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Transient partial response to sorafenib treatment in an adolescent patient with MEN2B syndrome and end-stage medullary thyroid carcinoma |
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| Authors: | Aller Steven Popescu Andrada Rao Sudha Morgan Elaine Gosiengfiao Yasmin |
| Affiliation: | Department of Pediatrics, Division of Hematology, Oncology, and Stem Cell Transplant, Children's Memorial Hospital, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA. |
| Abstract: | Metastatic medullary thyroid carcinoma (MTC) is an aggressive malignancy with an extremely poor prognosis. Currently no effective conventional systemic therapies exist to treat pediatric MTC. We describe an adolescent female with newly diagnosed MEN2B syndrome who presented with advanced stage metastatic MTC and demonstrated a partial transient response to sorafenib monotherapy. This clinical result supports further research into the use of sorafenib in the treatment of pediatric MTC. |
| Keywords: | MEN2B syndrome metastatic medullary thyroid carcinoma sorafenib |
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