在X-连锁低磷血症（XLH）青少年骨骼发育末期停止布罗苏单抗治疗的影响

IF 2.1 Q3 ENDOCRINOLOGY & METABOLISM

Bone Reports Pub Date : 2024-11-24 DOI:10.1016/j.bonr.2024.101819

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

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引用次数: 0

摘要

目前，许多患有 X 连锁低磷血症（XLH）的青少年不得不在骨骼生长期结束时停止使用布罗苏单抗。我们描述了一组 XLH 青少年在停用布罗苏单抗之前、期间和之后（中位疗程 37.5 个月）的经历。在布罗苏单抗治疗期间，患者的血清磷酸盐、疼痛、活动能力、功能和生活质量均有所改善，但在停止治疗后，这些改善又发生了逆转。我们需要更多真实世界的数据来探讨不间断布罗苏单抗治疗在青少年中的价值。

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

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.

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来源期刊

Bone Reports Medicine-Orthopedics and Sports Medicine

CiteScore

4.30

自引率

4.00%

发文量

444

审稿时长

57 days

期刊介绍： Bone Reports is an interdisciplinary forum for the rapid publication of Original Research Articles and Case Reports across basic, translational and clinical aspects of bone and mineral metabolism. The journal publishes papers that are scientifically sound, with the peer review process focused principally on verifying sound methodologies, and correct data analysis and interpretation. We welcome studies either replicating or failing to replicate a previous study, and null findings. We fulfil a critical and current need to enhance research by publishing reproducibility studies and null findings.