High-Dose Intravitreal Topotecan for Retinoblastoma With Subretinal Seeds: A Well-Tolerated and Efficacious Approach

Area of Science:

• Ophthalmology
• Pediatric Oncology
• Pharmacology

Background:

• Refractory or recurrent retinoblastoma presents a significant clinical challenge.
• Intravitreal chemotherapy offers a localized treatment approach to minimize systemic toxicity.
• High-dose intravitreal topotecan is being explored for advanced retinoblastoma cases.

Purpose of the Study:

• To evaluate the efficacy and safety of high-dose intravitreal topotecan (90 μg) in patients with refractory or recurrent retinoblastoma.
• To assess the potential for localized disease control in eyes with active tumor and subretinal seeds.
• To identify factors influencing treatment outcomes in this patient cohort.

Main Methods:

• A case series involving 5 patients with refractory or recurrent retinoblastoma.
• Administration of high-dose (90 μg) intravitreal topotecan.
• Monitoring for tumor response, adverse effects, and treatment outcomes.

Main Results:

• Tumor regression was observed in 4 out of 5 patients.
• No significant adverse effects were reported in the majority of treated patients.
• One patient experienced disease progression necessitating enucleation.
• Timely administration and consistent follow-up were crucial for successful outcomes.

Conclusions:

• High-dose intravitreal topotecan shows promise for localized tumor control in refractory retinoblastoma.
• The treatment exhibits an acceptable safety profile, suggesting its therapeutic potential.
• Further investigation is supported to establish the definitive role of this treatment modality.