Longitudinal assessment of physical function in adults with X-linked hypophosphatemia following initiation of burosumab therapy.

IF 4.2 2区 医学 Q1 ENDOCRINOLOGY & METABOLISM Osteoporosis International Pub Date : 2024-11-01 Epub Date: 2024-07-19 DOI:10.1007/s00198-024-07095-4
Giorgio Orlando, Matthew Roy, Judith Bubbear, Shane Clarke, Richard Keen, Muhammad Kassim Javaid, Alex Ireland
{"title":"Longitudinal assessment of physical function in adults with X-linked hypophosphatemia following initiation of burosumab therapy.","authors":"Giorgio Orlando, Matthew Roy, Judith Bubbear, Shane Clarke, Richard Keen, Muhammad Kassim Javaid, Alex Ireland","doi":"10.1007/s00198-024-07095-4","DOIUrl":null,"url":null,"abstract":"<p><p>We assessed multiple components of muscle function in ten adults with X-linked hypophosphatemia (XLH) receiving burosumab treatment. Lower limb power (+ 9%), short physical performance battery (SPPB) score (+ 1.2 points), and physical activity (+ 65%) increased following 6 months of treatment, and hand grip increased (+ 10%) between 6 and 12 months of treatment.</p><p><strong>Purpose: </strong>X-linked hypophosphatemia (XLH) is a rare genetic disorder of phosphate metabolism. Burosumab is a monoclonal antibody treatment shown to improve phosphate homeostasis and improve symptoms as well as fracture healing when used as a therapy for XLH in adults. However, little is known about its effects on the large deficits in multiple components of physical function previously reported in XLH.</p><p><strong>Methods: </strong>Ten adults (6 females, age 41.1 ± 15.7 y) were recruited from specialist centres in London and Bristol. During clinical visits for initial burosumab treatment and at 6-month and 12-month follow-up, physical function, and physical activity (PA) assessments were performed. In detail, lower limb power was assessed by mechanography via a countermovement jump, mobility by short physical performance battery (SPPB), functional capacity by 6-min walk test (6MWT), upper limb strength by hand grip dynamometry, and PA via an International Physical Activity Questionnaire (IPAQ). Differences between baseline and 6-month follow-up, and in a subset of 5 patients between 6- and 12-month follow-up, were assessed.</p><p><strong>Results: </strong>Lower limb power increased by 9% (P = 0.049) from baseline to 6 months, as did SPPB score (+ 1.2 points, P = 0.033) and total PA (+ 65%, P = 0.046) although hand grip and 6MWT did not differ. Only for hand grip was a significant improvement (+ 10%, P = 0.023) seen between 6 and 12 months.</p><p><strong>Conclusions: </strong>Burosumab treatment is associated with improved lower limb function and mobility at 6 months, with improvement in hand grip strength at 12 months. Future studies should explore the underlying mechanisms and describe on function and other patient outcomes.</p>","PeriodicalId":19638,"journal":{"name":"Osteoporosis International","volume":" ","pages":"2055-2060"},"PeriodicalIF":4.2000,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Osteoporosis International","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s00198-024-07095-4","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2024/7/19 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}
引用次数: 0

Abstract

We assessed multiple components of muscle function in ten adults with X-linked hypophosphatemia (XLH) receiving burosumab treatment. Lower limb power (+ 9%), short physical performance battery (SPPB) score (+ 1.2 points), and physical activity (+ 65%) increased following 6 months of treatment, and hand grip increased (+ 10%) between 6 and 12 months of treatment.

Purpose: X-linked hypophosphatemia (XLH) is a rare genetic disorder of phosphate metabolism. Burosumab is a monoclonal antibody treatment shown to improve phosphate homeostasis and improve symptoms as well as fracture healing when used as a therapy for XLH in adults. However, little is known about its effects on the large deficits in multiple components of physical function previously reported in XLH.

Methods: Ten adults (6 females, age 41.1 ± 15.7 y) were recruited from specialist centres in London and Bristol. During clinical visits for initial burosumab treatment and at 6-month and 12-month follow-up, physical function, and physical activity (PA) assessments were performed. In detail, lower limb power was assessed by mechanography via a countermovement jump, mobility by short physical performance battery (SPPB), functional capacity by 6-min walk test (6MWT), upper limb strength by hand grip dynamometry, and PA via an International Physical Activity Questionnaire (IPAQ). Differences between baseline and 6-month follow-up, and in a subset of 5 patients between 6- and 12-month follow-up, were assessed.

Results: Lower limb power increased by 9% (P = 0.049) from baseline to 6 months, as did SPPB score (+ 1.2 points, P = 0.033) and total PA (+ 65%, P = 0.046) although hand grip and 6MWT did not differ. Only for hand grip was a significant improvement (+ 10%, P = 0.023) seen between 6 and 12 months.

Conclusions: Burosumab treatment is associated with improved lower limb function and mobility at 6 months, with improvement in hand grip strength at 12 months. Future studies should explore the underlying mechanisms and describe on function and other patient outcomes.

Abstract Image

查看原文
分享 分享
微信好友 朋友圈 QQ好友 复制链接
本刊更多论文
对患有 X 连锁低磷血症的成人在开始接受布罗苏单抗治疗后的身体功能进行纵向评估。
我们对十名接受布罗苏单抗治疗的X连锁低磷血症(XLH)成人的肌肉功能进行了评估。治疗6个月后,下肢力量(+ 9%)、短期体能测试(SPPB)得分(+ 1.2分)和体力活动(+ 65%)均有所增加,治疗6至12个月期间,手部握力有所增加(+ 10%)。目的:X连锁低磷酸盐血症(XLH)是一种罕见的磷酸盐代谢遗传性疾病。布罗苏单抗是一种单克隆抗体疗法,用于治疗成人 XLH 时,可改善磷酸盐稳态,改善症状和骨折愈合。然而,人们对其对先前报道的 XLH 患者多种身体功能缺陷的影响知之甚少:从伦敦和布里斯托尔的专科中心招募了 10 名成人(6 名女性,年龄为 41.1 ± 15.7 岁)。在接受初始布罗苏单抗治疗的临床访问期间以及6个月和12个月的随访期间,进行了身体功能和体力活动(PA)评估。具体而言,下肢力量通过反向运动跳跃进行力学评估,活动能力通过短期体能测试(SPPB)进行评估,功能能力通过6分钟步行测试(6MWT)进行评估,上肢力量通过手部握力测定法进行评估,体力活动能力通过国际体力活动问卷(IPAQ)进行评估。评估了基线和 6 个月随访之间的差异,以及 5 名患者在 6 个月和 12 个月随访之间的差异:结果:从基线到 6 个月期间,下肢力量增加了 9% (P = 0.049),SPPB 评分(+ 1.2 分,P = 0.033)和总 PA(+ 65%,P = 0.046)也增加了,但手部握力和 6MWT 没有差异。只有手部握力在 6 个月和 12 个月之间有明显改善(+ 10%,P = 0.023):结论:布罗索单抗治疗可在6个月时改善下肢功能和活动能力,并在12个月时改善手部握力。未来的研究应探索其潜在机制,并描述其对功能和其他患者预后的影响。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
求助全文
约1分钟内获得全文 去求助
来源期刊
Osteoporosis International
Osteoporosis International 医学-内分泌学与代谢
CiteScore
8.10
自引率
10.00%
发文量
224
审稿时长
3 months
期刊介绍: An international multi-disciplinary journal which is a joint initiative between the International Osteoporosis Foundation and the National Osteoporosis Foundation of the USA, Osteoporosis International provides a forum for the communication and exchange of current ideas concerning the diagnosis, prevention, treatment and management of osteoporosis and other metabolic bone diseases. It publishes: original papers - reporting progress and results in all areas of osteoporosis and its related fields; review articles - reflecting the present state of knowledge in special areas of summarizing limited themes in which discussion has led to clearly defined conclusions; educational articles - giving information on the progress of a topic of particular interest; case reports - of uncommon or interesting presentations of the condition. While focusing on clinical research, the Journal will also accept submissions on more basic aspects of research, where they are considered by the editors to be relevant to the human disease spectrum.
期刊最新文献
Correction: Exposure to air pollution might decrease bone mineral density and increase the prevalence of osteoporosis: A mendelian randomization study. Type 2 diabetes incidence in patients initiating denosumab or alendronate treatment: a primary care cohort study. Real-world efficacy of a teriparatide biosimilar (RGB-10) compared with reference teriparatide on bone mineral density, trabecular bone score, and bone parameters assessed using quantitative ultrasound, 3D-SHAPER® and high-resolution peripheral computer tomography in postmenopausal women with osteoporosis and very high fracture risk. One versus 2 years of alendronate following denosumab: the CARD extension. Association of proton-density fat fraction with osteoporosis: a systematic review and meta-analysis.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
现在去查看 取消
×
提示
确定
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
已复制链接
已复制链接
快去分享给好友吧!
我知道了
×
扫码分享
扫码分享
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 联系我们:info@booksci.cn
Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。
Copyright © 2023 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1