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NEJM：新型药物组合或为肺部囊性纤维化患者带来福音

2015-05-19 佚名 生物谷

药物囊性纤维化
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近期，一项发表于国际杂志New England Journal of Medicine上的研究论文中，来自美国西北大学的研究人员通过研究表示，两种药物的新型组合或可有效改善肺部囊性纤维化患者的机体健康；相比安慰剂而言，这两种药物：lumacaftor和ivacaftor可以明显改善患者的呼吸及肺部感染状况。研究者Susanna McColley表示，这种新型药物组合疗法仅在美国就可使得1.5万名患

近期，一项发表于国际杂志New England Journal of Medicine上的研究论文中，来自美国西北大学的研究人员通过研究表示，两种药物的新型组合或可有效改善肺部囊性纤维化患者的机体健康；相比安慰剂而言，这两种药物：lumacaftor和ivacaftor可以明显改善患者的呼吸及肺部感染状况。

研究者Susanna McColley表示，这种新型药物组合疗法仅在美国就可使得1.5万名患者收益，而欧洲、北美及澳大利亚有将近7.5万人患有因基因突变而引发的囊性纤维化。文章中，研究者对年龄在12岁及以上的1108名个体进行长达6个月的新型药物组合治疗；而引发肺部囊性纤维化的原因和许多不同的基因突变相关，常见的情况是患者携带有两个拷贝的F508del基因突变，这种情况在大约一半的囊性纤维化病人机体中都存在。

囊性纤维化会引发患者机体产生过量的粘液，从而产生慢性肺部感染以及胰腺问题；研究者推测，在美国携带F508del基因突变的个体往往预期寿命仅为37年。药物Ivacaftor是2012年FDA批准一种新药，其可以帮助有效改善携带罕见遗传突变的囊性纤维化患者的机体健康，而另外一种名为lumacaftor的药物是由Vertex制药开发。

当两种药物相互结合后给患者带来的健康改善远大于单一药物的作用效果，而研究者指出，他们目前正在调节针对每一个病人的剂量，以及寻找药物联合后可能带来的副作用；后期还需要更多的研究来阐明两种药物联合的作用机制，研究人员希望本文研究或为后期开发新型治疗肺部囊性纤维化的个体化靶向疗法提供新的思路和希望。

原始出处：

Claire E. Wainwright, M.B., B.S., M.D., J. Stuart Elborn, M.D., Bonnie W. Ramsey, M.D. et al.Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.NEJM, May 17, 2015.DOI: 10.1056/NEJMoa1409547

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    2016-03-15 12498dd9m11暂无昵称

    #药物组合#

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    2016-02-10 wolongzxh

    #囊性#

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    2016-01-13 ms5906905816781247

    #囊性纤维化患者#

    0

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    2015-05-21 ms1260584454294838

    #新型药物#

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    2015-05-19 chenhui888

    联合用药

    0

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