In general, cancer in children is uncommon, so it can be hard for doctors to determine the best treatment unless they know what has been most effective in other children. That’s why more than 60% of children younger than 15 years old with cancer are treated as part of a clinical trial. Clinical trials are research studies that compare standard treatments (the best proven treatments available) with newer treatments that may be more effective or test new treatments. Investigating new treatments involves careful monitoring using scientific methods, and all participants are followed closely to track their health and 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. In many cases, a team of doctors works with a child and the family to provide care; this is called a multidisciplinary team.

Pediatric cancer centers often have extra support services for children and their families, such as child life specialists, nutritionists, social workers, counselors, and physical and speech therapists, if needed. Special activities and programs to help your child and family cope may also be available. As explained in the Overview, however, there are times when a teenager with cancer may need to be treated at an adult cancer center.

Descriptions of the most common treatment options for childhood cancer are listed below. The treatment of childhood cancer depends on several factors, including the type and stage of cancer, possible side effects, the family’s preferences, and the child’s overall health. The following are general descriptions of treatments that may be used as part of a child’s treatment plan. 

Surgery

Surgery is the removal of the tumor, either cancerous or noncancerous, and surrounding tissue during an operation. Many children with a tumor will need surgery at some point during their treatment. A surgical oncologist is a doctor who specializes in treating a tumor using surgery. The goal of surgery is to remove the entire tumor and the margin (tissue around the tumor), leaving a negative margin (no cancer in the healthy tissue). For most childhood tumors, there is microscopic tumor left after surgery, and then doctors will recommend chemotherapy, radiation therapy, or other treatments.

Side effects of surgery depend on the location and type of the tumor and whether it has metastasized. 

Chemotherapy

Chemotherapy is the use of drugs to kill cancer cells, usually by stopping the cancer cells’ ability to grow and divide. Systemic chemotherapy is given by mouth or delivered through the bloodstream (injected into the vein, muscle, spinal fluid, or under the skin) to reach cancer cells throughout the body. Chemotherapy is given by a pediatric oncologist, a doctor who specializes in treating children with cancer. A chemotherapy regimen (schedule) usually consists of a specific number of cycles given over a set period of time. A patient may receive one drug at a time or combinations of different drugs at the same time.

The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, increased risk of infection, increased risk of bleeding, nausea and vomiting, loss of appetite, and diarrhea. These side effects usually go away once treatment is finished.

 The medications used to treat cancer are continually being evaluated. Talking with your child’s 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. 

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles such as photons to kill cancer cells. A doctor who specializes in giving radiation therapy to treat cancer is called a radiation oncologist. The most common type of radiation treatment is called external-beam radiation therapy, which is radiation given from a machine outside the body. When radiation treatment is given using implants, it is called internal radiation therapy or brachytherapy. A radiation therapy regimen usually consists of a specific number of treatments given over a set period of time.

Side effects from radiation therapy may include fatigue, mild skin reactions, nausea, and loose bowel movements, depending on the part of the body that is receiving radiation. Most side effects go away soon after treatment is finished. 

Immunotherapy

Immunotherapy (also called biologic therapy) is designed to boost the body’s natural defenses to fight the cancer. It uses materials made either by the body or in a laboratory to bolster, target, or restore immune system function. Examples of immunotherapy include cancer vaccines, monoclonal antibodies, and interferons. 

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 it is the blood stem cells that are typically being transplanted, not the actual bone marrow tissue.

Before recommending transplantation, doctors will talk with the patient and family 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.

There are two types of stem cell transplantation depending on the source of the replacement blood stem cells: allogeneic (ALLO) and autologous (AUTO).

In both types, the goal of transplantation is to destroy cancer cells in the marrow, blood, and/or other parts of the body and allow replacement blood stem cells to 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. 

Palliative/supportive care

Cancer and its treatment often cause side effects. In addition to treatment to slow, stop, or eliminate the cancer, an important part of cancer care is relieving a person’s symptoms and side effects. This approach is called palliative or supportive care, and it includes supporting the patient with his or her physical, emotional, and social needs.

Palliative care can help a person at any stage of illness. People often receive treatment for the cancer and treatment to ease side effects at the same time. In fact, patients who receive both often have less severe symptoms, better quality of life, and report they are more satisfied with treatment.

Before treatment begins, talk with your child’s health care team about the possible side effects of the specific treatment plan and supportive care options. And during and after treatment, be sure to tell your child’s doctor or another health care team member if your child is experiencing a problem, so it is addressed as quickly as possible. 

Recurrent childhood cancer

A remission is when cancer cannot be detected in the body and there are no symptoms. This may also be called “no evidence of disease” or NED. 

A remission can be temporary or permanent. This uncertainty makes many patients and families worried or anxious that the cancer will come back. While many remissions are permanent, it’s important to talk with your child’s doctor about the possibility of the cancer returning. Understanding the risk of recurrence and the treatment options may help you feel more prepared if the cancer does return. 

If the cancer does return after the original treatment, it is called recurrent disease. It may come back in the same place (called a local recurrence), nearby (regional recurrence), or in another place in the body (distant recurrence).

When this occurs, a cycle of testing will begin again to learn as much as possible about the recurrence. After testing is done, your child’s doctor will talk with you about treatment options. Often the treatment plan will include the therapies described above (such as surgery, chemotherapy, and radiation therapy) but may be used in a different combination or given on a different schedule. Your child’s doctor may also suggest clinical trials that are studying new ways to treat this type of recurrent cancer.

When cancer recurs, patients and their families often experience emotions such as disbelief or fear. Families are encouraged to talk with their health care team about these feelings and ask about support services to help them cope. 

If treatment fails

Although treatment is successful for most children with cancer, sometimes it is not. If a child’s cancer cannot be cured or controlled, this is called advanced, terminal, or refractory cancer. This diagnosis is stressful, and it may be difficult to discuss. However, it is important to have open and honest conversations with your child’s doctor and health care team to express your family’s feelings, preferences, and concerns. The health care team is there to help, and many treatment centers have a palliative care service, with nurses and doctors who have special skills, experience, and knowledge to support patients and their families during this time.

Parents or guardians are encouraged to think about where the child would be most comfortable: at home, in a home-like setting elsewhere, in the hospital, or in a hospice environment. Nursing care and special equipment can make staying at home a workable alternative for many families. Some children may be happier if they can attend school part-time or keep up other activities and social connections for as long as possible. The child’s health care team can help parents or guardians decide on an appropriate level of activity. Making sure a child is physically comfortable and free from pain is an extremely important part of end-of-life care. 

The death of a child is an enormous tragedy, and families may need support to help them cope with the loss. Pediatric cancer centers often have professional staff and support groups to help with the process of grieving.

Coping with Side Effects

Fear of treatment side effects is common after a diagnosis of cancer, but it may help to know that preventing and controlling side effects is a major focus of your child’s health care team. This is called palliative or supportive care, and it is an important part of the overall treatment plan, regardless of the stage of disease.

Common side effects from each treatment option for childhood cancer are described in detail within the Treatment section. 

Before treatment begins, talk with your child’s doctor about possible side effects of each type of treatment your child will be receiving. Ask which side effects are most likely to happen, when they are likely to occur, and what can be done to prevent or relieve them. And, ask about the level of caregiving your child may need during treatment and recovery, as family members and friends often play an important role in the care of a person with childhood cancer. 

In addition to physical side effects, there may be psychosocial (emotional and social) effects as well. Patients and their families are encouraged to share their feelings with a member of their health care team who can help with coping strategies.

During and after treatment, be sure to tell the health care team about the side effects your child experiences, even if you feel they are not serious. Sometimes, side effects can last beyond the treatment period, called a long-term side effect. A side effect that occurs months or years after treatment is called a late effect. Treatment of both types of effects is an important part of survivorship care.

After Treatment

After treatment for cancer ends, talk with your child’s doctor about developing a follow-up care plan. This plan may include regular physical examinations and/or medical tests to monitor your child’s recovery for the coming months and years. All children treated for cancer should have life-long, follow-up care.

Many of the current treatments that are effective in curing childhood cancer are associated with short-term and long-term side effects, including cognitive (thought-process) and developmental side effects. These side effects can appear or change as a child grows older. As most children with cancer are cured, the schedule of follow-up care is based on the dosage and specific drugs used in treatment, as well as the dosage of radiation therapy. 

Based on the type of treatment the child received, the doctor will determine what examinations and tests are needed to check for long-term side effects and the possibility of secondary cancers. Your child’s doctor can recommend the necessary screening tests. Follow-up care should also address the child’s quality of life, including any developmental or emotional concerns. 

The child’s family is encouraged to organize and keep a record of the child’s medical information. That way, as the child enters adulthood, he or she has a clear, written history of the diagnosis, the treatment given, and the doctor’s recommendations about the schedule for follow-up care. The doctor’s office can help you create this. This information will be valuable to doctors who care for your child during his or her lifetime.

Children who have had cancer can also enhance the quality of their future by following established guidelines for good health into and through adulthood, including not smoking, maintaining a healthy weight, eating a balanced diet, and participating in regular physical activity. Talk with the doctor about developing a plan that is best for your child’s needs.

Latest Research

Doctors are working to learn more about childhood cancer, ways to prevent it, how to best treat it, and how to provide the best care to people diagnosed with this disease. The following areas of research may include new options for patients through clinical trials. Always talk with your child’s doctor about the diagnostic and treatment options best for your child.

Advances in treatment and follow-up care. The Children’s Oncology Group conducts large clinical trials for most types of pediatric cancer. It also conducts studies on quality of life and late effects of cancer after successful treatment. The Childhood Cancer Survivor Study conducts long-term, follow-up studies of patients who were treated many years ago to determine the late effects of cancer and its treatment, so new treatments can be developed to avoid serious side effects. Other groups, including the Pediatric Brain Tumor Consortium and the New Approaches to Neuroblastoma Therapy Consortium, perform studies of new drugs for specific types of cancer.

Reducing a child’s exposure to radiation. Because children have an increased risk of developmental damage and second cancers from radiation therapy, doctors prefer to use radiation therapy less often whenever possible. In place of radiation therapy, doctors may use chemotherapy with a combination of drugs after surgery or use new drug combinations. Researchers are also investigating new techniques such as proton therapy that more precisely focus radiation treatment at the tumor and not the surrounding healthy tissue.

Supportive care. Clinical trials are underway to find better ways of reducing other symptoms and side effects of current childhood cancer treatments in order to improve patients’ comfort and quality of life. For example, drugs called filgrastim (Neupogen) and pegfilgrastim (Neulasta) may help patients produce more white blood cells after radiation treatment and chemotherapy. Doctors are also studying chemoprotective drugs that may help protect the body from the harmful effects of chemotherapy, especially mucositis (mouth sores). Additionally, this area of research includes studies in cardioprotection (protecting the heart and cardiovascular system from chemotherapy) and otoprotection (protecting against damage to the ear).