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Treatment

Acute Lymphocytic Leukemia: Targeted Therapy

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Targeted therapy is the use of medicines that take aim on parts of cancer cells that make them different from healthy cells. They target proteins that control how cancer cells grow, divide, and spread. They can damage the cancer cells without affecting most healthy cells. These medicines are different from standard chemotherapy (chemo) medicines. They often have milder side effects.


When is targeted therapy used for ALL?

Targeted therapy may be used as the first treatment for acute lymphocytic leukemia (ALL). Or it may be used when chemo is not working. It may also be used along with chemo to help it work better.


Types of targeted therapy for ALL

Several types of medicines are used in targeted therapy for ALL. These medicines target certain abnormal proteins on ALL cells, such as those caused by the Philadelphia chromosome. They can be helpful if your ALL cells have this mutation. These medicines include tyrosine kinase inhibitors (TKIs) such as:

  • Imatinib mesylate
  • Nilotinib
  • Ponatinib
  • Dasatinib

A newer targeted therapy called a menin inhibitor is used for people with KMT2A gene changes. This medicine includes:

  • Revumenib

Other targeted medicines that may be used to treat ALL are monoclonal antibodies, including:

  • Blinatumomab
  • Inotuzumab ozogamicin
  • Rituximab


How targeted therapy is done

Most TKI medicines are taken as pills, once or twice a day. Take them exactly as your care team tells you. Taking these medicines as directed gives them the best chance to treat ALL.

Other medicines, like blinatumomab, are given through an I.V. They may be given in a doctor's office, infusion clinic, or in a hospital. These medicines sometimes cause an allergicor infusion-related reaction. This reaction may be serious in some people. Before treatment starts, you may be given medicine to help lower the chance of an allergic or infusion-related reaction. Treatments might be given anywhere from once a month to several times a week.

Tell your doctor about all other medicines you take. This includes over-the-counter medicines and supplements, such as herbs and vitamins. Some medicines and supplements can change the way targeted therapy medicines work.

During treatment, blood tests will be done. This is to check for a decrease in your white or red blood cells or platelet levels. Blood tests will be done more often at the start of treatment.


Possible side effects of targeted therapy

Side effects depend on the medicine you are given. Common side effects may include:

  • Diarrhea or constipation.
  • Fatigue.
  • Fever.
  • Headaches.
  • Itching or skin rashes.
  • Muscle pain.
  • Nausea or vomiting.
  • Swelling.
  • Lower blood cell counts, which can increase your risk of infections, bleeding, and bruising.
  • Infusion reactions with monoclonal antibodies.

Side effects from these medicines tend to be mild. But in some cases, they can cause more severe side effects depending on the medicine. Ask your doctor what symptoms to watch for and when to report them.


Partnering with your care team

It's important to know which medicines you're taking. Write down the names of your medicines. Ask your care team how they work, how to take them, and what side effects they might have.

Talk with your health care providers about what side effects to watch out for and when you should report them to your care team. Know what number to call with problems or questions, including after office hours and on holidays and weekends.

It may be helpful to keep a diary of your side effects. Write down any physical, thinking, and emotional changes. A written list will make it easier for you to remember your questions when you go to your appointments. It will also help your care team make a plan to manage your side effects.

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© 2026 The StayWell Company, LLC. All rights reserved. This information is not intended as a substitute for professional medical care. Always follow your healthcare provider's instructions.

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