{"title":"治疗A型血友病的新兴药物:II期和III期临床试验的见解。","authors":"Hande Kizilocak, Guy Young","doi":"10.1080/14728214.2021.1988073","DOIUrl":null,"url":null,"abstract":"<p><strong>Introduction: </strong>Hemophilia is a lifelong, genetic-bleeding disorder, which inadequately treated results in permanent joint damage. It is characterized by spontaneous and trauma-related bleeding episodes. In the last 50 years, treatment has seen dramatic improvements which have improved the quality of life of persons with hemophilia.</p><p><strong>Areas covered: </strong>This review will provide a summary of current pharmacological approaches for hemophilia A as well as discuss novel agents which are either approved recently or in phase II-III clinical trials, plasma-derived and recombinant factor VIII (FVIII) products, extended half-life FVIII products, bypassing agents and non-replacement therapies.</p><p><strong>Expert opinion: </strong>Novel therapies are already changing the way that hemophilia A is managed, and as more new therapies get approved, there will be a revolution in the management of this serious condition. Clinicians will have both the opportunities as well as the challenges of incorporating such new technologies into clinical practice.</p>","PeriodicalId":12292,"journal":{"name":"Expert Opinion on Emerging Drugs","volume":"26 4","pages":"337-350"},"PeriodicalIF":2.7000,"publicationDate":"2021-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"2","resultStr":"{\"title\":\"Emerging drugs for hemophilia A: insights into phase II and III clinical trials.\",\"authors\":\"Hande Kizilocak, Guy Young\",\"doi\":\"10.1080/14728214.2021.1988073\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Introduction: </strong>Hemophilia is a lifelong, genetic-bleeding disorder, which inadequately treated results in permanent joint damage. It is characterized by spontaneous and trauma-related bleeding episodes. In the last 50 years, treatment has seen dramatic improvements which have improved the quality of life of persons with hemophilia.</p><p><strong>Areas covered: </strong>This review will provide a summary of current pharmacological approaches for hemophilia A as well as discuss novel agents which are either approved recently or in phase II-III clinical trials, plasma-derived and recombinant factor VIII (FVIII) products, extended half-life FVIII products, bypassing agents and non-replacement therapies.</p><p><strong>Expert opinion: </strong>Novel therapies are already changing the way that hemophilia A is managed, and as more new therapies get approved, there will be a revolution in the management of this serious condition. Clinicians will have both the opportunities as well as the challenges of incorporating such new technologies into clinical practice.</p>\",\"PeriodicalId\":12292,\"journal\":{\"name\":\"Expert Opinion on Emerging Drugs\",\"volume\":\"26 4\",\"pages\":\"337-350\"},\"PeriodicalIF\":2.7000,\"publicationDate\":\"2021-12-01\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"2\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Expert Opinion on Emerging Drugs\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1080/14728214.2021.1988073\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2021/10/11 0:00:00\",\"PubModel\":\"Epub\",\"JCR\":\"Q2\",\"JCRName\":\"PHARMACOLOGY & PHARMACY\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Expert Opinion on Emerging Drugs","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1080/14728214.2021.1988073","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2021/10/11 0:00:00","PubModel":"Epub","JCR":"Q2","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
Emerging drugs for hemophilia A: insights into phase II and III clinical trials.
Introduction: Hemophilia is a lifelong, genetic-bleeding disorder, which inadequately treated results in permanent joint damage. It is characterized by spontaneous and trauma-related bleeding episodes. In the last 50 years, treatment has seen dramatic improvements which have improved the quality of life of persons with hemophilia.
Areas covered: This review will provide a summary of current pharmacological approaches for hemophilia A as well as discuss novel agents which are either approved recently or in phase II-III clinical trials, plasma-derived and recombinant factor VIII (FVIII) products, extended half-life FVIII products, bypassing agents and non-replacement therapies.
Expert opinion: Novel therapies are already changing the way that hemophilia A is managed, and as more new therapies get approved, there will be a revolution in the management of this serious condition. Clinicians will have both the opportunities as well as the challenges of incorporating such new technologies into clinical practice.
期刊介绍：
Expert Opinion on Emerging Drugs (ISSN 1472-8214 [print], 1744-7623 [electronic]) is a MEDLINE-indexed, peer-reviewed, international journal publishing structured reviews on Phase II and Phase III drugs/drug classes emerging onto the market across all therapy areas, providing expert opinion on their potential impact on the current management of specific diseases.
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