• Glioblastoma Multiforme (GBM)

    Glioblastoma Multiformes (GBMs) are high-grade gliomas that arise from the brain’s supportive tissue, known as glial cells. These are aggressive tumors that rapidly infiltrate adjacent healthy brain tissue and, as a result, are difficult to treat.

    The majority of GBMs, roughly 65%, occur in the cerebral hemispheres, which control higher functions like speech, movement, thought and sensation. They can also develop in the part of the brain that identifies sensations such as temperature, pain and touch and the region of the brain that controls balance and motor function.

    These tumors are usually diagnosed between the ages of 5 and 9, and occur in boys and girls equally. GBMs occur with increased frequency in children with certain genetic syndromes, including neurofibromatosis 1, Li-Fraumeni syndrome, hereditary nonpolyposis colon cancer and tuberous sclerosis. Most GBMs, however, have no known cause.

    GBM Treatment at Dana-Farber/Boston Children's

    Children with GBMs are treated Dana-Farber/Boston Children's through our Glioma Program, one of the world's largest treatment programs focused on this kind of cancer. Our glioma specialists have extensive expertise in treating all types of gliomas, including GBMs. Our patients receive multidisciplinary care from neuro-oncologists, neurosurgeons, neurologists, and pediatric subspecialists.

    How are GBMs classified?

    When doctors diagnose a brain tumor, they “stage” it, or give it a grade, according to whether it has spread, and if so, how far. This helps us determine treatment options and prognosis. The World Health Organization classification scheme includes four grades of gliomas, which are:

    Low-grade gliomas

    High-grade gliomas

    • grade III (anaplastic)
    • grade IV (glioblastoma multiforme)

    Glioblastoma multiforme is a grade IV tumor. These are aggressive tumors that spread to adjacent healthy brain tissue.

    What are the symptoms and causes of GBMs?

    Brain tumors can cause a variety of symptoms in children based primarily on the location of the tumor, its size and whether it has spread. Keep in mind that the symptoms of a brain tumor may resemble other more common conditions or medical problems. It is important to consult your child's physician for a diagnosis.

    Most GBM symptoms result from increased pressure within the head. Other symptoms relate to the tumor’s specific location, rate of growth and associated inflammation.

    Symptoms can develop slowly over time or begin very suddenly. The following are the most common:

    • headache and lethargy (generally upon awakening in the morning)
    • seizures, depending on tumor type and location
    • compression of surrounding brain structures. Depending on the location, this can cause weakness and other motor dysfunction, hormonal abnormalities or changes in behavior or thought processes.

    As a parent, you undoubtedly want to know what may have caused your child’s tumor. It’s important to understand that these and other brain tumors most often occur with no known cause. There’s nothing that you could have done or avoided doing that would have prevented the tumor from developing.

    There are certain hereditary conditions that are linked to these tumors. GBMs can occur with increased frequency in families with conditions including Li-Fraumeni syndrome, hereditary nonpolyposis colon cancer, tuberous sclerosis and neurofibromatosis Type 1.

    How are GBMs diagnosed?

    The first step in treating your child is forming an accurate and complete diagnosis. In the case of GBMs, this is primarily done with a combination of imaging and biopsy. Your child’s physician may order a number of different tests including:

    • A physical exam and complete medical history.
    • Computerized tomography scan (also called a CT or CAT scan), a diagnostic imaging procedure that uses a combination of x-rays and computer technology to produce cross-sectional images, often called slices, of the body from horizontal and vertical perspectives. CT scans are more detailed than general x-rays.
    • Magnetic resonance imaging (MRI), a diagnostic procedure that produces detailed images of the structures within the brain and spine. An MRI uses a combination of large magnets, radiofrequencies and a computer to analyze organs and structures within the body. No x-rays or radiation are used.
    • Magnetic resonance spectroscopy (MRS), done along with MRI at specialized facilities that can help identify tissue as either normal or tumorous, and which may be able to distinguish between different types of tumors.
    • Lumbar puncture (spinal tap), to remove a small sample of cerebrospinal fluid (CSF). This can determine if any tumor cells have started to spread. In young children, this procedure can safely be performed under sedation.
    • Positron emission tomography (or PET) scan, which measures the use of glucose (blood sugar) within organs and tissues. Tumors use glucose more quickly than normal tissues, causing them to show up brightly on these scans.
    • Biopsy or tissue sample, taken from the tumor to provide definitive information about the type of tumor. This is collected during surgery.

    There may be other diagnostic tests that your doctor will discuss with you depending on your child's individual situation. 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 possible treatment options. All patients undergoing resection of brain tumors in our program are also invited to participate in our ongoing research studies which allow genetic profiling of each child’s tumor. These studies may potentially guide personalized (precision) medicine.

    What are the treatments for GBMs?

    Your child's physician will determine a specific course of treatment based on several factors, including your child's age, overall health and medical history, the type, location, and size of the tumor and the extent of the disease.

    Treatments for GBMs include:

    • Neurosurgery: The first treatment is usually surgery to remove as much of the tumor as possible, ideally using advanced techniques to maximize tumor removal, such as intraoperative MRI (in which surgeons can visualize the tumor as they operate). Complete resection, or surgical removal, of the entire tumor is ideal when possible, though most high-grade gliomas cannot be completely removed because they tend to infiltrate into adjacent healthy tissues. In general, the more completely the tumor can be removed, the greater the chances for survival.
    • Radiation: High-energy waves from a specialized machine damage or shrink tumors. Your child also may receive precisely targeted and dosed radiation to kill cancer cells left behind after surgery. This is important to control the local growth of tumor, and it helps increase survival in high-grade gliomas.
    • Chemotherapy: Chemotherapy refers to drugs that interfere with the cancer cells' ability to grow or reproduce. To date, no chemotherapy regimen has been demonstrated to increase survival rates in children with GBM, though chemotherapy before surgery may help shrink the tumor, making it possible to remove. A variety of chemotherapy regimens have been tested in the treatment of newly diagnosed high-grade gliomas, and some have produced responses but none have improved survival. Studies in adults have suggested that certain drugs can produce modest responses in high-grade gliomas, but they have less effect in children. High-intensity chemotherapy in conjunction with a stem cell transplant also does not seem to improve chances of survival.

    What is the latest research on GBMs?

    Clinical trials, or research studies evaluating new treatment approaches, are a major offering at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. Clinical trials are very important for children with hard-to-treat or relapsed conditions.

    Clinical Trials

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

    It’s possible that your child will be eligible to participate in one of our brain tumor clinical trials. In addition to launching our own clinical trials, we also offer trials available through collaborative groups such as the Children's Oncology Group (COG), the Pacific Pediatric Neuro-Oncology Consortium (PNOC). If your child has progressive or recurrent tumor, she may be eligible for a number of experimental therapies available through these groups, or from one of our independent clinical investigators.

    Clinical and basic scientists at Dana-Farber/Boston Children’s are conducting numerous research studies to help clinicians better understand and treat malignant gliomas. Through the consortia of researchers to which we belong, a number of novel therapies are available for children with both newly diagnosed and current brain tumors. Participation in any clinical trial is completely voluntary. We will take care to fully explain all elements of the treatment plan prior to the start of the trial, and you may remove your child from the medical study at any time.

    What is the long-term outlook for GBMs?

    Unfortunately, the prognosis for GBM remains very poor. In general, more complete surgical removal of the tumor, when possible, results in greater chance of survival. Your child’s doctor will discuss treatment options with you, including experimental clinical trials and supportive care.

    For children with relapsed GBMs, we offer access to the latest clinical trials and experimental therapies. Current trials include novel medications, as well as new methods for the delivery of more traditional agents.

    Our Pediatric Advanced Care Team (PACT) is available to provide supportive treatments intended to optimize your child’s quality of life and promote comfort and healing for children with life-threatening illnesses. In addition, PACT can provide psychosocial support and help arrange end-of-life care, when necessary.

    Children successfully treated for a GBM should visit a survivorship clinic yearly to manage disease complications, screen for recurrence and manage late treatment side effects. A typical follow-up visit is likely to include a physical exam, laboratory testing and imaging scans.

    Through our Stop & Shop Family Pediatric Neuro-Oncology Outcomes Clinic, children are able to meet with their neurosurgeon, radiation oncologist, pediatric neuro-oncologist and neurologists at the same follow-up visit. Our multidisciplinary approach and depth of expertise will give your child on-site access to endocrinologists, neuro-psychologists and alternative/complementary therapy specialists. School liaison and psychosocial personnel from the pediatric brain tumor team are also available. In addition, children needing rehabilitation may meet with speech, physical, and occupational therapists during and after their visit.

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