Pediatric and Childhood Cancers

How is cancer diagnosed?

The first step in treating your child is forming an accurate and complete diagnosis. Your child’s physician may order a number of different tests to determine if your child has cancer and its specific type and location. These include:

• physical examination
• blood tests
• urine tests
• imaging scans,  including x-rays, magnetic resonance imaging (MRI),computerized tomography scan (also called a CT or CAT scan), positron emission tomography/CT (also called a PET/CT scan) and bone scans  produce detailed images of the inside of the body
• bone marrow biopsy/aspiration - a procedure that involves a small amount of bone marrow fluid and tissue to be taken, usually from part of the hip bones, to further examine the number, size and maturity of blood cells and/or abnormal cells
• lymph node biopsy - a sample of tissue is removed from a lymph node and examined under a microscope
• tumor biopsy (or tissue sample) from the tumor to provide definitive information about the type of tumor; this is collected during surgery
• lumbar puncture (spinal tap) to remove a small sample of cerebrospinal fluid and determine if any tumor cells have started to spread. In young children, this procedure is safely performed under sedation.
• chromosome analysis and other genetic testing – samples of tumor, blood and bone marrow can be sent to a special laboratory to look for certain changes, or mutations, in the chromosomes of  cancer cells

After we complete all necessary tests, our experts meet to review and discuss what they have learned about your child's condition. Then we will meet with you and your family to discuss the results and outline the best treatment options.

How is childhood cancer treated?

Children treated at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center receive the combined strengths of two internationally renowned Harvard teaching hospitals. A single team of experts delivers care at a Boston campus connected by pedestrian bridges, with inpatients treated at Boston Children's Hospital and outpatients at the Jimmy Fund Clinic at Dana-Farber Cancer Institute.

We base every child's specific course of treatment on several factors, including:

• age, overall health and medical history
• type, location and size of the tumor
• genetic changes, for some tumors
• extent of the disease
• tolerance for specific medications, procedures or therapies
• how your child's doctors expects the disease to progress

There are a number of treatments we may recommend. Some may help to treat the tumor while others are intended to address complications of the disease or side effects of the treatment.

Surgery

Surgery has multiple roles in the diagnosis and treatment of some types of pediatric cancers, including biopsy, tumor removal and placement of long-term intravenous lines.

Tumor specimens are examined by our pathologists to determine the exact diagnosis.

When possible, it’s best to completely remove the entire tumor. In general, the more of the tumor that is removed, the greater the chance for survival.

Using the latest molecular profiling techniques, some tumors (including brain tumors) are now processed to identify abnormal genes within the tumor.

Radiation therapy

Our doctors use precisely targeted and dosed radiation to kill cancer cells and control the growth of the tumor. Depending on the type of the cancer, some patients are treated with targeted focal radiation therapy. In those tumors that may have spread, radiation therapy can sometimes be delivered to larger areas of the body.

Chemotherapy

Chemotherapy (or chemo) refers to a group of drugs that interfere with the cancer cell’s ability to grow or reproduce.

• Different groups of chemotherapy drugs work in different ways to fight cancer cells and shrink tumors.
• Often, a combination of chemotherapy drugs is used.
• Certain chemotherapy drugs may be given in a specific order depending on the type of cancer it is being used to treat.

Chemotherapy is systemic treatment, meaning it is introduced to the bloodstream and travels throughout the body to kill cancer cells. It may be given:

• orally, as a pill 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

Chemotherapy also refers to the use of drugs that are made to specifically inhibit a genetic pathway required to keep the tumor going. These drugs are often referred to as “targeted” therapy. Rather than the standard side effects associated with radiation and chemotherapy (loss of hair, nausea and vomiting, damage of the blood producing cells), targeted drugs tend to be better tolerated.

Side effects of chemotherapy and radiation therapy

While chemotherapy can be quite effective in treating certain cancers, the drugs affect both cancerous and non-cancerous cells. In the same way, while radiotherapy can be quite effective in treating certain cancers, the radiation damages both cancerous and non-cancerous cells. Because of this, there can be many undesirable side effects during treatment. Being able to anticipate these side effects can help the care team, parents and child prepare, and in some cases prevent these symptoms from occurring.

Stem cell transplant

A stem cell (bone marrow) transplant is an infusion of healthy blood forming stem cells by IV (through a needle into a vein) to treat certain types of cancer, blood disorders and other conditions. Blood forming stem cells are a special type of cell found in the bone marrow that can form any type of blood cell: red blood cells, white blood cells and platelets. This is different from embryonic stem cells, which can form any type of cell in the body.

The cells for a stem cell transplant (also called a bone marrow transplant) can come from a related donor—usually a sibling, an unrelated donor—or the patient himself. The stem cells are infused into the bloodstream and travel to the bone marrow where they begin to make new blood cells.

A stem cell transplant can be used to:

• replace diseased, non-functional bone marrow with healthy functioning bone marrow (for conditions such as aplastic anemia and sickle cell disease)
• replace bone marrow and restore its normal function after high doses of chemotherapy or radiation (for diseases such as leukemia, lymphoma and neuroblastoma)
• replace bone marrow with healthy functioning bone marrow to prevent further damage from a genetic disease (such as Hurler's syndrome and adrenoleukodystrophy)

Stem cell transplantation has risks involved, some of which are life threatening. The risks and benefits must be weighed and thoroughly discussed with your child’s stem cell transplant team prior to the procedure.

Children receiving a stem cell transplant are treated through our Stem Cell Transplant Center, one of the largest and most experienced stem cell transplant programs in the United States.

What is the long-term outlook for childhood cancer?

Today, the five-year survival rate is 84 percent for all childhood cancers combined. However, it’s important to understand that your child’s short-term health and long-term survival varies greatly depending on the specific type of cancer. Prompt medical attention and intensive therapy are very important, as is continuous follow-up care. (Read Five Things Parents of Children Should Know.)

Your child’s prognosis greatly depends on:

• the type of cancer
• the extent of the disease
• size and location of the tumor(s)
• presence or absence of metastasis (spread to other areas in the body)
• the cancer’s response to therapy
• your child’s age and overall health
• your child’s tolerance of specific medications, procedures or therapies
• new developments in treatment

New methods are continually being discovered to improve treatment and to decrease side effects.

What kind of long-term follow-up care should my child get?

Some children who are treated for pediatric cancer will experience significant long-term problems as a result of their treatment. Children require ongoing specialized care to monitor for signs that the cancer may have returned and manage side effects of treatment.

Because of the possible long-term problems and the risk of a cancer returning, a child usually returns to us for care for years after she is initially treated for cancer.

One of the major goals of the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center is to maximize your child’s long-term health. We do this in many ways, including attending to your child’s:

• intellectual function and school performance
• endocrine evaluation and treatment
• neurologic assessment
• psychosocial care
• hearing and vision

To monitor for recurrence and address short and long-term side effects of treatment, children treated for pediatric cancer receive follow-up care at our cancer survivorship clinics.

Survivorship clinics

The long-term outcome for children with pediatric cancer is improving. How well each child does depends on various factors, including the type of tumor and whether the disease has spread to other parts of the body (metastasized) by the time of diagnosis. Our research continues to help us understand the causes of cancer so we can develop more targeted, effective therapies.

David B. Perini Jr. Quality of Life Clinic

To assist the many children successfully treated for malignant tumors, we have an established multidisciplinary, long-term follow-up program through Dana-Farber Cancer Institute's David B. Perini Quality of Life Clinic.

This program helps survivors of all ages address the medical, psychological and social consequences of their diagnosis and subsequent treatments. Childhood cancer survivors of any age are invited to visit our clinic, regardless of where they were initially treated.

Stop & Shop Family Pediatric Neuro-Oncology Outcomes Clinic

For survivors of childhood brain tumors, we have established the Stop & Shop Family Pediatric Neuro-Oncology Outcomes Clinic at Dana-Farber Cancer Institute. Through this service, your child will be able to meet with his neurosurgeon, radiation oncologist, pediatric neuro-oncologist and neurologists at the same follow-up visit:

• Our pediatric brain tumor survivorship clinic is held weekly.
• In addition to meeting with your pediatric neuro-oncologists, neurologist and neurosurgeon, your child also may see one of our endocrinologists and/or alternative/complementary therapy specialists.
• School liaisons and psychosocial personnel from the pediatric brain tumor team are also available.
• If your child needs rehabilitation, he also may meet with speech, physical and occupational therapists during and after treatments.

Supportive and palliative care

When necessary, our Pediatric Advanced Care Team (PACT) is available to provide supportive treatments intended to optimize the quality of life and promote healing and comfort for children with life-threatening illness. In addition, PACT can provide psychosocial support and help arrange end-of-life care, should that be necessary.

Resources and support

In addition to providing medical care, we strive to also meet all of your family’s emotional and quality-of-life needs. Our patient and family support services will help address many of your needs and concerns. Read about our resources and support services here.

The team at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center improves care in many ways. Some changes come from scientific research into childhood cancer and blood disorders. Understanding diseases deeply—even at the cellular or molecular level—leads to new drugs and therapies. Other improvements come from moments spent at the bedside, when doctors and nurses see opportunities to improve current treatment methods. Learn more at Dana-Farber/Boston Children’s.

Dana-Farber/Boston Children's Cancer and Blood Disorders Center participates the Children's Oncology Group (COG), a consortium of cancer treatment centers across the United States, Canada and other countries that conduct studies of nearly every kind of pediatric cancer. Our participation in the COG gives children with cancer unparalleled access to the newest clinical trials. We also participate in several other consortia, such as Therapeutic Advances in Childhood Leukemia (TACL), Pediatric Oncology Experimental Therapeutics Investigators' Consortium (POETIC), New Approaches to Neuroblastoma Therapy (NANT) and the DFCI Pediatric Leukemia Consortium.

If your child has progressive or recurrent disease, she may be eligible for a number of experimental therapies available through COG or other groups, or through one of our independent clinical investigators.

Read about our innovative research on the Cancer Care Center page.

Q: What is a clinical trial?

A: A clinical trial is a research study that uses volunteers to test new methods of screening, prevention, diagnosis or treatment of a disease. These studies are designed to answer important questions about cancer diagnosis, treatment and prevention. If enrolling in a clinical trial is an option, your child's primary oncologist will explain what the treatment involves, its goals and objectives, and answer any questions you have about enrolling on the study.

Q: What is the purpose of a clinical trial?

A: Most clinical trials are designed to ask a question that hasn't been answered yet such as, "Does a certain medicine cure a disease, or make some of the symptoms go away?" The results help us learn which treatments are currently most effective for certain diseases.

For cancer in particular, medical research is very important. Clinical trials test the efficacy of new medications, evaluate new approaches designed to minimize long-term effects, and provide treatments to preserve quality of life during and after treatment.

Q: Why should my child enroll in a clinical trial?

A: Most pediatric cancers are treated at major medical institutions, which use state-of-the-art treatments. Many of these treatments are based on clinical trials. Clinical trials rely on scientific findings to investigate the newest medications and most advanced procedures to treat childhood cancer.

Doctors recommend what they believe will be the best treatment. In many cases, your child may receive the best care possible by following a plan outlined in a clinical trial.

Q: What if I want to remove my child from a clinical trial?

A: Participation in clinical trials is completely voluntary. Before we begin treating your child in the trial, we will fully explain all parts of the treatment plan and fill out formal consent forms. You may remove your child from the clinical trial at any time.

Q: Are there long-term benefits of clinical trials?

A: Clinical trials are important in the long-term treatment of childhood cancer, and discoveries made years ago through clinical trials are now the standard of care for many of the curable cancers. By participating in a trial, your child will help us develop improved treatments for future children with the same condition.

Q: How can I obtain additional information?

A: To help patients and families understand more about clinical trials, Dana-Farber Cancer Institute produced an informational video, called "Entering a Clinical Trial: is it right for you?" The video explains how to evaluate, enroll and participate in a cancer clinical trial. If the video does not answer your question, our doctors and nurses are available to address your concerns.

How are clinical trials categorized?

Clinical trials are categorized as Phase III, II or I. Classification depends on the purpose of the study and determines the total number of patients who may receive treatment on that clinical trial. Newly-diagnosed patients are often treated using a Phase III trial.

Phase III studies compare the new treatment to a standard (current, state-of-the-art) treatment. At the beginning of the study, doctors don't know if the new treatment is better than the standard treatment. Phase III trials typically enroll hundreds of patients, and are carried out in treatment centers nationwide. If a drug successfully completes the Phase III level of testing, the pharmaceutical company that makes it can apply for FDA approval for this use.

In cases when the standard of care or Phase III treatment is not effective, your child's health care team may recommend a Phase II trial.

Phase II studies investigate whether the new treatment works for a specific type of cancer. They also continue to evaluate the safety of the treatment. It is important to realize that it is not yet known whether a Phase II drug will be effective against your cancer. Phase II studies generally enroll no more than 100 people.

If the standard of care, or a Phase III or Phase II treatment does not deliver positive results, your child's primary oncologist may suggest enrolling your child on a Phase I study.

Phase I studies primarily evaluate:

• the safety of a new drug
• how much and how often the drug needs to be given
• the side effects of the new drug are

This is the first level of testing for a new drug or a known drug given in a new way, but is generally based on laboratory testing or the safe dose of a similar drug given to other people. Very few patients are enrolled on Phase I trials and they are available only through select programs.

Two important aspects of Phase I trials are that:

• Medications used in pediatric cancer clinical trials have usually been tested in adult trials before they are used in children. However, because your child might be one of the first pediatric patients in the world to receive the new drug, the side effects in children may not be known.
• Although this drug has shown promise in the laboratory or in adults, it is not known if it will work against your child’s cancer.

If our physicians recommend enrolling your child on any clinical trial, they will explain its purpose, benefits, possible side effects and answer any questions you or your child have. You will also have to sign a consent form before any treatment begins.

For many children with rare or hard-to-treat conditions, clinical trials provide new options.

• Search our open pediatric cancer clinical trials
• Contact us: If you’re not sure which clinical trials might be right for your child, email us at clinicaltrials@danafarberbostonchildrens.org. We can help you navigate your options.