Endocrine Abstracts

Background: Patients with Multiple Endocrine Neoplasia type 2 (MEN2) without previous family history often present late with advanced Medullary Thyroid Cancer (MTC). Surgery is not always curative but RET tyrosine kinase pathway is a potential target for molecular treatment for progressive MTC.Methods: Retrospective review of clinical, genetic, biochemical (calcitonin, CEA) and imaging (US, CT/MRI, Gallium Dotate) data of children with MEN2 who developed...

Background and Demographics: Medullary thyroid carcinoma (MTC), in the context of Multiple Endocrine Neoplasia type 2 (MEN2), is caused by mutations in the RET proto-oncogene. For children with MEN2, both 2A and 2B subtypes, and advanced MTC, the RET tyrosine kinase (TK) pathway is a target for treatment with selpercatinib, a selective RET TK inhibitor (TKI). In the United Kingdom, 7 paediatric patients have been receiving named -patient, compassionate access ...