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Childhood Craniopharyngioma Treatment (PDQ®): Treatment - Health Professional Information [NCI] - 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

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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]

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