Bone marrow typically produces platelets (which help with blood clotting), red blood cells (which transport oxygen throughout the body), and white blood cells (which help fight infection).
Acute myeloid leukemia (AML) is a blood cancer that starts in the bone marrow. With AML, blood cells don’t form properly in the bone marrow and young, immature cells, called “blasts,” are produced instead. These young cells are unable to function the way healthy blood cells can. In most cases, AML can quickly move from the bone marrow into the blood and can sometimes spread to other parts of the body.
As the bone marrow continues to produce more and more blasts, there becomes no room for the production of normal red blood cells, white blood cells, and platelets. When there are too many blasts and not enough healthy cells, the symptoms of AML begin to occur.
Red Blood Cell
White Blood Cell
Young Blood Cell (blast)
What is relapsed or refractory AML?
The term “relapsed” means that the signs and symptoms of AML have returned after a period of being symptom free after initial therapy. It is also possible for AML to be “refractory,” which means that it does not respond to a particular treatment. It is common for a person with relapsed or refractory (R/R) AML to go through periods of treatment, response, and relapse over time.
Want more information?
Get more clarity on AML and IDHIFA therapy in the glossary
IDHIFA® (enasidenib) is a prescription medicine used to treat people with acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation whose disease has come back or has not improved after previous treatment(s). It is not known if IDHIFA is safe and effective in children.
What is the most important information I should know about IDHIFA?
IDHIFA may cause serious side effects, including:
Differentiation Syndrome. Differentiation syndrome is a condition that affects your blood cells which may be life-threatening or lead to death if not treated. Differentiation syndrome has happened within 10 days and up to 5 months after starting IDHIFA. Call your healthcare provider or go to the nearest hospital emergency room right away if you develop any of the following symptoms of differentiation syndrome while taking IDHIFA:
shortness of breath
swelling of arms and legs
swelling around neck, groin, or underarm area
fast weight gain (greater than 10 pounds within a week)
If you develop any of these symptoms of differentiation syndrome, your healthcare provider may start you on a medicine taken by mouth or given through a vein (intravenous) called corticosteroids and may monitor you in the hospital.
Before taking IDHIFA, tell your healthcare provider about all of your medical conditions, including if you:
Are pregnant or plan to become pregnant. IDHIFA can cause harm to your unborn baby if taken during pregnancy
If you are able to become pregnant, your healthcare provider will do a pregnancy test before you start taking IDHIFA
Females who are able to become pregnant and who take IDHIFA should use effective birth control (contraception) during treatment with IDHIFA and for at least 1 month after your last dose of IDHIFA
Males who have female partners that are able to become pregnant should use effective birth control during treatment with IDHIFA and for at least 1 month after your last dose of IDHIFA
IDHIFA may affect how hormonal contraceptives work and may cause them to not work as well
Talk to your healthcare provider about birth control methods that may be right for you while taking IDHIFA
IDHIFA may cause fertility problems in females and males, which may affect your ability to have children. Talk to your healthcare provider if you have concerns about fertility
Are breastfeeding or plan to breastfeed. It is not known if IDHIFA passes into your breast milk. You should not breastfeed during your treatment with IDHIFA and for at least 1 month after your last dose of IDHIFA
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
How should I take IDHIFA?
Take IDHIFA exactly as your healthcare provider tells you to
Take IDHIFA 1 time a day at the same time each day. IDHIFA can be taken with or without food
Swallow IDHIFA tablets whole with 8 ounces (one cup) of water. Do not chew or split the tablet
If you miss a dose of IDHIFA or vomit after taking a dose of IDHIFA, take the dose of IDHIFA as soon as possible on the same day. Then take your next dose the next day at your regularly scheduled time. Do not take 2 doses at the same time to make up for the missed dose
Your healthcare provider should do blood tests to check your blood counts before you start IDHIFA treatment and at a minimum of every 2 weeks for at least the first 3 months during treatment to check for side effects
What are the possible side effects of IDHIFA?
IDHIFA may cause serious side effects, including:
See "What is the most important information I should know about IDHIFA?"
The most common side effects of IDHIFA include:
Tell your healthcare provider if you have any changes to the color of your skin or the whites of your eyes.
Your healthcare provider will monitor you for side effects during treatment and may tell you to stop taking IDHIFA if you develop certain side effects. These are not all the possible side effects of IDHIFA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
General information about the safe and effective use of IDHIFA
Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Do not take IDHIFA for conditions for which it was not prescribed. Do not give IDHIFA to other people, even if they have the same symptoms you have. It may harm them. You can ask your pharmacist or healthcare provider for information about IDHIFA that is written for health professionals.