Vandetanib in Children and Adolescents with Multiple Endocrine Neoplasia Type 2B Associated Medullary Thyroid Carcinoma

• Published in: Clinical cancer research Vol. 19; no. 15; pp. 4239 - 4248
• Main Authors: Fox, Elizabeth, Widemann, Brigitte C., Chuk, Meredith K., Marcus, Leigh, Aikin, Alberta, Whitcomb, Patricia O., Merino, Maria J., Lodish, Maya, Dombi, Eva, Steinberg, Seth M., Wells, Samuel A., Balis, Frank M.
• Format: Journal Article
• Language: English
• Published: Philadelphia, PA American Association for Cancer Research 01.08.2013
• Subjects: Adolescent; Antineoplastic agents; Biological and medical sciences; Carcinoma, Medullary - drug therapy; Carcinoma, Medullary - genetics; Carcinoma, Neuroendocrine; Child; Child, Preschool; Drug-Related Side Effects and Adverse Reactions - pathology; Endocrinopathies; Gene Expression Regulation, Neoplastic; General aspects. Associated endocrine diseases. Endocrine paraneoplasic syndromes; Germ-Line Mutation; Humans; Malignant tumors; Medical sciences; Multiple Endocrine Neoplasia Type 2b - drug therapy; Multiple Endocrine Neoplasia Type 2b - genetics; Multiple Endocrine Neoplasia Type 2b - pathology; Multiple tumors. Solid tumors. Tumors in childhood (general aspects); Neoplasm Metastasis; Neoplasm Recurrence, Local - drug therapy; Neoplasm Recurrence, Local - pathology; Pharmacology. Drug treatments; Piperidines - administration & dosage; Piperidines - adverse effects; Protein-Tyrosine Kinases - genetics; Proto-Oncogene Mas; Proto-Oncogene Proteins c-ret - genetics; Quinazolines - administration & dosage; Quinazolines - adverse effects; Thyroid Neoplasms - drug therapy; Thyroid Neoplasms - genetics; Thyroid Neoplasms - pathology; Thyroid. Thyroid axis (diseases); Tumors; Endocrinopathy; Human; Antineoplastic agent; Vandetanib; Enzyme; Tyrosine kinase inhibitor; Transferases; Thyroid diseases; Enzyme inhibitor; Thyroid medullary carcinoma; Malignant tumor; Genetic disease; Thyroid cancer; Multiple endocrine neoplasia; Adolescent; Multikinase inhibitor; Antiangiogenic agent; Protein-tyrosine kinase; Child; Cancer

Purpose: Medullary thyroid carcinoma (MTC) is a manifestation of multiple endocrine neoplasia type 2 (MEN2) syndromes caused by germline, activating mutations in the RET (REarranged during Transfection) proto-oncogene. Vandetanib, a VEGF and EGF receptor inhibitor, blocks RET tyrosine kinase activit...