Craniopharyngioma

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    Craniopharyngioma is a benign brain tumor that may be considered malignant because it can damage the hypothalamus, which is the area of the brain that controls body temperature, hunger, and thirst. Learn about craniopharyngioma and find information on how we support and care for children and teens with craniopharyngioma before, during, and after treatment.

The Brain Tumor Center at Dana-Farber/Boston Children's Cancer and Blood Disorders Center cares for children with many different types of common and rare brain and spinal tumors, including astrocytomas, medulloblastomas, ependymoma, glioblastomas, and primitive neuroectodermal tumors (PNET).

Your child will receive care from some of the world’s most experienced pediatric brain tumor doctors and internationally recognized pediatric subspecialists.

Our team works closely together to develop a care plan that offers your child the highest possible quality of life after treatment, and takes the needs of your child and your family into account.

Children treated at the Brain Tumor Center have access to some of the most advanced diagnostics and therapies, including:

  • Quick and accurate diagnosis from our dedicated pediatric neuropathologist
  • Access to advanced technologies like the intraoperative MRI, which allows our neurosurgeons to see detailed images of the brain during surgery
  • Advanced pediatric radiation oncology services, including targeted radiosurgery and low-dose radiation therapy that minimize exposure to radiation
  • Outpatient and oral chemotherapy, which may minimize the number of times your child will need to visit the hospital
  • Innovative therapies offered through clinical trials at Dana-Farber, Boston Children's Hospital, and nationally
  • Specialized programs for the treatment of low- and high-grade gliomas, and medulloblastoma

Thanks to refined surgical techniques and improved chemotherapy and radiation therapy, the majority of children with brain and spinal cord tumors are now long-term survivors. However, they may face physical, social, and intellectual challenges that require specialized care.

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Information for: Patients | Healthcare Professionals

Craniopharyngioma

What is a craniopharyngioma?

A craniopharyngioma is a tumor of the brain that commonly affects children. It grows in the area of the pituitary gland and the nerves that relay vision from the eyes to the brain (optic nerves), and frequently grows up into the base of the brain. The tumors arise from cells that in the developing embryo had helped to form the normal pituitary gland. For reasons that are not understood, these cells begin to grow on their own, producing masses that often contain both solid tissue and fluid. In children, portions of the tumor frequently have calcium deposits. The fluid ("cystic") portions of the tumors can reach very large size, and occasionally extend into both sides of the brain.

How does Dana-Farber/Boston Children's approach craniopharyngioma treatment?

Children with craniopharyngiomas are treated through the Brain Tumor Center at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, an integrated pediatric oncology program through Dana-Farber Cancer Institute and Boston Children’s Hospital that provides—in one specialized program—all the services of both a leading cancer center and a pediatric hospital. Our Brain Tumor Center is a world-renowned destination for children with malignant and non-malignant brain and spinal cord tumors.

How does the craniopharyngioma cause symptoms?

These tumors cause symptoms in three major ways. Some children will initially have difficulties with hormonal functions since these tumors grow in or above the pituitary gland and because the pituitary gland regulates virtually all of the hormones in the body; including growth rate and the functions of the thyroid, adrenal, steroid, and sex glands. Perhaps the most common manifestation of this hormone effect is a fall-off in a normal growth rate due to a lack of growth hormone.

A second form of clinical symptom is related to increased pressure within the brain. These tumors can grow up into the base of the brain, obstructing the chambers in the brain ("ventricles") through which the fluid in the brain ("CSF") circulates. This obstruction results in headache, nausea, and vomiting - all symptoms of the fluid and pressure building up inside the brain; a condition called hydrocephalus.

A third, and unfortunately fairly common presentation of these tumors, is a loss of vision. Because children rarely complain about slowly developing health problems and because these tumors are located near the base of the brain where the optic nerves are located, it is possible for these tumors to put gradually increasing pressure on the optic nerves which leads to severe vision loss in one or both eyes in the peripheral or central part of the visual field. Many children will be initially diagnosed when they fail a vision test at school or when it is suddenly noted by their parents that their vision is dramatically reduced.

In a personal series of patients treated at the Dana-Farber/Boston Children's over the past decade, the majority of children had diagnosed when they had symptoms because of pressure within the brain and blockage of CSF circulation. A slightly smaller percentage had visual deterioration and the smallest group had endocrine changes.

My child has been diagnosed as having a possible craniopharyngioma; what diagnostic tests should be carried out?

When some children with large craniopharyngiomas are initially diagnosed, they may be quite ill with increased pressure in their brain and there really is not much time to do any special additional diagnostic testing. In this case, surgery must be carried out rapidly to reduce the brain pressure.

If time is available, however, there are some tests that are helpful to the treating physicians. We believe it is worthwhile to get a standard CT scan of the brain to determine the extent of the calcium build-up within the tumor. Calcium deposits, which are not seen well on MRI, often denote areas of the tumor that may be difficult to remove and knowing location of these areas can help the surgeon plan the operation more effectively. We like to obtain endocrinologic and ophthalmologic evaluations before the surgery, since baseline evaluations of both of these areas of body function can help to predict post-operative problems and suggest management strategies to the neurosurgeon, anesthesiologist and endocrinologist caring for the child.

Once the diagnosis has been made, what are the options for treatment of my child's tumor?

There continues to be a great deal of discussion among pediatric neurosurgeons throughout the world regarding the appropriate treatment for these tumors. The two most commonly used treatment options include complete excision of the tumor surgically or partial removal followed by radiation therapy.

There is no question that total removal of these tumors is perhaps the only way of guaranteeing their cure. Yet because of the nature of the tumors themselves, total removal may sometimes result in complications that affect the quality of life of the child following surgery. For example, the frequently intense scarring in the brain next to these tumors may make their removal difficult without injury to the adjacent brain. Because the brain centers near the tumor typically control not only hormonal function but also the control of appetite and emotions, any of these functions could be adversely affected if these centers are injured during an attempt at total removal of the tumor. In addition, these tumors are often quite adherent to arteries that supply blood to vital areas of the brain. If these arteries are injured during the removal of the tumor, the patient could suffer a stroke resulting in permanent paralysis or other severe neurologic deficit. The bleeding that occurs when large arteries at the base of the brain are torn may be very difficult to stop.

Therefore, we believe that the surgeon must have as much information as is practicable to obtain regarding the tumor's boundaries and be able to use the utmost clinical judgment when removing the tumor in order to avoid or minimize these potential complications. Despite the most careful planning and intraoperative technical expertise, however, inadvertent injury to these vital structures can still occur.

Radiation therapy is an important part of treatment strategy in many institutions. Although these tumors are "benign," their growth can be frequently slowed or stopped by radiation therapy. Various types of radiation therapy can be employed to treat craniopharyngioma including the delivery of external beam radiation to the tumor volume or the instillation of isotopes such as radioactive phosphorus ("P32") or Yttrium directly within cystic portions of the tumor. When these treatments are employed, a skull opening must be made for either the placement of a semi-permanent tube into the cyst or to provide an avenue for the direct injection of the radioactive isotope into the cyst.

Why aren't all craniopharyngiomas treated with radiation therapy if it is effective against the tumor?

Many children who come to the hospital with a craniopharyngioma are often very sick with pressure symptoms or visual deficits. The radiation therapy does not work quickly enough to reduce these symptoms and surgery is often required emergently to deal with the pressure of the tumor and cyst. Once radiation therapy is given, it unfortunately does not have a guaranteed cure rate and it is still possible for tumors to regrow even years after initial treatment. The radiation treatment in addition has the potential for significant long term side effects - including effects on the child's learning skills, hormonal status, and blood circulation to the brain - all because of the late damaging effects of radiation therapy to the brain of a developing child.

There have been many recent advances in the technological delivery of radiation therapy, however, that may lessen the long-term likelihood of these complications. It is beyond the scope of this presentation to discuss radiation therapy and its complications in detail, but this is an issue that you should explore in detail with your child's physicians when treatment options are being reviewed. The patient's age, symptoms, preoperative hormonal status, and size and configuration of the tumor are among the many factors which need to be taken into consideration when tumor treatment is planned.

What treatment options do you usually recommend for children with craniopharyngiomas?

Almost always, we attempt to remove as much of the tumor as is safely possible and, hopefully, cure the patient by removing all of it. We have been able to do this in about 65% of our operated patients. In the vast majority of children with craniopharyngiomas, the tumors grow from the pituitary stalk region; meaning that the removal of the tumor invariably results in a full and complete pituitary hormonal deficit. These children, therefore, require life-long replacement with hormones and must remain under the care of an endocrinologist throughout their growth and development. Even if surgery is completely successful in removing the tumor, there is still a possibility that the tumor could regrow from small fragments of the tumor inadvertently left behind at the operation. Some children will require reoperation to remove these recurrences.

Surgery does not sound very pleasant, but the effects of the tumor if left untreated will result in similar hormonal deficits eventually and in most cases the risks of surgery are well worth the benefits of reducing the intracranial pressure, preserving vision, and achieving a long term cure. If cysts formed by the tumor are extremely large and complex, we may drain them either as part of the initial operation or as a separate procedure using a tube directed through a hole in the skull into the cyst. Very rarely, we will utilize certain chemotherapeutic agents such as Bleomycin to slow or stop cyst growth by injecting them directly into the cyst themselves. There is relatively limited experience with this technique in North America, but there are several centers developing more experience with this technique and we have used it in a few patients in our own hospital with varying success.

What is the length of follow-up that is needed for a child following surgery for a craniopharyngioma?

Our experience is that these children will need life-time follow-up. Although many hospitals and surgeons state their cure rate in terms of 5 or 10 year follow up, recurrences of this tumor can occur remote from surgery. In a personal series, there has been a recurrence rate of 16% even after total removal as judged by post-operative scans and the surgeon's judgment at the initial operation. For this reason, we will follow our patients yearly until ten years after surgery and then on an every two year basis indefinitely.

The follow-up issues include not only the possibility of tumor regrowth, but also continued monitoring and treatment of side-effects related to radiotherapy and other adjuvant treatments. Most of our children with craniopharyngiomas are followed in the Pediatric Brain Tumor Clinic because in this clinic a continuing, ongoing relationship can be maintained with all of the specialists needed for long term follow-up including ophthalmologists, endocrinologists, neuropsychologists, neurologists, and neurosurgeons.

My surgeon has recommended focused radiation treatment (stereotactic radiosurgery, Gamma Knife, proton beam) for my child's tumor; is this recommendation reasonable?

For some patients, these highly focused, single-dose radiation techniques are a useful method to treat areas of tumor that cannot be successfully removed by any other means. Because these radiation modalities are intended to destroy all tissue that has been targeted, the technique is best used only for small tumor areas that are not touching structures whose function would be harmed by this type of radiation. For example, if the tumor is near or touching the brain stem or optic nerves, these treatments may not be safe. Prior radiotherapy also will affect radiation dose that can be used.

Learn more

Learn more about treatment options and the treatment team at our Brain Tumor Center.


General Information

The PDQ childhood brain tumor treatment summaries are organized primarily according to the World Health Organization classification of nervous system tumors.[1][2] 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.

Dramatic improvements in survival have been achieved for children and adolescents with cancer. Between 1975 and 2010, childhood cancer mortality has decreased by more than 50%.[3] Childhood and adolescent cancer survivors require close follow-up 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.)

Primary brain tumors are a diverse group of diseases that together constitute the most common solid tumor of childhood. Brain tumors are classified according to histology, but tumor location and extent of spread are important factors that affect treatment and prognosis. Immunohistochemical analysis, cytogenetic and molecular genetic findings, and measures of mitotic activity are increasingly used in tumor diagnosis and classification.

References:

  1. Louis DN, Ohgaki H, Wiestler OD, et al., eds.: WHO Classification of Tumours of the Central Nervous System. 4th ed. Lyon, France: IARC Press, 2007.

  2. Louis DN, Ohgaki H, Wiestler OD, et al.: The 2007 WHO classification of tumours of the central nervous system. Acta Neuropathol 114 (2): 97-109, 2007.

  3. Smith MA, Altekruse SF, Adamson PC, et al.: Declining childhood and adolescent cancer mortality. Cancer 120 (16): 2497-506, 2014.

Background Information About Childhood Craniopharyngioma

Incidence and Presentation

Craniopharyngiomas are relatively rare pediatric tumors, accounting for about 6% of all intracranial tumors in children.[1][2][3] They are believed to be congenital in origin, arising from ectodermal remnants, Rathke cleft, or other embryonal epithelium in the sellar and/or parasellar area. No predisposing factors have been identified.

Because craniopharyngiomas occur in the region of the pituitary gland, endocrine function and growth may be affected. Additionally, the close proximity of the tumor to the optic nerves and chiasm may result in vision problems. Some patients present with obstructive hydrocephalus due to tumor obstruction of the third ventricle. Extremely rarely, the tumor may predominate in the posterior fossa with presenting symptoms of headache, diplopia, ataxia, and hearing loss.[4]

Prognosis

Regardless of the treatment modality, long-term survival is approximately 85% in children [2][3] with 5- and 10-year overall survival rates greater than 90%.[5][6][7][8]

References:

  1. Bunin GR, Surawicz TS, Witman PA, et al.: The descriptive epidemiology of craniopharyngioma. J Neurosurg 89 (4): 547-51, 1998.

  2. Karavitaki N, Wass JA: Craniopharyngiomas. Endocrinol Metab Clin North Am 37 (1): 173-93, ix-x, 2008.

  3. Garnett MR, Puget S, Grill J, et al.: Craniopharyngioma. Orphanet J Rare Dis 2: 18, 2007.

  4. Zhou L, Luo L, Xu J, et al.: Craniopharyngiomas in the posterior fossa: a rare subgroup, diagnosis, management and outcomes. J Neurol Neurosurg Psychiatry 80 (10): 1150-4, 2009.

  5. Muller HL: Childhood craniopharyngioma. Recent advances in diagnosis, treatment and follow-up. Horm Res 69 (4): 193-202, 2008.

  6. Müller HL: Childhood craniopharyngioma--current concepts in diagnosis, therapy and follow-up. Nat Rev Endocrinol 6 (11): 609-18, 2010.

  7. Sanford RA, Muhlbauer MS: Craniopharyngioma in children. Neurol Clin 9 (2): 453-65, 1991.

  8. Zacharia BE, Bruce SS, Goldstein H, et al.: Incidence, treatment and survival of patients with craniopharyngioma in the surveillance, epidemiology and end results program. Neuro Oncol 14 (8): 1070-8, 2012.

Histopathologic Classification of Childhood Craniopharyngioma

Craniopharyngiomas are histologically benign and do not metastasize to remote brain locations or to areas outside the sellar region except by direct extension. They may be invasive, however, and may recur locally. They may be classified as adamantinomatous or squamous papillary, with the former being the predominant form in children.[1] They are typically composed of both a solid portion with an abundance of calcification, and a cystic component which is filled with a dark, oily fluid. Recent evidence suggests that adamantinomatous craniopharyngiomas are locally more aggressive with a significantly higher rate of recurrence compared with the squamous papillary subtype.[2]

The molecular basis for craniopharyngioma differs by histologic subtype. Activating beta-catenin gene mutations are found in virtually all adamantinomatous tumors.[3][4] Conversely, BRAF V600E mutations are observed in nearly all squamous papillary craniopharyngiomas.[4]

References:

  1. Karavitaki N, Wass JA: Craniopharyngiomas. Endocrinol Metab Clin North Am 37 (1): 173-93, ix-x, 2008.

  2. Pekmezci M, Louie J, Gupta N, et al.: Clinicopathological characteristics of adamantinomatous and papillary craniopharyngiomas: University of California, San Francisco experience 1985-2005. Neurosurgery 67 (5): 1341-9; discussion 1349, 2010.

  3. Sekine S, Shibata T, Kokubu A, et al.: Craniopharyngiomas of adamantinomatous type harbor beta-catenin gene mutations. Am J Pathol 161 (6): 1997-2001, 2002.

  4. Brastianos PK, Taylor-Weiner A, Manley PE, et al.: Exome sequencing identifies BRAF mutations in papillary craniopharyngiomas. Nat Genet 46 (2): 161-5, 2014.

Diagnostic Evaluation of Childhood Craniopharyngioma

The results of imaging studies (computerized tomography scans and magnetic resonance imaging [MRI] scans) are often diagnostic for childhood craniopharyngiomas, with most demonstrating intratumoral calcifications and a solid and cystic component. The most common location is suprasellar, with an intrasellar portion. Craniopharyngiomas without calcification may be confused with other tumor types, such as germinoma or hypothalamic/chiasmatic astrocytoma, and biopsy may be required.[1] MRI of the spinal axis is not routinely performed.

Apart from imaging, patients often undergo formal visual examination including visual field evaluation and endocrine testing.

References:

  1. Rossi A, Cama A, Consales A, et al.: Neuroimaging of pediatric craniopharyngiomas: a pictorial essay. J Pediatr Endocrinol Metab 19 (Suppl 1): 299-319, 2006.

Stage Information

There is no generally applied staging system for childhood craniopharyngiomas. For treatment purposes, patients are classified as having newly diagnosed or recurrent disease.

Treatment Options for Newly Diagnosed Childhood Craniopharyngioma

There is no consensus as to the optimal treatment of newly diagnosed craniopharyngioma, in part due to the lack of prospective randomized trials comparing different treatment options. A systematic review of 109 reports that described extent of resection found that subtotal resection plus radiation therapy was associated with similar rates of tumor control as gross total resection and that both approaches were associated with higher progression-free survival (PFS) rates than subtotal resection alone.[1][Level of evidence: 3iiiDiii] Treatment is individualized based on factors such as the size, location, and extension of the tumor and potential short-term and long-term toxicity.

Radical Surgery

Because these tumors are histologically benign, it may be possible to remove all the visible tumor resulting in long-term disease control.[2][Level of evidence: 3iA]; [3][Level of evidence: 3iiiB]; [4][Level of evidence: 3iiiC] A 5-year PFS rate of about 65% has been reported.[5] Many surgical approaches have been described, and the route should be determined by the size, location, and extension of the tumor. A transsphenoidal approach may be possible in some small tumors located entirely within the sella,[6][Level of evidence: 3iiiC] but this is not usually possible in children, in which case a craniotomy is usually required.

Gross total resection is 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. The tumor may be adherent to these structures, which may cause complications, and may limit the ability to remove the entire tumor. The surgeon often has limited visibility in the region of the hypothalamus and in the sella, and portions of the mass may be left in these areas, accounting for some recurrences. Almost all craniopharyngiomas have an attachment to the pituitary stalk, and of the patients who undergo radical surgery, virtually all will require life-long pituitary hormone replacement with multiple medications.[3][7]

Complications of radical surgery include the need for hormone replacement, obesity (which can be life threatening),[8] severe behavioral problems,[9] blindness, seizures, spinal fluid leak, false aneurysms, and difficulty with eye movements. Rare complications include death from intraoperative hemorrhage, hypothalamic damage, or stroke. Hypothalamic-sparing surgical techniques may show a decrease in severe postoperative obesity without an increase in tumor recurrence.[10][Level of evidence: 3iiDi]

If the surgeon feels that tumor remains, or if postoperative imaging reveals residual craniopharyngioma that was not resected, radiation therapy may be recommended to prevent early progression.[11][Level of evidence: 3iiiDiii] It can be difficult to determine whether a tumor is progressive; carbon-11 methionine positron emission tomography is being evaluated for its use in these cases.[12] Periodic surveillance magnetic resonance imaging is performed for several years after radical surgery because of the possibility of tumor recurrence.

Surgery with Cyst Drainage

For large cystic craniopharyngiomas, particularly in children younger than 3 years and in those with recurrent cystic tumor after initial surgery, stereotactic or open implantation of an intracystic catheter with a subcutaneous reservoir may be a valuable alternative treatment option. The benefits of this procedure include temporary relief of fluid pressure by serial drainage, and in some cases, for intracystic instillation of sclerosing agents as a means to prolong the interval to or obviate the need for radiation. This procedure may also be helpful in allowing the surgeon to perform a two-staged approach, whereby first the cyst is drained by the implanted catheter to relieve pressure and complicating symptoms, followed by tumor resection.[13]

Limited Surgery and 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 certain late effects associated with radical surgery.[10] The surgical procedure is followed by radiation therapy, with a 5-year PFS rate of about 70% to 90% [5][14]; [15][Level of evidence: 3iDiii] and 10-year overall survival rates higher than 90%.[16][Level of evidence: 3iiA]; [1][Level of evidence: 3iiiDiii] Transient cyst enlargement may be noted soon after radiation therapy but generally resolves without further intervention.[17][Level of evidence: 3iDiv] Conventional radiation is fractionated external-beam radiation with a recommended dose of 54 Gy to 55 Gy in 1.8 Gy fractions.[18] Surgical complications are less likely than with radical surgery. Complications of radiation include loss of pituitary hormonal function, cognitive dysfunction, development of late strokes and vascular malformations, delayed blindness, development of second tumors, and, rarely, malignant transformation of the primary tumor within the radiation field.[19][20] Newer radiation technologies such as intensity-modulated proton therapy may reduce scatter whole-brain and whole-body irradiation and result in the sparing of normal tissues. It is unknown whether such technologies result in decreased late effects from irradiation.[15][21][22] Tumor progression remains a possibility, and it is usually not possible to repeat the radiation dose. In selected cases, stereotactic radiation therapy can be delivered as a single large dose of radiation to a very small field.[23][Level of evidence: 3iC] Proximity of the craniopharyngioma to vital structures, particularly the optic nerves, limits this to very small tumors that are in the sella.[24][Level of evidence: 3iiiDiii]

Intracavitary Radiation Therapy and/or Chemotherapy

Some craniopharyngiomas with a large cystic component may be treated by stereotaxic delivery of P-32 or other radioactive compounds.[25][26]; [27][Level of evidence: 2A]; [28][Level of evidence: 3iiiDiii] Nonradioactive agents such as bleomycin and interferon-alpha have also been used.[29][30][31]; [32][Level of evidence: 2C] These strategies have been found to be useful in certain cases and are with low reported risk of complications. However, none have shown efficacy against solid portions of the tumor.

References:

  1. Clark AJ, Cage TA, Aranda D, et al.: A systematic review of the results of surgery and radiotherapy on tumor control for pediatric craniopharyngioma. Childs Nerv Syst 29 (2): 231-8, 2013.

  2. Mortini P, Losa M, Pozzobon G, et al.: Neurosurgical treatment of craniopharyngioma in adults and children: early and long-term results in a large case series. J Neurosurg 114 (5): 1350-9, 2011.

  3. Elliott RE, Hsieh K, Hochm T, et al.: Efficacy and safety of radical resection of primary and recurrent craniopharyngiomas in 86 children. J Neurosurg Pediatr 5 (1): 30-48, 2010.

  4. Zhang YQ, Ma ZY, Wu ZB, et al.: Radical resection of 202 pediatric craniopharyngiomas with special reference to the surgical approaches and hypothalamic protection. Pediatr Neurosurg 44 (6): 435-43, 2008.

  5. Yang I, Sughrue ME, Rutkowski MJ, et al.: Craniopharyngioma: a comparison of tumor control with various treatment strategies. Neurosurg Focus 28 (4): E5, 2010.

  6. Locatelli D, Massimi L, Rigante M, et al.: Endoscopic endonasal transsphenoidal surgery for sellar tumors in children. Int J Pediatr Otorhinolaryngol 74 (11): 1298-302, 2010.

  7. Sands SA, Milner JS, Goldberg J, et al.: Quality of life and behavioral follow-up study of pediatric survivors of craniopharyngioma. J Neurosurg 103 (4 Suppl): 302-11, 2005.

  8. Müller HL, Gebhardt U, Teske C, et al.: Post-operative hypothalamic lesions and obesity in childhood craniopharyngioma: results of the multinational prospective trial KRANIOPHARYNGEOM 2000 after 3-year follow-up. Eur J Endocrinol 165 (1): 17-24, 2011.

  9. Clark AJ, Cage TA, Aranda D, et al.: Treatment-related morbidity and the management of pediatric craniopharyngioma: a systematic review. J Neurosurg Pediatr 10 (4): 293-301, 2012.

  10. Elowe-Gruau E, Beltrand J, Brauner R, et al.: Childhood craniopharyngioma: hypothalamus-sparing surgery decreases the risk of obesity. J Clin Endocrinol Metab 98 (6): 2376-82, 2013.

  11. Lin LL, El Naqa I, Leonard JR, et al.: Long-term outcome in children treated for craniopharyngioma with and without radiotherapy. J Neurosurg Pediatr 1 (2): 126-30, 2008.

  12. Laser BS, Merchant TE, Indelicato DJ, et al.: Evaluation of children with craniopharyngioma using carbon-11 methionine PET prior to proton therapy. Neuro Oncol 15 (4): 506-10, 2013.

  13. Schubert T, Trippel M, Tacke U, et al.: Neurosurgical treatment strategies in childhood craniopharyngiomas: is less more? Childs Nerv Syst 25 (11): 1419-27, 2009.

  14. Winkfield KM, Tsai HK, Yao X, et al.: Long-term clinical outcomes following treatment of childhood craniopharyngioma. Pediatr Blood Cancer 56 (7): 1120-6, 2011.

  15. Merchant TE, Kun LE, Hua CH, et al.: Disease control after reduced volume conformal and intensity modulated radiation therapy for childhood craniopharyngioma. Int J Radiat Oncol Biol Phys 85 (4): e187-92, 2013.

  16. Schoenfeld A, Pekmezci M, Barnes MJ, et al.: The superiority of conservative resection and adjuvant radiation for craniopharyngiomas. J Neurooncol 108 (1): 133-9, 2012.

  17. Shi Z, Esiashvili N, Janss AJ, et al.: Transient enlargement of craniopharyngioma after radiation therapy: pattern of magnetic resonance imaging response following radiation. J Neurooncol 109 (2): 349-55, 2012.

  18. Kiehna EN, Merchant TE: Radiation therapy for pediatric craniopharyngioma. Neurosurg Focus 28 (4): E10, 2010.

  19. Ishida M, Hotta M, Tsukamura A, et al.: Malignant transformation in craniopharyngioma after radiation therapy: a case report and review of the literature. Clin Neuropathol 29 (1): 2-8, 2010 Jan-Feb.

  20. Aquilina K, Merchant TE, Rodriguez-Galindo C, et al.: Malignant transformation of irradiated craniopharyngioma in children: report of 2 cases. J Neurosurg Pediatr 5 (2): 155-61, 2010.

  21. Beltran C, Roca M, Merchant TE: On the benefits and risks of proton therapy in pediatric craniopharyngioma. Int J Radiat Oncol Biol Phys 82 (2): e281-7, 2012.

  22. Boehling NS, Grosshans DR, Bluett JB, et al.: Dosimetric comparison of three-dimensional conformal proton radiotherapy, intensity-modulated proton therapy, and intensity-modulated radiotherapy for treatment of pediatric craniopharyngiomas. Int J Radiat Oncol Biol Phys 82 (2): 643-52, 2012.

  23. Kobayashi T: Long-term results of gamma knife radiosurgery for 100 consecutive cases of craniopharyngioma and a treatment strategy. Prog Neurol Surg 22: 63-76, 2009.

  24. Hasegawa T, Kobayashi T, Kida Y: Tolerance of the optic apparatus in single-fraction irradiation using stereotactic radiosurgery: evaluation in 100 patients with craniopharyngioma. Neurosurgery 66 (4): 688-94; discussion 694-5, 2010.

  25. Julow J, Backlund EO, Lányi F, et al.: Long-term results and late complications after intracavitary yttrium-90 colloid irradiation of recurrent cystic craniopharyngiomas. Neurosurgery 61 (2): 288-95; discussion 295-6, 2007.

  26. Barriger RB, Chang A, Lo SS, et al.: Phosphorus-32 therapy for cystic craniopharyngiomas. Radiother Oncol 98 (2): 207-12, 2011.

  27. Kickingereder P, Maarouf M, El Majdoub F, et al.: Intracavitary brachytherapy using stereotactically applied phosphorus-32 colloid for treatment of cystic craniopharyngiomas in 53 patients. J Neurooncol 109 (2): 365-74, 2012.

  28. Zhao R, Deng J, Liang X, et al.: Treatment of cystic craniopharyngioma with phosphorus-32 intracavitary irradiation. Childs Nerv Syst 26 (5): 669-74, 2010.

  29. Ierardi DF, Fernandes MJ, Silva IR, et al.: Apoptosis in alpha interferon (IFN-alpha) intratumoral chemotherapy for cystic craniopharyngiomas. Childs Nerv Syst 23 (9): 1041-6, 2007.

  30. Linnert M, Gehl J: Bleomycin treatment of brain tumors: an evaluation. Anticancer Drugs 20 (3): 157-64, 2009.

  31. Steinbok P, Hukin J: Intracystic treatments for craniopharyngioma. Neurosurg Focus 28 (4): E13, 2010.

  32. Cavalheiro S, Di Rocco C, Valenzuela S, et al.: Craniopharyngiomas: intratumoral chemotherapy with interferon-alpha: a multicenter preliminary study with 60 cases. Neurosurg Focus 28 (4): E12, 2010.

Treatment Options for Recurrent Childhood Craniopharyngioma

Recurrence of craniopharyngioma occurs in approximately 35% of patients regardless of primary therapy.[1] Management is determined in large part by prior therapy. Repeat attempts at gross total resection are difficult and long-term disease control is less often achieved.[2][Level of evidence: 3iiiDi] Complications are more frequent than with initial surgery.[3][Level of evidence: 3iiiDi] External-beam radiation therapy is an option if this has not been previously employed, including consideration of radiosurgery in selected circumstances.[4][Level of evidence: 3iiiDiii] Cystic recurrences may be treated with intracavitary instillation of radioactive P-32, bleomycin,[5][Level of evidence: 3iiiDiii] or interferon-alpha,[6][Level of evidence: 3iiiB] and a reservoir may be placed to permit intermittent outpatient aspiration. Although systemic therapy is generally not utilized, a small series has shown that the use of subcutaneous pegylated interferon alpha-2b to manage cystic recurrences can result in durable responses.[7][Level of evidence: 3iiiDiii]

Treatment Options Under Clinical Evaluation

The following is an example of a national and/or institutional clinical trial that is currently being conducted. Information about ongoing clinical trials is available from the NCI Web site.

  • PBTC-039 (NCT01964300) (Peginterferon Alfa-2b in Treating Younger Patients With Craniopharyngioma That is Recurrent or Cannot Be Removed By Surgery): This is a phase II clinical trial evaluating how well peginterferon alfa-2b works in treating children with craniopharyngioma that is recurrent or cannot be removed by surgery. This trial follows a small (N = 5) single-institution experience with peginterferon alfa-2b, in which prolonged complete responses were observed in some patients.[7]

References:

  1. Yang I, Sughrue ME, Rutkowski MJ, et al.: Craniopharyngioma: a comparison of tumor control with various treatment strategies. Neurosurg Focus 28 (4): E5, 2010.

  2. Vinchon M, Dhellemmes P: Craniopharyngiomas in children: recurrence, reoperation and outcome. Childs Nerv Syst 24 (2): 211-7, 2008.

  3. Jang WY, Lee KS, Son BC, et al.: Repeat operations in pediatric patients with recurrent craniopharyngiomas. Pediatr Neurosurg 45 (6): 451-5, 2009.

  4. Xu Z, Yen CP, Schlesinger D, et al.: Outcomes of Gamma Knife surgery for craniopharyngiomas. J Neurooncol 104 (1): 305-13, 2011.

  5. Hukin J, Steinbok P, Lafay-Cousin L, et al.: Intracystic bleomycin therapy for craniopharyngioma in children: the Canadian experience. Cancer 109 (10): 2124-31, 2007.

  6. Cavalheiro S, Di Rocco C, Valenzuela S, et al.: Craniopharyngiomas: intratumoral chemotherapy with interferon-alpha: a multicenter preliminary study with 60 cases. Neurosurg Focus 28 (4): E12, 2010.

  7. Yeung JT, Pollack IF, Panigrahy A, et al.: Pegylated interferon-α-2b for children with recurrent craniopharyngioma. J Neurosurg Pediatr 10 (6): 498-503, 2012.

Late Effects in Patients Treated for Childhood Craniopharyngioma

Quality-of-life issues are important in this group of patients and are difficult to generalize due to various treatment modalities. Whereas intelligence quotient is usually maintained, behavioral issues and memory deficits attributed to the frontal lobe and hypothalamus are common.[1] Patients with hypothalamic involvement showed impairment in memory and executive functioning.[2] Other common problems include visual loss, obesity (which can be life threatening),[3] and the almost universal need for life-long endocrine replacement with multiple pituitary hormones.[4][5][6][Level of evidence: 3iiiC] Vasculopathies and secondary tumors may also result from local irradiation.[7] A recent report indicated that adults on long-term growth hormone replacement secondary to childhood craniopharyngioma involving the hypothalamus were at increased cardiovascular risk.[8]

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.

References:

  1. Winkfield KM, Tsai HK, Yao X, et al.: Long-term clinical outcomes following treatment of childhood craniopharyngioma. Pediatr Blood Cancer 56 (7): 1120-6, 2011.

  2. Özyurt J, Thiel CM, Lorenzen A, et al.: Neuropsychological outcome in patients with childhood craniopharyngioma and hypothalamic involvement. J Pediatr 164 (4): 876-881.e4, 2014.

  3. Elowe-Gruau E, Beltrand J, Brauner R, et al.: Childhood craniopharyngioma: hypothalamus-sparing surgery decreases the risk of obesity. J Clin Endocrinol Metab 98 (6): 2376-82, 2013.

  4. Vinchon M, Weill J, Delestret I, et al.: Craniopharyngioma and hypothalamic obesity in children. Childs Nerv Syst 25 (3): 347-52, 2009.

  5. Dolson EP, Conklin HM, Li C, et al.: Predicting behavioral problems in craniopharyngioma survivors after conformal radiation therapy. Pediatr Blood Cancer 52 (7): 860-4, 2009.

  6. Kawamata T, Amano K, Aihara Y, et al.: Optimal treatment strategy for craniopharyngiomas based on long-term functional outcomes of recent and past treatment modalities. Neurosurg Rev 33 (1): 71-81, 2010.

  7. Kiehna EN, Merchant TE: Radiation therapy for pediatric craniopharyngioma. Neurosurg Focus 28 (4): E10, 2010.

  8. Holmer H, Ekman B, Björk J, et al.: Hypothalamic involvement predicts cardiovascular risk in adults with childhood onset craniopharyngioma on long-term GH therapy. Eur J Endocrinol 161 (5): 671-9, 2009.


This information is provided by the National Cancer Institute.

This information was last updated on August 12, 2014.

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