Treatment

The treatment of ALL depends on its classification and the patient’s overall health. In many cases, a team of doctors will work with the patient to determine the best treatment plan.

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

Descriptions of the most common treatment options for ALL are listed below.

Chemotherapy

Chemotherapy is the use of drugs to kill cancer cells. Systemic chemotherapy is delivered through the bloodstream, targeting 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 blood diseases. Some people may receive chemotherapy in their doctor's office; others may go to the hospital. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a specific time.

Patients with ALL receive several different drugs throughout their treatment period, 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:

Induction. This stage of treatment refers to the initial course of treatment given during the first three to four weeks. It is designed to destroy most of the detectable leukemia cells, eliminate symptoms of the disease, and restore normal blood counts. The goal of induction therapy is a complete remission (CR), which means that the blood counts have returned to normal, the leukemia is not visible when a marrow sample is examined under the microscope, and the signs and symptoms related to the ALL are gone. A CR is accomplished in more than 95% of children and 75% to 80% of adults with ALL. However, it is known that small amounts of leukemia remain, and it is necessary to give additional therapy to prevent the ALL from coming back.

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

Maintenance therapy. This stage of therapy refers to treatment 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 of therapy is used to treat ALL if it has come back after treatment.

Central nervous system prophylaxis (preventive treatment). This treatment involves the use of drugs, given directly in the spinal fluid by spinal tap 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 and supportive treatment

Induction therapy usually begins in the hospital and often requires a hospitalization of three to four weeks. However, depending on the circumstances, many patients can leave the hospital and are monitored closely as outpatients. Hospitalization is sometimes needed to give consolidation therapy, but patients are generally treated as outpatients thereafter. Maintenance therapy rarely requires hospitalization; many patients with ALL can return to school or work while receiving maintenance therapy.

Because chemotherapy attacks rapidly dividing cells, including those in normal tissues 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 require transfusions of red blood cells and platelets at some point during their treatment. Treatment with antibiotics to prevent or treat infection is usually required 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 disturbances in blood clotting, and cause fatigue by lowering the number of red blood cells, which carry oxygen. Chemotherapy may affect fertility (ability to conceive a child or maintain a pregnancy) and increase the risk of developing a second cancer.

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

In addition to standard chemotherapy, targeted therapy directed against faulty genes or proteins that contribute to cancer growth and development is sometimes recommended, especially for Philadelphia chromosome-positive ALL (Ph+ ALL). Such drugs include imatinib (Gleevec), dasatinib (Sprycel), and nilotinib (Tasigna) for Philadelphia chromosome-positive ALL, and nelarabine (Arranon) for the treatment of T-cell ALL. Learn more about targeted treatments.

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to kill cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. In ALL, radiation therapy to the brain is sometimes used to kill 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 diseased bone marrow is replaced by highly specialized cells, called hematopoietic stem cells. Hematopoietic stem cells are found both in the bloodstream and in the bone marrow. Today, this procedure is more commonly called a stem cell transplant, rather than bone marrow transplant, because blood stem cells are typically what is being transplanted, not the actual bone marrow tissue.

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 an ALLO transplant, stem cells are obtained from a donor whose tissue matches the patient’s on a genetic level; this testing is called HLA-typing. Most often, a patient’s brother or sister serves as the donor, although unrelated donors can serve as the donor too. Millions of people worldwide have volunteered to donate stem cells for patients who do not have matched family members; your health care team will search a computer registry to look for a match. In addition, stem cells derived from umbilical cord blood are sometimes used if family donors are not available.

In an AUTO transplant, the patient’s own stem cells are used. The stem cells are obtained from the patient when he or she is in remission from previous treatment. The stem cells are then frozen until they are needed after the high-dose treatment (explained below) is completed. AUTO transplants are typically 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 have replacement blood stem cells 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. This also destroys the patient’s bone marrow tissue and suppresses the patient’s immune system so that, in an ALLO transplant, the donor cells are not rejected by the body. After the high-dose treatment is given, blood stem cells are infused into the patient’s vein to replace the bone marrow and restore normal blood counts from donor cells. Sometimes, ALLO transplants can also be performed after giving lower doses of chemotherapy and/or radiation therapy that are still sufficient to suppress the immune system and allow growth of the donor cells. (These transplants, sometimes termed “mini-transplants” or “reduced intensity transplants” have less immediate side effects, allowing the procedure to be used for older patients.)

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.

For both ALLO and AUTO transplant types, the replacement cells engraft (begin to make new blood cells) and turn into healthy, blood-producing tissue in 10 days to three weeks. Destroying the patient’s own marrow reduces the body’s natural defenses, temporarily leaving the patient at an increased risk of infection. Until the patient’s immune system is back to normal, patients may need antibiotics and blood transfusions.

In an ALLO transplant, another major risk is that the donor’s cells will recognize the patient’s body as foreign, causing graft-versus-host disease (GVHD). GVHD may be a serious complication of allogeneic transplants and can be fatal. Other side effects may include liver problems, diarrhea, infections, and rashes. However, GVHD can also be a benefit, in that the donor cells can recognize the cancer cells as foreign and destroy these cells, a mechanism that is one of the major reasons why ALLO transplantation generally works so well over the long term. The risk of GVHD can be reduced with exact HLA-type matching and the use of preventive drugs.

Learn more about bone marrow and stem cell transplantation.

Refractory/recurrent ALL

Refractory ALL occurs when a complete remission is not achieved because the drugs did not kill enough leukemia cells. Patients with refractory disease may be offered new drugs being tested in clinical trials or ALLO transplantation.

If the disease goes into remission but comes back later, it is called recurrent ALL. The treatment for recurrence depends on the length of remission. 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 transplant is often offered to patients whose leukemia has come back.

Find out more about common terms used during cancer treatment.