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JASN：伯舒替尼治疗常染色体显性多囊肾疾病疗效如何？

2017-08-27 xing.T MedSci原创

伯舒替尼ADPKD疗效
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总之，与安慰剂相比，采用伯舒替尼200毫克/天治疗可以减少ADPKD患者的肾脏肿大。整个胃肠道和肝脏的毒性与之前伯舒替尼的研究一致；并没有发现新的毒性反应。

Src激活与常染色体显性遗传性多囊肾病（ADPKD）的发病机制有关。近日，肾脏病领域权威杂志Journal of the American Society of Nephrology上发表一篇研究文章，这项2期、多中心研究评估了口服双Src/Bcr-Abl酪氨酸激酶抑制剂波舒替尼的安全性和疗效。

eGFR≥60ml/min每1.73m2、肾脏总体积≥750ml的ADPKD患者按1:1:1的比例分别给予伯舒替尼200毫克/天、伯舒替尼400毫克/天或安慰剂治疗24个月。该研究的主要终点为每年肾脏肿大的发生率。

在172名招募的患者中，169名患者接受了至少一个研究剂量的治疗。根据修订的协议，24例患者最初接受伯舒替尼400毫克/天的剂量随后降低到200毫克/天。采用伯舒替尼200毫克/天治疗的患者相比于安慰剂患者肾脏肿大每年的发生率减少了66%（分别为1.63% vs. 4.74%，P=0.01），并且接受伯舒替尼治疗的患者与安慰剂者相比，肾脏肿大每年的发生率减少了82%（分别为0.84% vs. 4.74%，P＜0.001）。在治疗期间，接受安慰剂或伯舒替尼的患者每年eGFR下降程度类似。胃肠道和肝脏相关的不良事件是最常见的毒性反应。

总之，与安慰剂相比，采用伯舒替尼200毫克/天治疗可以减少ADPKD患者的肾脏肿大。整个胃肠道和肝脏的毒性与之前伯舒替尼的研究一致；并没有发现新的毒性反应。

原始出处：

Vladimir Tesar,et al. Bosutinib versus Placebo for Autosomal Dominant Polycystic Kidney Disease.JASN,2017, http://jasn.asnjournals.org/content/early/2017/08/23/ASN.2016111232.abstract

本文系梅斯医学（MedSci）原创编译整理，转载需授权！


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    2017-12-25 yhy100200

    #常染色体#

    0

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    2018-02-10 chenhongpeng

    #ASN#

    0

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    2017-08-29 新生儿张玉军

    #染色体#

    0

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    2017-08-29 ms9608593228839890

    #多囊肾#

    0

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    2017-08-28 清风拂面

    谢谢分享.学习了

    0

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