{"id":144278,"date":"2022-09-02T01:33:59","date_gmt":"2022-09-02T01:33:59","guid":{"rendered":"https:\/\/labinsights.com\/acf_article\/%e6%96%b0%e8%88%88%e7%9a%84%e8%a1%80%e5%8f%8b%e7%97%85%e7%99%82%e6%b3%95%e5%87%b8%e9%a1%af%e4%ba%86%e5%b0%8d%e5%93%81%e8%b3%aa%e8%a8%ba%e6%96%b7%e7%9a%84%e9%9c%80%e6%b1%82\/"},"modified":"2026-02-03T07:22:29","modified_gmt":"2026-02-03T07:22:29","slug":"emerging-haemophilia-therapies-highlight-a-need-for-quality-diagnostics","status":"publish","type":"acf_article","link":"https:\/\/labinsights.com\/zhtw\/disease-area\/rare-diseases\/emerging-haemophilia-therapies-highlight-a-need-for-quality-diagnostics\/","title":{"rendered":"\u65b0\u8208\u7684\u8840\u53cb\u75c5\u7642\u6cd5\u51f8\u986f\u4e86\u5c0d\u54c1\u8cea\u8a3a\u65b7\u7684\u9700\u6c42"},"content":{"rendered":"

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\u8840\u53cb\u75c5\u7684\u4e00\u4e9b\u65b0\u8208\u7642\u6cd5\u662f\u4ec0\u9ebc\uff1f<\/h2>\n

\u4eca\u5929\uff0c\u8840\u53cb\u75c5\u7684\u6cbb\u7642\u5074\u91cd\u65bc\u66ff\u4ee3\u7642\u6cd5\uff0c\u8a72\u904e\u7a0b\u6d89\u53ca\u4f7f\u7528\u51dd\u56fa\u56e0\u5b50\u4f86\u6fc3\u7e2e\u4ee5\u88dc\u5145\u60a3\u8005\u7684\u8840\u6db2\u3002\u9019\u4e9b\u6cbb\u7642\u53ef\u80fd\u6703\u5c0e\u81f4\u4f75\u767c\u75c7\uff0c\u4f8b\u5982\u7522\u751f\u653b\u64ca\u8840\u6db2\u4e2d\u51dd\u8840\u56e0\u5b50\u4e26\u5c0e\u81f4\u8840\u53cb\u75c5\u767c\u751f\u7387\u5347\u9ad8\u7684\u4e2d\u548c\u6297\u9ad4\uff08\u963b\u65b7\u5291\uff09 [1]\u3002<\/p>\n

\u70ba\u4e86\u89e3\u6c7a\u9019\u4e9b\u554f\u984c\u53ca\u5176\u4ed6\u554f\u984c\uff0c\u5065\u5eb7\u7167\u8b77\u5c08\u696d\u4eba\u54e1\u6b63\u5728\u63a2\u7d22\u591a\u7a2e\u8840\u53cb\u75c5\u7642\u6cd5\u7684\u65b0\u65b9\u6cd5\uff0c\u5305\u62ec\u5ef6\u9577\u534a\u8870\u671f(EHL)\u51dd\u56fa\u56e0\u5b50\uff1b\u9776\u5411\u7d44\u7e54\u56e0\u5b50\u9014\u5f91\u963b\u65b7\u5291(TFPI)\u7684\u55ae\u682a\u6297\u9ad4\uff1b\u4f5c\u70ba\u975e\u56e0\u5b50\u66ff\u4ee3\u7642\u6cd5\u7684\u96d9\u7279\u7570\u6027\u6297\u9ad4\uff1b\u4ee5\u53ca\u6709\u671b\u6cbb\u7652\u8a72\u75c5\u75c7\u7684\u57fa\u56e0\u7642\u6cd5\u3002<\/p>\n

EHL\u51dd\u8840<\/strong><\/p>\n

\u56e0\u5b50\u81ea2010\u5e74\u4ee3\u8d77\uff0c\u91cd\u7d44\u6280\u8853\u5df2\u88ab\u5f15\u5165\u4ee5\u6539\u9020\u56e0\u5b50\uff0c\u4ee5\u5ef6\u9577\u5176\u5728\u5faa\u74b0\u4e2d\u7684\u6642\u9593\uff0c\u964d\u4f4e\u7d66\u85e5\u983b\u7387[2]\u3002\u76ee\u524d\uff0cEHL FVIII\u53caFXI\u7522\u54c1\u53ef\u4f9b\u4f7f\u7528\u3002\u5404\u7a2eEHL\u7522\u54c1\u7684\u85e5\u7269\u52d5\u529b\u5b78\u53ca\u529f\u6548\u5f97\u5230\u5145\u5206\u8b49\u660e\uff0c\u7279\u5225\u662f\u5728\u51fa\u8840\u7684\u9810\u9632\u548c\u6cbb\u7642\u4e2d\u3002<\/p>\n

\u9776\u5411\u7d44\u7e54\u56e0\u5b50\u9014\u5f91\u963b\u65b7\u5291(TFPI)\u7684\u55ae\u682a\u6297\u9ad4<\/strong><\/p>\n

\u9776\u5411TFPI\u963b\u65b7\u5291\u7684\u55ae\u682a\u6297\u9ad4\u6b63\u5728\u9032\u884c\u591a\u9805\u7814\u7a76\uff0c\u9019\u4e9b\u55ae\u682a\u6297\u9ad4\u6709\u671b\u589e\u5f37\u6b62\u8840\u6d3b\u6027\u3001\u66f4\u7a69\u5b9a\u7684\u51dd\u584a\u5f62\u6210\u4ee5\u53ca\u96a8\u5f8c\u7684\u51fa\u8840\u4e8b\u4ef6\u6e1b\u5c11\u3002\u4e00\u500b\u4f8b\u9805\u662f\u5eb7\u8cfd\u73e0\u55ae\u6297\uff0c\u9019\u662f\u4e00\u7a2e\u4eba\u55ae\u682a\u6297\u9ad4\uff0c\u6b63\u5728\u91dd\u5c0dA\u578b\u53caB\u578b\u8840\u53cb\u75c5\u60a3\u8005\u76842\u671f\u81e8\u5e8a\u8a66\u9a57\u4e2d\u9032\u884c\u8a55\u4f30\u3002\u00a0\u6b50\u76df\u3001\u82f1\u570b\u548c\u7f8e\u570b\u9810\u8a08\u65bc2023\/4\u5e74\u6279\u51c6\u5eb7\u8cfd\u73e0\u55ae\u6297\u7528\u65bc\u6cbb\u7642A\u578b\u8840\u53cb\u75c5\u6216B\u578b\u8840\u53cb\u75c5\u3002\u00a0 BAY1093884 \uff08\u53e6\u4e00\u7a2e\u985e\u4f3c\u65bcconcizumab\u7684\u55ae\u682a\u6297\u9ad4\uff09\u7684I\u671f\u81e8\u5e8a\u8a66\u9a57\u7684\u62db\u52df\u5de5\u4f5c\u4e5f\u5728\u9032\u884c\u4e2d\u3002<\/p>\n

\u4f5c\u70ba\u975e\u56e0\u5b50\u66ff\u4ee3<\/strong><\/p>\n

\u7642\u6cd5\u7684\u96d9\u7279\u7570\u6027\u6297\u9ad4\u5176\u4ed6\u65b0\u8208\u7642\u6cd5\u5305\u62ec\u975e\u56e0\u5b50\u66ff\u4ee3\u7522\u54c1\u3002\u9019\u4e9b\u901a\u5e38\u662f\u96d9\u7279\u7570\u6027\u6297\u9ad4\uff0c\u5982emicizumab\uff0c\u5176\u6a21\u64ec\u51dd\u8840\u56e0\u5b50\uff0c\u6709\u6548\u5730\u6a4b\u63a5FXIa\u53caFX\u3002\u00a0emicizumab\u9069\u7528\u65bc\u5177\u6709FVIII\u963b\u65b7\u5291\u7684\u6210\u4eba\u53ca\u5152\u79d1\u60a3\u8005\u7684\u5e38\u898f\u9810\u9632\u30022018\u5e744\u6708\u88ab\u6388\u4e88A\u578b\u8840\u53cb\u75c5\u7a81\u7834\u6027\u7642\u6cd5\u8a8d\u5b9a[3]\u3002<\/p>\n

\u6709\u671b\u6cbb\u7652\u8a72\u75c5<\/strong><\/p>\n

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\u9019\u5c0d\u5be6\u9a57\u5ba4\u8a3a\u65b7\u610f\u5473\u8457\u4ec0\u9ebc\uff1f<\/h2>\n

\u81e8\u5e8a\u5be6\u9a57\u5ba4\u8a3a\u65b7\u5728\u76e3\u6e2c\u4e0d\u540c\u8840\u53cb\u75c5\u7642\u6cd5\u7684\u6709\u6548\u6027\u65b9\u9762\u767c\u63ee\u8457\u91cd\u8981\u4f5c\u7528\u3002\u4f8b\u5982\uff0c\u7576\u6295\u4e88\u66ff\u4ee3\u7642\u6cd5\u6642\uff0c\u56e0\u5b50\u6fc3\u7e2e\u7269\u4e4b\u9078\u64c7\u81f3\u95dc\u91cd\u8981\uff0c\u56e0\u70ba\u5b83\u5f71\u97ff\u7528\u65bc\u6e2c\u91cf\u4e4b\u6e2c\u5b9a\u7684\u9078\u64c7\u3002<\/p>\n

\u96a8\u8457\u975e\u56e0\u5b50\u6cbb\u7642\u5291(\u8af8\u5982emicizumab)\u7684\u5f15\u5165\uff0c\u5be6\u9a57\u5ba4\u5c07\u9996\u5148\u9700\u8981\u77ad\u89e3\u6cbb\u7642\u4f5c\u7528\u6a21\u5f0f\u53ca\u4e0d\u540c\u578b\u5225\u6e2c\u5b9a\u7684\u751f\u5316\u5f71\u97ff\u3002\u4e4b\u5f8c\uff0c\u4ed6\u5011\u53ef\u4ee5\u9078\u64c7\u6700\u5408\u9069\u7684\u6e2c\u5b9a\u9032\u884c\u76e3\u6e2c\uff1a<\/p>\n

\u7121\u8ad6\u9078\u64c7\u4f55\u7a2e\u7642\u6cd5\uff0c\u4ecd\u53ef\u80fd\u7522\u751f\u963b\u65b7\u5291\u3002\u81e8\u5e8a\u5be6\u9a57\u5ba4\u9700\u8981\u76e3\u6e2c\u963b\u65b7\u5291\u7684\u767c\u5c55\uff0c\u56e0\u70ba\u5b83\u5011\u5f71\u97ff\u6cbb\u7642\u7684\u6709\u6548\u6027\u3002\u4ee5\u4e0b\u662f\u963b\u65b7\u5291\u6e2c\u8a66\u9700\u8981\u6ce8\u610f\u7684\u4e00\u4e9b\u8981\u9ede\u3002<\/p>\n

\u5118\u7ba1\u8840\u6f3f\u884d\u751f\u56e0\u5b50\u7522\u7269\u53ca\u91cd\u7d44\u56e0\u5b50\u7522\u7269\u5f62\u5f0f\u7684\u66ff\u4ee3\u7642\u6cd5\u4ecd\u7136\u662f\u7576\u4eca\u8840\u53cb\u75c5\u7684\u4e3b\u8981\u6cbb\u7642\u65b9\u6cd5\uff0c\u4f46\u5c0d\u65bc\u65b0\u85e5\u5291\u7684\u767c\u73fe\uff0c\u986f\u7136\u6b63\u5728\u9032\u884c\u6709\u5e0c\u671b\u7684\u7814\u7a76\u3002\u9019\u4e9b\u85e5\u5291\u4f7f\u7528\u4e0d\u540c\u7684\u5206\u5b50\u85e5\u7269\u6a19\u9776\u3001\u53c3\u8207\u51dd\u8840\u7d1a\u806f\u904e\u7a0b\u7684\u57fa\u56e0\u548c\u86cb\u767d\u8cea\u3002\u7d50\u679c\u5df2\u7522\u751f\u5408\u7406\u7684\u6a02\u89c0\u60c5\u7dd2\uff0c\u8a8d\u70ba\u5728\u4e0d\u4e45\u7684\u5c07\u4f86\u53ef\u80fd\u5b58\u5728\u6cbb\u7652\u8840\u53cb\u75c5\u7684\u9014\u5f91[6]\u3002<\/p>\n

\u53c3\u8003\u6587\u737b\uff1a<\/em><\/p>\n

[1]\u75be\u75c5\u7ba1\u5236\u8207\u9810\u9632\u4e2d\u5fc3\uff0c\u8840\u53cb\u75c5\u9996\u9801: \u6291\u5236\u7269\u8207\u8840\u53cb\u75c5 https:\/\/www.cdc.gov\/ncbddd\/hemophilia\/inhibitors.html<\/p>\n

[2]<\/a> Mannucci\uff0cP.\uff0c2020. \u8840\u53cb\u75c5\u7642\u6cd5\uff1a\u672a\u4f86\u5df2\u7d93\u958b\u59cb\u3002Haematologica\uff0c105(3)\uff0c\u7b2c545-553\u9801\u3002<\/p>\n

[3]<\/a> FDA\u6279\u51c6emicizumab-kxwh\u7528\u65bc\u9810\u9632\u548c\u6e1b\u5c11\u5177\u6709\u56e0\u5b50VIII\u6291\u5236\u7269\u7684A\u578b\u8840\u53cb\u75c5\u60a3\u8005\u7684\u51fa\u8840\u3002https:\/\/www.fda.gov\/drugs\/resources-information-approved-drugs\/fda-approves-emicizumab-kxwh-prevention-and-reduction-bleeding-patients-hemophilia-factor-viii<\/p>\n

[4]<\/a> CSL Behring\u57fa\u56e0\u7642\u6cd5\u5728\u66ab\u7de9\u5f8c\u91cd\u65b0\u56de\u6b78\uff0c\u4ee5\u4f9bFDA\u512a\u5148\u5be9\u67e5\u3002https:\/\/www.fiercebiotech.com\/biotech\/2020-trial-pause-isnt-holding-csl-behring-back-fda-oks-priority-review-application<\/p>\n

[5]<\/a> BioMarin\u7684\u8840\u53cb\u75c5\u57fa\u56e0\u7642\u6cd5Roctavian\u5728FDA\u5ef6\u9072\u5f8c\u8d0f\u5f97\u6709\u689d\u4ef6\u7684EU\u652f\u63f4\u3002https:\/\/www.fiercepharma.com\/pharma\/biomarins-hemophilia-gene-therapy-roctavian-wins-conditional-eu-backing-fda-plan-delayed<\/p>\n

[6] <\/a>\u4e16\u754c\u8840\u53cb\u75c5\u806f\u76df\u8840\u53cb\u75c5\u7ba1\u7406\u6307\u5357\u3002https:\/\/www1.wfh.org\/publications\/files\/pdf-1863.pdf<\/p>\n

[7] \u570b\u5bb6\u8840\u53cb\u75c5\u57fa\u91d1\u6703\u6240\u6709\u51fa\u8840\u6027\u75be\u60a3-\u7576\u524d\u6cbb\u7642\u3002https:\/\/www.hemophilia.org\/bleeding-disorders-a-z\/treatment\/current-treatments<\/p>\n

[8]<\/a> \u4e16\u754c\u8840\u53cb\u75c5\u806f\u76df\u8840\u53cb\u75c5\u7ba1\u7406\u6307\u5357\u3002 http:\/\/www1.wfh.org\/publications\/files\/pdf-1863.pdf<\/p>\n","protected":false},"excerpt":{"rendered":"

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https:\/\/www.cdc.gov\/ncbddd\/hemophilia\/inhibitors.html [2] Mannucci\uff0cP.\uff0c2020. \u8840\u53cb\u75c5\u7642\u6cd5\uff1a\u672a\u4f86\u5df2\u7d93\u958b\u59cb\u3002Haematologica\uff0c105(3)\uff0c\u7b2c545-553\u9801\u3002 [3] FDA\u6279\u51c6emicizumab-kxwh\u7528\u65bc\u9810\u9632\u548c\u6e1b\u5c11\u5177\u6709\u56e0\u5b50VIII\u6291\u5236\u7269\u7684A\u578b\u8840\u53cb\u75c5\u60a3\u8005\u7684\u51fa\u8840\u3002https:\/\/www.fda.gov\/drugs\/resources-information-approved-drugs\/fda-approves-emicizumab-kxwh-prevention-and-reduction-bleeding-patients-hemophilia-factor-viii [4] CSL Behring\u57fa\u56e0\u7642\u6cd5\u5728\u66ab\u7de9\u5f8c\u91cd\u65b0\u56de\u6b78\uff0c\u4ee5\u4f9bFDA\u512a\u5148\u5be9\u67e5\u3002https:\/\/www.fiercebiotech.com\/biotech\/2020-trial-pause-isnt-holding-csl-behring-back-fda-oks-priority-review-application [5] BioMarin\u7684\u8840\u53cb\u75c5\u57fa\u56e0\u7642\u6cd5Roctavian\u5728FDA\u5ef6\u9072\u5f8c\u8d0f\u5f97\u6709\u689d\u4ef6\u7684EU\u652f\u63f4\u3002https:\/\/www.fiercepharma.com\/pharma\/biomarins-hemophilia-gene-therapy-roctavian-wins-conditional-eu-backing-fda-plan-delayed [6] \u4e16\u754c\u8840\u53cb\u75c5\u806f\u76df\u8840\u53cb\u75c5\u7ba1\u7406\u6307\u5357\u3002https:\/\/www1.wfh.org\/publications\/files\/pdf-1863.pdf [7] \u570b\u5bb6\u8840\u53cb\u75c5\u57fa\u91d1\u6703\u6240\u6709\u51fa\u8840\u6027\u75be\u60a3-\u7576\u524d\u6cbb\u7642\u3002https:\/\/www.hemophilia.org\/bleeding-disorders-a-z\/treatment\/current-treatments [8] \u4e16\u754c\u8840\u53cb\u75c5\u806f\u76df\u8840\u53cb\u75c5\u7ba1\u7406\u6307\u5357\u3002 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