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Enasidenib is a medication used in the treatment of acute myeloid leukemia (AML) with a specific genetic mutation. Like any medication, it carries certain risks and side effects. Here are some of the risks associated with taking Enasidenib:

  • Differentiation Syndrome: This is a potentially life-threatening condition where there’s a rapid increase in white blood cells and other symptoms such as fever, cough, difficulty breathing, swelling, rapid weight gain, or bone pain.
  • Liver Toxicity: Enasidenib can cause liver damage, leading to symptoms like yellowing of the skin or eyes (jaundice), dark urine, abdominal pain, nausea, or vomiting.
  • Electrolyte Abnormalities: It can cause changes in electrolyte levels, such as low potassium or magnesium levels, which may lead to muscle weakness, confusion, irregular heartbeat, or seizures.
  • Cardiac Events: There’s a risk of cardiac problems such as QT prolongation (an abnormal heart rhythm) which can lead to fainting, palpitations, or sudden cardiac death.
  • Bone Marrow Suppression: Enasidenib can decrease the production of blood cells in the bone marrow, leading to anemia (low red blood cell count), thrombocytopenia (low platelet count), or neutropenia (low white blood cell count), which increases the risk of infections and bleeding.
  • Tumor Lysis Syndrome: In some cases, Enasidenib treatment can lead to the rapid breakdown of cancer cells, causing a release of substances into the bloodstream that can lead to kidney failure, abnormal heart rhythms, seizures, or even death.
  • Embryo-Fetal Toxicity: Enasidenib can harm the fetus if taken during pregnancy, leading to birth defects or miscarriage. Thus, it’s essential for women to avoid becoming pregnant while taking this medication and to use effective contraception.

It’s crucial for patients taking Enasidenib to be closely monitored by their healthcare providers for any signs of these potential risks or side effects. It’s also essential to discuss the benefits and risks of Enasidenib treatment with a healthcare professional before starting the medication.

Why is this medication prescribed?

Enasidenib is a medication used in the treatment of a specific type of acute myeloid leukemia (AML) called relapsed or refractory AML with an isocitrate dehydrogenase-2 (IDH2) mutation. This mutation causes an overproduction of a protein called 2-hydroxyglutarate, which disrupts the normal development of blood cells, leading to the development of AML.

How should this medicine be used?

Enasidenib works by inhibiting the mutated IDH2 enzyme, thereby reducing the levels of 2-hydroxyglutarate and potentially allowing for the restoration of normal blood cell development.

Enasidenib is usually prescribed by a hematologist or oncologist and should be used according to their instructions. Typically, it is taken orally once daily, with or without food. The dosage and duration of treatment may vary depending on individual factors such as the patient’s overall health, the severity of the leukemia, and their response to the medication.

It’s important for patients to follow the prescribed dosage and schedule closely and not to stop taking the medication without consulting their healthcare provider. Additionally, they should report any side effects or concerns to their doctor promptly.

Other uses for this medicine

Enasidenib is primarily used for the treatment of relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation, as mentioned earlier. However, it is important to note that medications are sometimes prescribed “off-label” for conditions not specifically approved by regulatory agencies like the FDA. Off-label use refers to the use of a medication for a purpose other than what it has been officially approved for. Healthcare providers may sometimes prescribe Enasidenib off-label based on their clinical judgment and the available evidence.

What special precautions should I follow?

Regarding special precautions for Enasidenib, it’s crucial to follow the guidance provided by your healthcare provider. Here are some general precautions to consider:

  • Pregnancy and breastfeeding: Enasidenib may cause harm to a developing fetus, so it’s essential to use effective contraception during treatment and for a period after discontinuing the medication, as advised by your doctor. It’s also not recommended to breastfeed while taking Enasidenib.
  • Liver function: Enasidenib may cause liver problems, so your doctor may perform regular blood tests to monitor your liver function during treatment.
  • Interactions with other medications: Inform your healthcare provider about all the medications, vitamins, and supplements you are currently taking, as Enasidenib may interact with certain drugs. This includes prescription and over-the-counter medications.
  • Side effects: Be aware of potential side effects of Enasidenib, such as nausea, diarrhea, fatigue, decreased appetite, and others. Report any unusual symptoms or side effects to your healthcare provider promptly.
  • Regular follow-ups: Attend all scheduled appointments with your healthcare provider for monitoring and evaluation of your response to treatment and to address any concerns or questions you may have.
  • Special populations: Enasidenib safety and efficacy in pediatric patients or individuals with severe renal impairment have not been established. Use caution and consult your doctor if you fall into these categories.

Always follow your healthcare provider’s instructions and guidelines for taking Enasidenib, and don’t hesitate to ask any questions or seek clarification regarding your treatment plan.

What special dietary instructions should I follow?

Regarding special dietary instructions, there are no specific dietary restrictions associated with Enasidenib. However, it’s essential to maintain a balanced and healthy diet to support your overall well-being during treatment.

What should I do if I forget a dose?

If you forget to take a dose of Enasidenib, take it as soon as you remember, unless it is almost time for your next scheduled dose. In that case, skip the missed dose and continue with your regular dosing schedule. Do not take a double dose to make up for a missed one. If you are unsure about what to do, contact your healthcare provider or pharmacist for advice.

What side effects can this medication cause?

Enasidenib, like any medication, can cause side effects, although not everyone experiences them. Common side effects associated with Enasidenib may include:

  • Nausea
  • Vomiting
  • Diarrhea
  • Fatigue
  • Decreased appetite
  • Fevers
  • Constipation
  • Differentiation syndrome: This is a potentially serious side effect characterized by fever, cough, difficulty breathing, rapid weight gain, swelling, and/or fluid retention. It typically occurs within the first few months of treatment.

Less common but more serious side effects may include:

  • Liver problems: Enasidenib can cause liver toxicity, which may manifest as yellowing of the skin or eyes (jaundice), dark urine, abdominal pain, or unusual tiredness.
  • Increased risk of infections: Enasidenib may lower your body’s ability to fight infections.
  • Tumor lysis syndrome: This is a rare but serious complication that can occur when cancer cells are rapidly destroyed, leading to the release of large amounts of waste products into the bloodstream. Symptoms may include rapid heart rate, shortness of breath, nausea, vomiting, muscle weakness, seizures, and kidney failure.

It’s essential to report any new or worsening symptoms to your healthcare provider promptly, as they can help manage and alleviate side effects. Additionally, seek medical attention immediately if you experience any signs of a severe allergic reaction, such as rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, or trouble breathing.

Remember to discuss any concerns or questions about side effects with your healthcare provider before starting Enasidenib treatment. They can provide personalized guidance and support based on your individual health status and medical history.

What should I know about storage and disposal of this medication?

When it comes to storing and disposing of Enasidenib, here’s what you should know:


  • Keep Enasidenib tablets in the original container at room temperature away from light and moisture.
  • Do not store Enasidenib in the bathroom or near the kitchen sink where it might be exposed to moisture or heat.
  • Keep Enasidenib out of reach of children and pets.
  • Follow any specific storage instructions provided by your pharmacist or healthcare provider.


  • Dispose of Enasidenib tablets properly according to local regulations or guidelines.
  • Do not flush Enasidenib down the toilet or pour it down the drain unless instructed to do so by your pharmacist or healthcare provider.
  • Consult your pharmacist or healthcare provider for guidance on the proper disposal of unused or expired Enasidenib tablets.

In case of emergency/overdose

In case of emergency or overdose of Enasidenib, it’s important to seek immediate medical attention. Contact your local poison control center or emergency room for guidance. Be prepared to provide information such as the amount of Enasidenib ingested, the time of ingestion, and any symptoms experienced.

What other information should I know?

  • Regular monitoring: Your healthcare provider may perform regular blood tests to monitor your response to Enasidenib treatment and check for any potential side effects or complications.
  • Follow-up appointments: Attend all scheduled follow-up appointments with your healthcare provider to assess your progress and adjust your treatment plan as needed.
  • Patient support resources: Consider seeking out patient support groups or resources for individuals with acute myeloid leukemia (AML) or cancer in general. These groups can provide valuable support, information, and resources to help you cope with your diagnosis and treatment.

Always follow your healthcare provider’s instructions and guidance regarding the use, storage, and disposal of Enasidenib, and don’t hesitate to ask any questions or seek clarification if needed.

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