title: Transient partial response to sorafenib treatment in an adolescent patient with MEN2B syndrome and end-stage medullary thyroid carcinoma.
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.
authors: Department of Pediatrics, Division of Hematology, Oncology, and Stem Cell Transplant, Children's Memorial Hospital, Northwestern University Feinberg School of Medicine, Chicago, Illinois, USA.
authors: Steven
authors: S
authors: Aller
authors: Andrada
authors: A
authors: Popescu
authors: Sudha
authors: S
authors: Rao
authors: Elaine
authors: E
authors: Morgan
authors: Yasmin
authors: Y
authors: Gosiengfiao
mesh_terms: D000293:Adolescent; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D001557:Benzenesulfonates / Q000627:therapeutic use*; D018276:Carcinoma, Medullary / Q000188:drug therapy* / Q000209:etiology; D005260:Female; D006801:Humans; D018814:Multiple Endocrine Neoplasia Type 2b / Q000150:complications / Q000188:drug therapy*; D009536:Niacinamide / Q000031:analogs & derivatives; D010671:Phenylurea Compounds; D011379:Prognosis; D011725:Pyridines / Q000627:therapeutic use*; D000077157:Sorafenib; D013964:Thyroid Neoplasms / Q000188:drug therapy* / Q000209:etiology
chemical_list: D000970:Antineoplastic Agents; D001557:Benzenesulfonates; D010671:Phenylurea Compounds; D011725:Pyridines; D009536:Niacinamide; D000077157:Sorafenib