Leukemia - Acute Lymphocytic - ALL: Treatment Options

This section has been reviewed and approved by the Cancer.Net Editorial Board, 08/2013

ON THIS PAGE: You will learn about the different ways doctors use to treat people with ALL. To see other pages, use the menu on the side of your screen.

This section outlines treatments that are the standard of care (the best proven treatments available) for this specific type of cancer. When making treatment plan decisions, patients are also encouraged to consider clinical trials as an option. A clinical trial is a research study to test a new treatment to evaluate whether it is safe, effective, and possibly better than the standard treatment. Your doctor can help you review all treatment options. For more information, see the Clinical Trials and Latest Research sections.

Treatment overview

In cancer care, different types of doctors often work together to create a patient’s overall treatment plan that combines different types of treatments. This is called a multidisciplinary team.

Descriptions of the most common treatment options for ALL are listed below. Treatment options and recommendations depend on several factors, including the subtype of ALL, its classification, possible side effects, and the patient’s preferences and overall health. Take time to learn about your treatment options and be sure to ask questions about things that are unclear. Also, talk about the goals of each treatment with your doctor and what you can expect while receiving the treatment. Learn more about making treatment decisions.

Chemotherapy

Chemotherapy is the use of drugs to kill cancer cells, usually by stopping the cancer cells’ ability to grow and divide. Systemic chemotherapy is delivered through the bloodstream to reach cancer cells throughout the body. Chemotherapy is given by a medical oncologist, a doctor who specializes in treating cancer with medication, or a hematologist, a doctor who specializes in treating blood disorders. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time.

Patients with ALL receive several different drugs throughout their treatment, some of which are given orally (by mouth) as a pill, while others are injected into a vein (intravenously, IV). A patient may receive chemotherapy during different stages of treatment:

Remission Induction therapy. This is the first round of treatment given during the first three to four weeks after diagnosis. It is designed to destroy most of the leukemia cells, stop symptoms of the disease, and return the blood counts to normal levels.

The specific treatments used may include:

  • Doxorubicin (Adriamycin), cyclophosphamide (Neosar), or vincristine (Vincasar), given by an injection into a vein
  • Asparaginase (Elspar), given by injection into a muscle, under the skin or into a vein
  • Dexamethasone (multiple brand names) or prednisone (multiple brand names) by mouth
  • Methotrexate (multiple brand names) or cytarabine (Cytosar-U) as an injection into the spinal fluid

The goal of induction therapy is a complete remission (CR), which means that the blood counts have returned to normal, the leukemia cannot be seen when a bone marrow sample is examined under the microscope, and the signs and symptoms of the ALL are gone. More than 95% of children and 75% to 80% of adults with ALL will have a CR. However, small amounts of leukemia can remain after treatment even if it cannot be seen with a microscope, and it is necessary to give additional therapy to prevent the ALL from coming back. 

Remission Consolidation or Intensification therapy. This stage of therapy refers to the use of different drugs given in doses similar to or higher than the doses used to achieve remission. Depending on the subtype of the ALL, the doctor may recommend several courses of consolidation therapy.

Remission Maintenance or Continuation therapy. Treatment is given both orally and intravenously over a two-year to three-year period to keep the ALL from returning. These drugs are usually given in lower doses and have fewer side effects.

Re-induction chemotherapy. This stage is used to treat ALL if it has come back after treatment.

Central nervous system (CNS) prophylaxis (preventive treatment). This is the use of drugs, given directly in the spinal fluid by spinal tap (lumbar puncture; see Diagnosis) and/or by vein, to prevent the leukemia from spreading from the blood to the brain or spinal cord. This treatment is often given in combination with radiation therapy (see below) to the head. 

Side effects of chemotherapy

Induction therapy usually begins in the hospital, and patients will often need to stay in the hospital for three to four weeks during treatment. However, depending on the situation, many patients can leave the hospital and are monitored closely with regular visits to the doctor. Sometimes, patients will need to stay in the hospital for consolidation therapy, but most patients are able to go home. Many patients with ALL can return to school or work while receiving maintenance therapy.

Because chemotherapy attacks rapidly dividing cells, including those in normal tissue such as the hair, lining of the mouth, intestines, and bone marrow, patients receiving chemotherapy may lose their hair, develop mouth sores, or have nausea and vomiting. Because of changes in the blood counts, most patients will need transfusions of red blood cells and platelets at some point during their treatment. Treatment with antibiotics to prevent or treat infection is usually needed as well. Chemotherapy may lower the body’s resistance to infection by reducing the number of neutrophils, lead to increased bruising and bleeding because of the decrease in the number of platelets and other problems with blood clotting, and cause fatigue by lowering the number of red blood cells.

Chemotherapy may affect fertility (ability to have a child in the future) and increase the risk of developing a second cancer. Patients may want to talk with a fertility specialist before treatment begins.

Learn more about chemotherapy and preparing for treatment. The medications used to treat cancer are continually being evaluated. Talking with your doctor is often the best way to learn about the medications prescribed for you, their purpose, and their potential side effects or interactions with other medications. Learn more about your prescriptions by using searchable drug databases.

Targeted therapy

Targeted therapy is a treatment that targets the cancer’s specific genes, proteins, or the tissue environment that contributes to cancer growth and survival. This type of treatment blocks the growth and spread of cancer cells while limiting damage to normal cells.

Recent studies show that not all cancers have the same targets. To find the most effective treatment, your doctor may run tests to identify the genes, proteins, and other factors involved in your leukemia. As a result, doctors can better match each patient with the most effective treatment whenever possible. In addition, many research studies are taking place now to find out more about specific molecular targets and new treatments directed at them. Learn more about targeted treatments.

For ALL, targeted therapy is recommended in addition to standard chemotherapy for patients with Philadelphia chromosome-positive ALL (Ph+ ALL). Such drugs include imatinib (Gleevec), dasatinib (Sprycel), nilotinib (Tasigna), ponatinib (Iculsig) for Philadelphia chromosome-positive ALL. Currently, the safety of ponatinib is being re-evaluated by the FDA because of the risk of life-threatening blood clots and narrowing of blood vessels. Patients taking this drug should talk with their doctors about the risks and benefits of continuing to take the drug, as other treatment options may be available.

Nelarabine (Arranon) is a new drug that targets T-cell ALL. Monoclonal antibodies such as rituximab (Rituxan) are used in addition to chemotherapy for the treatment of B-cell ALL. Talk with your doctor about possible side effects for a specific medication and how they can be managed.

Radiation therapy

Radiation therapy is the use of high-energy x-rays to kill cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. A radiation therapy regimen usually consists of a specific number of treatments given over a set period of time. For ALL, radiation therapy to the brain is sometimes used to destroy cancerous cells around the brain and spinal column.

Side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most side effects go away soon after treatment is finished. Learn more about radiation therapy.

Stem cell transplantation/bone marrow transplantation

A stem cell transplant is a medical procedure in which bone marrow that contains leukemia is destroyed and then replaced by highly specialized cells, called hematopoietic stem cells, that develop into healthy bone marrow. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. These stem cells make all of the normal cells in the blood. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because it is the stem cells in the blood that are typically being transplanted, not the actual bone marrow tissue.

Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of cancer, results of any previous treatment, and patient’s age and general health.

There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO). However, AUTO transplants are generally not used to treat ALL.

In both types, the goal of transplantation is to destroy cancer cells in the marrow, blood, and other parts of the body and allow replacement blood stem cells to create healthy bone marrow. In most stem cell transplants, the patient is treated with high doses of chemotherapy and/or radiation therapy to destroy as many cancer cells as possible. Learn more about bone marrow and stem cell transplantation.

Getting care for symptoms and side effects

ALL and its treatment often cause side effects. In addition to treatment to slow, stop, or eliminate the disease, an important part of care is relieving a person’s symptoms and side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.

Palliative care can help a person at any stage of illness. People often receive treatment for the leukemia and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, and other therapies. You may also receive palliative treatments similar to those meant to eliminate the leukemia, such as chemotherapy and radiation therapy. Talk with your doctor about the goals of each treatment in the treatment plan.

Before treatment begins, talk with your health care team about the possible side effects of your specific treatment plan and supportive care options. And during and after treatment, be sure to tell your doctor or another health care team member if you are experiencing a problem so it is addressed as quickly as possible. Learn more about palliative care.

Recurrent ALL

A remission is when ALL cannot be detected in the body and there are no symptoms. This may also be called “no evidence of disease” or NED. 

A remission can be temporary or permanent. This uncertainty leads to many survivors feeling worried or anxious that the disease will come back. While many remissions are permanent, it’s important to talk with your doctor about the possibility of the leukemia returning. Understanding the risk of recurrence and the treatment options may help you feel more prepared if the disease does return. Learn more about coping with the fear of recurrence.   

If the leukemia does return after the original treatment, it is called recurrent or relapsed leukemia. When this occurs, a cycle of testing will begin again to learn as much as possible about the recurrence, including whether the leukemia’s subtype and classification have changed. After testing is done, you and your doctor will talk about your treatment options. Often the treatment plan will include the therapies described above (such as chemotherapy, targeted therapy, and radiation therapy) but they may be used in a different combination or given at a different pace. Your doctor may also suggest clinical trials that are studying new ways to treat recurrent ALL.

Treatment for recurrent ALL depends on the length of the remission and is usually given in cycles for two to three years. If a recurrence occurs after a long remission, the leukemia may respond again to the original treatment. If the remission was short, then other drugs are used, often in the form of new drugs being tested in clinical trials. An ALLO stem cell transplant is generally recommended for patients whose leukemia has come back after a second remission is achieved. The drug clofarabine (Clolar) may be used for patients between ages 1 and 21 who have relapsed or refractory ALL after already receiving at least two types of chemotherapy. Supportive care will also be important to help relieve symptoms and side effects.

People with recurrent leukemia often experience emotions such as disbelief or fear. Patients are encouraged to talk with their health care team about these feelings and ask about support services to help them cope. Learn more about dealing with cancer recurrence.

Refractory ALL

Refractory ALL occurs when a complete remission is not achieved because the drugs did not kill enough leukemia cells. These patients often continue to have low blood counts, need transfusions, and have a risk of bleeding or infection.

Patients with this diagnosis are encouraged to talk with doctors who are experienced in treating this type of leukemia, because there can be different opinions about the best treatment plan. Learn more about seeking a second opinion before starting treatment, so you are comfortable with the treatment plan chosen. This discussion may include clinical trials.

Your health care team may recommend a treatment plan that includes new drugs being tested in clinical trials or ALLO stem cell transplantation. Supportive care will also be important to help relieve symptoms and side effects.

For most patients, a diagnosis of refractory leukemia is very stressful and, at times, difficult to bear. Patients and their families are encouraged to talk about the way they are feeling with doctors, nurses, social workers, or other members of the health care team. It may also be helpful to talk with other patients, including through a support group.

If treatment fails

Recovery from leukemia is not always possible. If treatment is not successful, the disease may be called advanced or terminal leukemia.

This diagnosis is stressful because the disease is not curable, and it may be difficult to discuss. However, it is important to have open and honest conversations with your doctor and health care team to express your feelings, preferences, and concerns. The health care team is there to help, and many team members have special skills, experience, and knowledge to support patients and their families. Making sure a person is physically comfortable and free from pain is extremely important.

Patients who have advanced leukemia and who are expected to live less than six months may want to consider a type of palliative care called hospice care. Hospice care is designed to provide the best possible quality of life for people who are near the end of life. You and your family are encouraged to think about where you would be most comfortable: at home, in the hospital, or in a hospice environment. Nursing care and special equipment can make staying at home a workable alternative for many families. Learn more about advanced cancer care planning.

After the death of a loved one, many people need support to help them cope with the loss. Learn more about grief and bereavement.

The next section helps explain clinical trials, which are research studies. Use the menu on the side of your screen to select About Clinical Trials, or you can select another section, to continue reading this guide.