Watchful Waiting
Chemotherapy
Targeted Therapy
Biological Therapy
Radiation Therapy
Stem Cell Transplant

People with leukemia have many treatment options. The options are watchful waiting, chemotherapy, targeted therapy, biological therapy, radiation therapy, and stem cell transplant. If your spleen is enlarged, your doctor may suggest surgery to remove it. Sometimes a combination of these treatments is used.

The choice of treatment depends mainly on the following:

• The type of leukemia (acute or chronic)
  
  
• Your age
  
  
• Whether leukemia cells were found in your cerebrospinal fluid

It also may depend on certain features of the leukemia cells. Your doctor also considers your symptoms and general health.

People with acute leukemia need to be treated right away. The goal of treatment is to destroy signs of leukemia in the body and make symptoms go away. This is called a remission. After people go into remission, more therapy may be given to prevent a relapse. This type of therapy is called consolidation therapy or maintenance therapy. Many people with acute leukemia can be cured.

If you have chronic leukemia without symptoms, you may not need cancer treatment right away. Your doctor will watch your health closely so that treatment can start when you begin to have symptoms. Not getting cancer treatment right away is called watchful waiting.

When treatment for chronic leukemia is needed, it can often control the disease and its symptoms. People may receive maintenance therapy to help keep the cancer in remission, but chronic leukemia can seldom be cured with chemotherapy. However, stem cell transplants offer some people with chronic leukemia the chance for cure.

Your doctor can describe your treatment choices, the expected results, and the possible side effects. You and your doctor can work together to develop a treatment plan that meets your medical and personal needs.

You may want to talk with your doctor about taking part in a clinical trial, a research study of new treatment methods. See the Taking Part in Cancer Research ¹ section.

Your doctor may refer you to a specialist, or you may ask for a referral. Specialists who treat leukemia include hematologists, medical oncologists, and radiation oncologists. Pediatric oncologists and hematologists treat childhood leukemia. Your health care team may also include an oncology nurse and a registered dietitian.

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.

Before treatment starts, ask your health care team to explain possible side effects and how treatment may change your normal activities. Because cancer treatments often damage healthy cells and tissues, side effects are common. Side effects may not be the same for each person, and they may change from one treatment session to the next.

Watchful Waiting

People with chronic lymphocytic leukemia who do not have symptoms may be able to put off having cancer treatment. By delaying treatment, they can avoid the side effects of treatment until they have symptoms.

If you and your doctor agree that watchful waiting is a good idea, you'll have regular checkups (such as every 3 months). You can start treatment if symptoms occur.

Although watchful waiting avoids or delays the side effects of cancer treatment, this choice has risks. It may reduce the chance to control leukemia before it gets worse.

You may decide against watchful waiting if you don't want to live with an untreated leukemia. Some people choose to treat the cancer right away.

If you choose watchful waiting but grow concerned later, you should discuss your feelings with your doctor. A different approach is nearly always available.

Chemotherapy

Many people with leukemia are treated with chemotherapy. Chemotherapy uses drugs to destroy leukemia cells.

Depending on the type of leukemia, you may receive a single drug or a combination of two or more drugs.

You may receive chemotherapy in several different ways:

• By mouth: Some drugs are pills that you can swallow.
  
  
• Into a vein (IV): The drug is given through a needle or tube inserted into a vein.
  
  
• Through a catheter (a thin, flexible tube): The tube is placed in a large vein, often in the upper chest. A tube that stays in place is useful for patients who need many IV treatments. The health care professional injects drugs into the catheter, rather than directly into a vein. This method avoids the need for many injections, which can cause discomfort and injure the veins and skin.
  
  
• Into the cerebrospinal fluid: If the pathologist finds leukemia cells in the fluid that fills the spaces in and around the brain and spinal cord, the doctor may order intrathecal chemotherapy. The doctor injects drugs directly into the cerebrospinal fluid. Intrathecal chemotherapy is given in two ways:
  • Into the spinal fluid: The doctor injects the drugs into the spinal fluid.
  • Under the scalp: Children and some adult patients receive chemotherapy through a special catheter called an Ommaya reservoir. The doctor places the catheter under the scalp. The doctor injects the drugs into the catheter. This method avoids the pain of injections into the spinal fluid.
    
    
  Intrathecal chemotherapy is used because many drugs given by IV or taken by mouth can't pass through the tightly packed blood vessel walls found in the brain and spinal cord. This network of blood vessels is known as the blood-brain barrier.

Chemotherapy is usually given in cycles. Each cycle has a treatment period followed by a rest period.

You may have your treatment in a clinic, at the doctor's office, or at home. Some people may need to stay in the hospital for treatment.

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

• Blood cells: When chemotherapy lowers the levels of healthy blood cells, you're more likely to get infections, bruise or bleed easily, and feel very weak and tired. You'll get blood tests to check for low levels of blood cells. If your levels are low, your health care team may stop the chemotherapy for a while or reduce the dose of drug. There also are medicines that can help your body make new blood cells. Or, you may need a blood transfusion.
  
  
• Cells in hair roots: Chemotherapy may cause hair loss. If you lose your hair, it will grow back, but it may be somewhat different in color and texture.
  
  
• Cells that line the digestive tract: Chemotherapy can cause poor appetite, nausea and vomiting, diarrhea, or mouth and lip sores. Ask your health care team about medicines and other ways to help you cope with these problems.
  
  
• Sperm or egg cells: Some types of chemotherapy can cause infertility.
  • Children: Most children treated for leukemia appear to have normal fertility when they grow up. However, depending on the drugs and doses used and the age of the patient, some boys and girls may be infertile as adults.
  • Adult men: Chemotherapy may damage sperm cells. Men may stop making sperm. Because these changes to sperm may be permanent, some men have their sperm frozen and stored before treatment (sperm banking).
  • Adult women: Chemotherapy may damage the ovaries. Women may have irregular menstrual periods or periods may stop altogether. Women may have symptoms of menopause, such as hot flashes and vaginal dryness. Women who may want to get pregnant in the future should ask their health care team about ways to preserve their eggs before treatment starts.

You may find it helpful to read NCI's booklet Chemotherapy and You ².

Targeted Therapy

People with chronic myeloid leukemia and some with acute lymphoblastic leukemia may receive drugs called targeted therapy. Imatinib (Gleevec) tablets were the first targeted therapy approved for chronic myeloid leukemia. Other targeted therapy drugs are now used too.

Targeted therapies use drugs that block the growth of leukemia cells. For example, a targeted therapy may block the action of an abnormal protein that stimulates the growth of leukemia cells.

Side effects include swelling, bloating, and sudden weight gain. Targeted therapy can also cause anemia, nausea, vomiting, diarrhea, muscle cramps, or a rash. Your health care team will monitor you for signs of problems.

You may want to read the NCI fact sheet Targeted Cancer Therapies: Questions and Answers.

³Biological Therapy

Some people with leukemia receive drugs called biological therapy. Biological therapy for leukemia is treatment that improves the body's natural defenses against the disease.

One type of biological therapy is a substance called a monoclonal antibody. It's given by IV infusion. This substance binds to the leukemia cells. One kind of monoclonal antibody carries a toxin that kills the leukemia cells. Another kind helps the immune system destroy leukemia cells.

For some people with chronic myeloid leukemia, the biological therapy is a drug called interferon. It is injected under the skin or into a muscle. It can slow the growth of leukemia cells.

You may have your treatment in a clinic, at the doctor's office, or in the hospital. Other drugs may be given at the same time to prevent side effects.

The side effects of biological therapy differ with the types of substances used, and from person to person. Biological therapies commonly cause a rash or swelling where the drug is injected. They also may cause a headache, muscle aches, a fever, or weakness. Your health care team may check your blood for signs of anemia and other problems.

You may find it helpful to read NCI's booklet Biological Therapy ⁴.

Radiation Therapy

Radiation therapy (also called radiotherapy) uses high-energy rays to kill leukemia cells. People receive radiation therapy at a hospital or clinic.

Some people receive radiation from a large machine that is aimed at the spleen, the brain, or other parts of the body where leukemia cells have collected. This type of therapy takes place 5 days a week for several weeks. Others may receive radiation that is directed to the whole body. The radiation treatments are given once or twice a day for a few days, usually before a stem cell transplant.

The side effects of radiation therapy depend mainly on the dose of radiation and the part of the body that is treated. For example, radiation to your abdomen can cause nausea, vomiting, and diarrhea. In addition, your skin in the area being treated may become red, dry, and tender. You also may lose your hair in the treated area.

You are likely to be very tired during radiation therapy, especially after several weeks of treatment. Resting is important, but doctors usually advise patients to try to stay as active as they can.

Although the side effects of radiation therapy can be distressing, they can usually be treated or controlled. You can talk with your doctor about ways to ease these problems.

It may also help to know that, in most cases, the side effects are not permanent. However, you may want to discuss with your doctor the possible long-term effects of radiation treatment.

You may find it helpful to read NCI's booklet Radiation Therapy and You ⁵.

Stem Cell Transplant

Some people with leukemia receive a stem cell transplant. A stem cell transplant allows you to be treated with high doses of drugs, radiation, or both. The high doses destroy both leukemia cells and normal blood cells in the bone marrow. After you receive highdose chemotherapy, radiation therapy, or both, you receive healthy stem cells through a large vein. (It's like getting a blood transfusion.) New blood cells develop from the transplanted stem cells. The new blood cells replace the ones that were destroyed by treatment.

Stem cell transplants take place in the hospital. Stem cells may come from you or from someone who donates their stem cells to you:

• From you: An autologous stem cell transplant uses your own stem cells. Before you get the high-dose chemotherapy or radiation therapy, your stem cells are removed. The cells may be treated to kill any leukemia cells present. Your stem cells are frozen and stored. After you receive high-dose chemotherapy or radiation therapy, the stored stem cells are thawed and returned to you.
  
  
• From a family member or other donor: An allogeneic stem cell transplant uses healthy stem cells from a donor. Your brother, sister, or parent may be the donor. Sometimes the stem cells come from a donor who isn't related. Doctors use blood tests to learn how closely a donor's cells match your cells.
  
  
• From your identical twin: If you have an identical twin, a syngeneic stem cell transplant uses stem cells from your healthy twin.

Stem cells come from a few sources. The stem cells usually come from the blood (peripheral stem cell transplant). Or they can come from the bone marrow (bone marrow transplant). Another source of stem cells is umbilical cord blood. Cord blood is taken from a newborn baby and stored in a freezer. When a person gets cord blood, it's called an umbilical cord blood transplant.

After a stem cell transplant, you may stay in the hospital for several weeks or months. You'll be at risk for infections and bleeding because of the large doses of chemotherapy or radiation you received. In time, the transplanted stem cells will begin to produce healthy blood cells.

Another problem is that graft-versus-host disease (GVHD) may occur in people who receive donated stem cells. In GVHD, the donated white blood cells in the stem cell graft react against the patient's normal tissues. Most often, the liver, skin, or digestive tract is affected. GVHD 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 find it helpful to read NCI's fact sheet Bone Marrow Transplantation and Peripheral Blood Stem Cell Transplantation: Questions and Answers ⁶.

Glossary Terms

A procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a genetically similar, but not identical, donor. This is often a sister or brother, but could be an unrelated donor.

A condition in which the number of red blood cells is below normal.

A procedure in which blood-forming stem cells (cells from which all blood cells develop) are removed, stored, and later given back to the same person.

Treatment to boost or restore the ability of the immune system to fight cancer, infections, and other diseases. Also used to lessen certain side effects that may be caused by some cancer treatments. Agents used in biological therapy include monoclonal antibodies, growth factors, and vaccines. These agents may also have a direct antitumor effect. Also called biological response modifier therapy, biotherapy, BRM therapy, and immunotherapy.

A network of blood vessels with closely spaced cells that makes it difficult for potentially toxic substances (such as anticancer drugs) to penetrate the blood vessel walls and enter the brain. Also called BBB.

A procedure to replace bone marrow that has been destroyed by treatment with high doses of anticancer drugs or radiation. Transplantation may be autologous (an individual's own marrow saved before treatment), allogeneic (marrow donated by someone else), or syngeneic (marrow donated by an identical twin).

A flexible tube used to deliver fluids into or withdraw fluids from the body.

Treatment with drugs that kill cancer cells.

A type of research study that tests how well new medical approaches work in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. Also called clinical study.

Treatment that is given after cancer has disappeared following the initial therapy. Consolidation therapy is used to kill any cancer cells that may be left in the body. It may include radiation therapy, a stem cell transplant, or treatment with drugs that kill cancer cells. Also called intensification therapy and postremission therapy.

A disease caused when cells from a donated stem cell graft attack the normal tissue of the transplant patient. Symptoms include jaundice, skin rash or blisters, a dry mouth, or dry eyes. Also called GVHD.

A doctor who specializes in treating blood disorders.

A drug used to treat different types of leukemia and other cancers of the blood, gastrointestinal stromal tumors, skin tumors called dermatofibrosarcoma protuberans, and a rare condition called systemic mastocytosis. It is also being studied in the treatment of other types of cancer. Imatinib mesylate blocks the protein made by the bcr/abl oncogene. It is a type of tyrosine kinase inhibitor. Also called Gleevec and STI571.

The complex group of organs and cells that defends the body against infections and other diseases.

The inability to produce children.

A method of putting fluids, including drugs, into the bloodstream. Also called intravenous infusion.

A biological response modifier (a substance that can improve the body's natural response to infections and other diseases). Interferons interfere with the division of cancer cells and can slow tumor growth. There are several types of interferons, including interferon-alpha, -beta, and -gamma. The body normally produces these substances. They are also made in the laboratory to treat cancer and other diseases.

Treatment in which anticancer drugs are injected into the fluid-filled space between the thin layers of tissue that cover the brain and spinal cord.

Treatment that is given to help keep cancer from coming back after it has disappeared following the initial therapy. It may include treatment with drugs, vaccines, or antibodies that kill cancer cells, and it may be given for a long time.

A doctor who specializes in diagnosing and treating cancer using chemotherapy, hormonal therapy, biological therapy, and targeted therapy. A medical oncologist often is the main health care provider for someone who has cancer. A medical oncologist also gives supportive care and may coordinate treatment given by other specialists.

The time of life when a woman’s ovaries stop producing hormones and menstrual periods stop. Natural menopause usually occurs around age 50. A woman is said to be in menopause when she hasn’t had a period for 12 months in a row. Symptoms of menopause include hot flashes, mood swings, night sweats, vaginal dryness, trouble concentrating, and infertility.

A type of protein made in the laboratory that can locate and bind to substances in the body, including tumor cells. There are many kinds of monoclonal antibodies. Each monoclonal antibody is made to find one substance. Monoclonal antibodies are being used to treat some types of cancer and are being studied in the treatment of other types. They can be used alone or to carry drugs, toxins, or radioactive materials directly to a tumor.

A device surgically placed under the scalp and used to deliver anticancer drugs to the fluid surrounding the brain and spinal cord.

A nurse who specializes in treating and caring for people who have cancer.

Having to do with children.

A method of replacing blood-forming cells destroyed by cancer treatment. Immature blood cells (stem cells) in the circulating blood that are similar to those in the bone marrow are given to the patient after treatment. This helps the bone marrow recover and continue producing healthy blood cells. Transplantation may be autologous (an individual's own blood cells saved earlier), allogeneic (blood cells donated by someone else), or syngeneic (blood cells donated by an identical twin). Also called peripheral stem cell support.

A doctor who specializes in using radiation to treat cancer.

The use of high-energy radiation from x-rays, gamma rays, neutrons, protons, and other sources to kill cancer cells and shrink tumors. Radiation may come from a machine outside the body (external-beam radiation therapy), or it may come from radioactive material placed in the body near cancer cells (internal radiation therapy). Systemic radiation therapy uses a radioactive substance, such as a radiolabeled monoclonal antibody, that travels in the blood to tissues throughout the body. Also called irradiation and radiotherapy.

A health professional with special training in the use of diet and nutrition to keep the body healthy. A registered dietitian may help the medical team improve the nutritional health of a patient.

The return of signs and symptoms of cancer after a period of improvement.

A decrease in or disappearance of signs and symptoms of cancer. In partial remission, some, but not all, signs and symptoms of cancer have disappeared. In complete remission, all signs and symptoms of cancer have disappeared, although cancer still may be in the body.

A problem that occurs when treatment affects healthy tissues or organs. Some common side effects of cancer treatment are fatigue, pain, nausea, vomiting, decreased blood cell counts, hair loss, and mouth sores.

Freezing sperm for use in the future. This procedure can allow men to father children after loss of fertility.

A method of replacing immature blood-forming cells that were destroyed by cancer treatment. The stem cells are given to the person after treatment to help the bone marrow recover and continue producing healthy blood cells.

A procedure in which a patient receives blood-forming stem cells (cells from which all blood cells develop) donated by his or her healthy identical twin.

A type of treatment that uses drugs or other substances, such as monoclonal antibodies, to identify and attack specific cancer cells. Targeted therapy may have fewer side effects than other types of cancer treatments.

The infusion of components of blood or whole blood into the bloodstream. The blood may be donated from another person, or it may have been taken from the person earlier and stored until needed.

Blood from the umbilical cord of a newborn baby. This blood contains high concentrations of stem cells (cells from which all blood cells develop).

The injection of umbilical cord blood to restore an individual's own blood production system suppressed by anticancer drugs, radiation therapy, or both. It is being studied in the treatment of cancer and severe blood disorders such as aplastic anemia. Cord blood contains high concentrations of stem cells (cells from which all blood cells develop).

Closely monitoring a patient's condition but withholding treatment until symptoms appear or change. Also called active surveillance, expectant management, and observation.