Medication Guide for Enasidenib

The dosage and administration of enasidenib must strictly follow medical advice and be conducted under the guidance of a qualified physician. Do not adjust the dose or stop taking the medication on your own. Proper administration helps maintain stable blood concentrations, maximizes therapeutic effects, and minimizes the risk of adverse reactions.

Standard Dosage for Adult Acute Myeloid Leukemia

Oral administration, 100 mg once daily, with or without food.

Treatment Duration

Continue treatment until disease progression or unacceptable toxicity occurs.

For patients who do not experience disease progression or unacceptable toxicity, treatment should be continued for at least 6 months to allow for a clinical response.

Patient Selection

Select patients based on the presence of isocitrate dehydrogenase 2 (IDH2) mutations in the blood or bone marrow, detected using an FDA-approved test.

Dosage Adjustments for Renal Impairment

No relevant data are available.

Dosage Adjustments for Hepatic Impairment

No relevant data are available.

Management of Hepatotoxicity During Treatment

For bilirubin elevation >3 × upper limit of normal (ULN) persisting for ≥2 weeks (in the absence of elevated transaminases or other liver diseases):

Reduce the dose to 50 mg/day.

If bilirubin elevation decreases to <2 × ULN, treatment may be resumed at 100 mg/day.

Dosage Adjustments for Adverse Reactions

Differentiation Syndrome

If differentiation syndrome is suspected, administer systemic corticosteroids and initiate hemodynamic monitoring.

Withhold treatment if severe pulmonary symptoms requiring intubation or mechanical ventilation and/or renal dysfunction persist for >48 hours after initiation of corticosteroids.

Resume treatment once symptoms improve to Grade 2 or lower.

Non-infectious Leukocytosis (White Blood Cell Count >30 × 10⁹/L)

Administer hydroxyurea according to standard institutional protocols.

If leukocytosis does not improve with hydroxyurea, withhold enasidenib. Resume treatment at a dose of 100 mg/day after the white blood cell count decreases to <30 × 10⁹/L.

Other Treatment-Related Grade 3 or Higher Toxicities (e.g., Tumor Lysis Syndrome)

Withhold treatment until the toxicity resolves to Grade 2 or lower.

Resume treatment at a dose of 50 mg/day upon recovery; may increase to 100 mg/day if the toxicity resolves to Grade 1 or lower.

Discontinue treatment if a Grade 3 or higher toxicity recurs.

Important Safety Information

Patients receiving enasidenib may experience differentiation syndrome, which can be fatal if not treated promptly. Symptoms may include fever, dyspnea, acute respiratory distress, pulmonary infiltrates, pleural or pericardial effusion, rapid weight gain or peripheral edema, lymphadenopathy, bone pain, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring immediately and continue until symptom resolution.