What are the side effects of gilteritinib (FLT3 inhibitor) in acute myeloid leukemia (AML) treatment?

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Last updated: June 6, 2025View editorial policy

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From the Guidelines

Gilteritinib, a FLT3 inhibitor used in acute myeloid leukemia treatment, commonly causes significant side effects, including febrile neutropenia, sepsis, pneumonia, and other infections, as reported in a 2022 study published in Leukemia 1. The most frequent adverse reactions associated with gilteritinib include:

  • Febrile neutropenia, which was documented in 40% of patients in an open-label phase 1/2 study 1
  • Sepsis, reported in 14% of patients in the same study 1
  • Pneumonia, occurring in 4% of patients 1
  • Clostridial colitis, affecting 2% of patients 1 These side effects are likely due to the inhibition of multiple kinases beyond FLT3, including AXL and other tyrosine kinases, which can affect various cellular processes throughout the body.

Key Considerations

  • Gilteritinib can cause QT interval prolongation, potentially leading to dangerous heart rhythm abnormalities, requiring regular ECG monitoring 1
  • Patients should report any new or worsening symptoms promptly, as early intervention can help manage these adverse effects effectively
  • The European Conference on Infections in Leukemia (ECIL) recommends standard diagnostic procedures and treatment for neutropenic fever and/or infections in patients receiving gilteritinib, as well as careful monitoring of hepatotoxicity 1

Management of Side Effects

  • Patients receiving gilteritinib should be closely monitored for signs of infection, such as fever, and should receive prompt treatment if an infection occurs
  • Regular ECG monitoring is recommended to detect potential QT interval prolongation
  • Patients should be educated on the importance of reporting any new or worsening symptoms promptly, to ensure early intervention and effective management of adverse effects.

From the Research

Side Effects of Gilteritinib

The side effects of gilteritinib, a FLT3 inhibitor used in the treatment of acute myeloid leukemia (AML), include:

  • Myelosuppression, which is a condition where the bone marrow's ability to produce blood cells is decreased 2
  • QTc prolongation, a condition that affects the heart's rhythm 2
  • Differentiation syndrome, a condition that occurs when the immature blood cells in the bone marrow start to mature too quickly 2
  • Diarrhea 3
  • Elevated aspartate aminotransferase, a sign of liver damage 3
  • Febrile neutropenia, a condition where the patient has a fever and a low white blood cell count 3
  • Anemia, a condition where the patient has a low red blood cell count 3
  • Thrombocytopenia, a condition where the patient has a low platelet count 3
  • Sepsis, a life-threatening condition that occurs when the body's response to an infection becomes uncontrolled 3
  • Pneumonia, an infection that inflames the air sacs in the lungs 3

Management of Side Effects

These side effects are usually manageable with dose reductions, interruption, or discontinuation of gilteritinib, and are often reversible 2. It is essential to monitor patients closely for these side effects and adjust the treatment plan accordingly.

Clinical Relevance

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. The drug is orally available and has become a standard of care for this patient population. Ongoing clinical trials are evaluating gilteritinib in combination with frontline chemotherapy and as a maintenance agent 4.

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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