New Treatment Modalities in Hemophilia
Hemophilia is a single gene disorder and as a genetical coagulation system problem it is a life-long bleeding disorder. Even though routine treatment modalities as plasma-derived and then recombinant factor concentrates available for last 50 years, unmet needs is continuing for hemophilia therapy....
Saved in:
| Main Author: | |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Aydın Pediatric Society
2022-03-01
|
| Series: | Trends in Pediatrics |
| Subjects: | |
| Online Access: | https://trendspediatrics.com/article/view/42 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1849718327106076672 |
|---|---|
| author | Kaan Kavaklı |
| author_facet | Kaan Kavaklı |
| author_sort | Kaan Kavaklı |
| collection | DOAJ |
| description |
Hemophilia is a single gene disorder and as a genetical coagulation system problem it is a life-long bleeding disorder. Even though routine treatment modalities as plasma-derived and then recombinant factor concentrates available for last 50 years, unmet needs is continuing for hemophilia therapy. Gold standart treatment is regularly prophylactic FVIII/FIX infusions. However, life-long and frequent intra-venous infusions become medical burden for patients and families. New agents as enhanced half-life (EHL) factor concentrates and non-factor therapies which are able to be used subcutaneously are very hopeful. In this review, EHL factor concentrates, FVIII mimetic agents and re-balancing therapies will be discussed. Although celluler gene therapy is very hopeful and successful phase-3 studies are reported, gene therapy for hemophilia will not be mentioned in this review.
|
| format | Article |
| id | doaj-art-87f68f58d9f74d6fb9822e1cb8c0a203 |
| institution | DOAJ |
| issn | 2792-0429 |
| language | English |
| publishDate | 2022-03-01 |
| publisher | Aydın Pediatric Society |
| record_format | Article |
| series | Trends in Pediatrics |
| spelling | doaj-art-87f68f58d9f74d6fb9822e1cb8c0a2032025-08-20T03:12:24ZengAydın Pediatric SocietyTrends in Pediatrics2792-04292022-03-013110.4274/TP.2022.03521New Treatment Modalities in HemophiliaKaan Kavaklı0https://orcid.org/0000-0003-1174-1958Ege University Children Hospital, Clinic of Hematology, İzmir, Turkey Hemophilia is a single gene disorder and as a genetical coagulation system problem it is a life-long bleeding disorder. Even though routine treatment modalities as plasma-derived and then recombinant factor concentrates available for last 50 years, unmet needs is continuing for hemophilia therapy. Gold standart treatment is regularly prophylactic FVIII/FIX infusions. However, life-long and frequent intra-venous infusions become medical burden for patients and families. New agents as enhanced half-life (EHL) factor concentrates and non-factor therapies which are able to be used subcutaneously are very hopeful. In this review, EHL factor concentrates, FVIII mimetic agents and re-balancing therapies will be discussed. Although celluler gene therapy is very hopeful and successful phase-3 studies are reported, gene therapy for hemophilia will not be mentioned in this review. https://trendspediatrics.com/article/view/42HemophiliaFVIIIFIXEHL-productsemicizumabfitusuran |
| spellingShingle | Kaan Kavaklı New Treatment Modalities in Hemophilia Trends in Pediatrics Hemophilia FVIII FIX EHL-products emicizumab fitusuran |
| title | New Treatment Modalities in Hemophilia |
| title_full | New Treatment Modalities in Hemophilia |
| title_fullStr | New Treatment Modalities in Hemophilia |
| title_full_unstemmed | New Treatment Modalities in Hemophilia |
| title_short | New Treatment Modalities in Hemophilia |
| title_sort | new treatment modalities in hemophilia |
| topic | Hemophilia FVIII FIX EHL-products emicizumab fitusuran |
| url | https://trendspediatrics.com/article/view/42 |
| work_keys_str_mv | AT kaankavaklı newtreatmentmodalitiesinhemophilia |