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Childhood Leukemia Treatment Options

Medically reviewed by Adam S. Levy, M.D.
Written by Maureen McNulty
Posted on May 6, 2021

Many treatments can lead to positive outcomes for children with leukemia. Treatments can help eliminate cancer and prevent it from returning. These treatments are sometimes aggressive and may lead to side effects, so long stays in the hospital are sometimes needed. It’s a good idea to seek treatment at a center that has expertise in treating childhood leukemia.

Treating Different Types of Leukemia in Children

When leukemia is diagnosed, your child’s doctor will also determine what type of leukemia they have. Different types of childhood leukemia may need different forms of treatment. When treatment works and signs of leukemia disappear, it is known as remission. If cancer comes back after being treated, it is called a relapse.

Childhood Acute Lymphoblastic Leukemia

The most common type of leukemia in children is acute lymphoblastic leukemia, also called acute lymphocytic leukemia (ALL). About 3 out of 4 cases of childhood leukemia are ALL.

Treatment of childhood ALL usually occurs in three phases:

  • Induction therapy kills as many leukemia cells as possible and usually puts leukemia into remission.
  • Consolidation therapy attacks any cancer cells that may be left behind.
  • Maintenance therapy continues to destroy any remaining leukemia cells and helps prevent a relapse.

Doctors recommend childhood ALL treatments based on a child’s risk group. Risk groups are determined based on factors like a child’s age, white blood cell count, gene changes, and which type of white blood cell is affected. Higher-risk ALL is often treated with more medications or higher drug doses.

Childhood Acute Myeloid Leukemia

Acute myeloid leukemia (AML) treatment generally happens in two phases: induction therapy and consolidation therapy. Which treatments your child receives depends on several factors. When recommending a treatment plan, your child’s doctor may consider:

  • Your child’s age
  • Your child’s white blood cell count
  • What subtype of AML your child has
  • Gene changes found within leukemia cells
  • Whether leukemia has spread
  • How well leukemia responds to initial treatment

If your child’s AML relapses or is refractory (doesn’t go away after treatment), then doctors may recommend different treatment options.

Other Types of Childhood Leukemia

Children may also be diagnosed with other types of blood cancer, including:

  • Acute promyelocytic leukemia (APL)
  • Juvenile myelomonocytic leukemia (JMML)
  • Myelodysplastic syndrome (MDS)
  • Chronic myelogenous leukemia (CML)

Treatments for these conditions also depend on factors like age, gene changes, how many cancer cells are in the blood, and whether cancer has relapsed.

Treatment Options for Childhood Leukemia

A doctor who specializes in types of childhood cancer (a pediatric oncologist) usually supervises treatment for children with leukemia. Many other health care professionals may also be involved in treatment, such as a pediatrician, hematologist, neurologist, pediatric nurse specialist, social worker, or psychologist. Your child’s health care team can help you, your child, and other family members decide on a treatment plan and manage side effects.

Chemotherapy Options for Children

Chemotherapy drugs are used to treat children with leukemia. These medications kill cancer cells or prevent them from making copies of themselves.

Most chemotherapy treatments are “systemic,” meaning the medication can travel through the blood to reach all parts of the body. Systemic chemotherapy may come in the form of a pill or capsule taken by mouth, a liquid injected into the muscle, or a liquid given directly into a vein (intravenously). Some children with leukemia are also treated with intrathecal chemotherapy, in which the drug is injected into the fluid that surrounds the central nervous system (brain and spinal cord). Intrathecal chemotherapy can kill cancer cells located in the brain or can help prevent leukemia cells from spreading there.

Treatment for childhood ALL often includes chemotherapy given over two or three years. During induction therapy, when a child receives their first doses of chemotherapy, they may need to stay in the hospital for a week or more. After this, a child often receives several more cycles of chemotherapy, which may require additional hospital stays.

Childhood AML is often treated with high doses of chemotherapy and lasts for five or six months. Managing this condition often requires a long hospital stay.

Targeted Therapies for Childhood Leukemia

Targeted therapies recognize and attack specific genes or proteins found in cancer cells. Targeted therapy drugs can kill cancer cells while mostly leaving the body’s healthy cells alone. Targeted therapy medications may cause fewer side effects than chemotherapy drugs. However, some targeted therapies can lead to serious health problems the first couple of times a child receives them. For this reason, the child may need to stay in the hospital when they first start taking a targeted therapy.

A doctor may recommend different targeted therapy drugs based on the type of leukemia and any gene changes found within leukemia cells. Several different targeted therapies can attack ALL or AML cells that have specific proteins. Additionally, children with CML almost always have a gene change called the Philadelphia chromosome that can be blocked with several different medications. Targeted therapies for CML usually work very well and keep leukemia away for a long time.

Surgery for Childhood Leukemia

No surgeries can cure or directly treat childhood leukemia. However, children may undergo minor surgery before using chemotherapy or other drugs that are delivered intravenously. During surgery, doctors place a small tube into a vein in the chest or arm. Intravenous drugs can later be given through this tube. Children who receive these tubes don’t need to get a needle stick each time they receive medication.

Childhood Leukemia Immunotherapy

Chimeric antigen receptor (CAR)-T cell therapy is a new type of treatment that helps the body’s immune system fight cancer. During CAR-T cell therapy, a child’s T cells (a type of immune cell) are removed, genetically changed, and then put back into the body. The new T cells are better at finding and attacking leukemia cells.

Radiation Therapy in Children

In radiation therapy, a machine is used to deliver high-energy beams that can kill cancer cells. Not all children with leukemia need radiation therapy. Radiation therapy is sometimes used to kill leukemia cells that spread to other places in the body, including the testicles or central nervous system. Children with AML may need radiation therapy to treat a chloroma (tumor made up of white blood cells).

Stem Cell Transplant

A stem cell transplant is a treatment of the bone marrow (tissue found inside of certain bones). Blood stem cells live in the bone marrow and are responsible for making all of the different types of blood cells.

Stem cell transplantation first destroys cancerous cells in the bone marrow with high doses of chemotherapy or radiation therapy. These aggressive chemotherapy or radiation treatments also usually kill the stem cells. Next, the person gets new stem cells so that their body can continue to make healthy blood cells. Leukemia is often treated using an allogeneic stem cell transplant, in which the new stem cells come from a donor.

A stem cell transplant can possibly cure childhood leukemia, making it go away and never relapse. However, transplantation can also have very serious side effects. It is often only recommended for children who are diagnosed with high-risk leukemia, those who have had a relapse, or children who are likely to have a relapse in the future.

Clinical Trials for Leukemia

Because leukemia in children is rare, researchers don’t always know exactly how to treat it. Participating in a clinical trial may be a good option for children with these conditions. Clinical trials allow a child to receive new therapies or take standard medications in new doses. These new treatment options may work better than currently available treatments.

Outlook for Childhood Leukemia

Treatments for childhood leukemia often cause all signs of cancer to disappear. About 98 percent of children with ALL and 90 percent of children with AML go into remission. Sometimes, leukemia will relapse after it initially goes away. Recurrent leukemia (leukemia that has come back) is often treated with different therapies or new medication doses than were used the first time.

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Posted on May 6, 2021
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Adam S. Levy, M.D. is co-director of pediatric neuro-oncology and medical director of the Pediatric Ambulatory Subspecialty Services at the Children’s Hospital at Montefiore, Bronx, NY. Review provided by VeriMed Healthcare Network. Learn more about him here.
Maureen McNulty studied molecular genetics and English at Ohio State University. Learn more about her here.

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