Background: Retinoblastoma is the most common primary malignant intraocular neoplasm of childhood. The poor outcomes of patients with metastatic retinoblastoma have encouraged the search for new therapies. In the current study, the efficacy of combination therapy with calcitriol and cisplatin in athymic mice with subcutaneous Y-79 human retinoblastoma tumours was assessed.
Methods: 60 athymic mice were subcutaneously injected with human Y79 retinoblastoma cells. Animals were randomised into four groups: group 1, 50 μg of cisplatin; group 2, 0.05 μg of calcitriol; group 3, 0.05 μg of calcitriol and 50 μg of cisplatin; group 4, control. The cisplatin was administered once a week, and the calcitriol was given five times a week.
Results: There was a significant inhibition of tumour growth in animals treated with the combination therapy of calcitriol and cisplatin as compared with controls and cisplatin alone (p = 0.0001 and p = 0.0041 respectively). In terms of toxicity, serum calcium levels were increased, but there was no mortality and minimal nephrotoxicity in any of the groups.
Conclusion: The present study shows that cisplatin given in combination with calcitriol may be a viable multidrug therapy option in the treatment of high-risk retinoblastoma.
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Funding The research reported in this paper was funded by NIH Grant R01-EY001917 and a Core Grant for Vision Research EY016665 with supplemental funding from Research to Prevent Blindness.
Competing interests None.
Presented in part at the Association for Research in Vision and Ophthalmology Annual Meeting in Fort Lauderdale, Florida in 2007.