{"title":"外源性 Dezaparvovec(Hemgenix)","authors":"Cadth","doi":"10.51731/cjht.2024.888","DOIUrl":null,"url":null,"abstract":"\nCADTH recommends that public drug plans reimburse Hemgenix for the treatment of hemophilia B if certain conditions are met. \nHemgenix should only be covered to treat patients (≥ 18 years of age) with moderately severe to severe hemophilia B (circulating coagulation factor IX [FIX] ≤ 2%) if their bleeding requires ongoing prophylactic treatment, their titre of the neutralizing antibody to variant adeno-associated virus 5 (AAV5) is below 1:900, they do not have FIX inhibitors, and if they have not previously received gene therapy to treat hemophilia B. \nHemgenix should only be reimbursed if it is prescribed by specialists who are experts in treating hemophilia B and the cost of Hemgenix is reduced. \n","PeriodicalId":505661,"journal":{"name":"Canadian Journal of Health Technologies","volume":" 21","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2024-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Etranacogene Dezaparvovec (Hemgenix)\",\"authors\":\"Cadth\",\"doi\":\"10.51731/cjht.2024.888\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"\\nCADTH recommends that public drug plans reimburse Hemgenix for the treatment of hemophilia B if certain conditions are met. \\nHemgenix should only be covered to treat patients (≥ 18 years of age) with moderately severe to severe hemophilia B (circulating coagulation factor IX [FIX] ≤ 2%) if their bleeding requires ongoing prophylactic treatment, their titre of the neutralizing antibody to variant adeno-associated virus 5 (AAV5) is below 1:900, they do not have FIX inhibitors, and if they have not previously received gene therapy to treat hemophilia B. \\nHemgenix should only be reimbursed if it is prescribed by specialists who are experts in treating hemophilia B and the cost of Hemgenix is reduced. \\n\",\"PeriodicalId\":505661,\"journal\":{\"name\":\"Canadian Journal of Health Technologies\",\"volume\":\" 21\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2024-05-08\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Canadian Journal of Health Technologies\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.51731/cjht.2024.888\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Canadian Journal of Health Technologies","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.51731/cjht.2024.888","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
引用次数: 0
摘要
CADTH 建议,如果符合特定条件,公共药品计划可报销 Hemgenix 用于治疗 B 型血友病的费用。Hemgenix 只能用于治疗中重度至重度 B 型血友病患者(年龄≥ 18 岁)(循环凝血因子 IX [FIX] ≤ 2%),前提是他们的出血情况需要持续的预防性治疗,他们的变异腺相关病毒 5 (AAV5) 中和抗体滴度低于 1:900,他们没有 FIX 抑制剂,并且之前没有接受过治疗 B 型血友病的基因疗法。Hemgenix 只有在治疗 B 型血友病的专家开具处方且 Hemgenix 的费用降低的情况下才能报销。
CADTH recommends that public drug plans reimburse Hemgenix for the treatment of hemophilia B if certain conditions are met.
Hemgenix should only be covered to treat patients (≥ 18 years of age) with moderately severe to severe hemophilia B (circulating coagulation factor IX [FIX] ≤ 2%) if their bleeding requires ongoing prophylactic treatment, their titre of the neutralizing antibody to variant adeno-associated virus 5 (AAV5) is below 1:900, they do not have FIX inhibitors, and if they have not previously received gene therapy to treat hemophilia B.
Hemgenix should only be reimbursed if it is prescribed by specialists who are experts in treating hemophilia B and the cost of Hemgenix is reduced.