Treatment of Leukemia

After diagnosing leukemia, your doctor will determine the subtype of leukemia and its classification. Factors that will also be considered include your age and symptoms, whether evaluation should occur for leukemia cells in the cerebrospinal fluid (the fluid in and around the spinal cord and brain), whether you received previous treatment for your leukemia and whether the disease has progressed. This information helps your doctor determine the best treatment options for you (see Forms of Leukemia).

This is an ideal time to seek a second opinion. Different specialists offer various levels of expertise and experience, and each may favor a different approach.

Watchful Waiting

People who have certain chronic forms of leukemia with no symptoms may not need immediate treatment, allowing them to postpone treatment-related side effects, hopefully without affecting their outcome.

If your doctor recommends watchful waiting, you should have regular checkups to look for signs and symptoms and to monitor blood counts. Treatment should begin as soon as the disease progresses or symptoms appear.


Chemotherapy is the use of drugs to destroy cancer cells by preventing them from growing and dividing. This form of treatment is known as systemic therapy, meaning the drugs travel through the bloodstream and affect cells throughout the entire body. Chemotherapy drugs work by attacking cancer cells that grow and multiply quickly. In doing so, they often damage healthy cells that also grow and multiply rapidly, resulting in side effects. Most people with leukemia receive some form of chemotherapy, which may consist of a single drug or multiple drugs given in combination. It may also be combined with other types of treatments, including targeted therapies or immunotherapies.

The goal of chemotherapy during leukemia treatment is complete remission of the disease, which means that all signs and symptoms of leukemia are gone and only normal cells are found in the blood and bone marrow.

Targeted Therapy

Like chemotherapy, targeted therapy uses drugs to block the growth of leukemia cells. However, instead of destroying all rapidly dividing cancerous and healthy cells, targeted treatments work by attacking a specific component of the leukemia cells, such as a mutated gene or an abnormal chromosome. For example, certain drugs target the proteins created by specific chromosomal or gene abnormalities found in some leukemia cells. These proteins help the leukemia cells grow. By targeting the proteins, these targeted therapy drugs stop the proteins from working, which helps stop the leukemia cells from growing. This allows your medical team to control the disease while limiting damage to healthy cells.


Immunotherapy is a treatment that works with or stimulates a person’s own immune system to recognize and destroy cancer cells. Training the immune system to respond to cancer has the potential for a more lasting response that can extend beyond the end of treatment. Immunotherapies can be used in combination with other treatments, as a maintenance therapy or by itself.

Monoclonal antibodies are one kind of immunotherapy used to treat certain types of leukemia. They are substances produced naturally by the body or made in a lab, and help the body’s immune system’s response to infections and disease. They are designed to attach to substances, called antigens, on the surface of cancer cells, which stops the cells from growing. One of the newest types of immunotherapy, chimeric antigen receptor (CAR) T-cell therapy, allows physicians to modify a patient’s own T-cells by adding a new gene that enables them to recognize and kill cancer cells. This one-time therapy is uniquely designed for each patient and is currently approved for patients up to 25 years of age who have refractory or relapsed B-cell precursor acute lymphocytic leukemia. Many other immunotherapies for leukemia are being studied in clinical trials.

Stem Cell Transplantation

Stem cell transplantation (also known as bone marrow transplantation) is an infusion of healthy stem cells into the body. The healthy cells can be collected from blood or bone marrow from the patient, a family member or another donor (see Figure 1). Umbilical cord blood that has been previously collected from a donated cord from a family (with no harm to baby or mother) and stored in a bank is also a potential option.

This procedure uses stem cells from a donor whose tissue type matches that of the patient. If available, a brother or sister could be a close tissue match because siblings share similar genes (there is about a 25 percent chance of each sibling being a complete match). If no family members are a tissue match to the patient, a matched unrelated donor (MUD) or matched donated umbilical cord blood may be found through a national registry.

Figure 1


Radiation Therapy

Radiation therapy involves the use of high-energy beams or particles to destroy cancer cells. It is not frequently used to treat leukemia, but some people may receive radiation to areas of the body where leukemia cells have accumulated, such as the spleen or brain. Others may receive radiation to the entire body (total body irradiation) before stem cell transplantation. It can also improve bone pain in certain situations.

When radiation therapy is used for leukemia, it is usually given as external-beam radiation therapy, during which radiation is directed at the cancer cells from an external source (a machine outside the body).

Clinical Trials

Many of the advances in cancer treatment are helping to save lives today because of the research conducted through clinical trials. They may offer opportunities for people with cancer to access cutting-edge treatments that are not yet widely available. Ask your doctor if you are a candidate for a clinical trial, and use the resources here to learn more about trials that are currently available.

Side Effects

Each type of treatment can cause different side effects. However, not everyone receiving the same treatment will experience the same effects. Doing your own research and talking to your doctor about the available options, along with your treatment goals and what to expect, will help you make the best treatment decisions (see Side Effects).

Pain Management

Diagnostic procedures, treatments and the disease itself may cause different types of pain. Although you can’t expect to be entirely free from pain, you can expect your health care team to do everything possible to make sure you’re comfortable. The more you share about how you are feeling, the better they will be able to help you.


Marrow registry – Be the Match

The National Marrow Donor Program operates Be the Match, the largest marrow registry in the world. For patients diagnosed with blood cancers and diseases, stem cell transplantation may be the best option for treatment and cure. The first option in finding a match is to seek someone in a person’s family, with the best chance being a sibling. However, 70 percent of patients in need of a stem cell transplant do not have a familial match donor available, leaving many people in need of an unrelated donor. Because of this, organizations such as Be the Match have created registries with millions of potential donors willing to donate bone marrow to someone in need. Marrow and stem cell donations can be collected from blood through a peripheral blood stem cell (PBSC) donation, bone marrow or umbilical cord blood.

For more information on becoming a donor for someone in need, visit https://bethematch.org


Additional Resources


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