Medical Management for Fracture Prevention in Children with Osteogenesis Imperfecta.

IF 3.3 3区 医学 Q2 ENDOCRINOLOGY & METABOLISM Calcified Tissue International Pub Date : 2024-12-01 Epub Date: 2024-03-29 DOI:10.1007/s00223-024-01202-7
Paul Arundel, Nick Bishop
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Abstract

There are no licensed treatments for children with osteogenesis imperfecta. Children currently receive off-label treatment with bisphosphonates, without any consistent approach to dose, drug or route of administration. Meta-analyses suggest that anti-fracture efficacy of such interventions is equivocal. New therapies are undergoing clinical trials, and it is likely that one or more will receive marketing authorisation within the next three to five years. The long-term outcome from such interventions will need to be studied carefully well beyond the period over which the clinical trials are conducted, and a consistent approach to the collection of data in this regard will be needed as a major collaborative effort.

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预防骨发育不全儿童骨折的医疗管理。
目前还没有针对成骨不全症儿童的特许治疗方法。目前,儿童接受的是标示外的双膦酸盐治疗,在剂量、药物或给药途径方面没有任何一致的方法。Meta 分析表明,此类干预措施的抗骨折疗效并不明显。新疗法正在进行临床试验,未来三到五年内很可能会有一种或多种疗法获得上市许可。在临床试验结束后,还需要对此类干预措施的长期疗效进行仔细研究,并且需要采取一致的方法来收集这方面的数据,这是一项重大的合作努力。
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来源期刊
Calcified Tissue International
Calcified Tissue International 医学-内分泌学与代谢
CiteScore
8.00
自引率
2.40%
发文量
112
审稿时长
4-8 weeks
期刊介绍: Calcified Tissue International and Musculoskeletal Research publishes original research and reviews concerning the structure and function of bone, and other musculoskeletal tissues in living organisms and clinical studies of musculoskeletal disease. It includes studies of cell biology, molecular biology, intracellular signalling, and physiology, as well as research into the hormones, cytokines and other mediators that influence the musculoskeletal system. The journal also publishes clinical studies of relevance to bone disease, mineral metabolism, muscle function, and musculoskeletal interactions.
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