American Society of Hematology, Blood, 10(126), p. 1214-1223, 2015
DOI: 10.1182/blood-2015-05-647172
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Key Points Germ-line CEBPA mutations are highly penetrant, causing early-onset de novo AML associated with favorable survival outcomes. Familial CEBPA-mutated AML displays a unique model of disease progression, with recurrence caused by novel, independent leukemic episodes.