Blood:促进红细胞生成联合铁限制可明显改善β-地中海贫血的贫血

2020-06-19 医刀 MedSci原创

增加促红细胞生成素的敏感性或活性可改善β-地中海贫血的促红细胞生成;红系刺激剂与红系铁限制剂联合使用可纠正β-地中海贫血的贫血。

中度地中海贫血是一种以红细胞生成无效(IE)、贫血、脾肿大和全身性铁超负荷为特征的疾病。研究人员正在探索新的治疗方法,基于减少铁吸收,如使用铁调素激活剂:Tmprss6-反义寡核苷酸(ASO)等),或增加红细胞生成,如促红细胞生产素(EPO)或调节转铁蛋白受体2(Tfr2)控制RBC合成的能力。

用Tmprss6-ASO靶向Tmprss6 mRNA被证明可提高诱导铁限制有效地提高IE和脾肿大。但Casu等人推测联合疗法的疗效可能优于单一疗法。因此,Casu等人用Tmprss6-ASO联合EPO或敲除了受β-地中海贫血中间媒介影响的动物 (Hbbth3/+)骨髓中的单个Tfr2等位基因,对其推测进行研究。

单用EPO或敲除单个Tfr2等位基因可增加血红蛋白水平和RBC。但单用EPO或Tfr2单等位基因缺失分别加剧了或没有改善β-地中海贫血小鼠的脾肿大。

为克服这一状况,研究人员推测一定水平的铁限制(通过靶向Tmprss6)将改善脾肿大,同时保留由EPO或Tfr2缺失介导的对RBC产生的有益作用。虽然单用Tmprss6-ASO也可以改善贫血,但Tmprss6-ASO + EPO或Tmprss6-ASO + Tfr2单等位基因缺失处理后的血红蛋白水平显着升高,而且脾肿大明显缩小。

总而言之,本研究结果清楚地提示这些联合处理(Tmprss6-ASO + EPO或Tmprss6-ASO + Tfr2单等位基因缺失)在缓解β-地中海贫血的IE和贫血方面优于单一处理,为将这些联合方法转化到临床实际应用提供了指导。

原始出处:

Carla Casu,et al. Correcting β-thalassemia by combined therapies that restrict iron and modulate erythropoietin activity. Blood. June 17,2020.

 

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    2021-03-27 ms5000000426001883

    很有意思的研究

    0

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    2020-11-02 lovetcm

    #EPO#联合#铁#限制可明显改善#β-地中海贫血#的贫血,那么不用EPO,用#罗沙司他#呢?

    0

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  5. [GetPortalCommentsPageByObjectIdResponse(id=951693, encodeId=321b951693aa, content=很有意思的研究, beContent=null, objectType=article, channel=null, level=null, likeNumber=59, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20201016/e69ebf6de4ff4c58975eca21defba7f4/998f896c1f9b4f698397a269727651c6.jpg, createdBy=7b2b5391720, createdName=ms5000000426001883, createdTime=Sat Mar 27 20:47:40 CST 2021, time=2021-03-27, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=896312, encodeId=d750896312fa, content=<a href='/topic/show?id=7f086891ce' target=_blank style='color:#2F92EE;'>#EPO#</a>联合<a href='/topic/show?id=6521965286d' target=_blank style='color:#2F92EE;'>#铁#</a>限制可明显改善<a href='/topic/show?id=0a5a1918980' target=_blank style='color:#2F92EE;'>#β-地中海贫血#</a>的贫血,那么不用EPO,用<a href='/topic/show?id=d717e96216e' target=_blank style='color:#2F92EE;'>#罗沙司他#</a>呢?, beContent=null, objectType=article, channel=null, level=null, likeNumber=152, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=6891, encryptionId=7f086891ce, topicName=EPO), TopicDto(id=96528, encryptionId=6521965286d, topicName=铁), TopicDto(id=19189, encryptionId=0a5a1918980, topicName=β-地中海贫血), TopicDto(id=79621, encryptionId=d717e96216e, topicName=罗沙司他)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Mon Nov 02 16:50:00 CST 2020, time=2020-11-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1282072, encodeId=da9512820e26c, content=<a href='/topic/show?id=3dece71531e' target=_blank style='color:#2F92EE;'>#红细胞#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=77153, encryptionId=3dece71531e, topicName=红细胞)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c22f171, createdName=kord1982, createdTime=Sun Jun 21 10:13:47 CST 2020, time=2020-06-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1419967, encodeId=99c5141996e6c, content=<a href='/topic/show?id=c7d9e716941' target=_blank style='color:#2F92EE;'>#红细胞生成#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=77169, encryptionId=c7d9e716941, topicName=红细胞生成)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=489d3549369, createdName=许安, createdTime=Sun Jun 21 10:13:47 CST 2020, time=2020-06-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1575521, encodeId=125115e552134, content=<a href='/topic/show?id=cd9941326bc' target=_blank style='color:#2F92EE;'>#地中海#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=41326, encryptionId=cd9941326bc, topicName=地中海)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=303116130321, createdName=wgx311, createdTime=Sun Jun 21 10:13:47 CST 2020, time=2020-06-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=799184, encodeId=d712e99184b0, content=本研究结果清楚地提示这些联合处理(Tmprss6-ASO + EPO或Tmprss6-ASO + Tfr2单等位基因缺失)在缓解β-地中海贫血的IE和贫血方面优于单一处理,, beContent=null, objectType=article, channel=null, level=null, likeNumber=98, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200529/3611e9bb629c4e5b81fbd8ce784b06cc/128487eae357460bb98f67d86230c218.jpg, createdBy=ab235268530, createdName=神盾医疗局局长Jack, createdTime=Fri Jun 19 20:18:35 CST 2020, time=2020-06-19, status=1, ipAttribution=)]
    2020-06-21 wgx311
  6. [GetPortalCommentsPageByObjectIdResponse(id=951693, encodeId=321b951693aa, content=很有意思的研究, beContent=null, objectType=article, channel=null, level=null, likeNumber=59, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20201016/e69ebf6de4ff4c58975eca21defba7f4/998f896c1f9b4f698397a269727651c6.jpg, createdBy=7b2b5391720, createdName=ms5000000426001883, createdTime=Sat Mar 27 20:47:40 CST 2021, time=2021-03-27, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=896312, encodeId=d750896312fa, content=<a href='/topic/show?id=7f086891ce' target=_blank style='color:#2F92EE;'>#EPO#</a>联合<a href='/topic/show?id=6521965286d' target=_blank style='color:#2F92EE;'>#铁#</a>限制可明显改善<a href='/topic/show?id=0a5a1918980' target=_blank style='color:#2F92EE;'>#β-地中海贫血#</a>的贫血,那么不用EPO,用<a href='/topic/show?id=d717e96216e' target=_blank style='color:#2F92EE;'>#罗沙司他#</a>呢?, beContent=null, objectType=article, channel=null, level=null, likeNumber=152, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=6891, encryptionId=7f086891ce, topicName=EPO), TopicDto(id=96528, encryptionId=6521965286d, topicName=铁), TopicDto(id=19189, encryptionId=0a5a1918980, topicName=β-地中海贫血), TopicDto(id=79621, encryptionId=d717e96216e, topicName=罗沙司他)], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20220519/c2ab253484ee4527a2d4e9589a4821ac/45de9bf494a54becb2ea4369c9d11e85.jpg, createdBy=7a3710, createdName=lovetcm, createdTime=Mon Nov 02 16:50:00 CST 2020, time=2020-11-02, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1282072, encodeId=da9512820e26c, content=<a href='/topic/show?id=3dece71531e' target=_blank style='color:#2F92EE;'>#红细胞#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=77153, encryptionId=3dece71531e, topicName=红细胞)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=c22f171, createdName=kord1982, createdTime=Sun Jun 21 10:13:47 CST 2020, time=2020-06-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1419967, encodeId=99c5141996e6c, content=<a href='/topic/show?id=c7d9e716941' target=_blank style='color:#2F92EE;'>#红细胞生成#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=31, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=77169, encryptionId=c7d9e716941, topicName=红细胞生成)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=489d3549369, createdName=许安, createdTime=Sun Jun 21 10:13:47 CST 2020, time=2020-06-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1575521, encodeId=125115e552134, content=<a href='/topic/show?id=cd9941326bc' target=_blank style='color:#2F92EE;'>#地中海#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=28, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=41326, encryptionId=cd9941326bc, topicName=地中海)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=303116130321, createdName=wgx311, createdTime=Sun Jun 21 10:13:47 CST 2020, time=2020-06-21, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=799184, encodeId=d712e99184b0, content=本研究结果清楚地提示这些联合处理(Tmprss6-ASO + EPO或Tmprss6-ASO + Tfr2单等位基因缺失)在缓解β-地中海贫血的IE和贫血方面优于单一处理,, beContent=null, objectType=article, channel=null, level=null, likeNumber=98, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=https://img.medsci.cn/20200529/3611e9bb629c4e5b81fbd8ce784b06cc/128487eae357460bb98f67d86230c218.jpg, createdBy=ab235268530, createdName=神盾医疗局局长Jack, createdTime=Fri Jun 19 20:18:35 CST 2020, time=2020-06-19, status=1, ipAttribution=)]
    2020-06-19 神盾医疗局局长Jack

    本研究结果清楚地提示这些联合处理(Tmprss6-ASO + EPO或Tmprss6-ASO + Tfr2单等位基因缺失)在缓解β-地中海贫血的IE和贫血方面优于单一处理,

    0

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