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骨折前预防性静脉注射双膦酸盐治疗的潜在可接受性:杜氏肌营养不良症青少年、父母及健康专业人员的观点

The prospective acceptability of preventative IV bisphosphonate therapy prior to fracture: Perspectives of young people with Duchenne muscular dystrophy, parents and health professionals.

作者信息

Mountain Danielle C, Crabtree Nicola, Wood Claire L, Wong Sze Choong, Bray Lucy

机构信息

Faculty of Health, Social Care and Medicine, Edge Hill University, Ormskirk, United Kingdom.

Department of Diabetes and Endocrinology, Birmingham Women's and Children's NHS Trust, Birmingham, United Kingdom.

出版信息

PLoS One. 2025 Jun 2;20(5):e0324124. doi: 10.1371/journal.pone.0324124. eCollection 2025.

Abstract

Young people with Duchenne muscular dystrophy (DMD) commonly experience osteoporosis and fractures which can lead to chronic pain and reduced quality of life. Initiating IV bisphosphonate therapy prior to first fracture may be a logical primary preventative approach given the extent and related morbidity of osteoporosis, although there is limited evidence for this. This qualitative study using semi-structured interviews and focus groups, aimed to explore the opinions and prospective acceptability of young people with DMD, parents and health professionals in the UK on initiating bisphosphonate therapy prior to first fracture. Four boys with DMD (aged 15-17 years) and 20 parents participated in semi-structured interviews. Twenty-seven health professionals involved in the care of young people with DMD participated in focus groups. A framework analysis was conducted. Three categories were identified which represented a continuum of opinions on the endorsement of preventative bisphosphonate therapy: 1) "It buys them time", endorsement of preventative bisphosphonate therapy; 2) Uncertainty and the importance of "choice"; and 3) "Worry about... starting bisphosphonates even earlier", not endorsing the use of preventative bisphosphonate therapy. Young people with DMD and parents discussed a range of opinions about the prospective acceptability of IV bisphosphonate as preventative therapy, highlighting the importance of family choice before initiation of therapy. Health professionals called for future research exploring the risks and benefits of preventative IV bisphosphonate therapy for young people with DMD to inform clinical practice.

摘要

患有杜氏肌营养不良症(DMD)的年轻人通常会出现骨质疏松和骨折,这可能导致慢性疼痛并降低生活质量。鉴于骨质疏松的程度和相关发病率,在首次骨折前开始静脉注射双膦酸盐治疗可能是一种合理的一级预防方法,尽管这方面的证据有限。这项定性研究采用半结构化访谈和焦点小组,旨在探讨英国患有DMD的年轻人、父母和健康专业人员对于在首次骨折前开始双膦酸盐治疗的意见和预期接受度。四名患有DMD的男孩(年龄在15 - 17岁)和20名父母参与了半结构化访谈。27名参与DMD患儿护理的健康专业人员参加了焦点小组。进行了框架分析。确定了三类意见,代表了对预防性双膦酸盐治疗认可程度的连续意见:1)“为他们争取时间”,认可预防性双膦酸盐治疗;2)不确定性和“选择”的重要性;3)“担心……更早开始使用双膦酸盐”,不认可使用预防性双膦酸盐治疗。患有DMD的年轻人和父母讨论了关于静脉注射双膦酸盐作为预防性治疗的预期接受度的一系列意见,强调了在开始治疗前家庭选择的重要性。健康专业人员呼吁未来进行研究,探索预防性静脉注射双膦酸盐治疗对患有DMD的年轻人的风险和益处,以为临床实践提供参考。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f00d/12129184/50b86dda1d84/pone.0324124.g001.jpg

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