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X连锁低磷血症(XLH)青少年骨骼生长结束时停用布罗索尤单抗治疗的影响。

Impact of stopping burosumab treatment at the end of skeletal growth in adolescents with X-linked hypophosphatemia (XLH).

作者信息

Jarvis Charlotte, Ramakrishnan Renuka, Dharmaraj Poonam, Mushtaq Talat, Gupta Sanjay, Williams Angela, Rylands Angela J, Barham Helen, Nixon Annabel, Uday Suma

机构信息

Alder Hey Children's NHS Foundation Trust, Liverpool L12 2AP, UK.

Leeds Teaching Hospitals NHS Trust, Leeds LS9 7TP, UK.

出版信息

Bone Rep. 2024 Nov 24;24:101819. doi: 10.1016/j.bonr.2024.101819. eCollection 2025 Mar.

DOI:10.1016/j.bonr.2024.101819
PMID:39679164
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11638637/
Abstract

Many adolescents with X-linked hypophosphatemia (XLH) currently have to stop treatment with burosumab at the end of skeletal growth. We describe the experience of a cohort of adolescents with XLH before, during, and after stopping burosumab (median treatment duration 37.5 months). Improvements in serum phosphate, pain, mobility, function, and quality of life noted during burosumab treatment were reversed after treatment cessation. Further real-world data are needed to explore the value of uninterrupted burosumab treatment in adolescents.

摘要

许多患有X连锁低磷血症(XLH)的青少年在骨骼生长结束时不得不停止布罗索尤单抗治疗。我们描述了一组患有XLH的青少年在停止布罗索尤单抗治疗前、治疗期间和治疗后的经历(中位治疗持续时间37.5个月)。布罗索尤单抗治疗期间血清磷酸盐、疼痛、活动能力、功能和生活质量的改善在治疗停止后出现逆转。需要更多真实世界的数据来探索布罗索尤单抗在青少年中持续治疗的价值。

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本文引用的文献

1
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Eur J Med Genet. 2024 Aug;70:104958. doi: 10.1016/j.ejmg.2024.104958. Epub 2024 Jun 29.
2
The International X-Linked Hypophosphatemia (XLH) Registry: first interim analysis of baseline demographic, genetic and clinical data.国际 X 连锁低磷血症(XLH)登记处:基线人口统计学、遗传学和临床数据的首次中期分析。
Orphanet J Rare Dis. 2023 Sep 27;18(1):304. doi: 10.1186/s13023-023-02882-4.
3
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.
4
Burosumab for X-linked hypophosphatemia in children and adolescents: Opinion based on early experience in seven European countries.用于治疗儿童和青少年 X 连锁低磷血症的布罗索尤单抗:基于七个欧洲国家早期经验的观点。
Front Endocrinol (Lausanne). 2023 Jan 31;13:1034580. doi: 10.3389/fendo.2022.1034580. eCollection 2022.
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Impact of X-Linked Hypophosphatemia on Muscle Symptoms.X 连锁低磷血症对肌肉症状的影响。
Genes (Basel). 2022 Dec 19;13(12):2415. doi: 10.3390/genes13122415.
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Interdisciplinary management of FGF23-related phosphate wasting syndromes: a Consensus Statement on the evaluation, diagnosis and care of patients with X-linked hypophosphataemia.成纤维细胞生长因子 23(FGF23)相关磷代谢紊乱的综合管理:X 连锁低磷血症患者评估、诊断和治疗的专家共识声明。
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Osteoporos Int. 2022 Jul;33(7):1485-1491. doi: 10.1007/s00198-022-06318-w. Epub 2022 Feb 5.
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