Prognostische Relevanz genetischer Aberrationen der akuten myeloischen Leukämie bei Kindern und Jugendlichen

The survival rate of children and adolescents suffering acute myeloid leukemia (AML) has been significantly improved within the last decades. This has been achieved by a continuously intensified therapy and progress in supportive care to prevent and treat complications. In Germany, the AML-BFM trials 98 (n = 413) and 2004 (n = 499) enrolled 912 children and adolescents as protocol patients (1998 - 2010). The 5-year-overall survival was 71 ± 2 %. In the previous studies prognosis and subsequent treatment stratification based on morphology, cytochemistry and white blood cell count. Today, the identification of new genetic aberrations in AML enables a genetically determined estimation of prognosis, although treatment response must be considered for treatment stratification. The group with a favorable prognosis summarized AML with t(8;21), inv(16), t(15;17), t(1;11), and AML with normal karyotype and NPM1-mutation (n = 253; EFS 74 ± 3 %, OS 88 ± 2 %). A poor prognosis (HR-group) must be expected in AML with t(4;11), t(5;11), t(6;11), t(6;9), t(7;12), t(9;22), Monosomy 7, combined FLT3 / WT1-mutation, and AML with der(12 p)-aberration (n = 101; EFS 30 ± 5 %; OS 56 ± 5 %). The intermediate group summarizes all other subgroups especially AML with normal karyotyp, AML with FLT3-ITD or t(9;11) (n = 558; EFS 43 ± 2 %; OS 64 ± 2 %). The validation of the internationally identified, genetically determined prognostic factors within the AML-BFM (Germany) study population will support treatment recommendations.