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JAMA：非甾体类抗炎药物减慢家族性淀粉样多发性神经病的进展

2013-12-30 sunight dxy

非甾体类家族性多发性神经病
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研究要点：1、家族性淀粉样多发性神经病往往会出现进行性神经缺损和残疾。2、非甾体类抗炎药物diflunisal减慢FAP进程。家族性淀粉样多发性神经病是一类以进行性神经缺损和残疾为特征的疾病，如果不能积极治疗，往往会有致命性后果。2013年12月25日JAMA报道，在这种具有致死性、遗传性神经变性病家族性淀粉样多发性神经病患者中，使用非甾体类抗炎药物diflunisal两年与使用安慰剂组患者相比，

研究要点：

1、家族性淀粉样多发性神经病往往会出现进行性神经缺损和残疾。

2、非甾体类抗炎药物diflunisal减慢FAP进程。

家族性淀粉样多发性神经病是一类以进行性神经缺损和残疾为特征的疾病，如果不能积极治疗，往往会有致命性后果。2013年12月25日JAMA报道，在这种具有致死性、遗传性神经变性病家族性淀粉样多发性神经病患者中，使用非甾体类抗炎药物diflunisal两年与使用安慰剂组患者相比，减少神经损害发生率，改善其生活质量。【原文下载】

根据该文献的研究背景，在世界范围内，有近1万例的患者估计有此临床表现。Diflunisal在1期临床试验中显示有一定疗效。来自波士顿大学医学院的John L. Berk及其同事随机纳入来自瑞典、日本、英格兰及美国的130例患者，随机分为diflunisal组和安慰剂组，每天两次服用两年，最后验证diflunisal对家族性淀粉样多发性神经病患者神经功能的影响。

与安慰剂组相比，使用diflunisal的患者显示神经受损的进展较安慰剂患者减慢。diflunisal抑制神经受损的效应在服用1年及2年时均有体现，diflunisal组中29.7%的患者，安慰剂组中9.4%的患者变现出神经功能稳定。diflunisal治疗的患者在开始服用至服用两年过程中与安慰剂相比均表现出生活质量的稳定。

文章作者提出该研究有几个重要的原因，首先该研究为此疾病的首次随机横断面研究，其提供了该疾病的神经功能受损的自然史；其次，其验证了diflunisal，一种低价治疗药物，能够被家族性淀粉样神经病患者很好耐受。尽管需要长期随访研究，这些发现显示该种治疗方式对家族性淀粉样神经病患者有效。

研究背景：

家族性淀粉样多发性神经病变(familial amyloid polyneuropathy)，在1952年由葡萄牙医师Andrade所发现，为一种罕见的体染色体显性遗传疾病，因为某些蛋白的基因突变，导致其结构出现变化，使得这些原本为水溶性的蛋白形成类淀粉纤维在细胞外沉积而致病。Diflunisal，主要成分是二氟尼柳，为水杨酸衍生物，属非甾体抗炎药，具有镇痛、抗炎及解热作用，其机理可能是抑制前列腺素合成。

原文出处：

Berk JL1, Suhr OB2, Obici L3, Sekijima Y4, Zeldenrust SR5, Yamashita T6, Heneghan MA7, Gorevic PD8, Litchy WJ5, Wiesman JF1, Nordh E2, Corato M9, Lozza A10, Cortese A10, Robinson-Papp J8, Colton T11, Rybin DV12, Bisbee AB12, Ando Y6, Ikeda S4, Seldin DC1, Merlini G3, Skinner M1, Kelly JW13, Dyck PJ5; Diflunisal Trial Consortium.Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.JAMA. 2013 Dec 25;310(24):2658-67. doi: 10.1001/jama.2013.283815.【原文下载】

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    2014-02-22 ms3892185887221420

    #多发性神经病#

    0

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    2014-09-02 jml2009

    #多发性#

    0

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    2014-01-01 zhouqu_8

    #非甾体类抗炎药#

    0

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    2014-01-01 xinmeili

    #抗炎药#

    0

  5. [GetPortalCommentsPageByObjectIdResponse(id=1709203, encodeId=7c2f1e092039d, content=<a href='/topic/show?id=dd2f43108e4' target=_blank style='color:#2F92EE;'>#多发性神经病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=43108, encryptionId=dd2f43108e4, topicName=多发性神经病)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=849831619609, createdName=ms3892185887221420, createdTime=Sat Feb 22 00:14:00 CST 2014, time=2014-02-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1889582, encodeId=f69d188958264, content=<a href='/topic/show?id=760c430895a' target=_blank style='color:#2F92EE;'>#多发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=62, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=43089, encryptionId=760c430895a, topicName=多发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04d6114, createdName=jml2009, createdTime=Tue Sep 02 04:14:00 CST 2014, time=2014-09-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1314366, encodeId=30bc13143661f, content=<a href='/topic/show?id=13669956408' target=_blank style='color:#2F92EE;'>#非甾体类抗炎药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=81, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=99564, encryptionId=13669956408, topicName=非甾体类抗炎药)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Wed Jan 01 03:14:00 CST 2014, time=2014-01-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1346709, encodeId=a3f71346e0978, content=<a href='/topic/show?id=f20155404fc' target=_blank style='color:#2F92EE;'>#抗炎药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=55404, encryptionId=f20155404fc, topicName=抗炎药)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=f49517, createdName=xinmeili, createdTime=Wed Jan 01 03:14:00 CST 2014, time=2014-01-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1491220, encodeId=809a149122026, content=<a href='/topic/show?id=5316e4387aa' target=_blank style='color:#2F92EE;'>#神经病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=74387, encryptionId=5316e4387aa, topicName=神经病)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=1ab48885684, createdName=axin012, createdTime=Wed Jan 01 03:14:00 CST 2014, time=2014-01-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1629462, encodeId=f2db1629462b2, content=<a href='/topic/show?id=c61d4641821' target=_blank style='color:#2F92EE;'>#家族性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=57, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=46418, encryptionId=c61d4641821, topicName=家族性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=26fc21339997, createdName=wetgdt, createdTime=Wed Jan 01 03:14:00 CST 2014, time=2014-01-01, status=1, ipAttribution=)]
    2014-01-01 axin012

    #神经病#

    0

  6. [GetPortalCommentsPageByObjectIdResponse(id=1709203, encodeId=7c2f1e092039d, content=<a href='/topic/show?id=dd2f43108e4' target=_blank style='color:#2F92EE;'>#多发性神经病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=66, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=43108, encryptionId=dd2f43108e4, topicName=多发性神经病)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=849831619609, createdName=ms3892185887221420, createdTime=Sat Feb 22 00:14:00 CST 2014, time=2014-02-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1889582, encodeId=f69d188958264, content=<a href='/topic/show?id=760c430895a' target=_blank style='color:#2F92EE;'>#多发性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=62, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=43089, encryptionId=760c430895a, topicName=多发性)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=04d6114, createdName=jml2009, createdTime=Tue Sep 02 04:14:00 CST 2014, time=2014-09-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1314366, encodeId=30bc13143661f, content=<a href='/topic/show?id=13669956408' target=_blank style='color:#2F92EE;'>#非甾体类抗炎药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=81, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=99564, encryptionId=13669956408, topicName=非甾体类抗炎药)], attachment=null, authenticateStatus=null, createdAvatar=https://wx.qlogo.cn/mmopen/aLGWoFXAyMbIu3qymFOyheQLjPSX3OUs5GmkyBlcCOwTPIeq3why9NGibxxUqYo6hcx8qZLHZFgNPnBK1yzWeOFpyg2OnWOt0/0, createdBy=fa4716, createdName=zhouqu_8, createdTime=Wed Jan 01 03:14:00 CST 2014, time=2014-01-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1346709, encodeId=a3f71346e0978, content=<a href='/topic/show?id=f20155404fc' target=_blank style='color:#2F92EE;'>#抗炎药#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=55404, encryptionId=f20155404fc, topicName=抗炎药)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=f49517, createdName=xinmeili, createdTime=Wed Jan 01 03:14:00 CST 2014, time=2014-01-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1491220, encodeId=809a149122026, content=<a href='/topic/show?id=5316e4387aa' target=_blank style='color:#2F92EE;'>#神经病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=63, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=74387, encryptionId=5316e4387aa, topicName=神经病)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=1ab48885684, createdName=axin012, createdTime=Wed Jan 01 03:14:00 CST 2014, time=2014-01-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1629462, encodeId=f2db1629462b2, content=<a href='/topic/show?id=c61d4641821' target=_blank style='color:#2F92EE;'>#家族性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=57, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=46418, encryptionId=c61d4641821, topicName=家族性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=26fc21339997, createdName=wetgdt, createdTime=Wed Jan 01 03:14:00 CST 2014, time=2014-01-01, status=1, ipAttribution=)]
    2014-01-01 wetgdt

    #家族性#

    0

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