每2周布若单抗治疗成人x连锁低磷血症的疗效:我们是否应该向儿童学习?

Q3 Medicine
Alessia Marcellino, Silvia Bloise, Carmelo Pirone, Giulia Brandino, Pietro Gizzone, Roberta Fraternali, Anna Dilillo, Emanuela Del Giudice, Vanessa Martucci, Mariateresa Sanseviero, Leone Maria Rita, Flavia Ventriglia, Riccardo Lubrano
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引用次数: 0

摘要

x连锁低磷血症是一种遗传性疾病,可导致成纤维细胞生长因子23 (FGF23)增加,导致磷酸盐肾消耗。自2018年以来,抗fgf23抗体burosumab已用于儿童和成人的不同剂量治疗该疾病。我们每2周报告一次布罗单抗给药的病例,通常在儿童中进行。我们回顾性评估了一名患有肾钙化症和三期甲状旁腺功能亢进的29岁男性患者每2周的甲状旁腺激素(PTH)、碱性磷酸酶、血清磷酸盐、磷酸盐小管重吸收(TRP)和25OH维生素D,该患者对布罗单抗标准治疗和最大剂量治疗均无反应,每2周用布罗单抗90mg治疗。与4周频率相比,该方案患者血清磷酸盐和TRP升高(分别为1.74±0.26 mg/dL对2.3±0.19 mg/dL [p 0.0004]和71.3%±4.8%对83.9%±7.9% [p 0.01]), PTH降低(183±24.7 pg/mL对109±12.2 pg/mL [p 0.04])。对于成人x连锁低磷血症患者,布若单抗可能是一个不错的选择;为实现疾病控制,需要新的数据来说明通常在儿童中增加剂量和/或给药频率。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Efficacy of Burosumab Every 2 Weeks in an Adult with X-Linked Hypophosphatemia: Should We Learn from Children?

X-linked hypophosphatemia is a genetic condition that leads to fibroblast-growth-factor 23 (FGF23) increase, causing phosphate renal wasting. Since 2018, burosumab, an anti-FGF23 antibody, has been used for this disease with different dosage in children and adults. We report the case of burosumab administration every 2 weeks, as usually done in children. We retrospectively evaluated parathormone (PTH), alkaline phosphatase, serum phosphate, tubular reabsorption of phosphate (TRP), and 25OH vitamin D every 2 weeks in a 29-year-old man with nephrocalcinosis and tertiary hyperparathyroidism who did not respond to standard treatment with burosumab nor to maximum dosage and was treated with burosumab 90 mg every 2 weeks. His serum phosphate and TRP increased with this regimen compared with 4 weeks frequency (respectively 1.74 ± 0.26 mg/dL vs. 2.3 ± 0.19 mg/dL [p 0.0004] and 71.3% ± 4.8% vs. 83.9% ± 7.9% [p 0.01]) with decrease in PTH (183 ± 24.7 pg/mL vs. 109 ± 12.2 pg/mL [p 0.04]). Burosumab may be a good choice in adult patients with X-linked hypophosphatemia; new data are needed regarding the increase in dosage and/or frequency of administration as usually done in children, to achieve disease control.

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来源期刊
CiteScore
4.80
自引率
0.00%
发文量
49
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