Treatment

The following are used to describe the state of disease for children with ALL:

Untreated ALL. The child hasn’t yet received any treatment.

ALL in remission. There are normal levels of white and other blood cells after treatment. Physical examinations, blood counts, and bone marrow aspirates show no detectable leukemia. Remission is the absence of signs or symptoms of leukemia.

Recurrent ALL. Recurrent ALL is cancer that comes back after the child has had some period of remission. The leukemia may recur in the bone marrow, spinal fluid, testicles (for boys), or, less commonly, other areas of the body. 

Refractory ALL. The leukemia did not go into remission, despite remission induction treatment (see below).

Treatment Overview

Clinical trials are the standard of care for the treatment of children with cancer. In fact, more than 60% of children with cancer are treated as part of a clinical trial. Clinical trials are research studies that compare standard treatments (the best treatments available) with newer treatments that may be more effective. Cancer in children is rare, so it can be hard for doctors to plan treatments unless they know what has been most effective in other children. Investigating new treatments involves careful monitoring using scientific methods, and all participants are followed closely to track progress.

To take advantage of these newer treatments, all children with cancer should be treated at a specialized cancer center. Doctors at these centers have extensive experience in treating children with cancer and have access to the latest research. Many times, a team of doctors treats a child with cancer. Pediatric cancer centers often have extra support services for children and their families, such as nutritionists, social workers, and counselors. Special activities for kids with cancer may also be available.

Three types of treatments are used to treat ALL for children: chemotherapy, radiation treatment, and stem cell transplantation/bone marrow transplantation. Sometimes, these treatments are used in combination. Each treatment option is described 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 the primary treatment for ALL. Several drugs are used in chemotherapy for ALL. Chemotherapy may be given by mouth, injected into a vein or muscle, or injected into the CSF.

Chemotherapy is generally done in four phases:

Remission induction therapy uses chemotherapy to kill as many of the cancer cells as possible to cause the cancer to go into remission.

Central nervous system directed therapy kills any cancer cells in the central nervous system and prevents the spread of the cancer to the spinal fluid.

Consolidation therapy begins once the child’s cancer has gone into remission. Higher doses of chemotherapy, or drugs not used during previous treatment, are used to kill the majority of the remaining cancer cells.

Continuation or maintenance therapy lasts for two to three years to kill any residual leukemia.

The side effects of chemotherapy depend on the individual and the dose used, but can include fatigue, risk of infection, nausea and vomiting, loss of appetite, bone and joint damage, and diarrhea. Less commonly, chemotherapy may cause learning problems and liver dysfunction. These side effects usually go away once treatment is finished. The severity of the side effects depends on the type and amount of the drug being given and the length of time the child receives the drug. The degree to which children experience side effects may also be affected by other factors, including genetic differences in the way the medications are metabolized, the child or teen’s age when diagnosed, and their overall health and well-being.

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 your child, their purpose, and their potential side effects or interactions with other medications. Learn more about your prescriptions through Cancer.Net's Drug Information Resources, which provides links to searchable drug databases.

Radiation therapy is the use of high-energy x-rays or other particles to kill cancer cells. The most common type of radiation treatment is called external-beam radiation therapy, which is radiation given from a machine outside the body. Radiation therapy for ALL is generally used only when the cancer has spread to the brain, spinal fluid, or a boy’s testicles, or in high-risk disease to help prevent the spread of leukemia to the spinal fluid. Radiation therapy is more often used for patients with T-cell leukemia. 

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. However, long-term side effects of radiation treatment to the brain and body can occur and may potentially include learning disabilities and a risk of developing a brain tumor (see After Treatment).

Stem cell transplantation/bone marrow transplantation

Stem cell transplantation is most often used as a treatment for ALL after a child has had a second, third, or fourth remission. 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).

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; matches can be made by searching a computer registry. In addition, a donation of stem cells derived from umbilical cord blood is sometimes considered 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, usually after the high-dose treatment (explained below) is completed.

In both types, the goal of transplantation is to prevent the cancer cells in the marrow, blood, and other parts of the body from returning. The transplant allows the patient’s marrow to be replaced with healthy blood stem cells from another source. 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 mild or serious complication of allogeneic transplants, and can even 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 preventative drugs.

In an AUTO transplant, there is little risk of GVHD because the replacement stem cells are the patient’s own cells. However, there is a risk in an autologous transplant that some of the cells that are put back into the patient could still be cancerous.

Learn more by reading the Cancer.Net Feature series, Understanding Bone Marrow and Stem Cell Transplantation.

Recurrent ALL

Treatment for recurrent ALL depends on many factors, including the type of treatment the child received originally, the length of time between the initial diagnosis and the recurrence, and whether leukemic cells are found in the bone marrow, CSF, testicles, or in a combination of these sites at the time of recurrence. Doctors will plan treatment based on each child’s circumstances; just as for newly diagnosed patients, clinical trials typically offer the best chance of cure.

To learn about the terms used in this section, read the Cancer.Net Feature: Cancer Terms to Know: During Treatment.