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Questions About Cancer? 1-800-4-CANCER

What You Need To Know About™


  • Posted: 12/23/2013

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The treatment that’s right for you depends mainly on the type of leukemia, your age, and your general health. People with leukemia have many treatment options, and you may receive more than one type of treatment.

Treatment options may include:

If you have chronic leukemia without symptoms, you may not need treatment right away. This option is called watchful waiting. When the disease worsens, treatment can often control the disease and its symptoms. After treatment controls leukemia, people may receive therapy known as maintenance therapy, which helps keep the leukemia from coming back.

However, people with acute leukemia need to be treated right away. The goal of treatment is to destroy signs of leukemia in the body and to make symptoms go away. Maintenance therapy may be given after signs of leukemia are gone.

At any time, care is available to relieve the side effects of treatment and to control pain and other symptoms. For example, antibiotics are given for infections, and transfusions of blood, platelets, or granulocytes are given for bleeding and other blood problems. You can get information about coping on NCI’s website at

Also, you can get information about coping from NCI’s Cancer Information Service at 1-800-4-CANCER (1-800-422-6237). Or, chat using NCI’s instant messaging service, LiveHelp.

Doctors Who Treat Leukemia

Whenever possible, people should be treated at a medical center that has doctors experienced in treating leukemia. If this isn’t possible, your doctor may discuss the treatment plan with a specialist at such a center.

Resources are available to help you find doctors who treat leukemia:

  • Your doctor may be able to refer you to specialists.
  • You can ask a local or state medical society, or a nearby hospital or medical school for names of specialists.
  • NCI’s Cancer Information Service can give you information about treatment centers near you. Call 1-800-4-CANCER (1-800-422-6237). Or, chat using LiveHelp, NCI’s instant messaging service.
  • Other sources can be found in the NCI fact sheet How To Find a Doctor or Treatment Facility If You Have Cancer.

Your health care team may include the following specialists:

Your health care team may also include an oncology nurse, a social worker, and a registered dietitian. For a child with leukemia, the health care team may include a pediatric oncologist.

Your health care team can describe your treatment options, the expected results of each option, and possible side effects. Because cancer treatments often damage healthy cells and tissues, side effects are common. These side effects depend on many factors, including the type of treatment. The specific side effects may not be the same for everyone, and they may even change from one treatment session to the next.

You may want to talk with your doctor about taking part in a treatment research study (clinical trial). Research studies are an important option for people with leukemia. See the Treatment Clinical Trials section.

Questions you may want to ask your doctor about treatment options
  • What are my treatment options? Which do you recommend for me? Why?
  • What are the expected benefits of each kind of treatment?
  • What are the risks and possible side effects of each treatment? How can side effects be managed?
  • What can I do to prepare for treatment?
  • Will I need to stay in the hospital? If so, for how long?
  • What is the treatment likely to cost? Will my insurance cover it?
  • How will treatment affect my normal activities?
  • Would a treatment research study be right for me?

Second Opinion

Before starting treatment, you might want a second opinion about your diagnosis and your treatment options. Some people worry that the doctor will be offended if they ask for a second opinion. Usually the opposite is true. Most doctors welcome a second opinion. Many health insurance companies will pay for a second opinion if you or your doctor requests it. Some insurance companies actually require a second opinion.

If you get a second opinion, the second doctor may agree with your first doctor’s diagnosis and treatment recommendation. Or, the second doctor may suggest another approach. Either way, you have more information and perhaps a greater sense of control. You can feel more confident about the decisions you make, knowing that you’ve looked at all of your options.

It may take some time and effort to gather your medical records and see another doctor. Because some people with leukemia need treatment right away, ask your doctor whether you can delay treatment for a couple weeks.

Treatment Clinical Trials

For anyone thinking about cancer treatment, clinical trials are an option. Clinical trials are research studies that involve people. They are an option for all stages of cancer.

Cancer research has led to real progress in the treatment of leukemia. Because of research, adults and children with leukemia can look forward to a better quality of life and a better chance of living longer. Doctors continue to search for new and better ways to treat leukemia.

Like all other treatment options, clinical trials have possible benefits and risks. But, by looking closely at all options, including clinical trials, you are taking an active role in a decision that affects your life.

Even if you don’t benefit directly from the treatment being studied, you may still make an important contribution by helping doctors learn more about leukemia and how to control it. If you’re interested in being part of a clinical trial, talk with your doctor.

NCI’s website has a section called Learn About Clinical Trials. You can learn about:

  • What clinical trials are and why they are important
  • How your safety is protected
  • Who pays for clinical trials
  • What to think about if you’re deciding whether to ake part in a clinical trial
  • What to ask your doctor

In addition, NCI’s Cancer Information Service can answer your questions and provide information about clinical trials. Contact CIS at 1–800–4–CANCER (1–800–422–6237) or at LiveHelp. You can search for clinical trials of leukemia at on NCI’s website.

Watchful Waiting

Your doctor may suggest watchful waiting if you’re diagnosed with chronic lymphocytic leukemia (CLL) but you don’t have symptoms. Watchful waiting means delaying treatment until you have symptoms. The purpose is to avoid the side effects of treatment as long as possible.

If you and your doctor agree that watchful waiting is a good idea, you’ll get exams and blood tests every 3 to 6 months. Your doctor may suggest starting treatment if you develop symptoms.

Some people worry that waiting to start treatment may reduce the chance to control leukemia before it gets worse. Having regular checkups reduces this risk.

If you choose watchful waiting but later become concerned about delaying treatment, you should talk with your doctor. You can change your mind and have treatment at any time.

Questions you may want to ask your doctor about watchful waiting
  • Is it safe for me to delay treatment? Does it mean I won’t live as long as if I started treatment right away?
  • Can I change my mind later on?
  • How often will I have checkups?
  • How will we know if the leukemia is getting worse?


Most people with leukemia are treated with chemotherapy. Chemotherapy uses drugs to kill leukemia cells.

Several drugs are used for leukemia, and they may be given in different ways. The drugs used for leukemia may be given through a thin needle directly into a vein (intravenously) and as pills and liquids that you swallow. The drugs enter the bloodstream and can kill leukemia cells almost all over the body.

However, many drugs given directly into a vein or taken by mouth can’t pass through the tightly packed blood vessel walls found in the brain and spinal cord. If leukemia affects the brain or spinal cord, the drugs may be given through a needle into the fluid that fills the spaces in and around the brain and spinal cord. This method is known as intrathecal chemotherapy, and it’s given in two ways:

  • Into the spinal fluid: The doctor may inject drugs into the spinal fluid. Injections into the spinal fluid can be painful.
  • Under the scalp: The surgeon may place a device known as an Ommaya reservoir under the scalp during surgery. The doctor injects drugs into the device. This method usually doesn’t hurt. The doctor may suggest this method when many doses of intrathecal chemotherapy are planned.

You may receive chemotherapy in a clinic, at the doctor’s office, or at home. Some people need to stay in the hospital during treatment.

The side effects depend mainly on which drugs are given and how much. Chemotherapy kills fast-growing leukemia cells, but the drugs can also harm normal cells that divide rapidly:

  • Blood cells: When drugs lower the levels of healthy blood cells, you’re more likely to get infections, bruise or bleed easily, and feel very weak and tired. Your health care team will check for low levels of blood cells. If your levels are low, your health care team may stop the chemotherapy for a while, reduce the dose of the drug, or give you a blood transfusion. They may also give you medicines that help your body to make new blood cells.
  • Cells in hair roots: Chemotherapy may cause hair loss. If you lose your hair, it will grow back after treatment, but the color and texture may be changed.
  • Cells that line the digestive tract: Chemotherapy can cause a poor appetite, nausea and vomiting, diarrhea, or mouth blisters. Your health care team can give you medicines and suggest other ways to help with these problems.

Ask whether chemotherapy could make you unable to have children. Chemotherapy can cause infertility in adults, but most children treated with chemotherapy for leukemia seem to have normal fertility when they grow up. If teens or adults want to have a child someday after treatment, they may choose to store sperm or eggs before treatment starts.

The NCI booklet Chemotherapy and You has helpful ideas for coping with chemotherapy side effects.

Targeted Therapy

Targeted therapies are drugs that can block the growth of leukemia cells. For example, a targeted therapy may block the action of an abnormal protein that causes leukemia cells to grow.

Several targeted therapies are used for leukemia. The type of targeted therapy depends on the type of leukemia:

  • For CML and ALL: People with the Philadelphia chromosome may receive a targeted therapy. Nearly everyone with CML (chronic myeloid leukemia) and some people with ALL (acute lymphocytic leukemia) have this chromosome. The targeted therapy used for CML and ALL is a pill that you swallow. Possible side effects include nausea, diarrhea, rash, and swelling. This drug may also cause headache, hair loss, or joint pain.
  • For CLL: People who have CLL (chronic lymphocytic leukemia) may receive a targeted therapy that is different from the one used for CML and ALL. This drug is given directly into a vein through a thin needle. It may cause nausea, vomiting, diarrhea, night sweats, or joint pain. Side effects usually go away after treatment ends.

You may want to read the NCI fact sheet Targeted Cancer Therapies to learn more about targeted therapies for leukemia.

Questions you may want to ask your doctor about chemotherapy or targeted therapy
  • Which drug or drugs do you suggest for me? What will they do?
  • What are the possible side effects? What can we do about them?
  • When will treatment start? When will it end? How often will I have treatments?
  • How will we know the treatment is working?
  • Will there be lasting side effects?

Radiation Therapy

Some people with leukemia receive radiation therapy along with chemotherapy. You’ll lie down on a treatment table, and a large machine will aim high-energy rays at your body to kill cancer cells. The machine may be aimed at the brain or other parts of the body where leukemia cells have been found. Or, the machine may be aimed at the whole body.

You’ll go to a hospital or clinic for treatment. Radiation therapy for the brain or other areas is usually given 5 days a week for several weeks. Radiation therapy for the whole body is given once or twice a day for a few days, usually before a stem cell transplant.

Side effects depend mainly on how much radiation is given and the part of your body that is treated. Ask your health care team what to expect. Side effects may develop during radiation therapy or months or years later.

Radiation therapy aimed at the brain may cause you to feel tired or to lose hair from your head. Your health care team can suggest ways to manage these problems, which usually go away when treatment ends. However, some side effects, such as memory loss or other problems, may be permanent.

It’s common for skin in the treated area to become red, dry, and itchy. Check with your doctor before using lotion or cream on that area. After treatment is over, the skin will slowly heal.

You’re likely to become tired during radiation therapy, especially in the later weeks of treatment. Although getting enough rest is important, most people say they feel better when they exercise every day. Try to go for a short walk, do gentle stretches, or do yoga.

The NCI booklet Radiation Therapy and You has helpful ideas for coping with radiation therapy side effects.

Questions you may want to ask your doctor about radiation therapy
  • Why do I need this treatment?
  • When will treatment start? When will it end? How often will I have treatments?
  • How will we know the treatment is working?
  • What side effects should I expect? What should I tell you about?
  • Are there any lasting effects?

Stem Cell Transplant

Some people with leukemia receive a stem cell transplant. This treatment is done after radiation therapy aimed at the whole body, a large dose of chemotherapy, or both. Radiation therapy and chemotherapy will destroy both leukemia cells and normal blood stem cells in the bone marrow.

Stem cell transplants take place in the hospital. You may need to stay in the hospital for several weeks.

To replace the normal blood stem cells that are destroyed by radiation therapy and chemotherapy, you’ll receive healthy blood stem cells through a vein. It’s like getting a blood transfusion. The transplanted stem cells will move to the bone marrow and make new blood cells.

People who receive stem cells from a donor, such as a sister or brother, may get graft-versus-host disease. The donated white blood cells may react against your normal issues and harm your liver, skin, or digestive tract. This disease can be mild or very severe. It can occur any time after the transplant, even years later. Steroids or other drugs may help.

You may want to read the NCI fact sheet Bone Marrow Transplantation and Peripheral Blood Stem Cell Transplantation. It tells about the types of transplants and their side effects.

Questions you may want to ask your doctor about stem cell transplants
  • What kind of stem cell transplant do you recommend? Where will the stem cells come from?
  • How long will I be in the hospital? Will I need special care? How will I be protected from germs?
  • What care will I need when I leave the hospital?
  • What are the risks and side effects of this treatment? What can we do about them?
  • How will we know if the treatment is working?
  • What is my chance of a full recovery? How long will that take?

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