e-ISSN: 2792-0429
New Treatment Modalities in Hemophilia [TP]
TP. 2022; 3(1): 1-4 | DOI: 10.4274/TP.2022.03521

New Treatment Modalities in Hemophilia

Kaan Kavaklı
Ege 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.

Keywords: Hemophilia, FVIII, FIX, EHL-products, emicizumab, fitusuran, concizumab

Kaan Kavaklı. New Treatment Modalities in Hemophilia. TP. 2022; 3(1): 1-4

Corresponding Author: Kaan Kavaklı, Türkiye
Manuscript Language: English