Intravenous Chemotherapy Plus Intra-Arterial Chemotherapy Versus Intra-Arterial Chemotherapy in Patients with Advanced Retinoblastoma Cancer: A Retrospective, Multicentre-Based Study

Abstract

Background: Intra-arterial chemotherapy (IAC) was found to be effective for the management of advanced retinoblastoma. However, the value of the addition of intravenous chemotherapy (IVC) to IAC in advanced retinoblastoma is unclear. Methods: Kaplan-Meier analyses were performed to compare 2-year rates of ocular survival, overall survival and event-free survival between the groups. Cox proportional hazards model was used to test the independent significance of different factors. Details of enucleated eyes and their histopathological results were recorded. Findings: Between July 2008 and December 2016, 213 patients at five institutions in China with advanced retinoblastoma (group D and E eyes) were included for analysis. They were treated with IVC plus IAC (n=103) or IAC (n=110). The proportion of patients with ocular survival at 2 years was 68% (95%CI 57-76) in the IVC plus IAC group and 72% (95%CI 60-80) in the IAC group (hazard ratio [HR] 0.88 [95%CI 0.55-1.43], P=0.61).The proportion of patients with overall survival at 2 years was 97% (95%CI 92-99) in the IVC plus IAC group and 96% (95%CI 90-99) in the IAC group (HR 1.56 [95%CI 0.41-5.90], P=0.51). The proportion of patients with event-free survival at 2 years was 76% (95%CI 67-83) in the IVC plus IAC group and 79% (95%CI 69-86) in the IAC group (HR 0.96 [95%CI 0.56-1.65], P=0.89). Multivariate analyses showed eye staging and local therapy were significant prognostic factors of ocular survival. High-risk histopathologic features were identified in 10.5% (4/38) of the IVC plus IAC group and 16.7% (5/30) of the IAC group, with no statistical difference (P=0.46). Interpretation: Addition of IVC to IAC did not significantly improve survival outcomes and lower the risk of metastasis in patients with advanced retinoblastoma cancer. Longer follow-up is needed to determine long-term efficacy, but at present, additional IVC is not recommended for using outside the scope of clinical research. Funding Statement: The National Natural Science Foundation of China; the National Natural Science Foundation of Guangdong Province; the Science and Technology Planning Project of Guangdong Province; the Scientific Research General Project of Guangzhou Science Technology and Innovation Commission; and the China Postdoctoral Science Foundation. Declaration of Interests: The authors declare that they have no competing interests. Ethics Approval Statement: All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.