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The FDA has approved midostaurin (Rydapt) for the treatment of adult patients with newly diagnosed FLT3-positive acute myeloid leukemia in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation. The drug has also been approved for the treatment of patients with advanced systemic mastocytosis (SM), including aggressive systemic mastocytosis, SM with associated hematological neoplasm, and mast cell leukemia.

Results of a phase II trial showed that more than 80% of patients with refractory non-Hodgkin lymphoma achieved objective responses to treatment with the chimeric antigen receptor (CAR) T-cell therapy axicabtagene ciloleucel.

The FDA has granted a priority review designation to tisagenlecleucel-T as a treamtnet for pediatric and young adult patients with relapsed and refractory B-cell acute lymphoblastic leukemia, making it the first CAR T-cell therapy to enter regulatory review.