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

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


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: NCT00514046.

Publication types

  • Case Reports
  • Research Support, N.I.H., Intramural

MeSH terms

  • Adolescent
  • Adrenocorticotropic Hormone / metabolism
  • Carcinoma, Neuroendocrine
  • Clinical Trials, Phase I as Topic
  • Clinical Trials, Phase II as Topic
  • Cushing Syndrome / etiology*
  • Humans
  • Male
  • Multiple Endocrine Neoplasia Type 2b / complications*
  • Neoplasms, Second Primary / complications*
  • Neoplasms, Second Primary / metabolism
  • Neoplasms, Second Primary / secondary
  • Piperidines / therapeutic use*
  • Protein-Tyrosine Kinases / antagonists & inhibitors*
  • Quinazolines / therapeutic use*
  • Thyroid Neoplasms / complications*
  • Thyroid Neoplasms / metabolism
  • Thyroid Neoplasms / secondary


  • Piperidines
  • Quinazolines
  • Adrenocorticotropic Hormone
  • Protein-Tyrosine Kinases
  • vandetanib

Supplementary concepts

  • Thyroid cancer, medullary

Associated data