Treatment of leukemia

To determine the treatment most likely to work for your leukemia, your doctor will consider your type of leukemia and its classification. Most cancer types are staged based on the size of the tumor, whether lymph nodes contain cancer, and whether the cancer has spread beyond the original site. Leukemia is different because it typically involves the blood and the bone marrow in a person’s body. Because of this, your doctors will use several different tests on your leukemia cells to help classify the disease accurately; this will help select treatment at the beginning and also later on after the initial phase of treatment is completed. Some “chronic” leukemias are still staged similarly to solid tumors, for the purpose of predicting outcomes and deciding when to start treatment.

Other factors that affect treatment options include your age and symptoms, whether leukemia cells are found in cerebrospinal fluid (the fluid in and around the spinal cord and brain), whether your leukemia was previously treated and whether the disease has progressed.

Treatment options for leukemia (depending on the type of leukemia) include watchful waiting, chemotherapy, targeted therapy, immunotherapy, stem cell transplantation and (less commonly) radiation therapy. Participating in a clinical trial also may be a treatment option for you too. Ask your doctor about any clinical trials for which you may qualify.

Different treatments can cause different side effects. However, not everyone getting the same treatment will experience the same side effects. Talk with your doctor about possible side effects to help you make the most informed treatment decision.

Watchful Waiting

People who have certain chronic forms of leukemia with no symptoms may not need immediate treatment. Waiting to start treatment until after symptoms begin is called watchful waiting. It allows people to avoid the side effects of treatment as long as possible.

During watchful waiting, people should have regular checkups with their doctor to look for symptoms. These doctor visits are important because treatment will need to start as soon as the disease progresses.


Chemotherapy drugs are used to stop the growth of cancer either by killing cancer cells or by preventing them from dividing and growing. Chemotherapy is sometimes referred to as conventional chemotherapy to distinguish it from targeted therapy.

The goal of chemotherapy for leukemia is complete remission of the disease, which occurs when all signs and symptoms of leukemia are gone and only normal cells are found in the blood and bone marrow. Remission means that the disease cells are not detectable under the microscope and that production of normal blood cells has been restored. Many people with leukemia receive chemotherapy as a part of their treatment plan. Chemotherapy may consist of a single drug or multiple drugs given in combination. Chemotherapy drugs are either taken by mouth, administered into a vein (IV) or injected under the scalp or through the back into the cerebrospinal fluid. Oral drugs (those taken by mouth) can be taken at home. Other types of chemotherapy are typically administered in a doctor’s office, clinic or hospital. Chemotherapy is usually given in cycles that consist of a treatment period followed by a break to allow normal cells to recover.

Chemotherapy can cause side effects because it works by attacking cells that divide quickly. These cells include not only cancer cells but also normal cells that divide quickly, such as those in the hair follicles and the lining of the mouth and intestines. When these normal cells are damaged, side effects occur. Some common side effects of chemotherapy include hair loss, mouth sores, loss of appetite, nausea and vomiting, skin and nail changes, fatigue and an increased risk of infection.

Targeted Therapy

Targeted therapy uses drugs to block the growth of leukemia cells by targeting specific parts of leukemia cells. For example, tyrosine kinase inhibitors target the proteins created by a specific chromosomal abnormality found in some leukemia cells. These proteins help the leukemia cells grow. By targeting the proteins, tyrosine kinase inhibitors stop the proteins from working, which helps stop the leukemia cells from growing.

Targeted therapy is most commonly used for chronic myeloid leukemia (CML), chronic lymphocytic leukemia (CLL) and some forms of acute lymphocytic leukemia (ALL). Targeted therapy drugs currently approved to treat leukemia are given as pills taken by mouth.

Common side effects of targeted therapy drugs include diarrhea, nausea, fatigue, constipation, cough, respiratory infections, low levels of red blood cells (anemia), low levels of white blood cells (neutropenia) and low levels of platelets (thrombocytopenia).


Immunotherapy uses the body’s own immune system to fight cancer. It 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 ALL and CLL. These are laboratory-made versions of antibodies, or proteins made by the immune system. They are designed to attach to substances on the surface of cancer cells, which stops the cells from growing. Interferon is a kind of immunotherapy sometimes used to treat CML. Many other immunotherapies for leukemia are being studied in clinical trials.

Monoclonal antibodies are usually given as an injection into a vein (IV). Interferon is most often given as an injection into the skin.

Side effects of immunotherapy drugs can include itching, chills, fever, nausea, rashes and fatigue.

Stem Cell Transplantation

Stem cell transplantation (also known as a bone marrow transplant) is an infusion of healthy stem cells into the body. The healthy cells can be collected from blood or bone marrow from the patient. It can also be collected from umbilical cord blood, a family member or another donor. Autologous stem cell transplants are transplants of the patient’s own stem cells, and allogeneic stem cell transplants are those from a donor whose tissue matches that of the patient’s (see Figure 1).

Figure 1


Allogeneic transplants are the most common type of transplant used to treat most forms of leukemia. In fact, stem cell transplantation from a donor is most accurately described as a form of immunotherapy, because the donor cells can attack residual leukemia cells (whether or not they are detectable or simply at high risk to relapse). For most patients, this type of transplant is preferable to an autologous transplant because the success of an autologous transplant depends only on the intensity of the chemotherapy; if the leukemia cells are resistant to even high-dose chemotherapy, an autologous transplant will not be curative. The best chance for a close tissue match for a donor transplant is a sibling, but the chance of a sibling match is 25 percent. Often, no family members are a tissue match to the patient. In these cases, a matched unrelated donor (MUD) may be necessary. To find a volunteer MUD, your doctor will most likely reach out to a marrow registry, such as Be the Match (see Lifesaving Donations above). Umbilical cord stem cells may also be an option in some cases. Increasingly, use of a half-matched (called haploidentical) family member as a donor is an effective option, but the transplant process is unique in these cases to try to prevent donor vs. recipient rejection (called graft-versus-host-disease, GVHD).

Prior to stem cell transplantation, most patients receive high-dose chemotherapy to kill cancer cells in the bone marrow. For people unable to tolerate high-dose chemotherapy, a reduced-intensity transplant may be an option. With this option, lower doses of chemotherapy and radiation therapy are used before the transplant. After either high-dose or reduced-dose chemotherapy, healthy stem cells are given through a catheter in a large vein in the neck or chest, much like a blood transfusion. The infused stem cells allow healthy new blood cells to develop in the bone marrow.

The high-dose chemotherapy before a stem cell transplant causes the usual side effects of chemotherapy. Infection can happen after stem cell transplantation. People receiving any type of allogeneic transplant may have GHVD, which occurs when the donor cells attack the patient’s cells.

Radiation Therapy

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

When radiation therapy is used for leukemia, it is usually given as external-beam radiation therapy, during which a machine delivers radiation to a specific part of the body. Your health care team will take careful measurements to determine the best position and dosage of the radiation to avoid damaging healthy cells.

Side effects of radiation therapy depend largely on the part of the body treated, but fatigue is common. Skin changes and hair loss can occur in the treated area. Radiation to the head can irritate the lining of the mouth, and radiation to the pelvis can cause diarrhea and nausea and vomiting.


Marrow registry – Be the Match

The National Marrow Donor Program operates Be the Match (https://bethematch.org), 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


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