Preclinical efficacy of gemcitabine in MLL-rearranged infant acute lymphoblastic leukemia
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The treatment of children diagnosed with acute lymphoblastic leukemia (ALL) has improved significantly over recent decades, with 5-year event-free survival (EFS) approaching 85% . However, 5-year EFS for infants diagnosed at less than 1 year of age with MLL-rearranged ALL remains less than 40% . MLL-rearranged infant ALL is both clinically and biologically distinct from other childhood ALL subtypes, which is reflected by a unique gene expression profile , a remarkably silent mutational landscape , and pronounced resistance to currently applied chemotherapeutics .