Tom 3, Supl. C (2023)
Opis przypadku
Opublikowany online: 2023-04-28
Wyświetlenia strony
314
Wyświetlenia/pobrania artykułu
25
Zastosowanie ruksolitynibu w leczeniu włóknienia szpiku. Opis przypadku oraz przegląd literatury
Streszczenie
Mielofibroza (MF) to najgorzej rokujący nowotwór mieloproliferacyjny (MPN) BCR-ABL-negatywny. Jedyną opcją terapeutyczną umożliwiającą̨ pełne wyleczenie MF pozostaje allogeniczne przeszczepienie krwiotwórczych komórek macierzystych (allo-HSCT). Poznanie zaburzeń szlaku sygnałowego JAK-STAT w patogenezie MPN stało się̨ podstawą do syntezy inhibitorów JAK-STAT, w tym ruksolitynibu. W przypadku MF głównymi wskazaniami do terapii ruksolitynibem są splenomegalia oraz obecność objawów ogólnych. Symptomy te zwykle korelują z niekorzystnym wskaźnikiem prognostycznym według skal IPSS i DIPPS, dlatego lek ten wykorzystuje się w terapii pomostowej do finalnej terapii, jaką jest allo-HSCT. W ostatnich latach ruksolitynib został również zarejestrowany w terapii steroidoopornej postaci choroby przeszczep przeciwko gospodarzowi (GvHD). W publikacji przedstawiono opis przypadku 56-letniego pacjenta z poczerwieniczą MF, u którego ostatecznie zadecydowano o wykonaniu allo-HSCT. Nie mniejszym wyzwaniem klinicznym pozostawała u chorego ciężka postać GvHD, która wymagała zastosowania leczenia skojarzonego — ruksolitynibemu z fotoferezami pozaustojowymi.
Słowa kluczowe: włóknienie szpikuchoroba przeszczep przeciwko gospodarzowiinhibitory JAKruksolitynib
Referencje
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