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.
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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