Frontiers in Oncology (Nov 2019)
The Efficacy and Safety of Tyrosine Kinase Inhibitors for Von Hippel–Lindau Disease: A Retrospective Study of 32 Patients
- Kaifang Ma,
- Kaifang Ma,
- Kaifang Ma,
- Kaifang Ma,
- Baoan Hong,
- Baoan Hong,
- Jingcheng Zhou,
- Jingcheng Zhou,
- Jingcheng Zhou,
- Jingcheng Zhou,
- Yanqing Gong,
- Yanqing Gong,
- Yanqing Gong,
- Yanqing Gong,
- Jiangyi Wang,
- Shengjie Liu,
- Xiang Peng,
- Bowen Zhou,
- Bowen Zhou,
- Bowen Zhou,
- Bowen Zhou,
- Jiufeng Zhang,
- Jiufeng Zhang,
- Jiufeng Zhang,
- Jiufeng Zhang,
- Haibiao Xie,
- Haibiao Xie,
- Haibiao Xie,
- Haibiao Xie,
- Kenan Zhang,
- Kenan Zhang,
- Kenan Zhang,
- Kenan Zhang,
- Lei Li,
- Lei Li,
- Lei Li,
- Lei Li,
- Desheng Cai,
- Desheng Cai,
- Desheng Cai,
- Desheng Cai,
- Zixin Wang,
- Zixin Wang,
- Zixin Wang,
- Zixin Wang,
- Lin Cai,
- Lin Cai,
- Lin Cai,
- Lin Cai,
- Kan Gong,
- Kan Gong,
- Kan Gong,
- Kan Gong
Affiliations
- Kaifang Ma
- Department of Urology, Peking University First Hospital, Beijing, China
- Kaifang Ma
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Kaifang Ma
- Institute of Urology, Peking University, Beijing, China
- Kaifang Ma
- National Urological Cancer Center, Beijing, China
- Baoan Hong
- Department of Urology, Beijing Cancer Hospital, Beijing, China
- Baoan Hong
- Beijing Institute for Cancer Research, Beijing, China
- Jingcheng Zhou
- Department of Urology, Peking University First Hospital, Beijing, China
- Jingcheng Zhou
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Jingcheng Zhou
- Institute of Urology, Peking University, Beijing, China
- Jingcheng Zhou
- National Urological Cancer Center, Beijing, China
- Yanqing Gong
- Department of Urology, Peking University First Hospital, Beijing, China
- Yanqing Gong
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Yanqing Gong
- Institute of Urology, Peking University, Beijing, China
- Yanqing Gong
- National Urological Cancer Center, Beijing, China
- Jiangyi Wang
- Department of Urology, Fudan University Shanghai Cancer Center, Shanghai, China
- Shengjie Liu
- Department of Urology, National Center of Gerontology, Beijing Hospital, Beijing, China
- Xiang Peng
- Department of Urology, The Second Affiliated Hospital of Nanchang University, Nanchang, China
- Bowen Zhou
- Department of Urology, Peking University First Hospital, Beijing, China
- Bowen Zhou
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Bowen Zhou
- Institute of Urology, Peking University, Beijing, China
- Bowen Zhou
- National Urological Cancer Center, Beijing, China
- Jiufeng Zhang
- Department of Urology, Peking University First Hospital, Beijing, China
- Jiufeng Zhang
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Jiufeng Zhang
- Institute of Urology, Peking University, Beijing, China
- Jiufeng Zhang
- National Urological Cancer Center, Beijing, China
- Haibiao Xie
- Department of Urology, Peking University First Hospital, Beijing, China
- Haibiao Xie
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Haibiao Xie
- Institute of Urology, Peking University, Beijing, China
- Haibiao Xie
- National Urological Cancer Center, Beijing, China
- Kenan Zhang
- Department of Urology, Peking University First Hospital, Beijing, China
- Kenan Zhang
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Kenan Zhang
- Institute of Urology, Peking University, Beijing, China
- Kenan Zhang
- National Urological Cancer Center, Beijing, China
- Lei Li
- Department of Urology, Peking University First Hospital, Beijing, China
- Lei Li
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Lei Li
- Institute of Urology, Peking University, Beijing, China
- Lei Li
- National Urological Cancer Center, Beijing, China
- Desheng Cai
- Department of Urology, Peking University First Hospital, Beijing, China
- Desheng Cai
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Desheng Cai
- Institute of Urology, Peking University, Beijing, China
- Desheng Cai
- National Urological Cancer Center, Beijing, China
- Zixin Wang
- Department of Urology, Peking University First Hospital, Beijing, China
- Zixin Wang
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Zixin Wang
- Institute of Urology, Peking University, Beijing, China
- Zixin Wang
- National Urological Cancer Center, Beijing, China
- Lin Cai
- Department of Urology, Peking University First Hospital, Beijing, China
- Lin Cai
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Lin Cai
- Institute of Urology, Peking University, Beijing, China
- Lin Cai
- National Urological Cancer Center, Beijing, China
- Kan Gong
- Department of Urology, Peking University First Hospital, Beijing, China
- Kan Gong
- Hereditary Kidney Cancer Research Center, Peking University First Hospital, Beijing, China
- Kan Gong
- Institute of Urology, Peking University, Beijing, China
- Kan Gong
- National Urological Cancer Center, Beijing, China
- DOI
- https://doi.org/10.3389/fonc.2019.01122
- Journal volume & issue
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Vol. 9
Abstract
Background: Von Hippel-Lindau (VHL) disease is an autosomal-dominant hereditary cancer syndrome. Currently, studies on tyrosine kinase inhibitor (TKI) therapy for VHL disease are scarce. In this study, we retrospectively evaluated the efficacy and safety of four TKIs in patients with VHL disease.Methods: Patients diagnosed with VHL disease who were receiving TKIs were recruited. Patients were treated with sunitinib (n = 12), sorafenib (n = 11), axitinib (n = 6), or pazopanib (n = 3). The therapeutic response was evaluated according to the Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1.Results: From July 2009 to September 2018, 32 patients with VHL disease were eligible and included in this study. The median duration of TKI therapy was 22 months (IQR 8.5–44.75), and the median follow-up period was 31.5 months (IQR 13.5–63.5). According to the RECIST, 9 (28%) of 32 patients showed a partial response, 15 (47%) achieved stable disease, and eight exhibited continued disease progression. A partial response was observed in 11 (31%) of 36 renal cell carcinomas, 4 (27%) of 15 pancreatic lesions, and 1 (20%) of five central nervous system (CNS) hemangioblastomas. The average tumor size decreased significantly for renal cell carcinomas (P = 0.0001), renal cysts (P = 0.027), and pancreatic lesions (P = 0.003) after TKI therapy. Common side effects included hand–foot skin reactions, diarrhea, alopecia, thrombocytopenia, and fatigue.Conclusions: Partial alleviation of VHL disease-related tumors can be achieved by TKI therapies in some patients, providing an alternative treatment strategy, and the side effects of TKIs are acceptable. Larger prospective studies are warranted to further evaluate the efficacy and safety of TKIs in patients with VHL disease.
Keywords