布罗苏马布与X链接低血症儿童的常规治疗:随机,主动控制,开放的第3期试验
Erik A Imel1, Francis H Glorieux2, Michael P Whyte3
1Department of Medicine and Department of Pediatrics, Indiana University School of Medicine, Indianapolis, IN, USA.
Lancet (London, England)
|May 21, 2019
概括
与传统疗法相比,布罗苏马布显著改善了X相关低血症儿童的恶心病的严重程度和生长. 这项研究强调了布罗苏马布作为这种罕见遗传疾病的更有效治疗方法.
科学领域:
- 儿童内分泌学
- 罕见的遗传疾病
- 骨发育不良症
背景情况:
- 结合X的低血症 (XLH) 是一种罕见的遗传性疾病,儿童的特征是FGF23升高,低血症,病和生长障碍.
- 常规治疗包括口服酸盐和活性维生素D, 但其在严重病例中的有效性有限.
研究的目的:
- 为了比较抗 FGF23 抗体 burosumab 的疗效和安全性,与传统疗法在治疗儿科 X 结合低血症.
- 评估布罗苏马布对XLH儿童的恶心病严重程度,生长和生化参数的影响.
主要方法:
- 一个随机的,主控的,开放的,第三阶段试验招募了61名儿童 (年龄在1-12岁).
- 患者被随机分为接受皮下布罗苏马布或常规治疗64周.
- 主要终点是40周病严重程度的变化,该变化由放射性全球变化印象 (RGIC) 评分进行评估.
主要成果:
- 与传统治疗组相比,布罗苏马布组在40周的RGIC得分显著改善 (1. 9 vs 0. 8, p< 0. 0001).
- 用布罗苏马布治疗的儿童在病的严重程度,生长和生化方面有显著的改善.
- 在布罗苏马布组中,不良事件更频繁 (59% vs 22%),但严重的不良事件与治疗无关.
结论:
- 与传统治疗相比,布罗苏马布在改善X相关低血症儿童的恶心病严重程度和生长方面表现出更好的效果.
- 布罗苏马布是治疗儿科XLH的一个有前途的疗法,具有显著的临床益处.
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