Abstract

(1) Background: To analyze changes in treatment patterns for advanced retinoblastoma over time and differences in globe salvage rates; (2) Methods: Retrospective, observational case-control study of 97 eyes of 91 patients with advanced retinoblastoma (Group D and E).; (3) Results: Patients were divided into two groups based on whether they were treated before or after intraarterial chemotherapy (IAC) was introduced in our center in 2010. Before 2010, primary treatment pattern was enucleation, which was performed in 57.6% of cases, whereas primary treatment pattern after 2010 was IAC combined with intravenous chemotherapy (IVC), which was performed in 78.1%. Intravitreal chemotherapy (IVitC) has been performed to treat vitreous and subretinal seeding since 2015. The 5-year globe salvage rate of IVC alone was 24.0% for Group D and 0% for Group E, whereas that of IVC–IAC was 50.4% for Group D and 49.7% for Group E. Whether IVitC was performed or not did not significantly contribute to globe salvage rate. There was one metastatic death in the IVC alone group.; (4) Conclusions: Primary treatment pattern changed from enucleation to IAC-based treatment, which can now save nearly half of eyes with advanced retinoblastoma with excellent safety profile and survival rate.

Highlights

  • Retinoblastoma is the most common primary intraocular malignant tumor in children.Patients who develop metastatic disease often exhibit histopathological high-risk features including tumor invasion into the optic nerve and/or uvea [1,2]

  • Introduced intra-arterial chemotherapy (IAC), which is a treatment strategy to inject anticancer drugs at the ostium of the ophthalmic artery, has shown promising results for the conservation of the eyeball, even those with advanced retinoblastoma, but by IAC alone, there still exists the concern for the development of metastatic disease [10,11,12]

  • Patients received an average of 10.6 ± 5.2 cycles of intravenous chemotherapy (IVC) treatments, 4.1 ± 2.0 IAC treatments, and 8.8 ± 8.0 Intravitreal chemotherapy (IVitC) treatments

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Summary

Introduction

Retinoblastoma is the most common primary intraocular malignant tumor in children.Patients who develop metastatic disease often exhibit histopathological high-risk features including tumor invasion into the optic nerve and/or uvea [1,2]. Patients with advanced Group D or E retinoblastoma have been generally recommended to have enucleation for fear of tumor involvement of the central nervous system or hematogenous spread of the disease to other organs. Treatment results of IVC on advanced retinoblastoma are not satisfactory, as local recurrence rate is high, resulting in eventual enucleation [8,9]. Introduced intra-arterial chemotherapy (IAC), which is a treatment strategy to inject anticancer drugs at the ostium of the ophthalmic artery, has shown promising results for the conservation of the eyeball, even those with advanced retinoblastoma, but by IAC alone, there still exists the concern for the development of metastatic disease [10,11,12]

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