Successful treatment of paraganglioma with sorafenib: a case report and brief review of the literature.
BACKGROUND
To date, no effective systemic therapies have been made available for paraganglioma. However, multiple mutations in susceptibility genes have been identified that are potential targets for sorafenib, an oral multitargeted tyrosine-kinase inhibitor.


METHODS
We report the case of a 69-year-old Chinese man with mediastinal paraganglioma that had metastasized to the bone. The paraganglioma responded to sorafenib, a novel multi-tyrosine kinase inhibitor that targets angiogenesis, the Raf-kinase pathway, the platelet-derived growth factor Ret, and c-Kit. The patient was diagnosed as having paraganglioma after biopsy of the mediastinal mass. We first treated the patient with radiotherapy. Then he tolerated an etoposide-and-cisplatin chemotherapy regimen. Subsequently, he received 6 months of maintenance treatment with sorafenib (400 mg twice daily). A dramatic reduction in tumor volume was observed. At present, the patient has achieved a partial response, and his clinical status remains unchanged.


CONCLUSIONS
We suggest that sorafenib should be further investigated in the management of patients with paraganglioma.
Department of Oncology, Shanghai East Hospital, People's Republic of China.
Yun
Y
Lin
Qun
Q
Li
Wei
W
Huang
Xinyan
X
Jia
Hong
H
Jiang
Yong
Y
Gao
Qi
Q
Li
molecular-targeted drug; paraganglioma; sorafenib