Blood:用浆细胞靶向疗法治疗单克隆性丙种球蛋白相关的硬化性粘液水肿

2020-02-09 QQY MedSci原创

中心点:HDIVig似乎可有效治疗非重症硬化性黏液水肿。需维持治疗以避免复发。靶向分泌MG的浆细胞克隆的化疗似乎可治疗这种难治性的重症病例。摘要:硬化性黏液水肿是一种罕见全身性皮肤粘蛋白增多症,常与单克隆性丙种球蛋白病(MG)有关。Mahévas等研究人员在法国开展了一项多中心的回顾性研究,共招募了33位患者,评估MG相关性硬化性黏液水肿的临床和治疗特征。采用转录组学方法分析皮肤分子特征。皮肤症状

中心点:

HDIVig似乎可有效治疗非重症硬化性黏液水肿。需维持治疗以避免复发。

靶向分泌MG的浆细胞克隆的化疗似乎可治疗这种难治性的重症病例。

摘要:

硬化性黏液水肿是一种罕见全身性皮肤粘蛋白增多症,常与单克隆性丙种球蛋白病(MG)有关。Mahévas等研究人员在法国开展了一项多中心的回顾性研究,共招募了33位患者,评估MG相关性硬化性黏液水肿的临床和治疗特征。

采用转录组学方法分析皮肤分子特征。皮肤症状包括丘疹(100%)、硬皮病样(91%)和狮面(39%)。MG涉及所有患者的IgG亚型,主要是λ轻链(73%)。4/33例患者被诊断为相关血液恶性肿瘤(12%;2例阴燃骨髓瘤、1例慢性淋巴细胞白血病和1例伴多系发育不良的难治性血细胞减少)。腕管综合征(33%)、关节痛(25%)和皮肤神经综合征(18%)是最常见的全身并发症。

1位粘液性心脏病患者死于急性心力衰竭。单纯静脉注射免疫球蛋白(HDIVig)或联合类固醇治疗似乎对非重症患者很有效(13/31例患者获得临床完全缓解)。重症病例(HDIVig难治性或有中枢神经系统或心脏受累的病例)采用来那度胺和(或)硼替佐米联合地塞米松和HDIVig可获得明星改善。

4/5位患者采用血浆置换、HDIVig和高剂量皮质类固醇紧急处理DNS时,神经症状完全缓解。采用定量反向转录-PCR (RT-PCR)对6例硬化性黏液水肿皮肤样品进行分析发现,硬化性黏液水肿皮肤的促纤维化通路水平(转化生长因子β[TGFβ]和胶原蛋白-1)明显高于健康皮肤。

原始出处:

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    2020-09-17 ms4000000956890434

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createdName=ms4000000956890434, createdTime=Thu Sep 17 12:36:58 CST 2020, time=2020-09-17, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2083536, encodeId=8d1f20835361a, content=<a href='/topic/show?id=dedfe359034' target=_blank style='color:#2F92EE;'>#硬化性粘液水肿#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=24, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=73590, encryptionId=dedfe359034, topicName=硬化性粘液水肿)], attachment=null, authenticateStatus=null, createdAvatar=http://thirdwx.qlogo.cn/mmopen/vi_32/Q0j4TwGTfTKQBvJI9WesEs5nl29JIJ7mAquShibzBHQmTeHHwZMDlIzc5qoQUhTJOpOwiaxYa9UcibrZviaM7dL7aQ/132, createdBy=35cb2500499, createdName=ms1343860338762272, createdTime=Mon Dec 07 06:48:00 CST 2020, time=2020-12-07, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=2035210, encodeId=4fac203521073, content=<a href='/topic/show?id=5a426866eae' target=_blank style='color:#2F92EE;'>#球蛋白#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=68667, encryptionId=5a426866eae, topicName=球蛋白)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=2317301, createdName=xzw113, createdTime=Fri May 01 06:48:00 CST 2020, time=2020-05-01, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1660212, encodeId=054b16602122e, content=<a href='/topic/show?id=8d0d28912db' target=_blank style='color:#2F92EE;'>#克隆#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=16, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=28912, encryptionId=8d0d28912db, topicName=克隆)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=596825099872, createdName=aids221, createdTime=Thu Jul 09 15:48:00 CST 2020, time=2020-07-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1333453, 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attachment=null, authenticateStatus=null, createdAvatar=, createdBy=b676418, createdName=yanghan, createdTime=Tue Feb 11 13:48:00 CST 2020, time=2020-02-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1466633, encodeId=839f14666335c, content=<a href='/topic/show?id=8d3e65434d4' target=_blank style='color:#2F92EE;'>#浆细胞#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=65434, encryptionId=8d3e65434d4, topicName=浆细胞)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=181c6846356, createdName=huangshifeng, createdTime=Tue Feb 11 13:48:00 CST 2020, time=2020-02-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1496365, encodeId=083d14963651c, content=<a href='/topic/show?id=43b6e358710' target=_blank style='color:#2F92EE;'>#硬化性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=17, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=73587, encryptionId=43b6e358710, topicName=硬化性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d8119265602, createdName=Smile2683, createdTime=Tue Feb 11 13:48:00 CST 2020, time=2020-02-11, status=1, ipAttribution=)]
    2020-05-01 xzw113
  5. [GetPortalCommentsPageByObjectIdResponse(id=1689283, encodeId=e4e41689283ce, content=<a href='/topic/show?id=cffa20922a2' target=_blank style='color:#2F92EE;'>#丙种球蛋白#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=20, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=20922, encryptionId=cffa20922a2, topicName=丙种球蛋白)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=04a428781144, createdName=ms1490123962508630, createdTime=Fri Jan 22 10:48:00 CST 2021, time=2021-01-22, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=886008, encodeId=cbbb88600895, content=👌, beContent=null, objectType=article, channel=null, level=null, likeNumber=60, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200918/133985b200ad44c386bfd27aa18100b5/cbb14ae7b1304dd59e34783b3c9b826d.jpg, createdBy=3dd05414640, 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attachment=null, authenticateStatus=null, createdAvatar=, createdBy=b676418, createdName=yanghan, createdTime=Tue Feb 11 13:48:00 CST 2020, time=2020-02-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1466633, encodeId=839f14666335c, content=<a href='/topic/show?id=8d3e65434d4' target=_blank style='color:#2F92EE;'>#浆细胞#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=65434, encryptionId=8d3e65434d4, topicName=浆细胞)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=181c6846356, createdName=huangshifeng, createdTime=Tue Feb 11 13:48:00 CST 2020, time=2020-02-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1496365, encodeId=083d14963651c, content=<a href='/topic/show?id=43b6e358710' target=_blank style='color:#2F92EE;'>#硬化性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=17, 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    2020-07-09 aids221
  6. 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attachment=null, authenticateStatus=null, createdAvatar=, createdBy=b676418, createdName=yanghan, createdTime=Tue Feb 11 13:48:00 CST 2020, time=2020-02-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1466633, encodeId=839f14666335c, content=<a href='/topic/show?id=8d3e65434d4' target=_blank style='color:#2F92EE;'>#浆细胞#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=29, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=65434, encryptionId=8d3e65434d4, topicName=浆细胞)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=181c6846356, createdName=huangshifeng, createdTime=Tue Feb 11 13:48:00 CST 2020, time=2020-02-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1496365, encodeId=083d14963651c, content=<a href='/topic/show?id=43b6e358710' target=_blank style='color:#2F92EE;'>#硬化性#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=17, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=73587, encryptionId=43b6e358710, topicName=硬化性)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=d8119265602, createdName=Smile2683, createdTime=Tue Feb 11 13:48:00 CST 2020, time=2020-02-11, status=1, ipAttribution=)]
    2020-02-11 小华子
  7. 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