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Treatment Name: Enasidenib (IDHIFA®)

Enasidenib (IDHIFA®) is a Chemotherapy Regimen for Acute Myeloid Leukemia (AML)

How does enasidenib (IDHIFA®) work?
A protein known as isocitrate dehydrogenase-2, commonly referred to as “IDH2”, can become mutated within immature myeloid white blood cells. A mutated IDH2 protein can prevent these cells from maturing into normal functional cells, and cause them to accumulate leading to acute myeloid leukemia (AML).

Enasidenib (IDHIFA®) is designed to bind to mutated IDH2 proteins in myeloid leukemia cells. By blocking the function of the mutated IDH2 proteins, enasidenib allows the cells to mature properly. Patients without a known IDH2 mutation may not benefit from enasidenib.

Goals of therapy:
Enasidenib is given to help increase normal blood cell counts such as red blood cells and platelets if they are low, decrease the risk of infection, decrease the amount of blood transfusions needed, and decrease the risk of bleeding. It is not commonly given with the goal of cure. In clinical trials, 11% of patients taking enasidenib were able to receive a potentially curative bone marrow transplant.


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  • Usual Enasidenib (IDHIFA®) starting dose: 100 mg oral tablet by mouth once daily

Enasidenib is usually taken at home until the drug no longer works or until unacceptable side effects occur. It may take as long as six months of therapy with enasidenib to see a response to treatment.

Note: Individual doses may vary based upon your Doctor's recommendation, or drug availability. The 50 mg tablet may be prescribed if a dose reduction is recommended by your doctor.

Side Effects

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

  • Increase in serum bilirubin (81%)
  • Serum calcium decrease (74%)
  • Nausea (50%)
  • Diarrhea (43%)
  • Serum potassium decrease (41%)
  • Vomiting (34%)
  • Decreased appetite (34%)
  • Serum phosphorous decrease (27%)
  • Differentiation syndrome (14%)
  • Altered taste (12%)
  • Increased white blood cell count (12%)
  • Tumor lysis syndrome (6%)

*Important note: Contact your doctor or go to the nearest emergency room immediately if you are experiencing any signs or symptoms of differentiation syndrome such as fever, cough, trouble breathing, bone pain, rapid weight gain, or swelling in your arms or legs.

If differentiation syndrome is suspected, a potent steroid such as dexamethasone 10 mg every 12 hours is usually given until improvement, then slowly decreased to make sure the symptoms do not come back. Treatment with enasidenib may need to be temporarily suspended. Differentiation syndrome may occur as early as 10 days after starting therapy with enasidenib or as late as 5 months after starting therapy. Hospitalization may be required to manage symptoms of differentiation syndrome.

Roughly 43% of patients require at least one temporary interruption of treatment and 17% of patients discontinue treatment permanently due to unacceptable side effects.

Side effect videos Side Effect Videos
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How often is monitoring needed?
Labs (blood tests) may be checked before treatment, at least every 2 weeks during the first 3 months of treatment (maybe more often), then periodically thereafter until stable. Labs often include: Complete Blood Count (CBC), Comprehensive Metabolic Panel (CMP), lactate dehydrogenase (LDH), serum phosphorus, uric acid, plus any others your doctor may order.

How often is imaging needed?
Imaging may be checked during treatment if there is concern for an infection, bleeding, or a blood clot. 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 enasidenib as planned, reduce the dose of future treatments, temporarily delay treatment until the side effect goes away, or switch to an alternative therapy.

Tumor lysis labs may be done more frequently when first stating therapy to protect your heart and kidneys from rapidly dying cancer cells.

ChemoExperts Tips

  • Approximately 12% of patients will experience an increase in white blood cell count (WBC) that is not due an infection while taking enasidenib. If the WBC rises to a high range (greater than 30 x 109/L) and there is no evidence of an infection, a medication known as hydroxyurea (Hydrea®) may be given to lower the WBC. If the WBC does not improve with hydroxyurea, treatment with enasidenib may need to be temporarily suspended until the WBC improves
  • Allopurinol may be prescribed to help prevent tumor lysis syndrome
  • Enasidenib may cause an increase in blood bilirubin, a common blood test used to monitor the function of your liver. In most cases, increases in blood bilirubin are due to enasidenib affecting the removal of bilirubin from your body and not because your liver is being damaged. Contact your doctor immediately if you notice yellowing of your skin or eyes which may be a sign that your bilirubin is increased
  • Be sure to keep enasidenib tablets in the original bottle and do not store in places where there is a lot of moisture such as the bathroom. Do not take tablets out of the bottle until right before taking your dose
  • 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 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 Enasidenib (IDHIFA®), 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 Enasidenib (IDHIFA®). Depending upon your income, they may be able to help cover the cost of:

  • Enasidenib

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 Enasidenib (IDHIFA®) 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 Enasidenib (IDHIFA®)

Individual Drug Label Information

Enasidenib (IDHIFA®)

  • Is an oral tablet available in 50 mg and 100 mg strengths
  • May be taken with or without food with a large glass of water. Swallow whole and do not split or crush tablets
  • If you miss a dose, take the dose as soon as possible if it is the same day and resume your normal schedule the following day. If its not the same day, take your normal dose as scheduled and do not double your dose to make up for the missed dose
  • Store tablets at in a dry place at room temperature (68°F to 77°F) in the original bottle. Do not take tablets out of the bottle until right before taking your dose
  • Dosage adjustments may be required for liver injury or other serious side effects
  • May cause fetal harm if taken while pregnant. Men and women taking enasidenib should use effective contraception during treatment and for one month after the last dose. Avoid breast feeding during enasidenib treatment and for one month after the last dose
General side effects from enasidenib (IDHIFA®)
  • Nausea and vomiting
  • Diarrhea
  • Decreased appetite
  • Differentiation syndrome
  • Altered taste
  • Increased white blood cell count
  • Tumor lysis syndrome
  • Changes in liver function, such as bilirubin
  • Click on the Enasidenib (IDHIFA®) package insert below for reported side effects, possible drug interactions, and other enasidenib prescribing information

Side Effect Videos
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See DailyMed package insert.

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1) Stein EM, et al. Enasidenib in mutant-IDH2 relapsed or refractory acute myeloid leukemia. Blood. 2017 Jun 6. [Epub ahead of print]

Created: August 2, 2017 Updated: November 8, 2017

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 Differentiation Syndrome?

Differentiation syndrome may include a combination of the following signs or symptoms: fever, shortness of breath, water in sac surrounding heart or lungs (pericardial or pleural effusion), kidney dysfunction, low blood pressure, and unexplained weight gain greater than 10 pounds.

Dexamethasone should be given at the first sign of differentiation syndrome and continue until symptoms resolve.

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

14) Serum calcium

What is Tumor Lysis Syndrome (TLS)?

Tumor lysis syndrome, or TLS, occurs when many cancer cells die quickly and release their contents into the bloodstream. Many times the body has the ability to flush these substances out through the kidneys or metabolize them via the liver. However, sometimes the body needs medicines to help eliminate these substances and to prevent organ damage.