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.
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。
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本文引用的文献
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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。
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