Patient and Disease Characteristics
Thirteen patients were identified, 8 (62%) with AML, 3 (23%) with B-ALL, and 2 (15%) with MDS. The median age upon initiation of venetoclax was 14 years (range: 4 months to 27 years). Six (46%) patients were male. Three patients had a history of a prior malignancy: one patient with T-ALL, one with AML, and one with neuroblastoma. The median number of lines of therapy was 3 (range 0 to 5). Five (38%) patients had received a hematopoietic stem cell transplant prior to receiving venetoclax therapy. Two patients had a defined predisposition to developing malignancy: one patient had Schwachman-Diamond Syndrome (SDS) who developed AML, and one had a germline GATA2 mutation who developed MDS. Patient and disease characteristics are summarized in