Venetoclax and azacytidine in childhood primary advanced myelodysplastic syndromes, refractory/relapsed Acute Myeloid Leukemia and therapy-related myeloid diseases (ASH 2025) - Nov 4, 2025 - Abstract #abs25-4370; 289; Pres time: Dec 7, 2025; 06:00 PM - 06:15 PM; Location: OCCC - W414CD; "Two of these NR cases were rescued withrevumenib achieving CR...A cytofluorimetric CR was also reported in 2 DEK-NUP214 AML patientsand one CBFA2T3-GLIS2 AML patient who receives ven/aza with STRO002...Our cohort is the largest reportedto date in the difficult-to-treat subgroups of pediatric MDS-EB and t-MDS/AML, supporting futureprospective protocols using ven/aza. Further biological characterization of specific genetic subgroups,such as KMT2A-rearranged malignancies, may help refine the role of Bcl-2 inhibition and improve theprediction of treatment response."
Clinical • Metastases • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • CBFA2T3 • DEK • FLT3 • GLIS2 • KMT2A • NUP214
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Francesco Baccelli1,2; Pietro Merli3; Gennaro Pagano1; Marco Becilli3; Katia Girardi3; Valeria Paganelli3; Davide Leardini1; Edoardo Muratore1; Francesca Vendemini4; Giuliana Beneduce5; Manuela Tumino6; Francesco Saglio7; Elena Sieni8; Elena Mastrodicasa9; Francesco Paolo Tambaro5; Riccardo Masetti1,2; Franco Locatelli3,10
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