Premium Nahrungsergänzungsmittel | artgerecht

Gilteritinib: Uses, Dosage & Side Effects

Gilteritinib is a targeted cancer drug used to treat acute myeloid leukemia with a FLT3 mutation. It blocks specific enzymes that drive tumor growth in leukemia cells.

Regular tips about health Regular tips about health

Things worth knowing about "Gilteritinib"

Gilteritinib is a targeted cancer drug used to treat acute myeloid leukemia with a FLT3 mutation. It blocks specific enzymes that drive tumor growth in leukemia cells.

What is Gilteritinib?

Gilteritinib is an orally administered kinase inhibitor approved for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) harboring a FLT3 mutation. It belongs to the class of targeted therapies, which are designed to interfere specifically with molecular pathways responsible for the uncontrolled growth of leukemia cells. In the European Union and the United States, gilteritinib is marketed under the brand name Xospata and has been approved by both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).

Indication

Gilteritinib is indicated for patients with FLT3-mutated AML whose disease has returned after prior treatment (relapsed) or did not respond to standard therapy (refractory). A FLT3 mutation is a genetic alteration in the FLT3 gene (FMS-like tyrosine kinase 3) found in approximately 25–30% of all AML patients and is associated with a poorer prognosis. The presence of a FLT3 mutation must be confirmed by a validated diagnostic test before initiating treatment.

Mechanism of Action

Gilteritinib acts as a dual FLT3 and AXL inhibitor. It selectively inhibits the activity of the mutated FLT3 receptor tyrosine kinase, which can occur in two main variants:

FLT3-ITD (internal tandem duplication): the most common mutation, leading to constitutive kinase activation
FLT3-TKD (tyrosine kinase domain): point mutations, such as D835Y

By inhibiting these kinases, gilteritinib blocks downstream signaling pathways including STAT5, MAPK, and PI3K/AKT, which promote the survival and proliferation of leukemic cells. In addition, gilteritinib inhibits AXL, a kinase associated with therapy resistance and disease progression.

Dosage and Administration

The recommended dose of gilteritinib is 120 mg once daily, taken orally as a tablet. It can be taken with or without food. Treatment is continued until disease progression or unacceptable toxicity occurs. Dose adjustments may be necessary in the event of certain adverse effects or drug interactions.

Side Effects

Like all cancer medications, gilteritinib can cause side effects. The most common include:

Differentiation syndrome (a potentially life-threatening complication requiring immediate medical attention)
Changes in liver enzyme levels (elevated transaminases)
Muscle pain and myalgia
Nausea, diarrhea, and vomiting
ECG changes (QT interval prolongation)
Fatigue
Swelling of the limbs (edema)
Elevated blood pressure

Differentiation syndrome is a particularly serious adverse effect and can manifest as fever, shortness of breath, pleural or pericardial effusions, and hypotension. Immediate medical attention should be sought if this condition is suspected.

Drug Interactions

Gilteritinib is primarily metabolized by the enzyme CYP3A4. Strong inhibitors or inducers of this enzyme can significantly alter the plasma levels of gilteritinib. Drugs that inhibit P-glycoprotein (P-gp) may also increase drug exposure. Patients should inform their physician about all medications, dietary supplements, and herbal preparations they are taking.

Clinical Studies

The approval of gilteritinib was based primarily on the Phase III ADMIRAL trial, which compared gilteritinib to salvage chemotherapy in patients with relapsed or refractory FLT3-mutated AML. Gilteritinib demonstrated a significantly longer overall survival (median survival: 9.3 months vs. 5.6 months with chemotherapy) as well as higher response rates.

Important Notes for Patients

Treatment should only be carried out under the supervision of a physician experienced in the management of leukemia.
Regular blood count and liver function monitoring is required during therapy.
Women of childbearing potential must use effective contraception during treatment and for at least 6 months after the last dose.
Gilteritinib must not be taken during pregnancy.

References

Perl AE et al. - Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML. New England Journal of Medicine, 2019; 381(18): 1728–1740.
European Medicines Agency (EMA) - Xospata (gilteritinib) - Summary of Product Characteristics. EMA, 2019. Available at: www.ema.europa.eu
Daver N et al. - Targeting FLT3 mutations in AML: review of current knowledge and evidence. Leukemia, 2019; 33(2): 299–312.

Building Blocks for a Healthy Life

As an integrated manufacturer, we develop & produce evidence-based, patented, and non-patented formulations. We work exclusively with plant and natural extracts according to the strictest purity & quality standards.
The combination of science & modern technology with the laws of nature creates solutions consistently tailored to humans – for the highest good: health.