Retinoblastoma management—Our fight is different!

Retinoblastoma treatment in India, as in the rest of the world, has come a long way—from saving lives to preserving the globe.[1,2]

Eye preservation using chemotherapy and focal treatments has become the bedrock of retinoblastoma treatment. Chemotherapy may be delivered intravenously (IVC) as well as intra-arterially (IAC), intravitreally, intracamerally, or periocularly. More recently, suprachoroidal injections and combination approaches such as ASIAC (alternate systemic IVC and IAC) are being explored to improve globe salvage by achieving higher intraocular drug concentrations.[2,3] Advances in molecular studies are laying the foundation for personalized care through liquid biopsy, prenatal diagnosis, and prognostic biomarkers. Newer surgical approaches, such as tylectomy and chemoplaque, are also being investigated.[2]

However, these advances are relevant only when parents are willing and able to pursue treatment, can afford it, and are sufficiently informed and involved in their child’s care. When the will to pursue treatment is weakened by an inability to reach healthcare facilities in time, financial constraints, or difficulty prioritizing life over vision or globe salvage, outcomes cannot be optimal.

Although studies from Europe and North America report excellent outcomes, with overall survival rates exceeding 95%, retinoblastoma patients in India continue to experience high rates of treatment abandonment.[1,2] This is not solely due to a lack of information or financial constraints. At times, families struggle to make decisions, resulting in delays and procrastination. Unfortunately, the decision-makers in these situations are not always the parents. The recent multicentric study from India reported a treatment default rate of 7.3%, with higher rates among girls and children with bilateral or extraocular disease.[4] Previous studies, however, report treatment abandonment ranging from 10% to 58%.[5]

The influence of cultural beliefs and socioeconomic circumstances has been highlighted repeatedly in the literature and remains a major obstacle to achieving optimal outcomes in retinoblastoma.[6] It is heartbreaking to see a child return after treatment has been abandoned, now presenting with extraocular disease. At that stage, all we can do is offer treatment again and hope that the family will complete it this time.

Several studies from India have documented delayed presentation and advanced disease at diagnosis. Outcome studies have consistently demonstrated poorer results than those reported from high-income countries, despite the availability of state-of-the-art treatment modalities at tertiary care centers.[5,7,8,9] Even after more than 20 years of working in this field, I still find it deeply distressing when parents switch off their phones and stop bringing their child for treatment.

As treating physicians, we need to ask ourselves: How should we treat our patients in light of the realities they face? Should we promise globe salvage through treatments that a family cannot afford, thereby prolonging an already painful journey and creating financial and emotional hardship for both the family and the treating team? Or should we offer a realistic pathway to cure and an earlier return to normal life?

Understanding each family—their cultural circumstances, socioeconomic realities, ability to travel, financial constraints, and capacity to adhere to prolonged treatment—and tailoring treatment accordingly may allow us to optimize outcomes and improve survival. With these considerations in mind we developed ICMR consensus guidelines for the treatment of retinoblastoma in India.[10,11] Our aim was to reduce mortality, even when this occasionally meant compromising globe salvage and to provide treatments that are accessible and affordable rather than simply those considered ideal in a high-income-country setting.[10,11]

Recognizing these limitations in our quest for both life and globe salvage, the importance of early detection becomes obvious. Retinoblastoma is the most common primary intraocular malignancy of childhood, despite its relatively low incidence of approximately 1 in 20,000. Early diagnosis and treatment through awareness campaigns and screening have the potential to improve both survival and globe salvage. The possibility of screening newborns, and combining routine immunization visits with screening for leukocoria is particularly attractive in addition to targeting improved awareness.

Our fight is different. We cannot simply mimic the West. Our healthcare strategies must reflect the realities of our patients and our healthcare system. This is something our policymakers need to recognize and act upon if we are to make a meaningful difference. With NGOs such as CANKIDS stepping in to support children with retinoblastoma and their families, such screening strategies may become a reality in the future.[12]

I do believe that statistics do not apply to individuals. When parents clearly understand the urgency, requirements, and costs of treatment, and when appropriate standard-of-care treatment modalities are available, we can achieve excellent outcomes.

At the same time, the biological variability in tumor aggressiveness limits our ability to predict treatment outcomes precisely. Research into tumor genetics and molecular markers is progressing rapidly, both in India and internationally, with the hope of improving prognostication for both life and globe salvage. Liquid biopsy of the eye itself is an exciting area of investigation.[13,14] Molecular studies aimed at developing targeted therapies are also underway, although the affordability and accessibility of such treatments for our patients remain important concerns.[15,16]

While we hope for a better future, our immediate responsibility is clear. We must invest our resources and energy in treating the patients in front of us with the tools we currently have. We must recognize their socioeconomic and cultural realities, adapt our treatment strategies accordingly, and strive to achieve the best possible outcomes within those constraints.

Our fight is different—and acknowledging that difference may be the first step towards winning it.

About the author

Dr Rachna Meel

Dr. Rachna Meel is a Professor of Ophthalmology at the Dr. Rajendra Prasad Centre for Ophthalmic Sciences, AIIMS, New Delhi. As a prominent medical researcher with over 160 highly cited publications, she specializes in ophthalmic oncology, oculoplasty, and retinoblastoma, bridging advanced surgical interventions with oncological care. Dr. Meel has been a key driver in establishing the Indian Council of Medical Research (ICMR) consensus guidelines for managing both intraocular and extraocular retinoblastoma. Furthermore, Dr. Meel has pioneered affordable, organ-sparing treatments for advanced Ocular Surface Squamous Neoplasia such as neoadjuvant systemic chemotherapy. Her work establishes vital treatment guidelines for complex orbital pathologies, aggressive sebaceous gland carcinomas, and rare sino-orbital infections. With a robust h-index and active participation in multi-centric trials, Dr. Meel’s insights serve as core global reference materials, optimizing management strategies for complex eye cancers while prioritizing eye-sparing outcomes