Newly Diagnosed Childhood Craniopharyngioma Treatment
Treatment Options for Newly Diagnosed Childhood Craniopharyngioma
There is no consensus on the optimal treatment for 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 factors that include the following:
- Tumor size.
- Tumor location.
- Extension of the tumor.
- Potential short-term and long-term toxicity.
Treatment options for newly diagnosed childhood craniopharyngioma include the following:
Radical surgery with or without radiation therapy
It may be possible to remove all visible tumor and achieve long-term disease control because these tumors are histologically benign.[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. Radical surgical approaches include the following:
- Transsphenoidal approach: A transsphenoidal approach may be possible for some small tumors located entirely within the sella.[Level of evidence: 3iiiC] The development of expanded endonasal techniques with endoscopic visualization have allowed this approach to be increasingly used, even for sizeable childhood tumors, which is similar to the experience in adults. When an endonasal approach is not possible, a craniotomy is required.
- Craniotomy: As noted above, gross-total resection may be technically challenging because the tumor is surrounded by vital structures. The surgeon often has a limited view of the hypothalamic and sellar regions, and portions of the mass may remain after surgery, 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.[2,7]
Complications of radical surgery include the following:
- Obesity, which can be life-threatening. Hypothalamic-sparing surgical techniques may result in reduced postoperative obesity without an increase in tumor recurrence.[Level of evidence: 3iiDi]
- Hormone replacement therapy.
- Severe behavioral problems.
- Spinal fluid leak.
- False aneurysms.
- Difficulty with eye movements.
- Death from intraoperative hemorrhage, hypothalamic damage, or stroke (rare).
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.[Level of evidence: 3iiiDiii]
Periodic surveillance using magnetic resonance imaging is performed for several years after radical surgery 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 radical surgery.
The surgical procedure is often followed by radiation therapy, with a 5-year PFS rate of about 70% to 90%[4,12]; [Level of evidence: 3iDiii] and 10-year overall survival rates higher than 90%.[Level of evidence: 3iiA]; [Level of evidence: 3iiiDiii] Conventional radiation therapy is fractionated external-beam radiation, with a recommended dose of 54 Gy to 55 Gy in 1.8-Gy fractions. 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 higher PFS rates than was subtotal resection alone.[Level of evidence: 3iiiDiii]
Surgical complications with a subtotal resection are less likely than with radical surgery. Complications of radiation therapy include the following:
Newer radiation technologies such as intensity-modulated proton therapy may reduce scatter during whole-brain and whole-body irradiation and result in the sparing of normal tissues. It is unknown whether such technologies result in reduced late effects from radiation.[13,20,21]
Tumor progression remains a concern, 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 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 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. Benefits include temporary relief of fluid pressure by serial drainage, and in some cases, for intracystic instillation of sclerosing agents as a means to postpone or obviate radiation treatment. This procedure may also allow the surgeon to use a two-staged approach: first draining the cyst via the implanted catheter, to relieve pressure and complicating symptoms; and then later resecting the tumor or employing radiation therapy.
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