Transsphenoidal surgery effective in children with Cushing’s disease
Most participants in single-center study achieved long-term remission
Transsphenoidal surgery, a minimally invasive procedure to remove pituitary tumors through the nose, achieved long-term remission in nearly all children with Cushing’s disease who participated in a single-center study in China.
“While recurrence occurred in a minority of patients, primarily those with [small tumors], durable disease control is attainable for the majority with appropriate surgical management,” the researchers wrote.
The study, “The neurosurgical outcome of pediatric Cushing’s disease in a single center from China: a 20-year experience,” was published in Frontiers in Endocrinology.
Transsphenoidal surgery in children poses challenges
Cushing’s disease is caused by a tumor in the brain’s pituitary gland, called a pituitary adenoma, that secretes abnormally high levels of the signaling molecule adrenocorticotropic hormone (ACTH). Too much ACTH ultimately leads to the overproduction of cortisol, giving rise to disease symptoms.
The typical first-line treatment for Cushing’s disease is a transsphenoidal adenomectomy, a surgical procedure to remove the disease-causing tumor in the pituitary gland via the nasal passages. This type of surgery has an overall cure, or complete remission, rate of up to 90%.
In children with Cushing’s disease, however, the pituitary tumor can be very small and hard to see on imaging tests like MRI. As a result, the transsphenoidal surgery poses significant technical challenges for neurosurgeons.
With this in mind, researchers reviewed the outcomes of 22 children with Cushing’s disease, ages 9 to 18, who underwent transsphenoidal surgery at a single center in China between 2002 and 2022.
Immediate postoperative remission in majority of patients
MRI and findings from bilateral inferior petrosal sinus sampling (BIPSS) guided the surgical strategy. This test can help determine whether the tumor is located on the right or left side of the pituitary gland by analyzing ACTH levels in the blood from the veins draining the gland.
Using MRI, adenomas were localized in 18 patients (81.8%), comprising 16 microadenomas (small tumors) and two macroadenomas (large tumors). Via BIPSS, tumor lateralization, which refers to determining the tumor’s precise location, was confirmed in 14 cases (73.7%). The agreement between BIPSS lateralization findings and MRI localization was 57.9%.
Half of the 22 patients underwent microscopic transsphenoidal surgery, which was performed before 2016 using microscopes to visualize tumors. Thereafter, the other half had endoscopic transsphenoidal surgery, which used an endoscope for a wider, more flexible view.
Twenty patients (90.9%) achieved immediate postoperative remission, defined as the normalization of cortisol levels in the blood and urine. The two patients who didn’t reach remission received additional treatments.
“Our immediate remission rate … compares favorably with contemporary pediatric [Cushing’s disease] surgical series, which typically report rates between 70% and 98%,” the team noted.
Of those with initial remission, Cushing’s recurred in two (10%) patients, one with a microadenoma and another with no MRI findings, during a follow-up period that ranged from two to 129 months, or more than 10 years. Therefore, the sustained long-term remission rate for the initial transsphenoidal surgery was 81.8%.
Transsphenoidal surgery performed in a specialized center achieves high rates of immediate and sustained remission.
In all cases, bleeding during the procedure was controlled, and no patient required a blood transfusion. All patients who achieved remission were treated with cortisone replacement therapy to help maintain proper cortisol levels.
In addition to cortisol, one child who underwent endoscopic and five who underwent microscopic surgeries received treatment with levothyroxine, a thyroid hormone replacement pill to treat low levels of thyroid hormone complications after surgery. For those with diabetes insipidus, a condition characterized by large amounts of dilute urine and an increased thirst, oral desmopressin was given to three patients in the endoscopic group and two in the microscopic group.
“Transsphenoidal surgery performed in a specialized center achieves high rates of immediate and sustained remission,” the researchers concluded. “While recurrence remains a concern necessitating vigilant long-term follow-up, the majority of children with [Cushing’s disease] can attain durable disease control with appropriate surgical management.”
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