Nowe leki ewolucją leczenia hemofilii A

Autorzy

Łukasz Grajcarek - Studenckie Koło Naukowe przy Katedrze i Zakładzie Biofizyki im. prof. Zbigniewa Religi, Wydział Nauk Medycznych w Zabrzu, Śląski Uniwersytet Medyczny w Katowicach; Tola Kotkiewicz; Marcin Jezierzański; Julia Gawron; Tomasz Furgoł; Witold Kimla

Słowa kluczowe:

hemofilia A, emicizumab, efanesoktokog alfa, FVIII

Streszczenie

Hemofilia A to choroba genetyczna związana z chromosomem płci, wywołana przez niedobory czynnika VIII. Celem jej leczenia jest zapobieganie krwawieniom oraz minimalizacja ryzyka powikłań spowodowanych przez niewystarczające krzepnięcie krwi. Obecnie wiadomo, że dawka i częstotliwość terapii zastępczych czynnikiem VIII wymaganych do ograniczenia krwawień różnią się u poszczególnych osób i mogą zmieniać się w przypadku tej samej osoby z biegiem czasu, co oznacza, że standaryzowana dawka i schemat leczenia mogą nie zapewniać optymalnego leczenia u wszystkich pacjentów. W tej pracy przyjrzymy się metodą leczenie tej choroby które są z nami obecne od pewnego czasu jak leczenie przez: administrowanie czynnika VIII, koncentratami rekombinowanymi FVIII czy stosowanie czynników omijających przez nieczynnikową terapię zastępczą aż po terapię genową. Zestawimy również ze sobą najbardziej obiecujące na ten moment leki efanesoktokog alfa oraz emicizumab.

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