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Common Pediatric Cancer Treatments Explained

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By: Vikramjit Kanwar, MD, FAAP

A diagnosis of cancer is alarming and often unfamiliar for both children and their parents. Fortunately, survival rates for childhood cancer have increased dramatically in recent decades. Today, more than 80% children diagnosed with cancer in the United States who are treated will be cured.

In this article, the American Academy of Pediatrics offers information on some of the common treatment options available. Keep in mind that not all of these treatments are recommended for every cancer type or in every child.

What is a cancer treatment plan?

After diagnosis, a doctor specializing in childhood cancer called a pediatric oncologist will work with the family and other cancer subspecialists to put together a treatment plan or "protocol."

A child's cancer treatment plan may include a combination of:

  • surgery to remove cancerous tissue

  • chemotherapy (also known as drug therapy)

  • radiation therapy (high energy X-rays)

  • targeted therapy, such as immunotherapy (proteins that cause the immune system to kill cancer or that target specific cancer genes or proteins)

  • hematopoietic stem cell transplantation (strong chemotherapy followed by replacement with new marrow blood cells)

What's included in a treatment plan depends on the type of cancer, the cancer's genetics, and where the cancer is located in the body.


Surgery may play a role in both cancer diagnosis and treatment, especially for solid tumors and lymphomas (lymph nodes that contain cancer). The surgeon may first take out a piece of the tumor, a procedure called a biopsy, so the tissue can be tested. This can reveal the exact type of cancer.

Surgeons may then remove as much of the cancer as possible before, during, or after other treatments are given that shrink the tumor for the best possible outcomes.

Sometimes surgery can damage the healthy tissue surrounding the tumor. A child may need supportive medication or care to help them heal from the surgery.

What surgery treats

Surgery is often part of treatment for solid tumors in the kidney (such as Wilms tumor), nervous system (such as neuroblastoma), bone, muscle or other soft tissue (types of sarcomas), and brain cancers, for example.

Childhood cancer treatment & clinical trials

More than half of children being treated for cancer are enrolled in clinical trials. Clinical trials are research studies that help doctors learn improve cure rates and the quality of life during and after cancer treatment. They're good for patients, too.

Patients in clinical trials get the latest information, testing, procedures, chemotherapy and other drugs. A pediatric oncologist will go over the risks and benefits of the clinical trial to help families decide if a clinical trial is a good option for their child.

Radiation therapy

Radiation therapy uses high energy X-rays (photons or protons) to kill cancer cells. Radiation is delivered by a machine called a linear accelerator, which sends high energy X-rays directly into the cancer. Radiation is not seen, tasted, or smelled, but the linear accelerator sometimes makes a clicking or humming noise during treatment.

Each radiation treatment can last 10-30 minutes, and may be given daily, for several days a week. Total treatment can last from 1-8 weeks, depending on the type of cancer.

Sometimes radiation can temporarily damage healthy tissue around the tumor being treated. Side effects, which often depend on the type and location of the cancer, may arise two- to three weeks after starting treatment and last for several weeks. Supportive medications are given to reduce many side effects.

What radiation therapy treats

Radiation therapy may be part of treatment for childhood cancers that develop in soft tissue (rhabdomyosarcoma and sarcoma), the bones (Ewing Sarcoma), eye (retinoblastoma), lymph nodes (Hodgkin lymphoma) and nervous system (neuroblastoma, ependymoma, pontine glioma and astrocytoma), among other types.


Chemotherapy (sometimes called "chemo") is the use of medicines to help kill cancer cells. Chemotherapy can be given in a number of different ways:

  • by mouth (orally)

  • in the vein (intravenously)

  • into a muscle (intramuscularly)

  • into the spinal fluid (intrathecally or intraventricularly)

  • under the skin (subcutaneously).

Often, a combination of chemotherapy drugs are used together. The length of treatment depends on the type of cancer and how quickly the cancer responds.

As with radiation, sometimes healthy cells in the body are affected, too—especially fast-growing ones like those in the blood, intestines and hair follicles—but the damage is usually temporary. Each chemotherapy has unique side effects but there are supportive medications available to reduce them.

What chemotherapy treats

Chemotherapy is often part of treatment for most types of cancer that start in the kidney, nervous system, ovaries or testes (germ cells in the ovaries or testes), muscle, bones (Ewing Sarcoma, osteosarcoma), liver (hepatoblastoma), eye (retinoblastoma), blood, lymph nodes (both Hodgkin and Non-Hodgkin lymphoma), immune system (Langerhans cell histiocytosis) and brain.

Hematopoietic stem cell & bone marrow transplant

Stem cell transplants replace diseased or damaged bone marrow cells with healthy stem cells from bone marrow or umbilical cord blood that can make healthy, new mature blood cells. There are two main types of bone marrow transplants used to treat childhood cancers:

Autologous transplant. This type of transplant collects or "harvests" the child's own stem cells and freezes them for later use. It starts with very high doses of chemotherapy to destroy cancer cells. After chemotherapy, a stimulant protein is given daily to force stem cells out from the bone marrow. The cells are then collected using a special machine in a process called apheresis, which separates blood into its different parts. The frozen stem cells are then thawed and put back or "infused" into the patient's bloodstream. These healthy stem cells travel to the healthy marrow to grow healthy blood cells.

Allogeneic transplant. This type of transplant collects stem cells from another person, often a brother or sister. If a family donor is not a genetic match, families can look for an unrelated donor through the National Marrow Donor Program. Patients may also receive donated umbilical cord blood.

Before donated stem cells are transplanted, chemotherapy with or without radiation (called conditioning) is used to destroy the patient's bone marrow. The new donated stem cells are infused into the bloodstream to produce new blood cells.

Since patients are vulnerable to infection and many other side effects during stem cell transplants, they usually need to stay in the hospital for a while. A hospital stay also helps doctors watch closely for any complications, such as the body rejecting the transplanted cells.

What stem cell transplant treats

Stem cell transplant may be used to treat children with high-risk neuroblastoma and certain types of brain tumors, lymphomas, germ cell tumors, and leukemia that have relapsed (returned) after earlier treatment.

Targeted therapy

Targeted therapy, sometimes called molecular therapy or precision medicine, works by targeting and destroying the cancer's specific mutated (changed) genes, proteins or surrounding tissue. Targeted therapy may be used in combination with other treatments such as chemotherapy.

Major types of targeted therapy

There are different types of targeted cancer therapies. The two major types include small molecule drugs that work inside cancer cells, and monoclonal antibodies that attach to the outside of cancer cells. Some mark cancer cells so that they are better seen and killed by the immune system. Others kill or stop cancer cells from growing. Still others carry toxins to cancer cells or target a specific genetic defect in the tumor.

Although the hope of targeted therapy is to reduce any side effects to healthy tissue, even targeted therapy has side effects that are manageable with supportive medication and care from the pediatric oncologist.

What targeted therapy treats

Targeted therapy so far has been most successful with some types of leukemia and lymphoma. As research leads to a better understanding of various genetic abnormalities in different types of childhood cancers, others may also be treated with different targeted therapies.


Immunotherapy treatment involves using medications to help the body's own germ-fighting immune system to target and destroy cancer cells. There are different types of immunotherapy that can treat cancer, including:

  • Monoclonal antibody treatment, which uses specific antibodies made by the immune system to bind to specific protein markers on tumor cells. This helps the immune system recognize and destroy them.

  • Immune checkpoint inhibitors are a newer class of drug that turn off the protective function of proteins that are produced by cancer cells previously missed in the immune system's attack.

  • Adoptive cell therapy, also called T-cell transfer therapy, is when T cells (a type of lymphocyte or white blood cell) are collected from a patient and grown in a laboratory under conditions that "trains" them to fight cancer cells. Chimeric antigen receptor T cell (CAR T cell) therapy is the best-known of this type of cell therapy. CAR T-cell therapy has many initial, severe side effects but can be highly effective against relapsed leukemia or lymphoma.

  • Cancer vaccines, available in clinical trials, helps the immune system to identify and destroy cancer cells.

As with other types of targeted therapy, immunotherapy also has side effects that can happen during and even after the treatment. Careful monitoring and supportive medical care help to reduce these side effects.

What immunotherapy treats

Immunotherapy may be part of the treatment plan many different types of cancers, including Non-Hodgkin lymphoma, skin cancer (melanoma), high risk or relapsed leukemia or neurblastoma that has spread to other parts of the body.


Starting cancer treatment can be a confusing time for patients and their families. Talk with your pediatrician and cancer team if you have any questions about the best treatment plan for your child.

More information

About Dr. Kanwar

Vikramjit Kanwar, MD, FAAP, is a Corresponding Fellow of the American Academy of Pediatrics Section on Hematology and Oncology. Formerly Chief of the Division of Pediatric Hematology-Oncology at Albany Medical Center and an Adjunct Professor of Pediatrics at Albany Medical College New York, Dr. Kanwar has been actively involved with the Childrens Oncology Group.

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American Academy of Pediatrics Section on Hematology/Oncology (Copyright © 2020)
The information contained on this Web site should not be used as a substitute for the medical care and advice of your pediatrician. There may be variations in treatment that your pediatrician may recommend based on individual facts and circumstances.
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