关键词: CNO CRMO Chronic nonbacterial osteomyelitis Diagnosis Monitoring Pathophysiology Treatment

Mesh : Adolescent Child Humans Bone Diseases Consensus Osteomyelitis / diagnosis therapy

来  源:   DOI:10.1186/s12969-023-00851-6   PDF(Pubmed)

Abstract:
OBJECTIVE: Chronic nonbacterial osteomyelitis (CNO) is an autoinflammatory bone disorder that predominantly affects children and young people. The pathophysiology and molecular mechanisms of CNO remain poorly understood, and diagnostic criteria and biomarkers are lacking. As a result, treatment is empiric and follows personal experience, case series and expert consensus plans.
METHODS: A survey was designed to gain insight on clinician and patient experiences of diagnosing and treating CNO and to collate opinions on research priorities. A version containing 24 questions was circulated among international expert clinicians and clinical academics (27 contacted, 21 responses). An equivalent questionnaire containing 20 questions was shared to explore the experience and priorities of CNO patients and family members (93 responses).
RESULTS: Responses were used to select topics for four moderated roundtable discussions at the \"International Conference on CNO and autoinflammatory bone disease\" (Liverpool, United Kingdom, May 25-26th, 2022). The group identified deciphering the pathophysiology of CNO to be the highest priority, followed by clinical trials, necessary outcome measures and classification criteria. Surprisingly, mental wellbeing scored behind these items.
CONCLUSIONS: Agreement exists among clinicians, academics, patients and families that deciphering the pathophysiology of CNO is of highest priority to inform clinical trials that will allow for the approval of medications for the treatment of CNO by regulatory agencies.
摘要:
目的:慢性非细菌性骨髓炎(CNO)是一种主要影响儿童和年轻人的自身炎症性骨病。CNO的病理生理学和分子机制仍然知之甚少。缺乏诊断标准和生物标志物。因此,治疗是经验性的,遵循个人经验,案例系列和专家共识计划。
方法:一项调查旨在了解临床医生和患者在诊断和治疗CNO方面的经验,并整理对研究重点的意见。在国际专家临床医生和临床学者中分发了包含24个问题的版本(已联系27个,21个答复)。共享了包含20个问题的等效问卷,以探索CNO患者和家庭成员的经验和优先事项(93份答复)。
结果:在“CNO和自身炎症性骨病国际会议”上,响应被用来选择四个主持圆桌讨论的主题(利物浦,英国,5月25日至26日,2022年)。该小组确定破译CNO的病理生理学是最高优先级,接下来是临床试验,必要的结果衡量标准和分类标准。令人惊讶的是,这些项目背后的心理健康得分。
结论:临床医生之间存在协议,学者,破译CNO病理生理学的患者和家庭是最优先考虑的事项,以告知临床试验,这些临床试验将允许监管机构批准用于治疗CNO的药物.
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