The oral small molecule is the first drug approved for a major driver of acute leukemia in infants.
KMT2AThe approval makes the oral small-molecule menin inhibitor the first pharmaceutical to carry the indication. It blocks the binding of menin to mutated KMT2A fusion proteins, tamping down the process that leads to the disease.
Approval was based on a single-arm of the open-label AUGMENT-101 trial with 104 adult and pediatric patients with the mutation. Pediatric patients were at least 30 days old. Eighty-three patients required blood cell and/or platelet transfusions at baseline; 12 did not need transfusions for 56 days afterward. Of the 21 who were transfusion-free at baseline, 10 remained so over the same period.
Acute Leukaemia Leukemia Leukaemia Blood Cancer Children Child Childhood Pediatrics Kids Transfusion Platelet Transfusion Plateletpheresis Thrombapheresis Thrombocytapheresis Pain Management Lipids Lipid Management Aminotransferase Ast Liver Enzymes Liver Studies Alt Transaminase Remission Bacteria Bacterial Infection Blood Edema Ecg Electrocardiogram Ekg
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