囊性纤维化相关骨病的治疗

IF 4.2 Q1 ENDOCRINOLOGY & METABOLISM
Jagdeesh Ullal , Katherine Kutney , Kristen M. Williams , David R. Weber
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引用次数: 1

摘要

高效CFTR调节疗法的出现减缓了囊性纤维化患者肺部并发症的进展。由于囊性纤维化患者的寿命增加,对囊性纤维化骨病(CFBD)的兴趣增加。CFBD是一种复杂的多因素疾病。CFBD是由于骨质低矿化导致强度、结构和质量差,易发生骨折。CFBD的发展跨越了不同的年龄组。管理必须根据现有的指导方针,在平衡治疗益处和长期使用骨活性药物的风险的同时,针对每个组进行细微差别的调整。目前,主要的治疗方法包括双膦酸盐。然而,双膦酸盐治疗CF患者的长期效果尚不完全清楚。我们描述可用于骨质疏松症治疗的新药。尽管如此,缺乏数据的单克隆抗体治疗,如Denosumab和Romozosumab,以及合成代谢骨治疗,如teriparatide和Abaloparatide,应该进行试验。在这篇综述中,我们还总结了CFBD的筛查和非药物治疗,并描述了CFBD药物治疗的各种选择。我们讨论CFTR调节剂对骨骼健康的前景,同时等待长期试验来描述这些药物对骨骼健康的影响。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Treatment of cystic fibrosis related bone disease

Treatment of cystic fibrosis related bone disease

The advent of highly effective CFTR modulator therapies has slowed the progression of pulmonary complications in people with cystic fibrosis. There is increased interest in cystic fibrosis bone disease (CFBD) due to the increasing longevity of people with cystic fibrosis. CFBD is a complex and multifactorial disease. CFBD is a result of hypomineralized bone leading to poor strength, structure and quality leading to susceptibility to fractures. The development of CFBD spans different age groups. The management must be tailored to each group with nuance and based on available guidelines while balancing therapeutic benefits to risks of long-term use of bone-active medication. For now, the mainstay of treatment includes bisphosphonates. However, the long-term effects of bisphosphonate treatment in people with CF are not fully understood. We describe newer agents available for osteoporosis treatment. Still, the lack of data behooves trials of monoclonal antibodies treatments such as Denosumab and Romozosumab and anabolic bone therapy such as teriparatide and Abaloparatide. In this review, we also summarize screening and non-pharmacologic treatment of CFBD and describe the various options available for the pharmacotherapy of CFBD. We address the prospect of CFTR modulators on bone health while awaiting long-term trials to describe the effects of these medications on bone health.

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来源期刊
CiteScore
6.10
自引率
0.00%
发文量
24
审稿时长
16 weeks
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