Successful treatment of paraganglioma with sorafenib: a case report and brief review of the literature

Yun Lin,^1,* Qun Li,^1,* Wei Huang,¹ Xinyan Jia,¹ Hong Jiang,¹ Yong Gao,¹ Qi Li<sup>1,2

1</sup>Department of Oncology, Shanghai East Hospital, ²Department of Oncology, Shanghai First People's Hospital, Shanghai, People's Republic of China

*These authors contributed equally to this work, and are joint first authors

Introduction: 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.
Case presentation: 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.
Conclusion: We suggest that sorafenib should be further investigated in the management of patients with paraganglioma.

Keywords: sorafenib, paraganglioma, molecular-targeted drug