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Updated: Nov 28, 2025
Endoscopic Endonasal Trans-sphenoidal Approach: Minimally Invasive Surgery for Pituitary Adenomas
Published on: January 17, 2018
Clinical Outcomes and Complications of Pituitary Blastoma
Anthony P Y Liu1, Megan M Kelsey2, Nelly Sabbaghian3
1Division of Neuro-Oncology, Department of Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee, USA.
Context:
Pituitary blastoma is a rare, dysontogenetic hypophyseal tumor of infancy first described in 2008, strongly suggestive of DICER1 syndrome.
Objective:
This work aims to describe genetic alterations, clinical courses, outcomes, and complications in all known pituitary blastoma cases.
Design And Setting:
A multi-institutional case series is presented from tertiary pediatric oncology centers.
Patients:
Patients included children with pituitary blastoma.
Interventions:
Genetic testing, surgery, oncologic therapy, endocrine support are reported.
Outcome Measures:
Outcome measures included survival, long-term morbidities, and germline and tumor DICER1 genotypes.
Results:
Seventeen pituitary blastoma cases were studied (10 girls and 7 boys); median age at diagnosis was 11 months (range, 2-24 months). Cushing syndrome was the most frequent presentation (n = 10). Cushingoid stigmata were absent in 7 children (2 with increased adrenocorticotropin [ACTH]; 5 with normal/unmeasured ACTH). Ophthalmoplegia and increased intracranial pressure were also observed. Surgical procedures included gross/near-total resection (n = 7), subtotal resection (n = 9), and biopsy (n = 1). Six children received adjuvant therapy. At a median follow-up of 6.7 years, 9 patients were alive; 8 patients died of the following causes: early medical/surgical complications (n = 3), sepsis (n = 1), catheter-related complication (n = 1), aneurysmal bleeding (n = 1), second brain tumor (n = 1), and progression (n = 1). Surgery was the only intervention for 5 of 9 survivors. Extent of resection, but neither Ki67 labeling index nor adjuvant therapy, was significantly associated with survival. Chronic complications included neuroendocrine (n = 8), visual (n = 4), and neurodevelopmental (n = 3) deficits. Sixteen pituitary blastomas were attributed to DICER1 abnormalities.
Conclusions:
Pituitary blastoma is a locally destructive tumor associated with high mortality. Surgical resection alone provides long-term disease control for some patients. Quality survival is possible with long-term neuroendocrine management.
