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Childhood Acute Lymphoblastic Leukemia
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How We Treat Childhood Acute Lymphoblastic Leukemia (ALL)
Your child's physician will determine a specific course of treatment for childhood leukemia based on several factors, including:
Your child's age, overall health, and medical history
The type of ALL (B-cell or T-cell)
Certain aspects of the ALL, such as the number of white blood cells in the blood when the leukemia is diagnosed, whether or not leukemia cells are seen in the spinal fluid, and whether or not specific chromosomal abnormalities are found in the leukemia cell
How well the leukemia responds to the first few weeks of treatment
Your child's tolerance for certain medications, procedures, or therapies
Whether your child has Down syndrome
How your child's physician expects the disease to progress
Treatment for ALL is a long-term process. Chemotherapy and other treatment for the disease may take two years or more to complete.
There are a number of treatments that your child's physician may recommend. Some of them help to treat the leukemia, while others are intended to address complications of the disease or side effects of the treatment. Treatment usually begins by addressing your child's symptoms, such as anemia, bruising, and/or fever. In addition, treatment may include the following (alone or in combination):
Chemotherapy is a drug treatment that interferes with the cancer cell's ability to grow or reproduce. For some types of cancer, chemotherapy is used alone, while in others it is used in conjunction with other therapy, such as radiation or surgery. Chemotherapy is the standard first treatment for acute lymphoblastic leukemia (ALL).
How is chemotherapy given to children?
Your child may receive chemotherapy:
Orally, as a pill or liquid to swallow
Intramuscularly (IM), as an injection into the muscle or fat tissue
Intravenously (IV), directly to the bloodstream
Intrathecally, with a needle directly into the fluid surrounding the spine
Does chemotherapy cause side effects in children?
While chemotherapy can be quite effective in treating certain cancers, the medications don't distinguish healthy cells from cancer cells. As a result, there can be side effects during treatment. Your child's care team will work to manage or prevent these side effects whenever possible.
Radiation therapy uses high-energy rays (radiation) from a specialized machine to damage or kill cancer cells and shrink tumors. Radiation is usually only given if your child is at a high risk of relapse occurring in the brain or central nervous system.
A stem cell (or bone marrow) transplant is a treatment that is rarely used to treat ALL except for:
Certain unusual subtypes of ALL
Relapsed leukemia — if the leukemia comes back (relapse) after initial treatment with chemotherapy
Stem cells are a specific type of cell from which all blood cells develop. They can develop into red blood cells to carry oxygen, white blood cells to fight disease and infection and platelets to aid in blood clotting. Stem cells are found primarily in bone marrow, but some also circulate in the blood stream.
In acute lymphoblastic leukemia, the cells for a stem cell transplant come from donors (other people). These donated cells are used to replace your child's stem cells after particularly intense treatment with chemotherapy and/or radiation.
CAR (chimeric antigen receptor) T-cell therapy is a promising new treatment for relapsed or refractory B-cell ALL. It is an immunotherapy that uses the body's immune cells to hunt and destroy cancer cells. Dana-Farber/Boston Children's is a certified treatment center for KYMRIAH®, a CAR T-cell therapy that the FDA approved in 2017.
Other Medications
We may give your child other medications to prevent or treat damage to other systems of his body caused by treatment or its side effects. Your child also may be given antibiotics to prevent or treat infections.
Sanctuary Therapy
In children with ALL, leukemia cells travel everywhere in the body, including into the brain and spinal fluid. Not all chemotherapy drugs that are given by mouth, by vein, or in the muscle can get into the brain effectively to treat any leukemia that is "hiding" there.
However, it is important to treat the leukemia cells hiding in the brain and spinal fluid to prevent the leukemia from coming back. To treat the leukemia hiding in the brain and spinal fluid, your child will receive:
Intrathecal chemotherapy: chemotherapy drugs delivered directly into the spinal canal to kill off cancerous cells that may hide in the central nervous system (CNS)
Your child may also receive:
Cranial radiation therapy: radiation treatment to the head to destroy leukemia cells that may have moved into the CNS
Treatment Phases in Childhood ALL
Phase 1: Remission induction
Remission induction is the first stage of ALL treatment. It consists of chemotherapy given over a four-week period to clear the marrow of visible leukemia cells. The goal of this phase is to achieve complete remission, which means leukemia cells are no longer visible under a microscope when examining either the bone marrow or blood.
The induction phase usually lasts approximately one month. Most children are in complete remission at the end of the month of treatment. If your child is not in remission after the first month (that is, leukemia cells can still be seen in the marrow under a microscope), another induction phase will be started using different chemotherapy drugs.
Even after remission is achieved, leukemia cells are still present in the body, which is why further therapy is necessary.
Phase 2: Intensification or consolidation
Intensification or consolidation is continued treatment with chemotherapy to kill additional leukemia cells. This phase will include chemotherapy drugs given in the blood, in the vein, in the spinal fluid, and sometimes in the muscle. Radiation therapy also may be given to the brain during this phase.
Phase 3: Maintenance or continuation
The goal of this stage is to eradicate all leukemia from the body. Usually during this phase, less intensive chemotherapy is used, much of which can be given at home. This phase will last for many months. Your child must visit his physician regularly during this stage to determine response to treatment, detect any recurrent disease, and manage any side effects of treatment.
What is minimal residual disease (MRD)?
Minimal residual disease (MRD) is a term that is used for the "invisible" leukemia that is left behind after your child is in complete remission. It means that leukemia cells are still in your child's body, but there are too few of them to see them under a microscope.
Doctors use special tests after the end of the induction phase to measure the amount of MRD remaining in a patient's marrow.
The results of MRD tests are used to help determine the type of treatment your child's doctor will recommend during the intensification phase of treatment. Children with higher levels of MRD at the end of induction are often given stronger chemotherapy drugs during the intensification phase to kill the remaining leukemia cells hiding in the body.
Clinical Trials for Childhood ALL
Dana-Farber/Boston Children's is actively engaged in leukemia clinical trials, leading to new therapies and aiming for eventual cures. Our clinical team works closely with our leukemia researchers to quickly develop new treatments based on the latest scientific discoveries.
Clinical trials at Dana-Farber/Boston Children's are designed to investigate new therapies for refractory or relapsed leukemia, novel combinations of chemotherapy drugs, and promising new treatment agents. Our researchers are conducting randomized clinical studies to reduce treatment intensity to lessen side effects and to test more intense and newer therapies to improve cure rates.
Learn more about clinical trials for pediatric cancer.
Long-term Outlook for Children Treated for ALL
The prognosis for children treated for ALL is very positive. The five-year, event-free childhood acute lymphoblastic leukemia survival rate (which refers to survival without relapse) is about 85 percent. Ultimately, more than 90 percent of patients are cured of the disease.
The prognosis (chance of recovery) and treatment options depend on:
Age and white blood cell count at diagnosis
How quickly and how low the leukemia cell count drops after initial treatment
The subtype of leukemia
Whether there are certain changes in the chromosomes of lymphocytes
Whether the leukemia is seen in the spinal fluid at diagnosis
As with any cancer, prognosis and long-term survival can vary greatly. Relapse can occur during any stage of treatment, even with aggressive therapy, or may occur months or years after treatment has ended.
For patients with relapsed ALL, CAR (chimeric antigen receptor) T-cell therapy is a promising new treatment. Dana-Farber/Boston Children's is a certified treatment center for providing this therapy. In general, relapsed ALL cells become resistant to chemotherapy drugs. CAR T-cell therapy instead utilizes genetic engineering of a patient's own T-cells to target and destroy cancer cells.
Prompt medical attention and aggressive therapy are important for the best prognosis, and continuous follow-up care is essential. New methods are continually being discovered to improve treatment and decrease side effects of the treatment for the disease.
Long-term follow-up
Childhood cancer was once considered to always be fatal, but today, the majority of children diagnosed with cancer can expect to be long-term survivors.
Survivorship comes with numerous complex issues: the long-term effects of treatment and the risk of second cancers, as well as social and psychological concerns.
Since 1993, physicians, nurses, researchers, and psychologists in our pediatric cancer survivorship programs at the David B. Perini Jr. Quality of Life Clinic at Dana-Farber/Boston Children's have helped thousands of survivors of childhood cancers, treated at the institute and at other hospitals in New England and elsewhere, to manage these long-term consequences.
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Childhood Cancer Clinical Trials FAQ
Find answers to common questions about clinical trials for childhood cancer, including whether or not a clinical trial may be the right choice for your child. You can also email your questions to clinicaltrials@danafarberbostonchildrens.org
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