Update on treatment responses and outcome measure development in chronic nonbacterial osteomyelitis.

IF 5.2 2区 医学 Q1 RHEUMATOLOGY Current opinion in rheumatology Pub Date : 2023-09-01 DOI:10.1097/BOR.0000000000000954
Shwetha Ramachandran, Yongdong Zhao, Polly J Ferguson
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Abstract

Purpose of review: To review recent trends in treatment and recent progress in developing outcome measures needed for chronic nonbacterial osteomyelitis (CNO) clinical trials.

Recent findings: CNO is an autoinflammatory bone disease. In a minority of patients, the disease is genetically driven, and diagnosis can be made by DNA sequencing. However, for nonsyndromic CNO there is no diagnostic test. The number of children with CNO appears to be increasing and damage is common. Increases in CNO diagnosis is due to raised awareness, increased availability of whole-body magnetic resonance imaging and rising incidence. Treatment remains empiric and it is unclear which second line treatment is superior. Tumor necrosis factor inhibitors (TNFi) and bisphosphonates continue to be used as second line agents for nonsteroidal anti-inflammatory drugs (NSAID) refractory CNO; newer immune modulatory medications are used if this fails. Validated classification criteria, clinical outcome measures and imaging scoring standards are needed for successful clinical trials.

Summary: Best treatment for NSAID refractory CNO remains unclear. Classification criteria, clinical outcomes measures and standardized imaging scoring have been developed or are near completion. This will facilitate robust clinical trials in CNO with the goal of having approved medications for this painful disease.

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慢性非细菌性骨髓炎治疗反应和结局指标进展的最新进展。
综述的目的:回顾慢性非细菌性骨髓炎(CNO)临床试验中治疗的最新趋势和发展结果指标的最新进展。最近发现:CNO是一种自身炎症性骨病。在少数患者中,这种疾病是由基因驱动的,可以通过DNA测序来诊断。然而,对于非综合征性CNO,没有诊断测试。CNO患儿的数量似乎在增加,损伤也很常见。CNO诊断的增加是由于意识的提高,全身磁共振成像的可用性增加以及发病率的上升。治疗仍然是经验性的,尚不清楚哪一种二线治疗更好。肿瘤坏死因子抑制剂(TNFi)和双膦酸盐继续被用作非甾体抗炎药(NSAID)难治性CNO的二线药物;如果这种方法失败,则使用更新的免疫调节药物。成功的临床试验需要经过验证的分类标准、临床结果测量和影像学评分标准。总结:NSAID难治性CNO的最佳治疗方法尚不清楚。分类标准、临床结果测量和标准化影像学评分已经制定或即将完成。这将促进CNO的强有力的临床试验,目标是获得针对这种痛苦疾病的批准药物。
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来源期刊
Current opinion in rheumatology
Current opinion in rheumatology 医学-风湿病学
CiteScore
9.70
自引率
2.00%
发文量
89
审稿时长
6-12 weeks
期刊介绍: A high impact review journal which boasts an international readership, Current Opinion in Rheumatology offers a broad-based perspective on the most recent and exciting developments within the field of rheumatology. Published bimonthly, each issue features insightful editorials and high quality invited reviews covering two or three key disciplines which include vasculitis syndromes, medical physiology and rheumatic diseases, crystal deposition diseases and rheumatoid arthritis. Each discipline introduces world renowned guest editors to ensure the journal is at the forefront of knowledge development and delivers balanced, expert assessments of advances from the previous year.
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