Medulloblastoma - Childhood

Treatment Options

In general, tumors in children are uncommon, so it can be hard for doctors to plan treatments unless they know what has been most effective in other children. That's why more than 60% of children with cancer are treated as part of a clinical trial. Clinical trials are research studies that compare the standard treatments (the best proven treatments available) with newer treatments that may be more effective. 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 a CNS tumor should be treated at a specialized cancer center. Doctors at these centers have extensive experience in treating children and have access to the latest research. A doctor who specializes in treating children with cancer is called a pediatric oncologist. 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, physical and occupational therapists, social workers, and counselors. Special activities and programs to help your child and family cope may also be available.

Treatment options and recommendations depend on several factors, including the type and stage of the tumor, possible side effects, the family's preferences, and the child's overall health.

Three types of treatment are typically used to treat medulloblastoma: surgery, radiation therapy, and chemotherapy. Sometimes, the treatments are used in combination. In some situations, stem cell or bone marrow transplantation may be recommended. Descriptions of these common treatment options for medulloblastoma are listed below.

Surgery

Surgery is the removal of the tumor and surrounding tissue during an operation. Most commonly, it is the first treatment used for medulloblastoma. In addition to removing or reducing the size of the tumor, surgery can provide a tissue sample to diagnose the tumor and analysis, as explained in the Diagnosis section.

Surgery to the brain requires the removal of part of the skull, a procedure called a craniotomy. After the surgeon removes the tumor, the patient's own bone will be used to cover the opening in the skull. There have been rapid advances in surgery for brain tumors, including the use of cortical mapping (which allows doctors to identify certain areas of the brain that control the senses, language, and motor skills) and enhanced imaging machines to give surgeons more tools to plan and perform the surgery.

Some tumors cannot be removed by surgery because of their location; these tumors are called inoperable. In these situations, the doctor will recommend other treatment options. If the tumor is cancerous, even if the cancer cannot be cured, its removal can relieve symptoms if it is pressing on parts of the brain.

Side effects from surgery for medulloblastoma can vary. Patients are encouraged to talk about possible short-term and long-term side effects with their doctor before surgery. Occasionally the surgeon will place a plastic tube (called a shunt) to move the fluid made inside the brain to the abdomen so that the fluid does not build up in the brain and cause problems. In most instances, the placement of a shunt is safe and most children do not have any problems from the procedure. Patients and their parents are encouraged to talk with the doctor about the possible side effects from placing a shunt.

Radiation therapy

Radiation therapy is the use of high-energy x-rays or other particles to kill tumor cells. A doctor who specializes in giving radiation therapy to treat a tumor 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 (schedule) usually consists of a specific number of treatments given over a set period of time.

Because radiation therapy can sometimes interfere with the normal growth and development of a child's brain and spine, the doctor may choose to treat the tumor in another way. If radiation therapy is recommended, the approach will be based on the child's age. For children older than three, radiation therapy includes a moderate dose to the entire brain and spine, followed by a higher dose aimed directly at the tumor and the surrounding area or the back part of the brain. For children younger than three, radiation therapy may be limited to the back part of the brain or the tumor and the surrounding area after surgery and chemotherapy (see below).

Short term side effects from radiation therapy may include fatigue, mild skin reactions, upset stomach, and loose bowel movements. Most of these side effects go away soon after treatment is finished. Long-term side effects of radiation therapy may include problems with growth, hormone deficiencies, and problems with learning, especially higher education.

Chemotherapy

Chemotherapy is the use of drugs to kill tumor cells, usually by stopping the tumor cells' ability to grow and divide. Systemic chemotherapy is delivered through the bloodstream to reach tumor cells throughout the body. Chemotherapy is given by a pediatric oncologist or a medical oncologist, a doctor who specializes in treating a tumor with medication. A chemotherapy regime (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.

Chemotherapy for medulloblastoma may be given orally (by mouth), intravenously (IV; by injection in a vein), or by injection into a muscle. Sometimes, it is delivered directly into the cerebrospinal fluid, which is fluid that circulates around the brain and spinal cord. Researchers are studying ways to use chemotherapy before, during, or after radiation therapy, as the two types of treatment can work better to treat the tumor when combined. High-dose chemotherapy may be used before or instead of radiation therapy for children younger than three to four years old. Several cycles of high-dose chemotherapy may be used before or after radiation therapy in children older than three to four years old. High-dose chemotherapy works best when there is little tumor left after surgery.

The side effects of chemotherapy depend on the individual and the dose used, but they can include fatigue, risk of infection, 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 the 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. 

Stem cell transplantation/bone marrow transplantation

A stem cell/bone marrow transplant may be used for children with recurrent medulloblastoma. 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 stems cells are typically being transplanted, not the actual bone marrow tissue.

Before recommending transplantation, doctors will talk with the patient about the risks of this treatment and consider several other factors, such as the type of tumor, 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). Only AUTO transplantation is used to treat medulloblastoma.

The goal of transplantation is to destroy cancer cells in the marrow, blood, and other parts of the body and allow replacement blood stem cells to create healthy bone marrow. In most stem cell transplants, the patient receives high doses of chemotherapy and/or radiation therapy to destroy as many cancer cells as possible. 

Palliative/supportive care

A tumor and its treatment often causes side effects. In addition to treatment to slow, stop, or eliminate the tumor, an important part of 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 tumor 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 families report they are more satisfied with treatment.

Before treatment begins, talk with your child's health care team about the possible side effects of your child's 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 medulloblastoma

A remission is when the tumor 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 leads to many people feeling worried or anxious that the tumor will come back. While many remissions are permanent, it's important to talk with your child's doctor about the possibility of the tumor returning. Understanding the risk of recurrence and the treatment options may help you feel more prepared if the disease does return. 

If the tumor does return after the original treatment, it is called a recurrent tumor. It may come back in the same place (called a local recurrence), nearby (regional recurrence), or in another place (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, you and your child's doctor will talk about the treatment options. Often the treatment plan will include the therapies described above (such as surgery, radiation therapy, or chemotherapy) but may be used in a different combination or given at a different pace. Your child's doctor may also suggest clinical trials that are studying new ways to treat this type of recurrent tumor.

Treatment of recurrent medulloblastoma depends on two factors:

Whether the tumor recurred in the place where it began or in another part of the brain

The type of treatment the child received for the original tumor

Depending on each child's situation, the doctor may recommend surgery, radiation therapy, chemotherapy, or stem cell/bone marrow transplantation. High-dose chemotherapy may be a part of “salvage” treatment for children with recurrent medulloblastoma. Salvage chemotherapy refers to the use of chemotherapy for a patient who experiences a recurrence of cancer following initial treatment, in the hope of providing a cure or prolonging life.

If your child is diagnosed with a recurrence, you and your family may experience emotions such as disbelief or fear. You are encouraged to talk with your child's health care team about these feelings and ask about support services to help your family cope. 

If treatment fails

Although treatment is successful for the majority of children with cancer, sometimes it is not. If a child's tumor cannot be cured or controlled, this is called advanced or terminal 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 team members have special skills, experience, and knowledge to support patients and their families.

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 arrange to attend school part-time or keep up other activities and social connections. 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 extremely important as 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 a tumor, 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 medulloblastoma are described in detail within the Treatment section. Side effects depend on a variety of factors, including the grade, the length and dosage of treatment(s), and your child's overall health.

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 child with medulloblastoma. 

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.

After Treatment

After treatment for medulloblastoma 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 a CNS tumor, including medulloblastoma, should have life-long, follow-up care.

Based on the type of treatment the child received, the doctor will determine what examinations and tests are need to check for long-term side effects, such as cognitive (thought-process) and endocrine (hormonal) symptoms caused by radiation therapy to the brain or spinal cord, side effects caused by surgery, and the possibility of secondary cancers. 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 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 medulloblastoma, ways to prevent it, how to best treat it, and how to provide the best care to children 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.

New staging systems. In addition to the staging criteria currently used, newer factors are being examined to help stage medulloblastoma in order to choose treatment. For example, tumors with certain features called anaplastic when examined under the microscope are being treated as high-risk tumors in some clinical trials.

Molecular testing of the tumor. Researchers are looking at laboratory tests for tumor samples to identify specific genes, proteins, and other factors unique to medulloblastoma. Results from these studies may help find new drugs that target these factors that help medulloblastoma grow and spread. Such drugs are being tested for patients whose tumors recur after initial treatment. These molecular features are also being looked at to predict how well treatment will work. Future studies will likely base patients' treatment on each tumor's specific molecular features. 

Improved methods of imaging and surgery. Imaging techniques have been developed that help surgeons pinpoint the tumor's location, which may reduce or prevent damage to the healthy parts of the brain during treatment.

Functional MRI (fMRI) is an imaging technique that identifies the parts of the brain that control speech, hearing, vision, touch, and movement. The specific locations of these functions are slightly different in each person, so fMRI allows surgeons to plan surgery around these areas.

Image-guided stereotaxis allows surgeons to visualize and operate on the brain using three-dimensional outlines of the brain and the tumor. Along with specialized software, these images help guide the surgeon to the tumor. A tumor that was once considered inoperable can now be removed by using this technique.

Improved methods of delivering radiation treatment. Conformal radiation therapy is a way to deliver high doses of radiation therapy more directly to a tumor and avoid healthy tissue. This technique produces detailed three-dimensional maps of the brain and tumor, so doctors know exactly where to deliver the radiation therapy.

Combination of therapies. Other areas of research include studies that examine the safety of reducing the doses of radiation therapy for children with a standard-risk tumor by using new approaches to chemotherapy. Other studies in infants and older children with high-risk tumors focus on new drugs and combinations of radiation therapy and chemotherapy to slow or stop tumor growth.

Supportive care. Clinical trials are underway to find better ways of reducing symptoms and side effects of current medulloblastoma treatments in order to improve patients' comfort and quality of life.