Intra-arterial chemotherapy for retinoblastoma: Treatment outcomes and globe preservation—A tertiary cancer center experience in India.

Abstract

e12622 Background: Intra-arterial chemotherapy (IAC) represents an important globe-preserving strategy for advanced retinoblastoma. However, comprehensive real-world data from Indian centers examining IAC across varied clinical scenarios remain scarce. We present treatment outcomes, recurrence rates, globe preservation success, and safety profiles from our institutional experience. Methods: We conducted a retrospective review of pediatric retinoblastoma patients who underwent IAC at our tertiary cancer center in Hyderabad, India, during 2020-2024. Primary end point is Treatment response. Treatment response was classified as complete response (CR), near-complete response (NCR), partial response (PR), poor response, or no response. Secondary endpoints included response rates, disease recurrence, enucleation frequency, globe preservation, and adverse events. Results: The cohort comprised approximately 160 patients with over 200 treated eyes. Median age at IAC initiation ranged from 20-70 months, with bilateral involvement in 60%. IAC indications distributed as follows: primary treatment (30%), bridge therapy (25%), post-VEC salvage (35%), and post-IAC salvage (10%). The majority presented with advanced disease (IIRC Groups D/E). CR/NCR was attained in approximately 65% of eyes, PR in 25%, and poor/no response in 10%. Response correlated with disease stage: Group B achieved 90% CR/NCR, Group C 70%, Group D 55%, and Group E 30%. Disease recurrence developed in 35-40% of treated eyes, predominantly among Group E and salvage cases. Globe preservation rates by group were: B (100%), C (85%), D (70%), and E (45-50%), yielding an overall preservation rate of 65-70%. Enucleation became necessary in 20-25% of Group D eyes and 40-50% of Group E eyes. Treatment related toxicity remained minimal, with isolated events. No treatment-related deaths occurred. Conclusions: This substantial Indian real-world cohort demonstrates that IAC offers high efficacy with favorable safety across multiple clinical indications in retinoblastoma management. Globe preservation outcomes were excellent for Groups B-D and clinically significant even for Group E disease. While recurrence poses challenges in advanced and multiply-treated eyes, repeat IAC proved viable and effective in appropriately selected patients. Our findings validate IAC as a cornerstone therapy for retinoblastoma care in diverse resource settings. Keywords: Retinoblastoma, intra-arterial chemotherapy, globe preservation, disease recurrence, pediatric oncology, India.