Primary brain tumors, including craniopharyngiomas, are a diverse group of diseases that together constitute the most common solid tumors of childhood. Brain tumors are classified according to histology, but tumor location and extent of spread are important factors that affect treatment and prognosis.
Craniopharyngiomas are uncommon pediatric brain tumors. They are believed to be congenital in origin, arising from ectodermal remnants, Rathke cleft, or other embryonal epithelium, and often occur in the suprasellar region with an intrasellar portion. Magnetic resonance imaging (MRI) and computed tomography (CT) imaging are used to diagnose craniopharyngiomas, but histologic confirmation is generally required before treatment.
The treatment of patients with newly diagnosed craniopharyngiomas may include a combination of surgery, radiation therapy, cyst drainage, and/or intracystic interferon-alpha. The treatment of patients with recurrent craniopharyngiomas depends on the initial treatment used. With current treatment strategies, the 5-year and 10-year survival rates exceed 90% for children between the ages of 0 and 14 years.
The PDQ childhood brain tumor treatment summaries are organized primarily according to the World Health Organization classification of nervous system tumors. For a full description of the classification of nervous system tumors and a link to the corresponding treatment summary for each type of brain tumor, refer to the PDQ summary on Childhood Brain and Spinal Cord Tumors Treatment Overview.
No predisposing factors have been identified.
Figure 1. Anatomy of the inside of the brain, showing the pineal and pituitary glands, optic nerve, ventricles (with cerebrospinal fluid shown in blue), and other parts of the brain. The tentorium separates the cerebrum from the cerebellum. The infratentorium (posterior fossa) is the region below the tentorium that contains the brain stem, cerebellum, and fourth ventricle. The supratentorium is the region above the tentorium and denotes the region that contains the cerebrum.
Craniopharyngiomas occur in the region of the pituitary gland, and endocrine function may be affected (refer to Figure 2). Additionally, their closeness to the optic nerves and chiasm may result in vision problems. Some patients present with obstructive hydrocephalus caused by tumor growth within the third ventricle. Rarely, tumors may extend into the posterior fossa, and patients may present with headache, diplopia, ataxia, and hearing loss.
Figure 2. Drawing showing a coronal view of the inside of the brain where craniopharyngiomas may form. The tumor usually occurs in the region of the pituitary gland, near the optic chiasm and optic nerves.
CT scans and MRI scans are often diagnostic for childhood craniopharyngiomas, with most tumors demonstrating intratumoral calcifications and a solid and cystic component. MRI of the spinal axis is not routinely performed.
Craniopharyngiomas without calcification may be confused with other tumor types, including germ cell tumors, hypothalamic/chiasmatic astrocytomas, or Langerhans cell histiocytosis. Biopsy or resection is required to confirm the diagnosis.
Apart from imaging, patients often undergo endocrine testing and formal vision examination, including visual-field evaluation.
Regardless of the treatment modality, the long-term event-free survival rate is approximately 65% in children;[3,4] the 5-year and 10-year overall survival rates exceed 90% in children between the ages of 0 and 14 years.[1,7,8,9]
Craniopharyngiomas are histologically benign and often occur in the suprasellar region, with an intrasellar portion. They may be locally invasive and typically do not metastasize to remote brain locations.
Craniopharyngiomas are classified as one of the following:
Table 1 describes the treatment options for newly diagnosed and recurrent childhood craniopharyngioma.
|Treatment Group||Treatment Options|
|Newly diagnosed childhood craniopharyngioma||Complete resection with or without radiation therapy|
|Subtotal resection with radiation therapy|
|Primary cyst drainage with or without radiation therapy|
|Intracystic therapy (i.e., interferon-alpha)|
|Recurrent childhood craniopharyngioma||Surgery|
|Radiation therapy, including radiosurgery|
|Intracystic therapy (intracavitary instillation of radioactive phosphorus P 32, bleomycin, or interferon-alpha, for those with cystic recurrences)|
|Systemic peginterferon alpha-2b and targeted therapy|
Dramatic improvements in survival have been achieved for children and adolescents with cancer. Between 1975 and 2010, childhood cancer mortality decreased by more than 50%. Childhood and adolescent cancer survivors require close monitoring because cancer therapy side effects may persist or develop months or years after treatment. (Refer to the PDQ summary on Late Effects of Treatment for Childhood Cancer for specific information about the incidence, type, and monitoring of late effects in childhood and adolescent cancer survivors.)
There is no consensus on the optimal treatment for patients with newly diagnosed craniopharyngioma, in part, because of the lack of prospective randomized trials that compare the different treatment options. Treatment is individualized on the basis of the following factors:
Treatment options for newly diagnosed childhood craniopharyngioma include the following:
Complete Resection With or Without Radiation Therapy
It may be possible to remove all visible tumor and achieve long-term disease control.[Level of evidence: 3iA]; [Level of evidence: 3iiiB]; [Level of evidence: 3iiiC] A 5-year progression-free survival (PFS) rate of about 65% has been reported. Gross-total resection is often technically challenging because the tumor is surrounded by vital structures, including the optic nerves and chiasm, the carotid artery and its branches, the hypothalamus, and the third cranial nerve. These structures may limit the ability to remove the entire tumor.
Many surgical approaches have been described, and the choice is determined by tumor size, location, and extension. Surgical approaches include the following:
Complications of complete resection using either approach include the following:
If the surgeon indicates that the tumor was not completely removed or if postoperative imaging reveals residual craniopharyngioma, radiation therapy may be recommended to prevent early progression (refer to the Subtotal Resection With Radiation Therapy section of this summary for more information).[Level of evidence: 3iiiDiii]
Periodic surveillance using magnetic resonance imaging is performed for several years after complete resection because of the possibility of tumor recurrence.
Subtotal Resection With Radiation Therapy
The goal of limited surgery is to establish a diagnosis, drain any cysts, and decompress the optic nerves. No attempt is made to remove tumor from the pituitary stalk or hypothalamus in an effort to minimize the late effects associated with complete resection.
The surgical procedure is often followed by radiation therapy; with this approach, the 5-year PFS rates are approximately 70% to 90%,[4,14]; [Level of evidence: 3iDiii] and the 10-year overall survival rates exceed 90%.[Level of evidence: 3iiA]; [Level of evidence: 3iiiDiii] The standard approach to radiation therapy involves fractionated external-beam radiation, with a recommended dose of 50 Gy to 54 Gy, in 1.8-Gy fractions, restricting the optic chiasm dose to 54 Gy.[18,19,20,21] Transient cyst enlargement may be noted soon after radiation therapy but generally resolves without further intervention.[Level of evidence: 3iDiv]
A systematic review of 109 reports that described extent of resection found that subtotal resection plus radiation therapy was associated with rates of tumor control similar to those for gross-total resection. It was also reported that both approaches were associated with PFS rates higher than those for subtotal resection alone.[Level of evidence: 3iiiDiii]
Surgical complications with a subtotal resection are less likely than with a complete resection.
Complications of radiation therapy include the following:
Newer radiation technologies such as intensity-modulated photon therapy and proton-beam radiation therapy may reduce the radiation dose to uninvolved parts of the brain and spare normal tissue. When these highly conformal radiation treatments are employed, interim imaging is commonly performed to detect changes in cyst volume, with treatment plans modified as appropriate.[21,25,26] It is unknown whether such technologies reduce late effects from radiation.[15,21,26,27]
Tumor progression remains a concern, and it is usually not possible to repeat a full course of standard fractionated radiation. In selected cases, stereotactic radiation therapy can be delivered as a single large dose of radiation to a small field.[Level of evidence: 3iC] Proximity of the craniopharyngioma to vital structures, particularly the optic nerves, limits this to small tumors within the sella.[Level of evidence: 3iiiDiii]
Primary Cyst Drainage With or Without Radiation Therapy
For predominantly cystic craniopharyngiomas, stereotactic drainage of the cyst followed by radiation therapy may be a viable alternative treatment to attempted surgical resection. This procedure may also allow the surgeon to use the following two-staged approach:
Intracystic interferon-alpha may also be a treatment option for primary cystic lesions, after ensuring the integrity of the cyst wall, to delay the need for alternative therapies.
Recurrence of craniopharyngioma occurs in approximately 35% of patients.
Treatment options for recurrent childhood craniopharyngioma include the following:
The management of recurrent craniopharyngioma is determined largely by previous therapy. Repeat attempts at gross-total resections are difficult, and long-term disease control is achieved less often.[Level of evidence: 3iiiDi] Complications are more frequent than with initial surgery.[Level of evidence: 3iiiDi]
If not previously employed, external-beam radiation therapy is an option, including the consideration of radiosurgery in selected circumstances.[Level of evidence: 3iiiDiii]
Cystic recurrences may be treated with intracavitary instillation of varying agents via placement of an Ommaya catheter. These agents have included radioactive 32P or other radioactive compounds,[6,7,8]; [Level of evidence: 2A] bleomycin,; [Level of evidence: 3iiiDiii] or interferon-alpha.; [Level of evidence: 3iiiB]; [Level of evidence: 3iiiDi] These strategies have been found to be useful in certain cases, and a low risk of complications has been reported. However, none of these approaches have shown efficacy against solid portions of the tumor.
Systemic Peginterferon Alpha-2b and Targeted Therapy
Although systemic therapy is generally not utilized, a small series has shown that the use of subcutaneous peginterferon alpha-2b to manage cystic recurrences can result in durable responses.[Level of evidence: 3iiiDiii] In addition, case reports demonstrating dramatic tumor response to BRAF inhibitors in adults with papillary craniopharyngiomas suggest that there may be a role for BRAF inhibitor therapy in the rare setting of a child with a papillary craniopharyngioma.[16,17]
Quality-of-life issues are important in pediatric patients with craniopharyngiomas and are difficult to generalize because of the various treatment modalities. In one series of 261 patients diagnosed with craniopharyngiomas before 2000, hypothalamic involvement was associated with lower overall survival (OS), impaired quality of life, and severe obesity.[Level of evidence: 3iA]
Late effects of treatment for childhood craniopharyngioma include the following:
Refer to the PDQ summary on Late Effects of Treatment for Childhood Cancer for specific information about the incidence, type, and monitoring of late effects in childhood and adolescent cancer survivors.
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Purpose of This Summary
This PDQ cancer information summary for health professionals provides comprehensive, peer-reviewed, evidence-based information about the treatment of childhood craniopharyngioma. It is intended as a resource to inform and assist clinicians who care for cancer patients. It does not provide formal guidelines or recommendations for making health care decisions.
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PDQ® Pediatric Treatment Editorial Board. PDQ Childhood Craniopharyngioma Treatment. Bethesda, MD: National Cancer Institute. Updated <MM/DD/YYYY>. Available at: https://www.cancer.gov/types/brain/hp/child-cranio-treatment-pdq. Accessed <MM/DD/YYYY>. [PMID: 26389330]
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Last Revised: 2021-05-19
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