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

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Kavaklı K. New Treatment Modalities in Hemophilia. Trends in Pediatrics. 2022;3(1):1-4. https://doi.org/10.4274/TP.2022.03521