Onkológia 2/2023
Gilteritinib v liečbe relabujúcej a/alebo refraktérnej FLT3 pozitívnej akútnej myeloblastovej leukémie
MUDr. Juraj Chudej, PhD., MBA, doc. MUDr. Juraj Sokol, PhD., MBA, MUDr. Ľudmila Lineková, MUDr. Monika Péčová, MUDr. Lenka Lisá, PhD.
Napriek pokrokom a schváleným novým liečivám v terapii akútnej myeloblastovej leukémie (AML) v prvej línii väčšina pacientov zrelabuje a/alebo sa stane refraktérnymi (R/R) na terapiu. Pri štandardných chemoterapeutických prístupoch je medián celkového prežívania 4 – 7 mesiacov, čo len zdôrazňuje potrebu nových terapeutických možností a nových liečiv. Mutácie v géne pre Fms-like tyrozínkinázový receptor 3 (FLT3) sú prítomné až u jednej tretiny pacientov s AML. Gilteritinib je vysokoúčinná druhá generácia inhibítora FLT3, o ktorom sa zistilo, že je účinný u FLT3-mutovaných pacientov s AML. Gilteritinib bol nedávno schválený FDA (Úrad pre kontrolu potravín a liečiv) a EMA (Európska lieková agentúra) v monoterapii u pacientov s R/R FLT3-mutovanou AML na základe výsledkov štúdie ADMIRAL. Gilteritinib bol spojený s predĺžením celkového prežívania. V tomto článku uvádzame prehľad súčasného stavu schválených inhibítorov FLT3 u pacientov s AML vrátane súčasného štandardu starostlivosti.
Kľúčové slová: akútna leukémia, FLT3, gilteritinib, terapia
Gilteritinib for the treatment of relapsed and/or refractory FLT3-mutated acute myeloid leukemia
Despite advances in frontline treatment with recently approved therapies for acute myeloid leukemia (AML), most patients experience relapsed/refractory (R/R) disease. R/R AML has a median overall survival (OS) of 4-7 months with standard chemotherapy approaches, emphasizing the importance of newly approved targeted therapies and the need for additional treatment options. Mutations in the Fms-like tyrosine kinase receptor 3 (FLT3) are found in up to one-third of AML patients. Gilteritinib is a highly potent second-generation FLT3 inhibitor that was found to be effective against FLT3-mutated AML. Gilteritinib has recently been approved by the FDA (The Food and Drug Administration) and EMA (European Medicines Agency) as monotherapy for patients with R/R FLT 3-mutated AML based on the results of the ADMIRAL trial. Gilteritinib was associated with improved overall survival. In this article, we review the current landscape of approved FLT3 inhibitors in AML, including the current standard of care.
Keywords: Despite advances in frontline treatment with recently approved therapies for acute myeloid leukemia (AML), most patients experience relapsed/refractory (R/R) disease. R/R AML has a median overall survival (OS) of 4-7 months with standard chemotherapy approaches, emphasizing the importance of newly approved targeted therapies and the need for additional treatment options. Mutations in the Fms-like tyrosine kinase receptor 3 (FLT3) are found in up to one-third of AML patients. Gilteritinib is a highly potent second-generation FLT3 inhibitor that was found to be effective against FLT3-mutated AML. Gilteritinib has recently been approved by the FDA (The Food and Drug Administration) and EMA (European Medicines Agency) as monotherapy for patients with R/R FLT 3-mutated AML based on the results of the ADMIRAL trial. Gilteritinib was associated with improved overall survival. In this article, we review the current landscape of approved FLT3 inhibitors in AML, including the current standard of care.