Generation of a comprehensive AML PDX resource
AML, thrombocytopenia, anemia, splenomegaly, AML PDX
DISEASE, THROMBOCYTOPENIA, AML, ANEMIA
To generate a representative AML PDX inventory, primary bone marrow or blood samples from 50 patients were tested for engraftment and development of AML in NOD/SCID/IL2gR−/−/hIL3,CSF2,KITLG (NSGS). The overall success rate for primary engraftment in NSGS was 70%, defined by bone marrow, spleen or peripheral blood donor chimerism of at least 20%, splenomegaly (spleen weight >70 mg), anemia (HCT < 35%) or thrombocytopenia (PLT < 400 × 10From the individual samples from patients with AML that successfully engrafted in NSGS, 30 were randomly selected and characterized based on clinical parameters, including patient age, sex, ELN2017 risk, World Health Organization (WHO) disease classification and molecular profiles obtained by transcriptional and mutational sequencing (Fig. 