Clinical Outcomes and Complications of Pituitary Blastoma
| Autor: | John R. Priest, Margaret Zacharin, Yomna H.E. Ahmed, Anthony P. Y. Liu, Anatoly Tiulpakov, Andrew C. Peet, Adam J. Esbenshade, Anastasia Lapshina, Pinaki Dutta, Sung Hye Park, Leanne de Kock, Nelly Sabbaghian, Kim E. Nichols, William D. Foulkes, Cheri Deal, Oswald Ploner, Márta Korbonits, Ashutosh Rai, Andrew W. Walter, Heidi Traunecker, Megan M. Kelsey |
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| Rok vydání: | 2020 |
| Předmět: |
Male
Ribonuclease III Pediatrics medicine.medical_specialty Endocrinology Diabetes and Metabolism Clinical Biochemistry Brain tumor Context (language use) Biochemistry DEAD-box RNA Helicases 03 medical and health sciences Cushing syndrome 0302 clinical medicine Endocrinology Postoperative Complications Internal medicine Biopsy medicine Adjuvant therapy Humans Pituitary Neoplasms Germ-Line Mutation Clinical Research Articles DICER1 Syndrome Retrospective Studies medicine.diagnostic_test business.industry Biochemistry (medical) Infant medicine.disease Prognosis 3. Good health Survival Rate 030220 oncology & carcinogenesis Child Preschool Blastoma Female Complication business Blast Crisis 030217 neurology & neurosurgery Follow-Up Studies |
| Zdroj: | J Clin Endocrinol Metab |
| ISSN: | 1945-7197 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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