Therapy of acute myeloid leukemia
Until 2017, the treatment of acute myeloid leukemia (AML) has always consisted of chemotherapy and bone marrow transplantation. Now available is midostaurin, the first drug that acts specifically only on leukemic cells and is indicated in FMS-like tyrosine kinase 3 (FLT3) mutation carriers, accounting for about one-third of all individuals with AML and the first treatment approved in Italy in 2 years.
Midostaurin is also reimbursed for the treatment of advanced systemic mastocytosis, a rare disease burdened by limited life expectancy with few treatment options. Diagnosis of the FLT3 mutation should be made early because therapy with the specific inhibitor, i.e., midostaurin), should be started as soon as possible (currently it is administered at day 8 after chemotherapy). Follow-ups to assess response to therapy should then be done after induction therapy and at the end of consolidation. It is also important to check with cytofluorimetric and molecular biology tests for residual disease status before and after stem cell transplantation (for patients who will undergo this procedure).
Source: Novartis
