Mylotarg (gemtuzumab ozogamicin) is an antibody-drug conjugate that’s used to treat a certain type of acute myeloid leukemia (AML) in adults and children. It specifically finds and kills leukemia cancer cells in the body. Mylotarg (gemtuzumab ozogamicin) is given as an injection into your vein (intravenously or IV) at an infusion center or provider’s office. This medication might cause side effects such as tiredness, low platelets, and liver problems.
Newly-diagnosed acute myeloid leukemia (AML) that’s CD33-positive in adults and children 1 month or older
Previously-treated acute myeloid leukemia (AML) that’s CD33-positive in adults and children 2 years and older
Mylotarg (gemtuzumab ozogamicin) is a type of cancer medication known as an antibody-drug conjugate. It’s made up of two parts: a monoclonal antibody and a chemotherapy drug called calicheamicin.
The antibody takes Mylotarg (gemtuzumab ozogamicin) to acute myeloid leukemia cells in the body that have a specific protein called CD33. Once there, the chemotherapy damages the leukemia cell’s DNA, which kills the cancer cell.
Source: DailyMed
Side effects that you should report to your care team as soon as possible:
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
First-choice treatment for people with CD33-positive AML
Specifically targets leukemia cells (the antibody part of the medication takes the chemotherapy to the cancer cells)
Approved to treat both children and adults
Given as an intravenous infusion, so requires a needle
Requires additional medications before each dose to prevent infusion-related reactions
Might cause serious liver problems including veno-occlusive disease (VOD)
It’s important to go to your appointments for your blood tests whenever your provider recommends doing so. Routine blood tests help monitor for certain Mylotarg (gemtuzumab ozogamicin) side effects, such as low blood cell counts and liver irritation. Sometimes, these side effects won’t cause symptoms and changes in your test results are the only sign of a side effect.
Mylotarg (gemtuzumab ozogamicin) might cause infusion reactions (e.g., fever, chills, fast heart rate). You’ll take several medications before each dose to prevent serious reactions to the infusion. These medications might include acetaminophen (Tylenol) and an antihistamine, like diphenhydramine (Benadryl).
Avoid getting sick by washing your hands often, avoiding crowds, and washing raw fruits and vegetables thoroughly before eating them. You might be more likely to get sick while you’re taking Mylotarg (gemtuzumab ozogamicin). The medication can sometimes make your white blood cell counts drop and raise your risk for infections.
It’s not recommended to breastfeed while you’re taking Mylotarg (gemtuzumab ozogamicin) and for 1 month after your last dose. We don’t know whether the medication passes into human milk. But because of the way Mylotarg works, it could harm a breastfeeding infant.
Mylotarg (gemtuzumab ozogamicin) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Risk factors: History of liver problems | People who’ve had a stem cell transplant
Some people taking Mylotarg (gemtuzumab ozogamicin) developed serious liver problems, including veno-occlusive disease (VOD). VOD is a condition that causes blockages in the blood vessels in the liver, which causes the liver to work less well. If not treated, it can lead to damage to other organs, like the lungs and kidneys.
The three main symptoms of VOD are high bilirubin levels in the blood; sudden weight gain, especially in the belly (ascites); and tenderness in the upper right side of your belly. Your providers will monitor you for liver problems, but be sure to let them know right away if you have sudden weight gain or belly pain. Depending on the type of liver problem, you might have to stop taking Mylotarg.
Mylotarg (gemtuzumab ozogamicin) can cause infusion-related reactions, including a severe allergic reaction called anaphylaxis. Most reactions happen during the infusion or within 24 hours after the infusion is finished. Typical symptoms include fever, fast heart rate, chills, trouble breathing, and low blood pressure.
You’ll take several medications before your Mylotarg infusion to prevent a reaction. These additional medications include acetaminophen (Tylenol) and diphenhydramine (Benadryl). Your healthcare team will watch for reactions during your infusion and for at least 1 hour after your infusion ends. Sometimes, people who experience a reaction can continue to take Mylotarg after a treatment break or after taking more preventative medications. But other times, the cancer medication must be permanently stopped if the reaction is serious. Get medical attention right away if you have symptoms of an infusion reaction after you’ve left the clinic.
Risk factors: Taking Mylotarg (gemtuzumab ozogamicin) together with chemotherapy
People taking Mylotarg (gemtuzumab ozogamicin) commonly develop low platelet counts in their body. Platelets are the cells in your blood that form blood clots and prevent bleeding. So when they’re low, your body can’t stop bleeding as well. Your provider will check your blood cell counts while you’re taking Mylotarg.
Contact your provider right away if you have signs of low platelets such as bruising, nose bleeds, bleeding that takes longer to stop, or blood in stool or urine. Your provider might need to hold off on giving you a dose of Mylotarg until your blood tests show that your body is starting to make more platelets and that your platelet count is going back up. Or sometimes, you’ll need to stop taking the medication altogether.
Risk factors: History of abnormal heart rhythms | Taking other medications that can affect heart rhythm
Some people taking medications containing calicheamicin, the chemotherapy part of Mylotarg (gemtuzumab ozogamicin), have experienced heart rhythm changes. It’s not clear whether Mylotarg causes these changes in heart rhythm. To be safe, your provider might follow up with you more often if you have heart rhythm problems (e.g., long QT syndrome) or if you take another medication that can change heart rhythm, such as amiodarone (Pacerone). This is because you might be at risk for these heart rhythm changes.
When you’re diagnosed with AML, your provider will typically perform a test to predict how well the cancer will likely respond to treatment and determine your risk group: favorable (good), intermediate, and adverse (poor). Knowing your risk group helps your provider determine which treatment is best for you.
Studies suggest that Mylotarg (gemtuzumab ozogamicin) might work better for people in the good- and intermediate-risk groups, but not as well for people in the poor-risk group. Providers might prefer to use a different treatment plan than with Mylotarg for some people in the poor-risk group. Your provider will consider the risks and benefits of treating your cancer with Mylotarg based on your risk group. Talk to your provider about what treatment choices you have and the risks and benefits for each one.
Based on animal studies, Mylotarg (gemtuzumab ozogamicin) might harm an unborn baby or raise the risk for miscarriage (pregnancy loss) when it’s given during pregnancy. For this reason, if you’re a female who can get pregnant, you should use birth control while you’re taking Mylotarg until at least 6 months after the last dose. If you’re a male who’s sexually active with a partner who’s able to have children, use condoms during treatment and for at least 3 months after your last dose. Let your provider know right away if you or your partner becomes pregnant while you’re taking Mylotarg.
The dose and treatment schedule depends on your age, your AML status, and whether you’re taking Mylotarg (gemtuzumab ozogamicin) along with other chemotherapy.
In general, Mylotarg (gemtuzumab ozogamicin) is given as an infusion through the vein (IV) by a healthcare provider.
First-choice treatment for newly-diagnosed AML
Adults
The dose is based on your body size using body surface area (BSA), which takes into account your weight and height.
When used together with other chemotherapy: A treatment course consists of 1 induction cycle and then 2 consolidation cycles.
Induction cycle: The typical dose is 3 mg/m2 given IV on days 1, 4 and 7.
Consolidation cycles: The typical dose is 3 mg/m2 given IV on day 1.
When used by itself: A treatment course consists of 1 induction cycle and up to 8 consolidation cycles.
Induction cycle: The typical dose is 6 mg/m2 given IV on day 1 and then 3 mg/m2 given IV on day 8.
Consolidation cycles: The typical dose is 2 mg/m2 given IV on day 1 every 4 weeks.
Children 1 month and older: Treatment days vary based on the treatment protocol that the healthcare team is following. The dose is based on body size, either using body surface area (mg/m2) or using body weight (mg/kg).
Previously-treated AML
People 2 years and older: The typical dose is 3 mg/m2 given IV on days 1, 4, and 7 for one course of treatment.
Allergic reaction to Mylotarg (gemtuzumab ozogamicin)
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American Cancer Society. (n.d.). Targeted therapy.
American Cancer Society. (2020). What are infusion or immune reactions?
American Cancer Society. (2023). Chemotherapy for acute myeloid leukemia (AML).
American Cancer Society. (2023). Typical treatment of acute myeloid leukemia (except APL).
American Cancer Society. (2024). Fevers.
American Liver Foundation. (2023). Ascites.
American Red Cross. (2021). The function of platelets: 5 things you might not know.
Centers for Disease Control and Prevention. (n.d.). What you need to know: Neutropenia and risk for infection.
Farzam, K., et al. (2023). QT prolonging drugs. StatPearls.
Fu, Z., et al. (2022). Antibody drug conjugate: The “biological missile” for targeted cancer therapy. Signal Transduction and Targeted Therapy.
Gupta, A., et al. (2018). Tumor lysis syndrome. Journal of the American Medical Association Oncology.
MedlinePlus. (2022). Bilirubin blood test.
MedlinePlus. (2022). Uric acid test.
Molica, M., et al. (2021). CD33 expression and gentuzumab ozogamicin in acute myeloid leukemia: Two sides of the same coin. Cancers.
National Cancer Institute. (2024). Acute myeloid leukemia treatment (PDQ®)–Patient version.
National Comprehensive Cancer Network. (2021). Anemia and neutropenia: Low red and white blood cell counts.
National Comprehensive Cancer Network. (2023). Acute myeloid leukemia.
National Heart, Lung, and Blood Institute. (2022). Long QT syndrome.
Patel, K., et al. (2017). Febrile neutropenia. Journal of the American Medical Association Oncology.
Richardson, P. G., et al. (2020). Veno-occlusive disease/sinusoidal obstruction syndrome in patients with prior gemtuzumab ozogamicin: Literature analysis of survival after defibrotide treatment. Blood Cancer Journal.
Senzolo, M., et al. (2007). Veno occlusive disease: Update on clinical management. World Journal of Gastroenterology.
Vollmar, B. S., et al. (2021). Calicheamicin antibody–drug conjugates with improved properties. Molecular Cancer Therapeutics.
Wyeth Pharmaceuticals LLC, a subsidiary of Pfizer Inc. (2021). Mylotarg- gemtuzumab ozogamicin injection, powder, lyophilized, for solution [package insert]. DailyMed.
Xu, Q., et al. (2021). Clinical benefits and safety of gemtuzumab ozogamicin in treating acute myeloid leukemia in various subgroups: An updated systematic review, meta-analysis, and network meta-analysis. Frontiers in Immunology.
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