Endocrine Abstracts

Background: Mutations in the RET proto-oncogene occur in about 70% of medullary thyroid cancers (MTC) and is central in its pathogenesis. Two highly selective RET inhibitors, selpercatinib and pralsetinib, are FDA/EMA-approved in RET-altered thyroid cancers. We share our experience of these drugs in metastatic MTC.Methods: Data were collected retrospectively from 19 patients commenced on selective RET inhibitors for MTC ...

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 ...