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Vandetanib Tumor Shrinkage in Metastatic Medullary Thyroid Cancer Allowing Surgical Resection of the Primary Site

A Case Report

Milner, Thomas D. MBChB, BMedSci, MRCS, MRCS(ENT)*; Ronghe, Milind MBBS, MD, DCH, DNB, FRCPCH; Shaikh, Mohamad G. BSc, MBChB, MD, FRCPCH; MacGregor, Fiona B. MBChB, FRCS, FRCS(ORL, H&N)*; Reed, Nicholas MBBS, MRCP, FRCR, FRCP§

Journal of Pediatric Hematology/Oncology: October 16, 2018 - Volume Publish Ahead of Print - Issue - p
doi: 10.1097/MPH.0000000000001330
Clinical and Laboratory Observations: PDF Only

Vandetanib has been shown to improve progression-free survival in adults with advanced medullary thyroid cancer. This article describes a pediatric patient with metastatic medullary thyroid cancer secondary to sporadic multiple endocrine neoplasia 2B, treated with vandetanib. At presentation, he had an inoperable primary tumor, with carotid encasement, and pulmonary metastases. Vandetanib induced a significant response: calcitonin and carcinoembryonic antigen levels both fell considerably, primary tumor maximal diameter decreased by 68%, and pulmonary metastases became no longer detectable. This allowed surgical resection of the primary tumor. The patient remains well after over 6 years of vandetanib therapy, with no treatment toxicity.

*Department of Otolaryngology, Queen Elizabeth University Hospital

Departments of Paediatric Haemato-Oncology

Paediatric Endocrinology, Royal Hospital for Sick Children

§Department of Clinical Oncology, Beatson West of Scotland Cancer Centre, Glasgow, UK

N.R. has previously sat on the advisory board of Astra-Zeneca. Vandetanib was provided by Astra-Zeneca on an expanded access program. The other authors declare no conflict of interest.

Reprints: Thomas D. Milner, MBChB, BMedSci, MRCS, MRCS (ENT), Department of Otolaryngology, Queen Elizabeth University Hospital, 1345 Govan Road, Glasgow G514TF, UK (e-mail:

Received January 17, 2018

Accepted August 19, 2018

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