布鲁苏单抗与口服磷酸盐和活性维生素D治疗x连锁低磷血症成人的实际有效性

IF 5.9 1区 医学 Q1 ENDOCRINOLOGY & METABOLISM
Pablo Florenzano, Erik A Imel, Aliya A Khan, Zhiyi Li, Marc Vincent, Takanobu Nomura, Stanley Krolczyk, Ben Johnson, Leanne Ward
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引用次数: 0

摘要

在x连锁低磷血症(XLH)中,PHEX基因变异导致FGF23生成升高,导致低磷血症、骨软化症、骨软化症相关骨折、骨关节炎、骨髓瘤病、椎管狭窄以及疼痛、僵硬和身体功能下降等症状。Burosumab是一种被批准用于XLH治疗的抗fgf23单克隆抗体。目前缺乏比较口服磷酸盐/活性维生素D (Pi/D)与布罗单抗在成人中的随机研究。该分析利用了来自前瞻性美国XLH疾病监测项目(NCT03651505)的真实数据,基于基线至1年就诊时血清磷酸盐、1,25-二羟基维生素D (1,25[OH]2D)、甲状旁腺激素(PTH)、西安大略省和麦克马斯特大学骨关节炎指数(WOMAC)评分、患者报告的结果测量信息系统物理功能(PROMIS PF)的变化,评估了布罗单抗与Pi/D的有效性。和计时起走(TUG)绩效结果。纳入了2组XLH成人患者,他们要么在基线和第1年就诊期间开始使用布罗单抗(n = 65),要么在研究开始时使用Pi/D (n = 74),没有接受布罗单抗。采用治疗加权的逆概率来调整由于基线队列差异造成的潜在混淆。在第1年的随访中,布罗单抗与Pi/D相比,基线的平均(标准误差)变化显著(0.78 [0.08]vs 0.15 [0.14] mg/dL;p
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Real-world effectiveness of burosumab vs oral phosphate and active vitamin D in adults with X-linked hypophosphatemia.

Real-world effectiveness of burosumab vs oral phosphate and active vitamin D in adults with X-linked hypophosphatemia.

Real-world effectiveness of burosumab vs oral phosphate and active vitamin D in adults with X-linked hypophosphatemia.

Real-world effectiveness of burosumab vs oral phosphate and active vitamin D in adults with X-linked hypophosphatemia.

In X-linked hypophosphatemia (XLH), PHEX gene variants lead to elevated FGF23 production, resulting in hypophosphatemia, osteomalacia, osteomalacia-related fractures, osteoarthritis, enthesopathy, spinal stenosis, and symptoms of pain, stiffness, and decreased physical function. Burosumab is an anti-FGF23 monoclonal Ab approved for XLH treatment. Randomized studies comparing oral phosphate/active vitamin D (Pi/D) to burosumab in adults are lacking. This analysis, which utilized real-world data from the prospective, Americas-based XLH Disease Monitoring Program (NCT03651505), evaluated the effectiveness of burosumab vs Pi/D, based on changes from baseline to the year 1 visit in serum phosphate, 1,25(OH)2D, PTH, Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) scores, Patient-Reported Outcomes Measurement Information System Physical Function (PROMIS PF), and Timed Up and Go performance outcome. Two cohorts of adults with XLH who either began burosumab between baseline and the year 1 visit (n = 65) or were on Pi/D (n = 74) at study entry and did not receive burosumab were included. Inverse probability of treatment weighting was employed to adjust for potential confounding due to baseline cohort differences. At the year 1 visit, mean (SE) change from baseline was significant for burosumab vs Pi/D in serum phosphate (0.78 [0.08] vs 0.15 [0.14] mg/dL; p < .001), 1,25(OH)2D (19.41 [3.39] vs 5.49 [3.43] pg/mL; p = .011), PTH (-13.82 [5.00] vs 11.79 [8.10] pg/mL; p = .006), WOMAC pain (-7.50 [2.34] vs 4.47 [3.23]; p = .004), WOMAC physical function (-5.68 [1.96] vs 6.77 [4.85]; p = .006), and WOMAC total (-7.78 [2.06] vs 3.15 [3.37]; p = .005) scores, PROMIS PF (1.51 [0.73] vs -1.64 [1.11], p = .018), and TUG (-1.19 [0.42] vs 0.55 [0.43] s, p = .011). A trend towards improved WOMAC stiffness was observed for burosumab (-10.16 [2.85] vs -1.79 [3.68]; p = .086). In this real-world analysis of adults with XLH, burosumab treatment was associated with improved biochemical parameters, pain, physical function, and mobility compared with Pi/D.

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来源期刊
Journal of Bone and Mineral Research
Journal of Bone and Mineral Research 医学-内分泌学与代谢
CiteScore
11.30
自引率
6.50%
发文量
257
审稿时长
2 months
期刊介绍: The Journal of Bone and Mineral Research (JBMR) publishes highly impactful original manuscripts, reviews, and special articles on basic, translational and clinical investigations relevant to the musculoskeletal system and mineral metabolism. Specifically, the journal is interested in original research on the biology and physiology of skeletal tissues, interdisciplinary research spanning the musculoskeletal and other systems, including but not limited to immunology, hematology, energy metabolism, cancer biology, and neurology, and systems biology topics using large scale “-omics” approaches. The journal welcomes clinical research on the pathophysiology, treatment and prevention of osteoporosis and fractures, as well as sarcopenia, disorders of bone and mineral metabolism, and rare or genetically determined bone diseases.
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