Central nervous system- Craniopharyngioma

Craniopharyngioma is a type of central nervous system (CNS) tumor. A CNS tumor may be either cancerous or benign. A cancerous tumor is malignant, meaning it is usually fast-growing and can spread to other parts of the body. A benign tumor means the tumor is usually slower-growing and will not spread. Craniopharyngioma is considered a benign tumor, which means that it is usually slow-growing and very unlikely to spread.

About the Central nervous system

The brain and spinal cord make up the CNS, where all vital functions of the body are controlled. The brain is the center of thought, memory, and emotion. It controls the 5 senses, which are smell, touch, taste, hearing, and sight. It also controls movement and other basic body functions, including consciousness, heartbeat, circulation, and breathing. The spinal cord is made up of nerves that carry information from the body to the brain and from the brain to the body.

Craniopharyngioma usually occurs in a part of the brain called the suprasellar region. This region is the area of the brain just above the pituitary gland. The pituitary gland is an important gland and is often called the “master endocrine gland” because it produces several different hormones that regulate many body functions. The optic nerves and a gland called the hypothalamus are located nearby. The hypothalamus regulates hunger, body temperature, thirst, sleep, fatigue, and other behaviors.

About craniopharyngioma

Craniopharyngioma is a slow-growing tumor that may be present for many years before it is found. It may be solid and/or cystic. A cystic tumor has a closed pouch or sac that contains fluid made by the tumor. The solid part often contains areas of calcium that can easily be seen on a computed tomography (CT) scan (see Diagnosis). The cystic part of the tumor often contains very high amounts of protein.

Every person is different, with different factors influencing their risk of being diagnosed with this tumor and the chance of recovery after a diagnosis. It is important to talk with your doctor about any questions you have about the general statistics provided below and what they may mean for your child individually. The original sources for these statistics are provided at the bottom of this page.

How many children are diagnosed with craniopharyngioma?

In 2023, an estimated 130 children under the age of 15 in the United States will be diagnosed with craniopharyngioma. This type of tumor is diagnosed most often between the ages of 5 and 14, but it is possible for a person of any age to be diagnosed with craniopharyngioma.

What is the survival rate for craniopharyngioma?

There are different types of statistics that can help doctors evaluate a child’s chance of recovery from craniopharyngioma. These are called survival statistics. A specific type of survival statistic is called the relative survival rate. It is often used to predict how having a tumor may affect life expectancy. The relative survival rate looks at how likely people with craniopharyngioma are to survive for a certain amount of time after their initial diagnosis or start of treatment compared to the expected survival of similar people without this tumor.

Example: Here is an example to help explain what a relative survival rate means. Please note that this is only an example and not specific to this type of tumor. Let’s assume that the 5-year relative survival rate for a specific type of tumor is 90%. “Percent” means how many out of 100. Imagine there are 1,000 people without this tumor, and based on their age and other characteristics, you expect 900 of the 1,000 to be alive in 5 years. Imagine that there are another 1,000 people similar in age and other characteristics as the first 1,000, but they all have the same specific type of tumor that has a 5-year survival rate of 90%. This means it is expected that 810 of the people with the specific tumor (90% of 900) will be alive in 5 years.

It is important to remember that statistics on the survival rates for children with craniopharyngioma are only an estimate. They cannot tell an individual person if the tumor will or will not shorten their life. Instead, these statistics describe trends in groups of people previously diagnosed with the same disease, including specific stages of the disease.

The 5-year relative survival rate for children under the age of 15 with craniopharyngioma in the United States is 96%.

The survival rates for craniopharyngioma vary based on several factors. These include age, general health, and how well the treatment plan works.

Experts measure relative survival rate statistics for craniopharyngioma every 5 years. This means the estimate may not reflect the results of advancements in how craniopharyngioma is diagnosed or treated from the last 5 years.

Craniopharyngioma: Childhood Risk Factors

A risk factor is anything that increases a person’s chance of developing a tumor. The cause of a craniopharyngioma is not known, and there are no proven risk factors.

Craniopharyngioma in Childhood: Symptoms and Signs

Children with craniopharyngioma may experience one or more of the following symptoms or signs:. Symptoms are changes that you can feel in your body. Signs are changes in something measured, like taking your blood pressure or doing a lab test. Together, symptoms and signs can help describe a medical problem. Sometimes, children with craniopharyngioma do not have any of the symptoms and signs described below. Or, the cause of a symptom or sign may be a medical condition that is not craniopharyngioma.

Symptoms of craniopharyngioma can be caused by hormonal changes, pressure building up in the brain, or by the tumor pressing on nerves or blood vessels, which can cause the brain to not function properly in those areas. Generally, craniopharyngioma is not diagnosed until a child has symptoms.

General symptoms include:

• Headaches, which may be severe and worse in the early morning
• Nausea and/or vomiting
• Difficulty with balance
• Increased sleepiness or fatigue
• Mood or behavior changes

Location-specific symptoms of craniopharyngioma include:

• Excessive thirst and increased urination
• Obesity or excessive weight gain
• Vision changes, blurriness, or loss of peripheral vision
• Slow or halted body growth
• Early or delayed puberty

If you are concerned about any changes your child experiences, please talk with your child’s doctor. Your doctor will try to understand what is causing your child’s symptom(s). They may do an exam and order tests to understand the cause of the problem, which is called a diagnosis.

If a central nervous system (CNS) tumor is diagnosed, relieving symptoms remains an important part of your child’s care and treatment. Managing symptoms may also be called “palliative or supportive care,” which is not the same as hospice care given at the end of life. You can receive palliative and supportive care at any time during treatment. This type of care focuses on managing symptoms and supporting people who face serious illnesses, such as a brain tumor. 

Doctors use many tests to find, or diagnose, a tumor. Doctors may also do tests to learn which treatments could work best.

For most tumors, a biopsy is the only sure way for the doctor to know if an area of the body has a tumor. In a biopsy, the doctor takes a small sample of tissue for testing in a laboratory. If a biopsy is not possible, the doctor may suggest other tests that will help make a diagnosis.

How craniopharyngioma is diagnosed

Your child’s doctor may consider these factors when choosing a diagnostic test:

• The type of tumor suspected
• Your child’s signs and symptoms
• Your child’s age and general health
• The results of earlier medical tests

There are different tests used for diagnosing craniopharyngioma. Not all the tests described here will be used for every child.

• Physical examination. The doctor will examine your child’s head and body and ask questions about the symptoms they are experiencing and their medical history. This may include tests to check your child’s vision, growth and development, and brain function.
• Blood and/or urine tests. The doctor may recommend different blood tests, including tests that measure the levels of certain hormones. If your child has excessive thirst and increased urination, the doctor may recommend a water deprivation test to evaluate the production of a hormone that helps concentrate urine.
• Biopsy. A biopsy is the only way to make a definite diagnosis, even if other tests can suggest that a tumor is present. During biopsy, part or all of the tumor is removed for examination under a microscope. For craniopharyngioma, a neurosurgeon may remove a piece of the tumor or the entire tumor during an operation. A neurosurgeon is a doctor who specializes in central nervous system (CNS) surgery. Then, a pathologist examines the tissue removed during the surgery under a microscope to make a diagnosis. A pathologist is a doctor who specializes in interpreting laboratory tests and evaluating cells, tissues, and organs to diagnose disease. A neuropathologist is a pathologist who specializes in CNS tissues and diseases. Usually, the neurosurgeon will try to safely remove as much of the tumor as possible while limiting damage to the brain

Imaging tests

results of the physical examination and blood and/or urine tests may suggest that imaging tests are needed to look for craniopharyngioma. Imaging tests show pictures of the inside of the body. There are 2 main types of imaging tests used to find craniopharyngioma.

• Computed tomography (CT or CAT) scan. A CT scan takes pictures of the brain using x-rays taken from different angles. A computer combines these pictures into a detailed, 3-dimensional image that shows abnormalities, including tumors. Sometimes, a special dye called a contrast medium is injected into a patient’s vein during the scan to provide better detail.
• Magnetic resonance imaging (MRI). An MRI produces detailed images of the inside of the body using magnetic fields, not x-rays. An MRI can be used to measure the tumor’s size. Often, a special dye called a contrast medium is injected into a patient’s vein before the scan to create a clearer picture.

After diagnostic tests are done, your child’s doctor will review the results with you. This information will be used to recommend treatment options

Craniopharyngioma: Childhood Stages

To take advantage of the special expertise necessary to treat a brain tumor, children with a brain tumor should receive treatment at a specialized pediatric center. Doctors at these centers have extensive experience in treating children with brain tumors and have access to the latest technology. A doctor who specializes in treating children with tumors is called a pediatric oncologist. A doctor who specializes in treating children with a brain tumor is called a pediatric neuro-oncologist. If a pediatric treatment center is not nearby, general cancer centers sometimes have pediatric specialists who are able to be part of your child’s care.

How craniopharyngioma is treated

In many cases, a team of doctors provides care to a child and their family. This is called a multidisciplinary team. Pediatric treatment centers often have extra support services for children and their families, such as child life specialists, nurse specialists, dietitians, physical therapists, occupational therapists, social workers, and counselors. Special activities and programs to help your child and family cope may also be available. Learn more about the types of clinicians who provide this type of care.

Treatment for craniopharyngioma is very often successful. The treatment plan used depends on whether the tumor can be completely removed with surgery. If the tumor cannot be completely removed, radiation therapy is usually recommended. However, the side effects of radiation therapy must be considered when deciding on the best treatment for very young children. Radiation therapy given to this area of the body can cause permanent learning and memory problems. Radiation therapy can also slow a child’s metabolism and decrease the levels of hormones needed for the body to function well. The doctor will consider several factors before recommending a treatment plan for your child.

There are treatments being studied in clinical trials for patients in whom the tumor cannot be completely removed surgically but who would like to avoid or delay radiation therapy. A clinical trial is a research study that tests a new approach to treatment. Talk with the doctor about whether this might be an option for your child. 

Take time to learn about all of your child’s treatment options and be sure to ask questions about things that are unclear. Talk with your child’s doctor about the goals of each treatment and what your child can expect during treatment. These types of conversations are called “shared decision-making.” Shared decision-making is when you and the doctors work together to choose treatments that fit the goals of your child’s care. Learn more about making treatment decisions.

The common types of treatments used for craniopharyngioma are described below. Your child’s care plan may also include treatment for symptoms and side effects, an important part of caring for a brain tumor. Learn more about preparing your child for treatment.

Read more below:

• Surgery
• Radiation therapy
• Physical, emotional, social, and financial effects of craniopharyngioma
• Remission and the chance of recurrence
• If treatment does not work

Physical, emotional, social, and financial effects of craniopharyngioma

Craniopharyngioma and its treatment cause physical symptoms and side effects, as well as emotional, social, and financial effects. Managing all of these effects is called palliative and supportive care. It is an important part of your child’s care that is included along with treatments intended to slow, stop, or eliminate the tumor.

Palliative and supportive care focuses on improving how your child feels during treatment by managing symptoms and supporting patients and their families with other, non-medical needs. Any person, regardless of age or type and extent of the brain tumor, may receive this type of care. And it often works best when it is started right after diagnosis. People who receive palliative and supportive care along with treatment for the tumor often have less severe symptoms, better quality of life, and report that they are more satisfied with treatment.

Palliative treatments vary widely and often include medication, nutritional changes, relaxation techniques, emotional and spiritual support, and other therapies. Your child may also receive palliative treatments, such as surgery or radiation therapy, to improve symptoms.

Before treatment begins, talk with your child’s doctor about the goals of each treatment in the recommended treatment plan. You should also talk about the possible side effects of the specific treatment plan and palliative and supportive care options. Many patients also benefit from talking with a social worker and participating in support groups. Ask your doctor about these resources, too.

Medical care is often expensive, and navigating health insurance can be difficult. Ask your doctor or another member of your health care team about talking with a financial navigator or counselor who may be able to help with your financial concerns.

During treatment, your child’s health care team may ask you to answer questions about your child’s symptoms and side effects and to describe each problem. Be sure to tell the health care team if your child is experiencing a problem. This helps the health care team treat any symptoms and side effects as quickly as possible. It can also help prevent more serious problems in the future.

Remission and the chance of recurrence

After treatment, when the tumor can no longer be seen on a magnetic resonance imaging (MRI) scan, this is called having “no evidence of disease” or NED. It may also be called a remission. A remission may be temporary or permanent. This uncertainty about whether the tumor will come back causes many patients and their families to feel worried or anxious. While the chance of the tumor coming back may be low, it is important to talk with your child’s doctor about the possibility of the tumor returning. This is also called a recurrence. Understanding your child’s risk of recurrence and the treatment options may help you and your family feel more prepared if the tumor does return. Learn more about coping with the fear of recurrence.

If a recurrence happens, craniopharyngioma most commonly comes back in the same place (called a local recurrence) or nearby (called a regional recurrence). When this occurs, a new cycle of testing may be done to learn as much as possible about the recurrence.

After this testing is done, your child’s doctor will talk about the treatment options. Often, the treatment plan will include the treatments described above, such as surgery and/or radiation therapy, but the same type of radiation therapy cannot usually be used more than once. There are also treatments being studied in clinical trials for children with recurrent or worsening craniopharyngioma. Talk with your child’s doctor about whether this might be an option for your child. Whichever treatment plan you choose, palliative and supportive care will be important for relieving symptoms and side effects.

When a tumor recurs or worsens despite treatment, patients and their families sometimes 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 you cope. Learn more about dealing with a recurrence.

If treatment does not work,

Although treatment is successful for the majority of children with craniopharyngioma, sometimes it is not. If a child’s tumor cannot be cured or controlled, this is called an advanced or terminal tumor. This diagnosis is stressful, and advanced craniopharyngioma may be difficult to discuss. However, it is important to have open and honest conversations with your child’s health care team to express your family’s feelings, preferences, and concerns. The health care team has special skills, experience, and knowledge to support patients and their families and is there to help.

Hospice care is designed to provide the best possible quality of life for people who are expected to live less than 6 months. Parents and guardians are encouraged to talk with the health care team about hospice options, which include hospice care at home, a special hospice center, or other health care locations. Nursing care and special equipment can make staying at home a workable option for many families. Some children may be happier and more comfortable if they can attend school part-time or keep up with 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. Learn more about caring for a terminally ill child and advanced care planning.

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

Patient safety and informed consent

To join a clinical trial, parents and children must participate in a process known as informed consent. Informed consent means that parents give permission for their child to participate in a clinical trial and that teenagers give their consent to participate. During informed consent, the doctor should:

• Describe all of the treatment options so that the person understands how the new treatment differs from the standard treatment.
• List all of the risks of the new treatment, which may or may not be different from the risks of standard treatment.
• Explain what will be required of each person in order to participate in the clinical trial, including the number of doctor visits, tests, and the schedule of treatment.
• Describe the purposes of the clinical trial and what researchers are trying to learn.

For craniopharyngioma, due to the general success of surgery and radiation therapy, current research focuses on treatments for recurrent tumors or when tumors are unable to be completely removed with surgery. There is also ongoing research to understand what allows craniopharyngioma to grow. Always talk with your child’s doctor about the best diagnostic and treatment options for your child.

• Treatment of cysts. Sometimes, the tumor itself is not actively growing, but it has a cyst that is growing and causing symptoms. These cysts can be treated with surgery or by injections of other treatments. Such treatments include radioactive phosphorous (also known as P-32), interferon (Roferon-A, Infergen, Intron A, and Alferon), and bleomycin (available as a generic drug).
• Immunotherapy. Immunotherapy, also called biologic therapy, is designed to boost the body’s natural defenses to fight the tumor. It uses materials made either by the body or in a laboratory to improve, target, or restore immune function. Interferon is a type of immunotherapy that helps regulate the body’s natural immune system. Interferon may help treat some childhood craniopharyngiomas. It is given either as a weekly injection under the skin or by injection directly into a cyst (see above).
• Improved methods of giving radiation therapy. For patients with a tumor that cannot be completely removed during surgery, doctors are studying new techniques for giving radiation therapy. The use of 3-dimensional radiation techniques allows high doses of radiation therapy to be delivered to the tumor with lower doses to nearby healthy brain tissue. These methods may help reduce damage to healthy tissue.
• Proton therapy. Proton therapy is a type of external-beam radiation therapy that uses protons rather than x-rays. At high energy, protons can destroy tumor cells. Doctors are studying proton therapy for people with a tumor that cannot be completely removed with surgery. Proton therapy allows for high doses of radiation therapy to be delivered to the tumor while reducing radiation doses to healthy brain tissue. Proton therapy is believed to be equally as effective as existing radiation therapy methods but may have fewer side effects.
• Molecular profiling and subtyping of craniopharyngioma. Recent studies have identified 2 subtypes of craniopharyngiomas: adamantinomatous craniopharyngioma (ACP) and papillary craniopharyngioma (PCP). ACPs are typically diagnosed in children and teens 5 to 15 years old and in people 45 to 60 years old, whereas PCPs are diagnosed mainly in adults in their 50s and 60s. ACPs are most commonly cystic in appearance and are driven by mutations in the CTNNB1 gene (encoding β-catenin) that are involved in regulation and coordination of cell-cell adhesion and gene transcription. PCPs are typically solid tumors and frequently have BRAF V600E mutations. Research is currently underway to better understand the biology of these subtypes, which may potentially lead to the development of molecular-based targeted therapy for craniopharyngiomas.
• Palliative and supportive care. Clinical trials are underway to find better ways of reducing the symptoms and side effects of current craniopharyngioma treatments to improve comfort and quality of life for patients.
• Managing long-term side effects. There are several ongoing studies to find more effective ways to treat obesity, which is a common long-term side effect for survivors of childhood craniopharyngioma

Craniopharyngioma, Childhood, Follow-Up Care

Care for children diagnosed with craniopharyngioma does not end when active treatment has finished. Your child’s health care team will continue to check that the tumor has not come back, manage any side effects, and monitor your child’s overall health. This is called follow-up care. All children treated for craniopharyngioma should have life-long, follow-up care.

Your child’s follow-up care may include regular physical examinations, magnetic resonance imaging (MRI) scans, or both. Doctors want to keep track of your child’s recovery in the months and years ahead. Follow-up care is important to find out whether the tumor has returned, called a recurrence, or is starting to grow again.

A child treated for craniopharyngioma should have regular MRI scans to check for any growth or recurrence of the tumor. Because craniopharyngioma is slow-growing, MRI scans are often only needed once or twice a year. If your child received radiation therapy, there is a small possibility that a different type of brain tumor may develop years later.

Rehabilitation may be recommended, and this could mean any of a wide range of services such as physical therapy, occupational therapy, family or individual counseling, nutritional planning, and/or educational assistance. The goal of rehabilitation is to help survivors and their families regain control over many aspects of their lives and remain as independent as possible.

Managing long-term and late side effects of a childhood brain tumor

Sometimes, side effects may linger beyond the active treatment period. These are called long-term side effects. In addition, other side effects called late effects may develop months or even years after treatment has ended. Late effects can occur almost anywhere in the body. They include physical problems, such as heart and lung problems and second cancers, which are new cancer that happen in someone who has had another type of tumor before. They also include emotional problems, such as anxiety and depression, and problems with memory, thinking, attention, and learning.

Based on the type of treatment your child received, the doctor will recommend the examinations and tests that are needed to check for late effects. For children with craniopharyngioma, it is important to monitor their weight, visual, endocrine (hormone), and metabolic functions. As a result of the tumor and its treatment, hormone replacement (see below) is often needed. 

It is also important that your child’s follow-up care addresses quality of life, including any developmental or emotional concerns.

Weight management

Children who receive treatment for craniopharyngioma often have problems with increased appetite, slow metabolism, weight gain, and obesity. A regular exercise program and dietary changes to the foods they eat are often recommended. Consider talking with a registered dietitian nutritionist (RD or RDN) who has experience in helping survivors manage this side effect. There are also clinical trials to help find new ways to manage weight gain for survivors of craniopharyngioma. Talk with your health care team to learn more about these resources.

Hormone replacement

A common presenting symptom and long-term side effect of children with craniopharyngiomas is diabetes insipidus. Diabetes insipidus is a result of a lack of production of a hormone called vasopressin by the pituitary gland, which is responsible for concentrating urine by the kidneys. Diabetes insipidus is treated with a hormone called desmopressin (DDAVP). Sometimes, other hormones produced by the pituitary gland are affected, too.

Hormone replacement is the use of medication to replace hormones that the body cannot produce enough of on its own. It is a type of hormone therapy. Hormone replacement is often necessary for children with craniopharyngioma because the tumor or its treatment may damage parts of the brain that make hormones, including the pituitary gland and the hypothalamus. An endocrinologist is a doctor who specializes in problems with glands and the endocrine system. This specialist will work with you and your child to determine the hormonal therapy that is needed.

Caring for a child with craniopharyngioma

Family members and friends often play an important role in taking care of a child with craniopharyngioma. This is called being a caregiver. As a parent or guardian, you are the primary caregiver for your child. However, friends and family can give you valuable support, even if they live far away.

When your child has craniopharyngioma, you may have an additional range of responsibilities. These may include giving medications or managing symptoms and side effects. However, it is important to seek help from others. Below are some of the responsibilities your family or friends could help with:

• Providing short-term care for your child
• Giving support and encouragement
• Assisting with meals or household chores
• Helping with insurance and billing issues

A caregiving plan can help caregivers stay organized and identify opportunities to delegate tasks to others. Ask how much care your child may need at home and with daily tasks during and after treatment.