Gilteritinib is an oral targeted anticancer medicine classified as a tyrosine kinase inhibitor (TKI). It is specifically designed to treat certain forms of acute myeloid leukemia (AML) driven by FLT3 gene mutations, which are associated with aggressive disease and poor prognosis.

Gilteritinib represents a precision oncology approach, targeting the molecular abnormality responsible for leukemia cell proliferation rather than using non-specific cytotoxic chemotherapy.

How Gilteritinib Works (Mechanism of Action)

Gilteritinib works by selectively inhibiting abnormal signaling pathways in leukemia cells.

Mechanistically, it:

• Inhibits FLT3-ITD and FLT3-TKD mutations

• Suppresses downstream signaling pathways involved in cell growth and survival

• Reduces uncontrolled proliferation of leukemic blasts

• Promotes apoptosis (programmed cell death)

By directly targeting FLT3-mutated cells, gilteritinib helps control disease progression while sparing many normal cells.

Approved Uses & Indications

Gilteritinib is primarily indicated for:

• Relapsed or refractory acute myeloid leukemia (AML)

• AML with confirmed FLT3 mutation (ITD or TKD)

Its use is strictly guided by molecular diagnostic testing to confirm the presence of FLT3 mutations before initiating therapy.

Dosage & Administration

Important: Dosage must be prescribed and adjusted by a qualified hematologist or oncologist.

General administration principles:

• Administered orally, once daily

• Can be taken with or without food

• Continuous daily dosing until disease progression or unacceptable toxicity

Patients require close monitoring during treatment, especially in the initial weeks.

Common Side Effects

Side effects may vary depending on patient condition and treatment duration.

Common Side Effects

• Fatigue

• Diarrhea

• Nausea

• Constipation

• Cough

• Peripheral edema

• Elevated liver enzymes

Serious or Less Common Side Effects

• Differentiation syndrome

• QT interval prolongation

• Pancreatitis

• Cardiac rhythm abnormalities

• Severe liver toxicity

Immediate medical attention is required if symptoms such as fever, shortness of breath, rapid weight gain, or chest discomfort occur.

Precautions, Warnings & Contraindications

Key precautions include:

• Differentiation syndrome: Potentially life-threatening; requires early recognition and treatment

• Cardiac monitoring: ECG monitoring recommended due to QT prolongation risk

• Liver function: Regular liver enzyme monitoring is required

• Drug interactions: Caution with CYP3A4 inhibitors or inducers

• Pregnancy: May cause fetal harm; effective contraception is required

• Breastfeeding: Not recommended during treatment

Available Formulations

Gilteritinib is available as an oral tablet formulation.

Commonly available strength:

• 120 mg tablets

Strength availability may vary slightly by country based on regulatory approvals.

Generic Availability & Alternatives

Generic versions of gilteritinib may be available in certain markets, subject to regulatory approval and patent status.

Therapeutic alternatives for FLT3-mutated AML may include:

• Other FLT3 inhibitors

• Combination chemotherapy regimens

• Stem cell transplantation

• Enrollment in clinical trials

Treatment selection depends on disease stage, prior therapies, and patient fitness.

Storage & Handling Information

• Store at controlled room temperature

• Protect from moisture and excessive heat

• Keep tablets in original packaging

• Do not crush or split tablets unless instructed

Standard handling precautions are sufficient; gilteritinib is not classified as a traditional cytotoxic agent.