Presentation Authors: Kaifang Ma*, Kan Gong, Baoan Hong, Haibiao Xie, Beijing, China, People's Republic of
Introduction: von Hippel-Lindau (VHL) disease is an autosomal-dominant, multi-organ, familial neoplastic syndrome. To date, studies about targeted therapy in Chinese patients associated with VHL disease are limited. In this study, we aimed to evaluate the efficacy and safety of four different tyrosine kinase inhibitors in patients with VHL disease.
Methods: In this retrospective study, patients who diagnosed with VHL disease and received targeted therapy were recruited from Peking University First Hospital. Patients were treated with four different tyrosine kinase inhibitors, respectively, including sunitinib (n=12), sorafenib (n=11), axitinib (n=6) and pazopanib (n=3). Assessments of clinical response and degrees of tumor changes were performed according to the Response Evaluation Criteria in Sold Tumor. Diverse adverse events related to the treatment of tyrosine kinase inhibitors were evaluated.
Results: From July 2009 to April 2018, 39 patients with genetically verified or clinical manifestations consistent with VHL disease, of whom 32 patients eligible to this study. The mean time of targeted therapy was 27.8 months (range 3-87 months) and mean follow-up time was 29.1Â±26.2 months (range 3-93 months). According to RECIST, 8 (25%) of 32 patients showed partial response to therapy, 16 (50%) of 32 patients achieved with stable disease and eight patients progressed. Lesion sites response were observed in 11 (31%) of 36 renal cell carcinomas, three (23%) of 13 renal cysts, four (27%) of 15 pancreatic lesions and one (20%) of five CNS hemangioblastomas. The study showed that the sizes of RCC (P=0.00013), renal cyst (P=0.027) and pancreatic lesion (P=0.003) were significant decreased after targeted therapy. The most common side-effects observed in this study including hand-foot syndrome, diarrhea, alopecia, back pain, thrombocytopenia and fatigue. Treatment-related serious adverse reactions included peridental abscess, hyperbilirubinemia and one case appeared perforation of nasal septum due to epistaxis repeatedly.
Conclusions: Tyrosine kinase inhibitors are effective in von Hippel-Lindau disease with acceptable side-effects, which are favorable options for VHL patients. Further evaluation of TKIs in patients with VHL disease in larger prospective studies is warranted.
Source of Funding: the National Natural Science Foundation of China (grant 81572506; 81872081)ï¼›Peking University Health Science Center 985 Action Plan (grant BMU2018WRLJ001)