Das A, Saiteja K, Shah PK, Prema S, Narendran V. Outcomes and adverse events following intra-arterial chemotherapy for retinoblastoma: A single center study in South India. World J Clin Pediatr 2025; 14(3): 103732 [DOI: 10.5409/wjcp.v14.i3.103732]
Corresponding Author of This Article
Parag K Shah, Professor, Department of Pediatric Retina and Ocular Oncology, Postgraduate Institute of Ophthalmology, Aravind Eye Hospital, Avinashi Road, Coimbatore 641014, Tamil Nādu, India. drshahpk2002@yahoo.com
Research Domain of This Article
Ophthalmology
Article-Type of This Article
Retrospective Study
Open-Access Policy of This Article
This article is an open-access article which was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Abhishek Das, Parag K Shah, Subramaniam Prema, Venkatapathy Narendran, Department of Pediatric Retina and Ocular Oncology, Postgraduate Institute of Ophthalmology, Aravind Eye Hospital, Coimbatore 641014, Tamil Nādu, India
Kothapally Saiteja, Department of General Ophthalmology, Aravind Eye Hospital, Coimbatore 641014, Tamil Nādu, India
Author contributions: Das A and Shah PK designed the study; Das A and Prema S conducted the study; Saiteja K analyzed the data and wrote the paper; Narendran V supervised the study; All authors read and approved the final version of the manuscript to be published.
Institutional review board statement: This study was reviewed and approved by the Institutional Ethics Committee of PSG Institute of Medical Sciences, Coimbatore, India.
Informed consent statement: Not required as it was a retrospective study.
Conflict-of-interest statement: The authors declare that there are no conflicts of interest.
Data sharing statement: Technical appendix, statistical code, and dataset available from the corresponding author at drshahpk2002@yahoo.com
Open Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Parag K Shah, Professor, Department of Pediatric Retina and Ocular Oncology, Postgraduate Institute of Ophthalmology, Aravind Eye Hospital, Avinashi Road, Coimbatore 641014, Tamil Nādu, India. drshahpk2002@yahoo.com
Received: December 3, 2024 Revised: March 1, 2025 Accepted: March 12, 2025 Published online: September 9, 2025 Processing time: 199 Days and 8 Hours
Abstract
BACKGROUND
Intra-arterial chemotherapy (IAC) has become a first-line standard treatment for retinoblastoma (RB). However, studies describing its adverse events are sparse, especially from the developing world. Our study described the outcomes and adverse events from a single center in South India.
AIM
To describe the challenges, treatment outcomes, and complications of selective IAC for RB in Indian eyes.
METHODS
This study was a single center, retrospective study that included 17 patients with RB who underwent IAC using melphalan (5/7.5 mg) and topotecan (1/2 mg) (n = 12) or melphalan (5 mg) alone (n = 3) or triple therapy that included carboplatin (30 mg) along with these drugs (n = 2) between January 2018 and December 2023. In all, 17 IAC procedures were performed using selective ophthalmic artery cannulation. Treatment outcomes were evaluated in terms of tumor control, vitreous and subretinal seed control, complications, and globe salvage rates.
RESULTS
Out of the 17 patients, 11 were diagnosed with unilateral RB and 6 were diagnosed with bilateral RB. The mean age at the time of diagnosis was 19.8 months. The mean interval between the first symptom and presentation was 6.5 months. IAC was employed as the primary (n = 9) or secondary (n = 8) modality of treatment. Each eye received a mean of 1.5 IAC sessions (median: 1 session; range: 1-3 sessions). Eyes were classified according to the international classification of RB as group B (n = 5), group C (n = 1), group D (n = 4), and group E (n = 7). Following IAC, complete regression of the main tumor was seen in 15 eyes (88%) and partial regression in 2 eyes (12%). Globe salvage was achieved in 15 eyes (88%). Adverse effects included vitreous hemorrhage (n = 3), rhegmatogenous retinal detachment (n = 2), choroidal ischemia (n = 1), isolated subretinal hemorrhage (n = 2), retinal pigment epithelium degeneration (n = 2), forehead pigmentation (n = 1), third nerve palsy with complete ptosis (n = 1), and 30-degree exotropia (n = 1). The mean follow-up period was 28.6 months (median: 24 months, range: 1–72 months).
CONCLUSION
IAC is an effective way to control RB and globe preservation. In the Indian context we encountered many challenges highlighting the importance of case selection. Further studies in India are required to thoroughly understand IAC as a treatment for RB.
Core Tip: Intra-arterial chemotherapy has become a first-line standard treatment for retinoblastoma. However, studies describing its adverse events are sparse, especially from the developing world. Our study described the challenges, treatment outcomes, and complications of selective intra-arterial chemotherapy for retinoblastoma in an Indian cohort. Our retrospective study showed that intra-arterial chemotherapy was effective in low resource settings with minimal adverse events.
