Midostaurin added to standard therapy in FLT3-positive acute myeloid leukemia treatment

Abstract

Acute myeloid leukemia (AML) is a complex disease with a dynamic course associated with a series of acquired and cumulative genetic changes. In recent years, significant advances have been made in understanding of its pathogenesis. Moreover, diagnostic and therapeutic options have expanded. The current classifications take into account cytogenetic and molecular disorders, including the presence of, among others, mutations within FLT3 transmembrane tyrosine kinase, regulating the proliferation and differentiation of hematopoietic cells at an early development stage. FLT3 mutation is detected in approximately 30% of newly diagnosed AML cases and concerns mutations: ITD (internal tandem duplication) or TKD (tyrosine kinase domain) gene. The high ratio of FLT3-ITD. mutation is associated with an unfavorable prognosis. The patients should be included into clinical trials due to insufficient standard therapy effects. The new AML treatment strategies include tyrosine kinase inhibitors, 1^st and 2^nd generation. Midostaurin is a non-specific kinase inhibitor registered for the treatment of newly diagnosed AML patients in 2017. The paper presents the experience of the Department of Hematology and Bone Marrow Transplantation in Poznan in the use of FLT3 tyrosine kinase inhibitors based on a case report of two patients with newly diagnosed AML.