HIGH-DOSE INTRAVITREAL TOPOTECAN FOR RECURRENT RETINOBLASTOMA, SUBRETINAL SEEDS, AND VITREOUS SEEDS
Carol L Shields1, Robert Medina, Haley Evans
1Ocular Oncology Service, Wills Eye Hospital, Thomas Jefferson University, Philadelphia, Pennsylvania.
Retina (Philadelphia, Pa.)
|October 8, 2024
Summary
High-dose intravitreal topotecan (IvitTopo) effectively controls recurrent retinoblastoma, achieving tumor regression and globe salvage in most cases. This treatment demonstrated no complications, suggesting it is a safe option for managing this eye cancer.
Area of Science:
- Ophthalmology
- Pediatric Oncology
- Retinoblastoma Research
Background:
- Retinoblastoma is the most common primary intraocular malignancy in children.
- Recurrent retinoblastoma poses a significant challenge, often requiring enucleation or systemic chemotherapy.
- Novel therapeutic strategies are needed to improve outcomes for recurrent retinoblastoma.
Purpose of the Study:
- To evaluate the efficacy and safety of high-dose intravitreal topotecan (IvitTopo) for treating recurrent retinoblastoma.
- To assess tumor control, globe salvage rates, and treatment-related complications.
Main Methods:
- A cohort of 13 patients with recurrent retinoblastoma received high-dose IvitTopo (90-100 µg).
- Patients had varying stages of recurrent disease, including solid tumors and vitreous/subretinal seeds.
- Treatment outcomes were evaluated based on tumor regression, globe salvage, and complications, with a mean follow-up of 9 months.
Main Results:
- Tumor regression was achieved in 92% of patients (12/13).
- Globe salvage was successful in all 13 patients (100%).
- High-dose IvitTopo, with or without additional therapies, showed no treatment complications.
Conclusions:
- High-dose intravitreal topotecan is an effective and safe therapeutic option for recurrent retinoblastoma.
- IvitTopo can be used in conjunction with other therapies or potentially as a standalone treatment.
- This approach offers a promising alternative for preserving the eye in cases of recurrent retinoblastoma.


