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J Clin Endocrinol Metab. 2014 Sep;99(9):3055-9. doi: 10.1210/jc.2013-4340. Epub 2014 Mar 11.

Vandetanib successfully controls medullary thyroid cancer-related Cushing syndrome in an adolescent patient.

Author information

1
Section on Endocrinology and Genetics (A.A.N., M.B.L., C.A.S.), Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland 20892; Pharmacology and Experimental Therapeutics Section, Pediatric Oncology Branch (E.F., F.M.B., P.O.W., J.D., B.C.W.), General Surgical Pathology Section (M.M.Q.), and Endocrine Oncology Branch (E.K.), Center for Cancer Research, National Cancer Institute, Bethesda, Maryland 20892; and Division of Oncology and Center for Childhood Cancer Research (E.F., F.M.B.), The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania 19104.

Abstract

CONTEXT:

Ectopic Cushing syndrome due to ACTH secretion from metastatic medullary thyroid cancer (MTC) is associated with significant morbidity and mortality.

OBJECTIVE:

The aim of the study was to describe the first case of Cushing syndrome associated with MTC in a pediatric patient and the successful reversal of Cushing syndrome with tyrosine kinase inhibitor (vandetanib) therapy.

PATIENT AND METHODS:

A 17-year-old Brazilian adolescent presented with metastatic MTC and associated ACTH-dependent ectopic Cushing syndrome in the context of multiple endocrine neoplasia type 2B. When the patient was treated with the tyrosine kinase inhibitor vandetanib, rapid decrease in serum cortisol and improvement of clinical symptoms were observed.

CONCLUSION:

We describe the first pediatric case of clinical and biochemical improvement of paraneoplastic MTC-related Cushing syndrome after treatment with vandetanib. Vandetanib and possibly other tyrosine kinase inhibitors may be a novel beneficial option in patients with neuroendocrine tumor-related ectopic Cushing syndrome.

TRIAL REGISTRATION:

ClinicalTrials.gov NCT00514046.

PMID:
24617713
PMCID:
PMC4154103
DOI:
10.1210/jc.2013-4340
[Indexed for MEDLINE]
Free PMC Article
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