Extended Half-Life Coagulation Factors: A New Era in the Management of Hemophilia Patients
Despite effective factor replacement and various treatment schedules, there remain several challenges and unmet needs in the prophylactic treatment of hemophilia limiting its adoption and thereby posing an increased risk of spontaneous bleeding. In this regard, extended half-life (EHL) recombinant f...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
Galenos Publishing House
2019-08-01
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| Series: | Turkish Journal of Hematology |
| Subjects: | |
| Online Access: | https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-33427 |
| _version_ | 1797909124833345536 |
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| author | Muhlis Cem Ar Can Balkan Kaan Kavaklı |
| author_facet | Muhlis Cem Ar Can Balkan Kaan Kavaklı |
| author_sort | Muhlis Cem Ar |
| collection | DOAJ |
| description | Despite effective factor replacement and various treatment schedules, there remain several challenges and unmet needs in the prophylactic treatment of hemophilia limiting its adoption and thereby posing an increased risk of spontaneous bleeding. In this regard, extended half-life (EHL) recombinant factor VIII (rFVIII) and factor IX (rFIX) products promise optimal prophylaxis by decreasing the dose frequency, increasing the compliance, and improving the quality of life without compromising safety and efficacy. EHL products might lead to higher trough levels without increasing infusion frequency, or could facilitate the ability to maintain trough levels while reducing infusion frequency. This paper aims to provide a comprehensive review of the rationale for developing EHL coagulation factors and their utility in the management of hemophilia, with special emphasis on optimal techniques for half-life extension and criteria for defining EHL coagulation factors, as well as indications, efficacy, and safety issues of the currently available EHL-rFVIII and EHL-rFIX products. Potential impacts of these factors on quality of life, health economics, and immune tolerance treatment will also be discussed alongside the challenges in pharmacokinetic-driven prophylaxis and difficulties in monitoring the EHL products with laboratory assays. |
| first_indexed | 2024-04-10T11:04:48Z |
| format | Article |
| id | doaj.art-ae166ad00cda4e2898a975a9e8b9e9b6 |
| institution | Directory Open Access Journal |
| issn | 1308-5263 |
| language | English |
| last_indexed | 2024-04-10T11:04:48Z |
| publishDate | 2019-08-01 |
| publisher | Galenos Publishing House |
| record_format | Article |
| series | Turkish Journal of Hematology |
| spelling | doaj.art-ae166ad00cda4e2898a975a9e8b9e9b62023-02-15T16:19:31ZengGalenos Publishing HouseTurkish Journal of Hematology1308-52632019-08-0136314115410.4274/tjh.galenos.2019.2018.0393TJH-33427Extended Half-Life Coagulation Factors: A New Era in the Management of Hemophilia PatientsMuhlis Cem Ar0Can Balkan1Kaan Kavaklı2İstanbul University-Cerrahpaşa, Cerrahpaşa Faculty of Medicine Department of Internal Medicine, Division of Hematology, İstanbul, Turkey; Members of the Executive Board of Hemophilia Scientific Subcommittee, Turkish Society of HematologyEge University Faculty of Medicine, Department of Pediatrics, Division of Hemato-Oncology, İzmir, Turkey; Members of the Executive Board of Hemophilia Scientific Subcommittee, Turkish Society of HematologyEge University Faculty of Medicine, Department of Pediatrics, Division of Hemato-Oncology, İzmir, Turkey; Members of the Executive Board of Hemophilia Scientific Subcommittee, Turkish Society of HematologyDespite effective factor replacement and various treatment schedules, there remain several challenges and unmet needs in the prophylactic treatment of hemophilia limiting its adoption and thereby posing an increased risk of spontaneous bleeding. In this regard, extended half-life (EHL) recombinant factor VIII (rFVIII) and factor IX (rFIX) products promise optimal prophylaxis by decreasing the dose frequency, increasing the compliance, and improving the quality of life without compromising safety and efficacy. EHL products might lead to higher trough levels without increasing infusion frequency, or could facilitate the ability to maintain trough levels while reducing infusion frequency. This paper aims to provide a comprehensive review of the rationale for developing EHL coagulation factors and their utility in the management of hemophilia, with special emphasis on optimal techniques for half-life extension and criteria for defining EHL coagulation factors, as well as indications, efficacy, and safety issues of the currently available EHL-rFVIII and EHL-rFIX products. Potential impacts of these factors on quality of life, health economics, and immune tolerance treatment will also be discussed alongside the challenges in pharmacokinetic-driven prophylaxis and difficulties in monitoring the EHL products with laboratory assays.https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-33427hemophiliafactor replacement therapyextended halflife productslaboratory assayspharmacokineticsquality of life |
| spellingShingle | Muhlis Cem Ar Can Balkan Kaan Kavaklı Extended Half-Life Coagulation Factors: A New Era in the Management of Hemophilia Patients Turkish Journal of Hematology hemophilia factor replacement therapy extended halflife products laboratory assays pharmacokinetics quality of life |
| title | Extended Half-Life Coagulation Factors: A New Era in the Management of Hemophilia Patients |
| title_full | Extended Half-Life Coagulation Factors: A New Era in the Management of Hemophilia Patients |
| title_fullStr | Extended Half-Life Coagulation Factors: A New Era in the Management of Hemophilia Patients |
| title_full_unstemmed | Extended Half-Life Coagulation Factors: A New Era in the Management of Hemophilia Patients |
| title_short | Extended Half-Life Coagulation Factors: A New Era in the Management of Hemophilia Patients |
| title_sort | extended half life coagulation factors a new era in the management of hemophilia patients |
| topic | hemophilia factor replacement therapy extended halflife products laboratory assays pharmacokinetics quality of life |
| url | https://jag.journalagent.com/z4/download_fulltext.asp?pdir=tjh&un=TJH-33427 |
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