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首页> 美国卫生研究院文献>other >Defining consensus opinion to develop randomised controlled trials in rare diseases using Bayesian design: An example of a proposed trial of adalimumab versus pamidronate for children with CNO/CRMO
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Defining consensus opinion to develop randomised controlled trials in rare diseases using Bayesian design: An example of a proposed trial of adalimumab versus pamidronate for children with CNO/CRMO

机译：定义共识意见以使用贝叶斯设计在罕见病中开展随机对照试验：阿达木单抗与帕米膦酸对CNO / CRMO儿童的拟议试验的示例

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IntroductionChronic nonbacterial osteomyelitis (CNO) is a rare autoinflammatory bone disorder primarily affecting children and adolescents. It can lead to chronic pain, bony deformities and fractures. The pathophysiology of CNO is incompletely understood. Scientific evidence suggests dysregulated expression of pro- and anti-inflammatory cytokines to be centrally involved. Currently, treatment is largely based on retrospective observational studies and expert opinion. Treatment usually includes nonsteroidal anti-inflammatory drugs and/or glucocorticoids, followed by a range of drugs in unresponsive cases. While randomised clinical trials are lacking, retrospective and prospective non-controlled studies suggest effectiveness of TNF inhibitors and bisphosphonates. The objective of the Bayesian consensus meeting was to quantify prior expert opinion.
机译：简介慢性非细菌性骨髓炎（CNO）是一种罕见的自身炎症性骨病，主要影响儿童和青少年。它会导致慢性疼痛，骨质畸形和骨折。 CNO的病理生理学尚未完全了解。科学证据表明，促炎症细胞因子和消炎细胞因子的表达失调可能是集中参与的。目前，治疗主要基于回顾性观察研究和专家意见。治疗通常包括非甾体类抗炎药和/或糖皮质激素，然后在无反应的情况下使用多种药物。尽管缺乏随机临床试验，但回顾性和前瞻性非对照研究表明TNF抑制剂和双膦酸盐的有效性。贝叶斯共识会议的目的是量化先前的专家意见。

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