LEČENJE PRIMARNE MIJELOFIBROZE, GDE SMO DANAS?
Sažetak
Uvod: Primarna mijelofibroza (PMF) je klonsko oboljenje hematopoeze koje odlikuju konstitutivne tegobe, uvećanje slezine, anemija i često trombocitopenija.
Cilj rada: prikazati uporedne podatke o sprovedenim studijama i lekovima koji su nam dostupni ali i novim terapijskim opcijama.
Metode: analiza publikovanih kliničkih studija i relevantnih radova.
Rezultati: u ovom trenutku zlatni standard lečenja bolesnika sa PMF je još uvek ruksolitinib, prvi predstavnik JAK inhibitora (JAKi). Postiže smanjenje volumena slezine (SVS≥35%) kod 41,9% bolesnika u odnosu na placebo i smanjuje tegobe >50% (TSS<50%) kod 49,5% lečenih. Nije idealan lek, jer se tokom primene javlja anemija, trombocitopenija i sklonost ka infekcijama, pa određeni broj bolesnika mora da prekine lečenje. Fedratinib je drugi JAKi, koji ima dobre rezultate kao druga linija nakon neuspeha ruksolitiniba. Primenjen u prvoj liniji, fedratinib postiže SVS≥35% od 47% uz TSS<50 od 40%.
Kod bolesnika sa transfuziono zavisnom anemijom, dobre rezultate je postigao momelotinib, koji nije bio inferioran u odnosu na ruksolitinib (SVS≥35% je sličan, 26.5% u odnosu na 29% za lečene ruksilitinibom). Primena momelotiniba je smanjila transfuzionu zavisnost za 17% u odnosu na ruksolitinib (66% prema 49%). Kod bolesnika sa trombocitopenijom (Tr 50-100x109/L), pakritinib predstavlja najnoviju terapijsku opciju; postiže smanjenje slezine u odnosu na modifikovane doze ruksolitiniba bez daljeg pogoršanja trombocitopenije.
Kombinacije sa ruksolitinibom (navitoklaks, pelabresib) imaju za cilj da značajno poboljšaju odgovor, prvenstveno smanjenjem slezine.
Zaključak: lečenje PMF je i dalje izazov. Primena ruksolitiniba omogućava dobru kontrolu bolesti kod skoro polovine bolesnika, a novi lekovi treba da omoguće lečenje refrakternih bolesnika ili da poboljšaju odgovor.
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