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NEJM：RNA干扰治疗遗传性转胸腺肽淀粉样变性

2018-07-06 zhangfan MedSci原创

遗传性转胸腺肽淀粉样变性RNAPatisiran
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研究认为Patisiran可改善遗传性转胸腺肽淀粉样变性患者的多种临床指标

Patisiran是一种在研RNA干扰药物，可特异性地抑制转胸腺肽的肝合成。

近日的III期临床研究中，研究人员招募遗传性经胸腺肽淀粉样变性伴多发性神经病患者，随机接受每公斤体重0.3mg的Patisiran或安慰剂，每3周1次，研究的主要终点为改良的神经病变损害评分+7(mNIS+7)，其他评估方法包括Norfolk活质量-糖尿病神经病评估(Norfolk QOL-DN)、10米步行测试以及白蛋白水平校准的BMI。

225名患者参与研究，其中Patisiran治疗组148人，安慰剂组77人，基线mNIS+7得分分别为 80.9和74.6。18个月后mNIS+7得分最小二乘平均变化，Patisiran治疗组减少6.0分，安慰剂组增加28.0分；基线 Norfolk QOL-DN得分，Patisiran治疗组59.6分，安慰剂组增加55.5分，18个月后，得分最小二乘平均变化，Patisiran治疗组减少6.7分，安慰剂组增加14.4分。Patisiran治疗对患者步行速度以及改良BMI均有作用，效果优于安慰剂。约20%的Patisiran治疗组患者出现轻中度输液反应，组间不良事件率及严重程度相近。

研究认为Patisiran可改善遗传性转胸腺肽淀粉样变性患者的多种临床指标。

原始出处：


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    2019-05-16 1508842480_96220445

    #胸腺#

    0

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    2019-06-02 般若傻瓜

    #遗传性#

    0

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    2019-01-02 1194695146_61857354

    #RNA干扰#

    0

  4. 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ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329699, encodeId=eaeb329699d2, content=知之为知之.学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTKibA62IFMeUJEmSyIe8DvYFeERuOsfrRd8iano7nENqQU1dRR3tqeQOUL3eqgYgEZRT2t2iaEsib0JEw/132, createdBy=66771693911, createdName=易水河, createdTime=Fri Jul 06 09:04:50 CST 2018, time=2018-07-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329688, encodeId=cec8329688c8, content=学习学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=112, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/jW482SpianMayicTRbRZ5RzR8ylhYfiaoAUx2BsaYHVia1IKhG3Cg8CT84rBrn6iaVRkyAibJxBcLEXabOQ5p19vWgDe34ib5ErxNgx/0, createdBy=8a701629084, createdName=jihuaijun1112, createdTime=Fri Jul 06 08:12:21 CST 2018, time=2018-07-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329682, encodeId=0b2f3296827d, content=一起学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=104, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/vi_32/TDiaAWyicHgedzX3wKo3qcxudVJib7AfibfvNeufHRF2jG3Bw8MmZyWjIhtBV1mpJI7Sy9n7RcBKK5t3CuOKuOFxkg/0, createdBy=dd702206933, createdName=wzb521zf, createdTime=Fri Jul 06 07:57:00 CST 2018, time=2018-07-06, status=1, ipAttribution=)]
    2018-07-07 xjy03

    #淀粉样变#

    0

  5. 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createdName=jihuaijun1112, createdTime=Fri Jul 06 08:12:21 CST 2018, time=2018-07-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329682, encodeId=0b2f3296827d, content=一起学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=104, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/vi_32/TDiaAWyicHgedzX3wKo3qcxudVJib7AfibfvNeufHRF2jG3Bw8MmZyWjIhtBV1mpJI7Sy9n7RcBKK5t3CuOKuOFxkg/0, createdBy=dd702206933, createdName=wzb521zf, createdTime=Fri Jul 06 07:57:00 CST 2018, time=2018-07-06, status=1, ipAttribution=)]
    2018-07-07 zhouqu_8

    #淀粉样变性#

    0

  6. 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ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329699, encodeId=eaeb329699d2, content=知之为知之.学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTKibA62IFMeUJEmSyIe8DvYFeERuOsfrRd8iano7nENqQU1dRR3tqeQOUL3eqgYgEZRT2t2iaEsib0JEw/132, createdBy=66771693911, createdName=易水河, createdTime=Fri Jul 06 09:04:50 CST 2018, time=2018-07-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329688, encodeId=cec8329688c8, content=学习学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=112, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/jW482SpianMayicTRbRZ5RzR8ylhYfiaoAUx2BsaYHVia1IKhG3Cg8CT84rBrn6iaVRkyAibJxBcLEXabOQ5p19vWgDe34ib5ErxNgx/0, createdBy=8a701629084, createdName=jihuaijun1112, createdTime=Fri Jul 06 08:12:21 CST 2018, time=2018-07-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329682, encodeId=0b2f3296827d, content=一起学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=104, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/vi_32/TDiaAWyicHgedzX3wKo3qcxudVJib7AfibfvNeufHRF2jG3Bw8MmZyWjIhtBV1mpJI7Sy9n7RcBKK5t3CuOKuOFxkg/0, createdBy=dd702206933, createdName=wzb521zf, createdTime=Fri Jul 06 07:57:00 CST 2018, time=2018-07-06, status=1, ipAttribution=)]
    2018-07-07 huangdf

    #变性#

    0

  7. 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ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329699, encodeId=eaeb329699d2, content=知之为知之.学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTKibA62IFMeUJEmSyIe8DvYFeERuOsfrRd8iano7nENqQU1dRR3tqeQOUL3eqgYgEZRT2t2iaEsib0JEw/132, createdBy=66771693911, createdName=易水河, createdTime=Fri Jul 06 09:04:50 CST 2018, time=2018-07-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329688, encodeId=cec8329688c8, content=学习学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=112, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/jW482SpianMayicTRbRZ5RzR8ylhYfiaoAUx2BsaYHVia1IKhG3Cg8CT84rBrn6iaVRkyAibJxBcLEXabOQ5p19vWgDe34ib5ErxNgx/0, createdBy=8a701629084, createdName=jihuaijun1112, createdTime=Fri Jul 06 08:12:21 CST 2018, time=2018-07-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329682, encodeId=0b2f3296827d, content=一起学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=104, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/vi_32/TDiaAWyicHgedzX3wKo3qcxudVJib7AfibfvNeufHRF2jG3Bw8MmZyWjIhtBV1mpJI7Sy9n7RcBKK5t3CuOKuOFxkg/0, createdBy=dd702206933, createdName=wzb521zf, createdTime=Fri Jul 06 07:57:00 CST 2018, time=2018-07-06, status=1, ipAttribution=)]
    2018-07-06 sunfeifeiyang

    阅

    0

  8. 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    2018-07-06 易水河

    知之为知之.学习

    0

  9. 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ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329699, encodeId=eaeb329699d2, content=知之为知之.学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=94, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTKibA62IFMeUJEmSyIe8DvYFeERuOsfrRd8iano7nENqQU1dRR3tqeQOUL3eqgYgEZRT2t2iaEsib0JEw/132, createdBy=66771693911, createdName=易水河, createdTime=Fri Jul 06 09:04:50 CST 2018, time=2018-07-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329688, encodeId=cec8329688c8, content=学习学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=112, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/jW482SpianMayicTRbRZ5RzR8ylhYfiaoAUx2BsaYHVia1IKhG3Cg8CT84rBrn6iaVRkyAibJxBcLEXabOQ5p19vWgDe34ib5ErxNgx/0, createdBy=8a701629084, createdName=jihuaijun1112, createdTime=Fri Jul 06 08:12:21 CST 2018, time=2018-07-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329682, encodeId=0b2f3296827d, content=一起学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=104, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/vi_32/TDiaAWyicHgedzX3wKo3qcxudVJib7AfibfvNeufHRF2jG3Bw8MmZyWjIhtBV1mpJI7Sy9n7RcBKK5t3CuOKuOFxkg/0, createdBy=dd702206933, createdName=wzb521zf, createdTime=Fri Jul 06 07:57:00 CST 2018, time=2018-07-06, status=1, ipAttribution=)]
    2018-07-06 jihuaijun1112

    学习学习学习

    0

  10. 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createdName=jihuaijun1112, createdTime=Fri Jul 06 08:12:21 CST 2018, time=2018-07-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=329682, encodeId=0b2f3296827d, content=一起学习学习, beContent=null, objectType=article, channel=null, level=null, likeNumber=104, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/vi_32/TDiaAWyicHgedzX3wKo3qcxudVJib7AfibfvNeufHRF2jG3Bw8MmZyWjIhtBV1mpJI7Sy9n7RcBKK5t3CuOKuOFxkg/0, createdBy=dd702206933, createdName=wzb521zf, createdTime=Fri Jul 06 07:57:00 CST 2018, time=2018-07-06, status=1, ipAttribution=)]
    2018-07-06 wzb521zf

    一起学习学习

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