In a phase IIB study of 95 patients with relapsed/refractory AML or myelodysplastic syndrome irrespective of FLT3 status, 50 mg or 100 mg midostaurin administered twice daily showed acceptable tolerability and high rates of blast reduction, with one FLT3-ITD+ patient achieving partial remission (PR) [151]. This evidence concerns the gene FLT3 and myelodysplastic syndrome.