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Content Update

November 17, 2019

Gilteritinib for Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia: In November 2018, the U.S. Food and Drug Administration (FDA) granted approval to gilteritinib (Xospata) for treatment of relapsed or refractory acute myeloid leukemia (AML) with an FLT3 mutation. Gilteritinib is a highly selective FLT3 inhibitor with activity against both types of FLT3 mutations. FDA approval was based on an interim analysis of response rate and duration of response in the gilteritinib arm of the ADMIRAL trial; the results of the phase 3 ADMIRAL trial were recently published. Patients randomized to receive gilteritinib had a higher complete remission rate and longer median overall survival than patients who received salvage chemotherapy. Grade 3 or higher adverse events occurred less frequently in the gilteritinib group. Based on these results, gilteritinib is now an NCCN category 1 therapy for patients with relapsed or refractory AML with FLT3 mutation.

Content Update

September 21, 2017

Midostaurin (Rydapt®) for FLT3-Positive Acute Myeloid Leukemia: Midostaurin is an FLT3 tyrosine kinase inhibitor that is FDA-approved for treatment of adults with newly-diagnosed acute myeloid leukemia (AML) who are FLT3 mutation-positive (FLT3+) in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation. In a phase 3 controlled clinical trial, midostaurin improved both overall and event-free survival when added to standard induction and consolidation chemotherapy. Midostaurin therapy is generally well tolerated with a potential increased risk of severe anemia and/or rash. The NCCN guidelines include midostaurin in addition to chemotherapy as a category 2A recommendation for AML patients with a known FLT3 mutation.



  • image Acute leukemias are the most common malignancies in children and the leading cause of cancer-related death in patients younger than age 20 years.

  • image Several risk factors correlate with prognosis for acute lymphoblastic leukemia (ALL). Poor prognostic factors include high white blood cell (WBC) count at presentation, very young or very old age at diagnosis, delayed remission induction and presence of certain cytogenetic abnormalities (eg, Philadelphia chromosome positive [Ph+]).

  • image For children with ALL, remission induction therapy includes vincristine, a corticosteroid, and asparaginase, with or without an anthracycline. For adults with ALL, vincristine, prednisone, and an anthracycline are given, and asparaginase is sometimes added.

  • image All patients with ALL require prophylactic therapy to prevent CNS disease because of the high risk of central nervous system (CNS) relapse. The choice for therapy includes a combination of the following: cranial irradiation, intrathecal chemotherapy, or high-dose systemic chemotherapy with drugs that cross the blood-brain barrier.

  • image Long-term maintenance therapy for 2 to 3 years is essential to eradicate residual leukemia cells and prolong the duration of remission. Maintenance therapy consists of oral methotrexate and mercaptopurine, with or without monthly pulses of vincristine and a corticosteroid.

  • image Disease-free survival is lower in adults with ALL and has been attributed to greater drug resistance, poor side effect tolerance with subsequent nonadherence, and possibly less-effective therapy. This population is also more likely to have ...

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