Gilteritinib vs Midostaurin in FLT3 Mutated Acute Myeloid Leukemia Eligible untreated patients with FLT3 acute myeloid leukemia (AML) between the ages of 18 and 70 will be randomized to receive gilteritinib or midostaurin during induction and consolidation. Collectively, these clinical data led to the approval of midostaurin by the US Food and Drug Administration and the European Medicines Agency for both newly diagnosed FLT3-mutated AML and advanced SM. Clinical Trials Search at Vanderbilt-Ingram Cancer Center. Around the same time, durable responses were also observed in other trials of midostaurin in patients with advanced SM.
This was the first study to show significant improvements in overall survival and event-free survival with the addition of a targeted therapy to standard chemotherapy in this population.
Through a series of collaborations between industry and academia, midostaurin in combination with standard chemotherapy was evaluated in the Cancer and Leukemia Group B 10603/RATIFY study, a large, phase 3, randomized, placebo-controlled trial in patients with newly diagnosed FLT3-mutated AML. Several years later, midostaurin was discovered to be a potent inhibitor of the FLT3 tyrosine kinase and to have activity against mutant forms of KIT proto-oncogene receptor tyrosine kinase, which drive advanced systemic mastocytosis (SM).
In 1996, the relatively frequent occurrence of fms-like tyrosine kinase 3 ( FLT3) activating mutations in acute myeloid leukemia (AML) was first recognized. The information may not cover all possible uses, actions, interactions, or side effects of this drug, or precautions to be taken while using it. It is not a substitute for medical advice. Important: The drug information on this page is meant to be educational. Despite promising preclinical data, early clinical trials in multiple diseases showed only modest efficacy. Find Clinical Trials for Midostaurin - Check for trials from NCIs list of cancer clinical trials now accepting patients. Midostaurin was a prototype kinase inhibitor, originally developed as a protein kinase C inhibitor and subsequently as an angiogenesis inhibitor, based on its inhibition of vascular endothelial growth factor receptor.