Nowa era w leczeniu ostrej białaczki szpikowej: cele molekularne i terapia szyta na miarę
DOI:
https://doi.org/10.12775/KOSMOS.2026.007Słowa kluczowe
ostra białaczka szpikowa, terapia celowana, mutacje genetyczne, wenetoklaks, midostaurynaAbstrakt
Ostra białaczka szpikowa (AML) to agresywny nowotwór układu krwiotwórczego, charakteryzujący się znaczną heterogennością klonalną i genetyczną, co wpływa na zróżnicowany przebieg kliniczny tej choroby oraz ograniczoną skuteczność terapii. W ostatnich latach nastąpił ogromny postęp w zrozumieniu podłoża molekularnego AML, czego następstwem jest rozwój nowych strategii jej leczenia. Niniejszy artykuł przedstawia aktualny stan wiedzy dotyczący patogenezy AML, ze szczególnym uwzględnieniem kluczowych mutacji genów zaangażowanych w leukemogenezę, takich jak FLT3, NPM1, IDH1/2, DNMT3A, CEBPA oraz TP53, a także mechanizmów chemiooporności komórek białaczkowych związanych z nadekspresją anty-apoptotycznych białek z rodziny BCL-2. Praca koncentruje się na przeglądzie najnowszych strategii terapeutycznych, ukierunkowanych na cele molekularne charakterystyczne dla AML. Szczególny nacisk położono na mechanizmy działania nowych leków dopuszczonych do stosowania klinicznego w AML, takich jak midostauryna, inhibitory IDH1/2 oraz wenetoklaks, podkreślając rosnącą rolę terapii personalizowanej w poprawie wyników klinicznych pacjentów.
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