NEJM:sutimlimab治疗冷凝集素病效果喜人

2021-04-09 MedSci原创 MedSci原创

sutimlimab通过选择性地对经典补体途径上游靶点抑制可迅速改善冷凝集素病患者溶血症状,增加血红蛋白水平并减少疲劳

冷凝集素病(CAD)一种罕见的自身免疫性溶血性贫血,以典型补体通路激活引起的溶血为特征,临床表现为慢性贫血、严重疲劳和急性溶血性危机,部分患者会出现手足发绀,甚至血栓。C1复合物是激活免疫系统经典补体途径的第一步,而人源化单克隆抗体Sutimlimab可选择性靶向和抑制C1复合物中的丝氨酸蛋白酶C1s, 阻止C1激活溶血。

近日研究人员考察了静脉注射Sutimlimab对CAD患者的治疗效果。

本次研究是一项为期26周的多中心、开放性、单组研究,以评估静脉注射sutimlimab治疗冷凝集素病的疗效和安全性。研究的主要终点是血红蛋白水平正常化(每分升12g或更高)、血红蛋白水平较基线增加每分升2g或更高、无需红细胞输注的复合终点。

24名患者接受至少一剂Sutimlimab治疗,13名患者(54%)符合复合主要终点。在治疗评估时(第23、25和26周),患者血红蛋白水平最小二乘均值变化为每分升2.6克。从第3周到研究结束,患者平均血红蛋白水平均保持在每分升11克以上,平均胆红素水平在第3周恢复正常。从第5周到第26周,17名患者(71%)未接受输血从治疗开始后第1周,即可观察到有临床意义的疲劳缓解,并持续至研究结束。功能评估显示,Sutimlimab可快速抑制经典补体途径,表现为血红蛋白水平升高、胆红素水平降低和疲劳减轻。22名(92%)患者在治疗期间至少发生一次不良事件,7名患者(29%)至少发生过一次严重不良事件,未确定不良事件与sutimlimab有关,未发生脑膜炎球菌性感染

Sutimlimab治疗后CAD患者血红蛋白水平变化

研究认为,sutimlimab通过选择性地对经典补体途径上游靶点抑制可迅速改善冷凝集素病患者溶血症状,增加血红蛋白水平并减少疲劳

原始出处:

Alexander Roth et al. Sutimlimab in Cold Agglutinin Disease. N Engl J Med, April 8, 2021.

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    2021-10-04 snf701207
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  5. [GetPortalCommentsPageByObjectIdResponse(id=1995787, encodeId=c5ef1995e87fa, content=<a href='/topic/show?id=31a61693e61' target=_blank style='color:#2F92EE;'>#sutimlimab#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=33, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16937, encryptionId=31a61693e61, topicName=sutimlimab)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=84fb152, createdName=xiongliangxl, createdTime=Sun May 30 15:37:35 CST 2021, time=2021-05-30, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1912583, encodeId=b33119125834e, content=<a href='/topic/show?id=4de7112046c' target=_blank style='color:#2F92EE;'>#mAb#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=46, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=11204, encryptionId=4de7112046c, topicName=mAb)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=25e7304, createdName=snf701207, createdTime=Mon Oct 04 04:37:35 CST 2021, time=2021-10-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1511282, encodeId=9432151128262, content=<a href='/topic/show?id=0b633113ee1' target=_blank style='color:#2F92EE;'>#冷凝集#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=31137, encryptionId=0b633113ee1, topicName=冷凝集)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=e72710318017, createdName=1249842em12(暂无昵称), createdTime=Sun Apr 11 10:37:35 CST 2021, time=2021-04-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1604919, encodeId=d8241604919f8, content=<a href='/topic/show?id=4d303113965' target=_blank style='color:#2F92EE;'>#冷凝集素病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=31139, encryptionId=4d303113965, topicName=冷凝集素病)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f92519296302, createdName=ms2999106592497887, createdTime=Sun Apr 11 10:37:35 CST 2021, time=2021-04-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=955643, encodeId=887b9556430d, content=学习了, beContent=null, objectType=article, channel=null, level=null, likeNumber=55, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20210410/7c0b976873854f29b81ead6925ffb660/60e27a9873264bada93f1ff647ccbaee.jpg, createdBy=c7995478790, createdName=我是猴子搬来的救兵, createdTime=Sat Apr 10 09:42:01 CST 2021, time=2021-04-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1035622, encodeId=a2a61035622c5, content=顶刊就是顶刊,谢谢梅斯带来这么高水平的研究报道,我们科里同事经常看梅斯,分享梅斯上的信息, beContent=null, objectType=article, channel=null, level=null, likeNumber=47, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=旺医, createdTime=Fri Apr 09 22:37:35 CST 2021, time=2021-04-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1047318, encodeId=9689104e318eb, content=疗效只是效果的众多方面之一,还要看对患者的获益,包括生活质量等因素共同决定效果的, beContent=null, objectType=article, channel=null, level=null, likeNumber=47, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=CHANGE, createdTime=Fri Apr 09 22:37:35 CST 2021, time=2021-04-09, status=1, ipAttribution=)]
    2021-04-10 我是猴子搬来的救兵

    学习了

    0

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    2021-04-09 旺医

    顶刊就是顶刊,谢谢梅斯带来这么高水平的研究报道,我们科里同事经常看梅斯,分享梅斯上的信息

    0

  7. [GetPortalCommentsPageByObjectIdResponse(id=1995787, encodeId=c5ef1995e87fa, content=<a href='/topic/show?id=31a61693e61' target=_blank style='color:#2F92EE;'>#sutimlimab#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=33, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=16937, encryptionId=31a61693e61, topicName=sutimlimab)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=84fb152, createdName=xiongliangxl, createdTime=Sun May 30 15:37:35 CST 2021, time=2021-05-30, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1912583, encodeId=b33119125834e, content=<a href='/topic/show?id=4de7112046c' target=_blank style='color:#2F92EE;'>#mAb#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=46, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=11204, encryptionId=4de7112046c, topicName=mAb)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=25e7304, createdName=snf701207, createdTime=Mon Oct 04 04:37:35 CST 2021, time=2021-10-04, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1511282, encodeId=9432151128262, content=<a href='/topic/show?id=0b633113ee1' target=_blank style='color:#2F92EE;'>#冷凝集#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=31137, encryptionId=0b633113ee1, topicName=冷凝集)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=e72710318017, createdName=1249842em12(暂无昵称), createdTime=Sun Apr 11 10:37:35 CST 2021, time=2021-04-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1604919, encodeId=d8241604919f8, content=<a href='/topic/show?id=4d303113965' target=_blank style='color:#2F92EE;'>#冷凝集素病#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=45, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=31139, encryptionId=4d303113965, topicName=冷凝集素病)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f92519296302, createdName=ms2999106592497887, createdTime=Sun Apr 11 10:37:35 CST 2021, time=2021-04-11, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=955643, encodeId=887b9556430d, content=学习了, beContent=null, objectType=article, channel=null, level=null, likeNumber=55, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20210410/7c0b976873854f29b81ead6925ffb660/60e27a9873264bada93f1ff647ccbaee.jpg, createdBy=c7995478790, createdName=我是猴子搬来的救兵, createdTime=Sat Apr 10 09:42:01 CST 2021, time=2021-04-10, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1035622, encodeId=a2a61035622c5, content=顶刊就是顶刊,谢谢梅斯带来这么高水平的研究报道,我们科里同事经常看梅斯,分享梅斯上的信息, beContent=null, objectType=article, channel=null, level=null, likeNumber=47, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=旺医, createdTime=Fri Apr 09 22:37:35 CST 2021, time=2021-04-09, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1047318, encodeId=9689104e318eb, content=疗效只是效果的众多方面之一,还要看对患者的获益,包括生活质量等因素共同决定效果的, beContent=null, objectType=article, channel=null, level=null, likeNumber=47, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=CHANGE, createdTime=Fri Apr 09 22:37:35 CST 2021, time=2021-04-09, status=1, ipAttribution=)]
    2021-04-09 CHANGE

    疗效只是效果的众多方面之一,还要看对患者的获益,包括生活质量等因素共同决定效果的

    0

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血浆ADAMTS13活性重度缺陷是血栓性血小板减少性紫癜(TTP)的主要原因,而补体通过替代途径过度激活又导致了非典型溶血性尿毒症综合征(aHUS),TTP和aHUS是血栓性微血管病的两种原型。但从临床和致病机制上区别TTP和aHUS非常具有挑战性。临床报道表明补体激活可能在血浆ADAMTS13活性重度缺陷所引起的TTP发展过程中发挥作用。但目前尚无确凿的实验证据。近日,《血液》杂志上发表一篇文章

NATURE:在各种疾病中,补体基因造成了"男女有别"  

许多常见的疾病,对男性和女性的影响是不同的,但是其背后的原因至今未知。