What is the impact of Gilteritinib (FLT3 inhibitor) on overall survival in patients with FLT3-mutated acute myeloid leukemia (AML)?

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Last updated: February 22, 2025View editorial policy

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From the Guidelines

Gilteritinib significantly improves overall survival in patients with FLT3-mutated acute myeloid leukemia (AML), with a median overall survival of 9.3 months compared to 5.6 months with standard chemotherapy. The use of gilteritinib in patients with relapsed or refractory FLT3-mutated AML has shown a favourable response rate and improved overall survival (OS) compared with chemotherapy, as reported in the 2020 ESMO clinical practice guidelines for diagnosis, treatment, and follow-up of acute myeloid leukaemia in adult patients 1. Some key points to consider when using gilteritinib include:

  • The recommended dose is 120 mg orally once daily, continued until disease progression or unacceptable toxicity.
  • Gilteritinib is particularly effective in patients with FLT3-ITD mutations, which are associated with poor prognosis in AML.
  • Patients should be monitored for side effects, including QT prolongation, differentiation syndrome, and posterior reversible encephalopathy syndrome.
  • Regular blood counts and liver function tests are necessary, and FLT3 mutation testing is required before starting treatment to ensure the drug's appropriateness for the patient. The improvement in overall survival with gilteritinib is a significant advancement in the treatment of FLT3-mutated AML, and its use should be considered in eligible patients, as supported by the guidelines 1.

From the Research

Impact of Gilteritinib on Overall Survival

  • The median overall survival in patients with relapsed or refractory FLT3-mutated acute myeloid leukemia (AML) treated with gilteritinib was significantly longer than those treated with salvage chemotherapy, with a median overall survival of 9.3 months vs. 5.6 months 2.
  • A real-world, multi-center, matched analysis found that the estimated median overall survival of patients treated with gilteritinib for FLT3-mutated relapsed or refractory AML was eight months, and was significantly higher in patients who achieved complete response compared to those who did not 3.
  • Gilteritinib has been shown to improve survival compared to salvage chemotherapy in relapsed and/or refractory FLT3-mutated AML, with a favorable toxicity profile 4.

Clinical Efficacy and Safety

  • Gilteritinib is a highly potent and selective FLT3 kinase inhibitor that has been approved as monotherapy for the treatment of relapsed and/or refractory FLT3-mutated AML 4.
  • The most common adverse events of grade 3 or higher in patients treated with gilteritinib were febrile neutropenia, anemia, and thrombocytopenia 2, 5.
  • Gilteritinib is generally well tolerated, but some clinically relevant adverse events should be monitored, especially myelosuppression, QTc prolongation, and differentiation syndrome 6.

Comparison with Salvage Chemotherapy

  • A phase 3 trial found that gilteritinib resulted in significantly longer survival and higher percentages of patients with remission than salvage chemotherapy among patients with relapsed or refractory FLT3-mutated AML 2.
  • A real-world, multi-center, matched analysis found that the response rates and median overall survival were similar between patients treated with gilteritinib and those treated with intensive salvage chemotherapy 3.

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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