ALC2 (NCT00651261): Midostaurin in patients with acute myeloid leukemia and FLT3-TKD mutations: a subanalysis from the RATIFY trial. Voso MT, Larson RA, Jones D, Marcucci G, Prior T, Krauter J, Heuser M, Lavorgna S, Nomdedeu J, Geyer SM, Walker A, Wei AH, Sierra J, Sanz MA, Brandwein JM, de Witte TM, Jansen JH, Niederwieser D, Appelbaum FR, Medeiros BC, Tallman MS, Schlenk RF, Ganser A, Amadori S, Cheng Y, Chen Y, Pallaud C, Du L, Piciocchi A, Ehninger G, Byrd J, Thiede C, Döhner K, Stone RM, Döhner H, Bloomfield CD, Lo-Coco F. Blood Adv 4: 4945-54, 2020. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7556122/ Findings: Midostaurin significantly improves event-free survival in patients with FLT3-TKD mutated AML. NPM1 mutations and CBF rearrangements were favorable independent predictors of OS and EFS in the multivariable analysis. The purpose of this study was to compare the effects, good and/or bad, of a standard chemotherapy regimen for AML that includes the drugs daunorubicin and cytarabine combined with or without midostaurin (also known as PKC412), to find out which is better. This research is being done because it is unknown whether the addition of midostaurin to chemotherapy treatment is better than chemotherapy treatment alone. Midostaurin has been tested in over 400 patients and is being studied in a number of illnesses, including AML, colon cancer, and lung cancer. Midostaurin blocks an enzyme, produced by a gene known as FLT3, that may have a role in the survival and growth of AML cells. Not all leukemia cells will have the abnormal FLT3 gene. This study focused only on patients with leukemia cells with the abnormal FLT3 gene. For more information please visit.
