X连锁低磷血症成人患者早期接受布芦单抗治疗后病情有所改善：三名患者的病例系列

IF 2.1 Q3 ENDOCRINOLOGY & METABOLISM Bone Reports Pub Date : 2024-11-12 DOI:10.1016/j.bonr.2024.101814

Julia Day , Chandrin Jayatilleke , Matthew Roy

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引用次数: 0

摘要

X连锁低磷血症（XLH）是一种终身性磷酸盐消耗性疾病，会导致骨骼畸形、疼痛、僵硬和疲劳，并影响生活质量。布罗苏单抗于 2020 年获准用于成人。我们描述了在现实世界中接受布罗苏单抗治疗的三名具有持续XLH症状的成人患者的情况。患者通过患者报告的结果指标报告了疼痛、活动能力、身体功能、精力、疲劳和精神健康的改善情况，并从书面证词中获得了更多细节。

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

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.

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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.