X连锁低磷酸盐血症(XLH)是一种遗传性疾病,可导致过度的FGF23,慢性低磷酸盐血症,肌肉骨骼异常,受影响的患者出现骨痛等症状,骨畸形,骨折,和假性骨折.Burosumab是一种与FGF23结合的完全人类单克隆抗体,可改善XLH患者血清1,25(OH)2D和磷酸盐水平的降低。关于使用burosumab的数据不足,其安全性,以及在现实世界中接受治疗的患者的结果。SUNFLOWER(与亚洲partnERs合作,对X连锁低磷酸盐血症/骨软化症患者的纵向观察研究)是一项正在进行的纵向研究,日本和韩国XLH患者的观察性队列研究。注册发生在2018年4月至2020年12月之间。该中期分析比较了接受burosumab的患者与未接受burosumab的患者的背景特征,并评估生物标志物的改善,身体和运动功能,健康相关生活质量(HRQOL)和其他患者报告结果(PRO)指标,以及6个月以上来自15个机构的143名日本患者的burosumab治疗的安全性。患者的中位[四分位距]年龄为17.5[11.0,38.8]岁,女性为98(68.5%)。在年龄<18岁和≥18岁的患者中,40/73(54.8%)和25/70(35.7%)收到burosumab,分别。与未接受burosumab治疗的患者相比,更多年龄≥18岁的患者在基线时出现骨痛(6/25,24.0%vs2/45,4.4%,p=.021)。使用burosumab治疗的患者血清磷酸盐和1,25(OH)2D水平得到改善;此外,病严重程度和HRQOL/PRO措施,如疼痛,似乎改善了超过6个月的burosumab治疗,没有发现新的安全问题。这项研究确定了在现实世界临床实践中接受burosumab的XLH患者的背景特征的趋势。此外,结果支持在现实环境中使用burosumab疗法.
X-linked hypophosphatemia (XLH) is a genetic disease that results in excessive FGF23, chronic hypophosphatemia, and musculoskeletal abnormalities, with affected patients experiencing symptoms such as bone pain, bone deformity, fracture, and pseudofracture. Burosumab is a fully human monoclonal antibody that binds to FGF23, improving lowered serum 1,25(OH)2D and phosphate levels in patients with XLH. There are insufficient data on the use of burosumab, its safety, and the outcomes of treated patients in a real-world setting. The SUNFLOWER (Study of longitUdinal observatioN For patients with X-Linked hypOphosphatemic
rickets/osteomalacia in collaboration With Asian partnERs) study is an ongoing longitudinal, observational cohort study of patients with XLH in Japan and South Korea. Enrollment occurred between April 2018 and December 2020. This interim analysis compared the background characteristics of patients who received burosumab with those who did not, and assessed improvements in biomarkers, physical and motor function, health-related quality-of-life (HRQOL) and other patient-reported outcome (PRO) measures, as well as the safety of burosumab treatment in 143 Japanese patients from 15 institutions over 6 mo. The patients had a median [interquartile range] age of 17.5 [11.0, 38.8] yr and 98 (68.5%) were female. Among patients aged <18 and ≥18 yr, 40/73 (54.8%) and 25/70 (35.7%) received burosumab, respectively. More patients aged ≥18 who received burosumab had bone pain at baseline vs those not treated with burosumab (6/25, 24.0% vs 2/45, 4.4%, p=.021). Patients treated with burosumab had improved serum phosphate and 1,25(OH)2D levels; moreover,
rickets severity and HRQOL/PRO measures, such as pain, appeared to improve over 6 mo of burosumab treatment, and no new safety concerns were identified. This study identified trends in the background characteristics of patients with XLH who receive burosumab in real-world clinical practice. Furthermore, the results support the use of burosumab therapy in real-world settings.