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Treatment Name: Gilteritinib (Xospata®)

Gilteritinib (Xospata®) is a Treatment Regimen for Acute Myeloid Leukemia (AML)

How does gilteritinib work?

A mutated FLT3 protein, (pronounced “flit three”), is sometimes found in myeloid white blood cells and can cause these cells to divide more rapidly and survive longer, leading to leukemia. Gilteritinib is designed to bind to mutated FLT3 (FLT3 stands for Fms-Like Tyrosine kinase 3) on the surface of myeloid leukemia cells. By blocking the function of the abnormally active FLT3 protein, gilteritinib slows the production of new leukemia cells and causes some of the leukemia cells to die. Patients without a known FLT3 mutation may not benefit from gilteritinib (Xospata®).

Goals of therapy:

Gilteritinib is taken to help patients achieve remission and possibly receive a bone marrow transplant. If a bone marrow transplant can be done, gilteritinib is commonly given with the goal of cure; however, if a bone marrow transplant cannot be done, gilteritinib is given to help patients live longer, but is not given with the goal of cure.

Schedule

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How is gilteritinib therapy for AML taken?

  • Usual starting dose: 120 mg (three 40 mg tablets) by mouth once daily, every day

Note: Individual doses may vary based upon your Doctor's recommendation, or drug availability.

Gilteritinib is usually taken at home. It is taken until the therapy no longer works, unacceptable side effects occur, or when a bone marrow transplant can be done. Therapy is typically given for at least six months to see if it works.

Side Effects

What are the most common side effects from gilteritinib for AML?

In clinical studies, the most commonly reported side effects of gilteritinib (Xospata®) are shown here:

  • Neutropenic fever (47%)
  • Low red blood cells [Anemia] (47%)
  • Fever (43%)
  • Liver injury (42%)
  • Diarrhea (33%)
  • Constipation (31%)
  • Low blood potassium (29%)
  • Fatigue (29%)
  • Cough (29%)
  • Low platelets [Thrombocytopenia] (26%)
  • Headache (26%)
  • Swelling in arms or legs (24%)
  • Shortness of breath (24%)
  • Vomiting (22%)

On average, 11% of patients discontinue treatment due to unacceptable side effects.

Importantly, not all people who experience a side effect from gilteritinib will experience it in the same way. It may be mild in some or severe in others, depending upon the individual. Everybody is different. Additionally, side effects may vary over time. For some, side effects may be a reason to delay or switch treatment, reduce the dose, or avoid treatment with a certain medication altogether.

Side effects may be treatable when they occur or preventable by taking certain medications before they happen. When medications are taken to prevent a problem, this is known as prophylaxis, or "prophy" for short.

After starting treatment with gilteritinib, be sure to come back and watch all of the side effect videos shown below. Each of these videos contain valuable information about side effect management that will hopefully help you to both feel better and stay out of the hospital.

Watch videos on common gilteritinib therapy side effects below

Side effect videos Side Effect Videos
Neutropenic FeverNeutropenic FeverAnemiaAnemiaDiarrheaDiarrheaConstipationConstipationFatigue Fatigue Nausea and VomitingNausea and Vomiting

Monitoring

How often is monitoring needed?

Labs (blood tests) may be checked before treatment, once weekly for the first month, then every other week for the second month. It may then be checked once monthly thereafter, if stable. Labs often include: Complete Blood Count (CBC), Comprehensive Metabolic Panel (CMP), creatine phosphokinase, plus any others your doctor may order. An EKG may be checked before treatment, then once weekly for two weeks, then monthly thereafter.

How often is imaging needed?

Imaging may be checked if there are concerns for an infection or to check for certain side effects. Imaging may include: X-rays, magnetic resonance imaging (MRI), or computerized tomography (CT) scans.

How might blood test results/imaging affect treatment?

Depending upon the results, your doctor may advise to continue gilteritinib as planned, reduce the dose, delay treatment until the side effect goes away, or switch to an alternative therapy.

ChemoExperts Tips

What are the most important things to know about gilteritinib while receiving therapy?

  • Differentiation syndrome may occur anytime during the first two months of treatment with gilteritinib. It is characterized by fever, shortness of breath, weight gain, or heart, liver, or kidney problems. It may be life threatening. It is best treated with a potent steroid known as dexamethasone if it develops
  • A pharmacist should ALWAYS review your medication list to ensure that drug interactions are prevented or managed appropriately
  • Clinical trials may exist for AML. Ask your doctor if any studies are currently enrolling in your area. If not, go to clinicaltrials.gov to search for other centers offering study medications

Patient Assistance & Co-payment Coverage

Patients under the age of 65 years, or those with private insurance plans:
If you have insurance and are looking for patient assistance or copay assistance for Gilteritinib (Xospata®), we have provided links that may help.

Visit our Patient Assistance page and click the links to various patient assistance programs for help paying for Gilteritinib (Xospata®). Depending upon your income, they may be able to help cover the cost of:

  • Gilteritinib

For Branded medications (may be available for generic medications too), check with the manufacturer to determine if a co-pay card is offered and if it could reduce your monthly copay.

  • If you are uninsured, check with the manufacturer to determine if you are eligible to receive medication at no cost.

Medicare and Medicaid patients (Patients 65 years or older):
The clinic providing treatment will likely pre-authorize medications and immune therapies such as Gilteritinib (Xospata®) and are the best source to help you understand drug cost.

  • Ask to speak with a patient assistance technician or financial counselor at the clinic or hospital administering this therapy.

Emotional Wellness

What is Emotional Wellness?
Emotional wellness is having a positive outlook balanced with a realistic understanding of current life events. This requires both an awareness and acceptance of your emotions. It is with this knowledge that you can develop a plan to take the necessary actions to positively impact your life.

Emotional wellness uses an ongoing process to continually reflect on the stressors of life in a constructive manner to move forward and create happiness.

Because emotional wellness is deeply connected with physical, social, and spiritual wellness, pursuing it often becomes particularly difficult in times of major illness. Despite this difficulty, working toward emotional wellness has been connected to improved treatment outcomes and a higher likelihood of achieving goals of therapy.

Learn more about pursuing emotional wellness while receiving treatment with Gilteritinib (Xospata®)

Individual Drug Label Information

Gilteritinib (Xospata®)

  • ​Is an oral tablet available in 40 mg
  • FDA Black Box Warning for differentiation syndrome. Contact your doctor if you experience fevers, trouble breathing, fast heartbeat, swelling in arms or legs, or dizziness 
  • Can be taken with or without food at the same time each day. Swallow tablets whole and do not crush or break tablets
  • If you miss a dose, take the dose if it has been less than 12 hours since the dose was supposed to be taken. If it has been more than 12 hours, skip the dose and take the next dose the following day at the regular scheduled time. DO NOT take two doses to make up for the missed dose
  • Should be stored at room temperature (68°F to 77°F). Protect from light, moisture, and humidity (do not store in bathroom)
  • Dosage adjustments may be required for inflammation of the pancreas, QT prolongation, or other serious side effects
  • May interact with certain antifungal and seizure medications. Ask your doctor or pharmacist to review your medications for any possible interactions
  • May interact with grapefruit and grapefruit juice, pomegranate, star fruit, or seville oranges (in marmalade) causing increased blood levels of gilteritinib. This could increase your risk of experiencing side effects. Avoid eating or drinking these foods during treatment 
  • Avoid therapy with St. John’s Wort, carbamazepine, phenytoin, or enzalutamide as they will decrease blood levels of gilteritinib. This could decrease the effectiveness 
  • May cause fetal harm if taken while pregnant. Use effective contraception during treatment and for at least 4 months after the last dose. Do not breastfeed while taking gilteritinib and for at least 2 months after the last dose
General side effects from gilteritinib
  • Fever
  • Low red blood cells and platelets
  • Liver injury
  • Diarrhea or constipation
  • Fatigue
  • Cough
  • Headache
  • Swelling in arms or legs
  • Shortness of breath
  • Vomiting
  • Click on the gilteritinib (Xospata®) package insert below for reported side effects, possible drug interactions, and other gilteritinib prescribing information

Side Effect Videos
Nausea and VomitingNausea and VomitingDiarrheaDiarrheaFatigue Fatigue ConstipationConstipationAnemiaAnemiaNeutropenic FeverNeutropenic Fever

See DailyMed package insert.

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References

1) Perl AE , Martinelli G, Cortes JE, et al. Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3-Mutated AML. N Engl J Med 2019;381:1728-1740

Created: August 17, 2020 Updated: August 17, 2020

What is Acute Myeloid Leukemia (AML)?

A disease of the myeloid cells found in the bone marrow. Myeloid cells are responsible for developing into mature white blood cells, red blood cells, and platelets. In AML, immature myeloid cells know as “blasts” replicate at a very fast rate. Sometimes blasts crowd out the normal cells in the bone marrow so that red blood cells or platelets are unable to develop. Common symptoms of this include fatigue, difficulty exercising, or easy bruising or bleeding.

Most cases of AML are considered “de novo” meaning that the cause is unknown. However, there are a few known risk factors for AML, such as exposure to radiation, various environmental toxins, and certain chemotherapy agents. There is no staging system for AML. Chromosomes (strands of DNA) are often analyzed to determine which mutations in the chromosomes exist. The effectiveness of the treatment may depend upon the specific chromosome mutations that are present.

NOTE: Treatment Options listed below are not all-inclusive. Other treatments may be available. ChemoExperts provides drug information and does not recommend any one treatment over another. Only your Doctor can choose which therapy is appropriate for you.

What does Cure mean?

The word “cure” means there are no cancer cells left in the body and cancer will never come back. Depending on the cancer type and stage, this may be the true goal of therapy. However, it is very difficult to prove all cancer cells are gone. Even though images, like X-rays and MRI’s, and blood tests may not show any signs of cancer, there can be a small amount of cancer cells still left in the body. Because of this, the word “remission” is used more often. This means there are no signs or symptoms of cancer. Patients in remission are followed closely for any signs of cancer returning. Sometimes, more chemotherapy may be given while in remission to prevent the cancer from coming back.

Doctors usually do not consider a patient “cured” until the chance of cancer returning is extremely low. If cancer does return, it usually happens within 5 years of having a remission. Because of this, doctors do not consider a patient cured unless the cancer has not come back within 5 years of remission. The five-year cutoff does not apply to all cancers.

What is a CBC?

A Complete Blood Count (CBC) is a frequently ordered blood test that tells clinicians the status of your: 1) White blood cell count, 2) Hemoglobin, and 3) Platelet count at the time the test was taken.

Common uses:
1) White blood cell count (WBC): is used to determine infection risk, or response to chemotherapy. Certain chemotherapy agents may harm our good infection-fighting cells. Sometimes chemotherapy may need to be delayed to allow these cells to recover.

2) Hemoglobin: is used to determine if someone is anemic. Anytime the hemoglobin is below 12 g/dL, the person is said to be anemic. Red blood cell transfusions, and sometimes iron can be given to restore the hemoglobin level, but anemia treatment should always aim at treating the underlying cause or condition.

3) Platelet count: is used to determine if the risk of bleeding is increased or if a platelet transfusion is required to prevent bleeding. Certain medications that increase bleeding risk, such as: aspirin, certain chemotherapy agents, and blood thinners, may need to be stopped temporarily until the platelet count is within a safe range.

What is a CMP?

A Comprehensive Metabolic Panel (CMP) is a frequently ordered blood test that tells clinicians the status of your: 1) Electrolytes & Acid/Base status2) Kidney function, 3) Liver function, 4) Blood sugar, and 5) Calcium at the time the test was taken. It is commonly used to monitor liver and kidney function when beginning new medications such as chemotherapy. A total of 14 tests are run simultaneously and are shown below.

Electrolytes & Acid/Base status:
1) Sodium, 2) Potassium, 3) Carbon dioxide, 4) Chloride

Kidney Function:
5) BUN (blood urea nitrogen), 6) Serum creatinine (Scr)

Liver Function:
7) AST, 8) ALT, 9) Total bilirubin, 10) Alk Phos, 11) Albumin, 12) Total protein

Blood sugar:
13) Serum glucose

Calcium:
14) Serum calcium