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Benefit of burosumab in adults with X-linked hypophosphataemia (XLH) is maintained with long-term treatment.布罗索尤单抗治疗成人性 X 连锁低磷血症（XLH）的疗效可长期维持。
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A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial Evaluating the Efficacy of Burosumab, an Anti-FGF23 Antibody, in Adults With X-Linked Hypophosphatemia: Week 24 Primary Analysis.一项评估抗 FGF23 抗体布罗索尤单抗在 X 连锁低磷血症成人患者中的疗效的随机、双盲、安慰剂对照、3 期临床试验：第 24 周主要分析。
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本文引用的文献

1
Benefit of burosumab in adults with X-linked hypophosphataemia (XLH) is maintained with long-term treatment.布罗索尤单抗治疗成人性 X 连锁低磷血症（XLH）的疗效可长期维持。
RMD Open. 2023 Feb;9(1). doi: 10.1136/rmdopen-2022-002676.
2
Patient-reported outcomes measures of X-linked hypophosphataemia participants: findings from a prospective cohort study in the UK.X 连锁低磷血症患者报告结局测量指标：来自英国前瞻性队列研究的结果。
Orphanet J Rare Dis. 2023 Feb 8;18(1):26. doi: 10.1186/s13023-023-02620-w.
3
Physical function and physical activity in adults with X-linked hypophosphatemia.X 连锁低磷血症成人的身体功能和身体活动。
Osteoporos Int. 2022 Jul;33(7):1485-1491. doi: 10.1007/s00198-022-06318-w. Epub 2022 Feb 5.
4
Musculoskeletal Features in Adults With X-linked Hypophosphatemia: An Analysis of Clinical Trial and Survey Data.伴 X 连锁低磷血症的成人生长骨骼肌肉特征：临床试验和调查数据分析。
J Clin Endocrinol Metab. 2022 Feb 17;107(3):e1249-e1262. doi: 10.1210/clinem/dgab739.
5
Burosumab treatment in adults with X-linked hypophosphataemia: 96-week patient-reported outcomes and ambulatory function from a randomised phase 3 trial and open-label extension.布罗索尤单抗治疗成人X连锁低磷血症：一项随机3期试验和开放标签扩展研究的96周患者报告结局及门诊功能
RMD Open. 2021 Sep;7(3). doi: 10.1136/rmdopen-2021-001714.
6
Exploring the burden of X-linked hypophosphatemia: a European multi-country qualitative study.探讨 X 连锁低磷血症的负担：一项欧洲多国定性研究。
Qual Life Res. 2020 Jul;29(7):1883-1893. doi: 10.1007/s11136-020-02465-x. Epub 2020 Mar 11.
7
The Lifelong Impact of X-Linked Hypophosphatemia: Results From a Burden of Disease Survey.X连锁低磷血症的终身影响：疾病负担调查结果
J Endocr Soc. 2019 May 7;3(7):1321-1334. doi: 10.1210/js.2018-00365. eCollection 2019 Jul 1.
8
Continued Beneficial Effects of Burosumab in Adults with X-Linked Hypophosphatemia: Results from a 24-Week Treatment Continuation Period After a 24-Week Double-Blind Placebo-Controlled Period.在 24 周双盲安慰剂对照期后继续治疗 24 周：X 连锁低磷血症成人中布罗索尤单抗的持续有益作用的结果。
Calcif Tissue Int. 2019 Sep;105(3):271-284. doi: 10.1007/s00223-019-00568-3. Epub 2019 Jun 4.
9
Clinical practice recommendations for the diagnosis and management of X-linked hypophosphataemia.X 连锁低磷血症的诊断和管理临床实践建议。
Nat Rev Nephrol. 2019 Jul;15(7):435-455. doi: 10.1038/s41581-019-0152-5.
10
FGF23 and its role in X-linked hypophosphatemia-related morbidity.成纤维细胞生长因子 23 及其在 X 连锁低磷血症相关发病机制中的作用。
Orphanet J Rare Dis. 2019 Feb 26;14(1):58. doi: 10.1186/s13023-019-1014-8.

布罗索尤单抗治疗X连锁低磷血症成人早期准入项目的疗效：3例患者的病例系列

Improvements with burosumab treatment in an early access programme for adults with X-linked hypophosphataemia: A case series of three patients.

作者信息

Day Julia, Jayatilleke Chandrin, Roy Matthew

机构信息

Department of Rheumatology, Bristol Royal Infirmary, Bristol, BS2 8HW, UK.

Department of Rheumatology, Royal United Hospital, Bath BA1 3NG, UK.

出版信息

Bone Rep. 2024 Nov 12;23:101814. doi: 10.1016/j.bonr.2024.101814. eCollection 2024 Dec.

DOI:10.1016/j.bonr.2024.101814
PMID:39624115
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11609550/
Abstract

X-linked hypophosphataemia (XLH) is a life-long phosphate-wasting disorder that causes skeletal deformities, pain, stiffness, and fatigue and impairs quality of life. Burosumab was approved for use in adults in 2020. We describe three adults with persistent XLH symptoms who received burosumab treatment in a real-world setting. Patients report improvements in pain, mobility, physical function, energy, fatigue, and mental wellbeing through patient-reported outcome measures, enriched with further detail from written testimonials.

摘要

X连锁低磷血症（XLH）是一种终身性的磷酸盐消耗性疾病，会导致骨骼畸形、疼痛、僵硬和疲劳，损害生活质量。布罗索尤单抗于2020年获批用于成人。我们描述了三名患有持续性XLH症状的成人，他们在现实环境中接受了布罗索尤单抗治疗。通过患者报告的结局指标，并结合书面证词中的更多细节，患者报告了疼痛、活动能力、身体功能、精力、疲劳和心理健康方面的改善。