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NEJM：新药Midostaurin可缓解肥大细胞增生症

2016-07-15 Andreas Reiter NEJM

增生靶向药物原癌基因
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德国

德国科学家日前发表的一项医学研究成果显示，肥大细胞增生症引起的严重病变，可以通过靶向药物米哚妥林得到缓解。

肥大细胞增生症是一种目前病因尚不完全明确的罕见疾病，特点是肥大细胞通过浸润性增生侵入周围组织，一般被认为与原癌基因KIT的激活性突变有关，这种基因的产物是Ⅲ型酪氨酸激酶。罹患此病后，病人会出现过敏、皮肤病变、体重下降和贫血等症状，严重时可转化为白血病。

最新研究覆盖了超过100名患有严重肥大细胞增生症的病人，是相关领域迄今规模最大的研究。研究人员发现，一种名为Midostaurin（米哚妥林）（PKC412）的酪氨酸激酶抑制剂，能够有选择性地抑制激活性突变KIT基因，通过这一靶向药物治疗，参与研究的约六成严重患者病情得到明显好转。

这项发表在美国《新英格兰医学杂志》上的研究显示，米哚妥林能遏制器质性病变和肥大细胞的浸润性增生，患者寿命能由此平均延长28.7个月。

原始出处

Jason Gotlib, M.D., Hanneke C. Kluin-Nelemans, M.D., Ph.D., Tracy I. George, M.D., Cem Akin, M.D., Ph.D., Karl Sotlar, M.D., Olivier Hermine, M.D., Ph.D., Farrukh T. Awan, M.D., Elizabeth Hexner, M.D., Michael J. Mauro, M.D., David W. Sternberg, M.D., Ph.D., Matthieu Villeneuve, M.Sc., Alice Huntsman Labed, Ph.D., Eric J. Stanek, Pharm.D., Karin Hartmann, M.D., Hans-Peter Horny, M.D., Peter Valent, M.D., and Andreas Reiter, M.D.Efficacy and Safety of Midostaurin in Advanced Systemic Mastocytosis.NEJM.2016

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  1. [GetPortalCommentsPageByObjectIdResponse(id=1703496, encodeId=aa0e1e03496f0, content=<a href='/topic/show?id=327e9425fc' target=_blank style='color:#2F92EE;'>#IDO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9425, encryptionId=327e9425fc, topicName=IDO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=3e3a30514791, createdName=tongyongming, createdTime=Sat Nov 19 18:23:00 CST 2016, time=2016-11-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=146034, encodeId=67ae146034b0, content=好文章，受益, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/jW482SpianMayicTRbRZ5RzdAIB7UwTkHt6fQkBP8ictliaXtc5gCUmYdAW1sAETC2nhO3q6YjRHkUibyOCtP8ibmXNObTyd63A076/0, createdBy=d4111948983, createdName=1e10c84am36(暂无匿称), createdTime=Tue Oct 11 22:34:26 CST 2016, time=2016-10-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=125509, encodeId=440712550957, content=赞一个！好文章拜读了，认真学习了。, beContent=null, objectType=article, channel=null, level=null, likeNumber=1, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/NUyjXTCJjo7s7ZCKOmmbBTD6nE7gcd5OZpvyGEzkxBP3S275k5ZJVrsPkotZhBglRHM9rKvcj1OgkssqhcnoPw8zIaMZTF8c/0, createdBy=4c441942088, createdName=ylzr123, createdTime=Tue Sep 13 16:32:00 CST 2016, time=2016-09-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=110678, encodeId=6f571106e809, content=学习了，好, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://cdnapi.center.medsci.cn/medsci/head/2017/11/23/ac01daf0962458a9eb8c0b1e2f6ce5bc.jpg, createdBy=04b81710322, createdName=1771ae4158m, createdTime=Sun Sep 04 08:56:00 CST 2016, time=2016-09-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1509249, encodeId=a59f150924937, content=<a href='/topic/show?id=19791e14471' target=_blank style='color:#2F92EE;'>#Tau#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=17144, encryptionId=19791e14471, topicName=Tau)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=55c610090940, createdName=szhvet, createdTime=Sun Jul 17 01:23:00 CST 2016, time=2016-07-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1569172, encodeId=5c2415691e2ca, content=<a href='/topic/show?id=13288188450' target=_blank style='color:#2F92EE;'>#肥大细胞#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81884, encryptionId=13288188450, topicName=肥大细胞)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c92315497125, createdName=gds2014, createdTime=Sun Jul 17 01:23:00 CST 2016, time=2016-07-17, status=1, ipAttribution=)]
    2016-11-19 tongyongming

    #IDO#

    0

  2. [GetPortalCommentsPageByObjectIdResponse(id=1703496, encodeId=aa0e1e03496f0, content=<a href='/topic/show?id=327e9425fc' target=_blank style='color:#2F92EE;'>#IDO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9425, encryptionId=327e9425fc, topicName=IDO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=3e3a30514791, createdName=tongyongming, createdTime=Sat Nov 19 18:23:00 CST 2016, time=2016-11-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=146034, encodeId=67ae146034b0, content=好文章，受益, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/jW482SpianMayicTRbRZ5RzdAIB7UwTkHt6fQkBP8ictliaXtc5gCUmYdAW1sAETC2nhO3q6YjRHkUibyOCtP8ibmXNObTyd63A076/0, createdBy=d4111948983, createdName=1e10c84am36(暂无匿称), createdTime=Tue Oct 11 22:34:26 CST 2016, time=2016-10-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=125509, encodeId=440712550957, content=赞一个！好文章拜读了，认真学习了。, beContent=null, objectType=article, channel=null, level=null, likeNumber=1, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/NUyjXTCJjo7s7ZCKOmmbBTD6nE7gcd5OZpvyGEzkxBP3S275k5ZJVrsPkotZhBglRHM9rKvcj1OgkssqhcnoPw8zIaMZTF8c/0, createdBy=4c441942088, createdName=ylzr123, createdTime=Tue Sep 13 16:32:00 CST 2016, time=2016-09-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=110678, encodeId=6f571106e809, content=学习了，好, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://cdnapi.center.medsci.cn/medsci/head/2017/11/23/ac01daf0962458a9eb8c0b1e2f6ce5bc.jpg, createdBy=04b81710322, createdName=1771ae4158m, createdTime=Sun Sep 04 08:56:00 CST 2016, time=2016-09-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1509249, encodeId=a59f150924937, content=<a href='/topic/show?id=19791e14471' target=_blank style='color:#2F92EE;'>#Tau#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=17144, encryptionId=19791e14471, topicName=Tau)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=55c610090940, createdName=szhvet, createdTime=Sun Jul 17 01:23:00 CST 2016, time=2016-07-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1569172, encodeId=5c2415691e2ca, content=<a href='/topic/show?id=13288188450' target=_blank style='color:#2F92EE;'>#肥大细胞#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81884, encryptionId=13288188450, topicName=肥大细胞)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c92315497125, createdName=gds2014, createdTime=Sun Jul 17 01:23:00 CST 2016, time=2016-07-17, status=1, ipAttribution=)]
    2016-10-11 1e10c84am36(暂无匿称)

    好文章，受益

    0

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    2016-09-13 ylzr123

    赞一个！好文章拜读了，认真学习了。

    0

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    2016-09-04 1771ae4158m

    学习了，好

    0

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    2016-07-17 szhvet

    #Tau#

    0

  6. [GetPortalCommentsPageByObjectIdResponse(id=1703496, encodeId=aa0e1e03496f0, content=<a href='/topic/show?id=327e9425fc' target=_blank style='color:#2F92EE;'>#IDO#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=39, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=9425, encryptionId=327e9425fc, topicName=IDO)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=3e3a30514791, createdName=tongyongming, createdTime=Sat Nov 19 18:23:00 CST 2016, time=2016-11-19, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=146034, encodeId=67ae146034b0, content=好文章，受益, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/jW482SpianMayicTRbRZ5RzdAIB7UwTkHt6fQkBP8ictliaXtc5gCUmYdAW1sAETC2nhO3q6YjRHkUibyOCtP8ibmXNObTyd63A076/0, createdBy=d4111948983, createdName=1e10c84am36(暂无匿称), createdTime=Tue Oct 11 22:34:26 CST 2016, time=2016-10-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=125509, encodeId=440712550957, content=赞一个！好文章拜读了，认真学习了。, beContent=null, objectType=article, channel=null, level=null, likeNumber=1, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/NUyjXTCJjo7s7ZCKOmmbBTD6nE7gcd5OZpvyGEzkxBP3S275k5ZJVrsPkotZhBglRHM9rKvcj1OgkssqhcnoPw8zIaMZTF8c/0, createdBy=4c441942088, createdName=ylzr123, createdTime=Tue Sep 13 16:32:00 CST 2016, time=2016-09-13, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=110678, encodeId=6f571106e809, content=学习了，好, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://cdnapi.center.medsci.cn/medsci/head/2017/11/23/ac01daf0962458a9eb8c0b1e2f6ce5bc.jpg, createdBy=04b81710322, createdName=1771ae4158m, createdTime=Sun Sep 04 08:56:00 CST 2016, time=2016-09-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1509249, encodeId=a59f150924937, content=<a href='/topic/show?id=19791e14471' target=_blank style='color:#2F92EE;'>#Tau#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=17144, encryptionId=19791e14471, topicName=Tau)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=55c610090940, createdName=szhvet, createdTime=Sun Jul 17 01:23:00 CST 2016, time=2016-07-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1569172, encodeId=5c2415691e2ca, content=<a href='/topic/show?id=13288188450' target=_blank style='color:#2F92EE;'>#肥大细胞#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=42, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=81884, encryptionId=13288188450, topicName=肥大细胞)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=c92315497125, createdName=gds2014, createdTime=Sun Jul 17 01:23:00 CST 2016, time=2016-07-17, status=1, ipAttribution=)]
    2016-07-17 gds2014

    #肥大细胞#

    0

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