On November 29, 2018, the U.S. Food and Drug Administration (FDA) issued a warning stating that signs and symptoms of a life-threatening side effect known as "differentiation syndrome" are not being adequately recognized in patients undergoing treatment with the acute myeloid leukemia (AML) drug enasidenib.
Warnings regarding differentiation syndrome are already included in the prescribing information and patient medication guide for enasidenib. However, the FDA has become aware of cases where differentiation syndrome went unrecognized, resulting in patients failing to receive necessary treatment.
Consequently, the FDA hereby reminds healthcare professionals and patients of the need for early recognition and aggressive, intensive treatment of differentiation syndrome to reduce the likelihood of severe illness and death. The FDA will continue to monitor this safety issue.
Information for Healthcare Professionals
1. When initiating treatment with Idhifa (enasidenib) and during subsequent follow-up visits, healthcare professionals should thoroughly review with patients the symptoms of differentiation syndrome listed in the medication guide.
2. Advise patients to contact their healthcare professional immediately if they experience any such symptoms.
3. Differentiation syndrome may manifest as early as 10 days after starting the medication, or as late as 5 months into treatment.
4. If a patient presents with unexplained respiratory distress or other symptoms, a diagnosis of differentiation syndrome should be considered.
5. Once differentiation syndrome is suspected or confirmed, immediate treatment with oral or intravenous corticosteroids should be initiated (please refer to the additional information provided to healthcare professionals for specific treatment regimens).
Information for Patients
While taking enasidenib, please contact your doctor immediately if you experience any of the following symptoms of differentiation syndrome:
Fever; cough; shortness of breath; swelling of the arms or legs; swelling in the neck, groin, or underarm areas; rapid weight gain of more than 10 pounds (approximately 4.5 kg) within one week; bone pain; or dizziness/lightheadedness.
