications, and the absence of agreed outcome measures. An international group of fourteen CNO experts and two patient/parent representatives was assembled to generate consensus to inform and conduct future RCTs. The exercise delivered consensus inclusion and exclusion criteria, patent protected (excludes TNF inhibitors) treatments of immediate interest (biological DMARDs targeting IL-1 and IL-17), primary (improvement of pain; physician global assessment) and secondary endpoints (improved MRI; improved PedCNO score which includes physician and patient global scores) for future RCTs in CNO.
慢性非细菌性骨髓炎（CNO）是一种自身炎症性骨病，主要影响儿童和青少年。CNO 与疼痛、骨肿胀、畸形和骨折有关。其病理生理学的特征是炎症小体组装增加和细胞因子表达失衡。目前的治疗方法基于个人经验、病例系列和由此产生的专家建议。由于 CNO 的罕见性、一些药物专利保护期已过以及缺乏公认的疗效评估指标，尚未开展随机对照试验（RCT）。一个由 14 名 CNO 专家和 2 名患者/家长代表组成的国际小组，旨在达成共识，为未来的 RCT 提供信息和指导。该研究达成了共识，纳入和排除标准、有专利保护（排除 TNF 抑制剂）的当前有兴趣的治疗方法（针对 IL-1 和 IL-17 的生物 DMARDs）、主要终点（疼痛改善；医生整体评估）和次要终点（改善 MRI；改善包括医生和患者整体评分的 PedCNO 评分）用于未来的 CNO RCT。
