Blood:BTK抑制剂泽布替尼治疗华氏巨球蛋白血症的疗效

2020-08-04 MedSci原创 MedSci原创

华氏巨球蛋白血症(WM)是一种罕见的非霍奇金淋巴瘤,淋巴浆细胞淋巴瘤。泽布替尼是中国百济神州公司自主研发的一种选择性BTK抑制剂,旨在评估泽布替尼用于WM的疗效。

华氏巨球蛋白血症(Waldenström's macroglobulinemia,WM),一种罕见的非霍奇金淋巴瘤,淋巴浆细胞淋巴瘤。由一名瑞典医生Jan G. Waldenström(1906-1996)发现,患者表现为口鼻出血、贫血、血液中纤维蛋白原水平降低、淋巴结肿大,骨髓中存在大量增殖的浆细胞,并且由于大量增加的巨球蛋白导致血液粘稠。

(血液粘稠难以回流,轻压手指就会变白,长时间不能恢复)

Bruton酪氨酸激酶(BTK)抑制剂在华氏巨球蛋白血症中展现出治疗活性。泽布替尼(Zanubrutinib)是中国百济神州公司自主研发的一种选择性BTK抑制剂,于2019年11月获得美国FDA批准用于治疗既往治疗过的套细胞淋巴瘤(MCL)患者。

Trotman等开展了一项1/2期的临床试验,旨在评估泽布替尼用于初治(TN)或复发性/难治性(R/R)WM的疗效。

两种泽布替尼口服方案:160 mg 2/日(n=50)或320 mg 1/日(n=23)。疗效终点包括总体缓解率(ORR)和非常好的部分缓解/完全缓解(VGPR/CR)。

2014年9月-2018年3月,共77位患者(24例TN和53例R/R)接受治疗。R/R患者和TN患者分别中位随访了36个月和23.5个月后,72.7%的患者仍在接受治疗。

终止治疗的原因有不良反应(13%,1例治疗相关)、疾病进展(10.4%)和其他(3.9%)。ORR和VGPR/CR率分别是95.9%和45.2%,VGPR/CR率还随着时间的推移而增加:6个月时 20.5%、12个月时 32.9%和24个月时 43.8%。

预计3年无进展生存率为80.5%,总体生存率为84.8%。不良反应由挫伤(32.5%,全为1级)、中性粒细胞减少(18.2%)、大出血(3.9%)、房颤/房扑(5.2%)和3级腹泻(2.6%)。
总结:部分WM患者长期采用泽布替尼单药治疗可获得较深的持久缓解。泽布替尼长期疗法用于这类患者的安全性可接受。

原始出处:

Judith Trotman,et al. Zanubrutinib for the treatment of patients with Waldenström macroglobulinemia: three years of follow-up. Blood. July 22,2020.

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    2020-08-08 xzw113
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    2021-01-23 jklm09
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    2020-08-06 fengting7
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    2020-08-05 ms3000000449926787

    学习了,很赞!

    0

  6. [GetPortalCommentsPageByObjectIdResponse(id=2035231, encodeId=d30b203523179, 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=35, 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=Sat Aug 08 23:56:32 CST 2020, time=2020-08-08, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1852454, encodeId=a3d0185245430, content=<a href='/topic/show?id=f0a254918f0' target=_blank style='color:#2F92EE;'>#抑制剂#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=26, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=54918, encryptionId=f0a254918f0, topicName=抑制剂)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=44a360, createdName=jklm09, createdTime=Sat Jan 23 21:56:32 CST 2021, time=2021-01-23, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1297397, encodeId=8e80129e39728, content=<a href='/topic/show?id=d6b83e724b' target=_blank style='color:#2F92EE;'>#BTK抑制剂#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=50, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3772, encryptionId=d6b83e724b, topicName=BTK抑制剂)], attachment=null, authenticateStatus=null, createdAvatar=, createdBy=1e72268, createdName=zhixianchen, createdTime=Thu Aug 06 08:56:32 CST 2020, time=2020-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1539117, encodeId=e808153911e9e, content=<a href='/topic/show?id=6bce3e7106' target=_blank style='color:#2F92EE;'>#BTK#</a>, beContent=null, objectType=article, channel=null, level=null, likeNumber=36, replyNumber=0, topicName=null, topicId=null, topicList=[TopicDto(id=3771, encryptionId=6bce3e7106, topicName=BTK)], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=61d813108447, createdName=fengting7, createdTime=Thu Aug 06 08:56:32 CST 2020, time=2020-08-06, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=806320, encodeId=0cb480632051, content=学习了,很赞!, beContent=null, objectType=article, channel=null, level=null, likeNumber=91, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=8f435296195, createdName=ms3000000449926787, createdTime=Wed Aug 05 15:16:13 CST 2020, time=2020-08-05, status=1, ipAttribution=), GetPortalCommentsPageByObjectIdResponse(id=1032601, encodeId=0069103260102, content=谢谢梅斯提供这么好的信息,学到很多, beContent=null, objectType=article, channel=null, level=null, likeNumber=0, replyNumber=0, topicName=null, topicId=null, topicList=[], attachment=null, authenticateStatus=null, createdAvatar=null, createdBy=f0620, createdName=内科新手, createdTime=Tue Aug 04 20:56:32 CST 2020, time=2020-08-04, status=1, ipAttribution=)]
    2020-08-04 内科新手

    谢谢梅斯提供这么好的信息,学到很多

    0

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