Let's achieve a fair world for all children affected by retinoblastoma

Vempuluru et al. and The Operation Eyesight Universal Institute for Eye Cancer and Duke Eye Center present a timely, important view of global retinoblastoma studies.1 They responded to the World Health Organization Global Initiative for Childhood Cancer prioritisation of retinoblastoma as a key indicator of the profound inequity in the survival of childhood cancer,2 with a careful review of the literature. They report on the global disparity in outcomes for retinoblastoma in 2024, pointing out the “complex dynamics of health tourism and the diverse challenges faced by children with retinoblastoma around the world.” The reports are largely retrospective views making very clear the dire situation for children depending on the economies and complexities of where they live.1 Retinoblastoma is rare, which was a major reason the World Health Organization Global Initiative for Childhood Cancer prioritised retinoblastoma, because the baseline of profound inequity in survival can be an opportunity for research to measure the impact of innovative intervention.2 The sharing of retrospective clinical data across world centres is an important first step. One immediate action towards real progress to address the crisis would be promotion of recognition that primary enucleation is the safest, most easily available treatment, which avoids expensive delays and quickly gets the child back to playing.3 Well-implemented, primary enucleation can avoid long-term complications and costs. The current widely popularised goal to salvage the eye infers that the parent's duty is to focus on eye salvage and travel, when the safest care may be well done at home.4 Teams of high-level experts, best developed with realistic coordinated local services, are required for advanced complex treatments, such as intra-arterial administration of chemotherapy and stem cell transplantation to rescue the child’s life after failure of eye salvage. At the country level, such advanced, expensive services may be appropriate only when real impact can be evaluated. Today, >5% of children worldwide with retinoblastoma, who could have local care at home for the disease when still amenable to globe salvage, instead pursue cross-border care and aspire to the quality of care available in high-income countries (HIC).5 When they do travel, mortality after returning home may be unreported for international patients treated in an HIC with inadequate follow-up. The consequence of the under-reporting is undue optimism of the next low- and middle-income country child seeking to travel in pursuit of eye salvage.4 Delay in accepting enucleation significantly impacts children’s lives.6 The retinoblastoma community has an exciting opportunity for prospective retinoblastoma studies. We can now take advantage of baseline data and look to design prospective “clinical trials” that measure the impact of novel interventions, compared with the dismal outcomes summarised by the currently available retrospective studies. For example, national and regional guideline development around the circle of care by communities has been undertaken in two countries (Canada and Kenya), with different levels of resources and healthcare technologies.7-9 Both countries worked over several years with strategy groups including ophthalmologists, oncologists, pathologists, child life specialists, health administrators, and patients/parents with lived experience. The Canadian guidelines did not consider pre- or post-guideline measures of outcomes for the children,8 but were very much driven by parents and those with lived experience. The Kenyan guidelines7 arose in part in response to poor outcomes before the guidelines10-14, and were developed over many years with wide input of clinicians, parents, patients and the Kenyan Ministry of Health. We look forward to publication of recent improved Kenyan survival rates. Another opportunity is patient-reported outcome measures (PROMs). One study of parental refusal of enucleation collected physician opinions of reasons for refusal of enucleation.3 We await a study of parents' reasons, using PROMs standards, to validate the results. Abandonment of care studies15, 16 largely surveyed the clinicians' opinions. Parents' reasons in their own words may be informative and even wise. In the presence of advanced disease, maybe the parents were ‘right’? Are clinicians sometimes focused overly on survival rather than quality of life? Also begging for PROMs studies is the impact of the COVID-19 pandemic on the outcomes of children affected by retinoblastoma. Unstudied is the SickKids retinoblastoma team observation of a positive benefit of the COVID-19 pandemic. We moved to virtual disclosures of clinical workup and recommended treatments, in full discussion with all the retinoblastoma team when the family was at home, joining virtually, seeing all images and fully engaging with extended family. Without research to measure this change in practice or seek parent opinions, we have continued with virtual disclosures, rather than the previous in-person discussions. We can also dream now of real-time data collection on retinoblastoma kids and the treatments and outcomes they experience. Coming soon is the capacity for clinical data to serve families everywhere, and simultaneously (with consent for coded data for research) inform national and World Health Organization interests. No funding sources to declare. Dr. Gallie has no conflicts of interest.

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