Enucleation and Globe Preservation Across Treatment Eras in Unilateral and Bilateral Retinoblastoma: A Retrospective Cohort Study
This retrospective cohort study demonstrates that the post-2012 era of targeted chemotherapy significantly reduced enucleation rates and improved globe preservation in both unilateral and bilateral retinoblastoma, with the most dramatic improvements observed in International Classification of Retinoblastoma group D eyes.
Original paper licensed under CC BY 4.0 (https://creativecommons.org/licenses/by/4.0/). This is an AI-generated explanation of the paper below. It is not written or endorsed by the authors. For technical accuracy, refer to the original paper. Read full disclaimer
Every year, thousands of children around the world are diagnosed with retinoblastoma, a rare cancer that begins in the eye. It is the most common eye tumor found in young children, usually appearing before the age of five. The disease can strike just one eye or both, and while modern medicine has made great strides in saving lives, the challenge has always been balancing survival with saving the eye itself. For decades, the standard approach to aggressive cases often involved removing the eye to ensure the cancer did not spread, a drastic but sometimes necessary step. However, as medical technology advanced, doctors began developing ways to deliver chemotherapy directly into the eye, aiming to kill the tumor without taking the organ out. The big question for specialists was whether these newer, more targeted methods were truly changing the outcome for children, and if the benefits were the same whether a child had one affected eye or two.
A team of researchers at the Iran University of Medical Sciences set out to answer this by looking back at more than two decades of patient records. They examined the medical histories of 491 children treated between 2001 and 2024. To make sense of the long timeline, the researchers divided the patients into two groups based on when they were treated: those treated before 2012 and those treated after. This split was chosen because the period around 2012 marked a shift in how doctors approached the disease, moving from treatments that relied mostly on chemotherapy given through the bloodstream to more precise methods that delivered drugs directly into the eye or the fluid inside it. The researchers wanted to see if this shift in strategy actually led to fewer children losing an eye.
The results showed a clear and significant change in outcomes. In the earlier period, before 2012, the majority of children with cancer in just one eye had to undergo surgery to remove the eye. Specifically, more than four out of every five children with a single affected eye lost that eye during the initial treatment phase. However, in the later period, after 2012, that number dropped considerably. For children with one affected eye, the rate of eye removal fell to roughly six out of ten. The improvement was even more dramatic for children with cancer in both eyes. In the earlier era, nearly half of the affected eyes in these children were removed, but in the later era, that number fell to about one-quarter. The researchers calculated that the risk of losing an eye decreased by nearly 30 percent for children with one affected eye and by nearly 45 percent for those with two affected eyes in the newer treatment era.
The study also looked closely at how the severity of the cancer influenced these results. The researchers found that the biggest improvements happened in eyes with advanced disease that was not yet at the most extreme stage. For these specific cases, the rate of eye removal plummeted from over 80 percent in the earlier years to just 12 percent in the later years for children with one affected eye. For children with two affected eyes, the rate for this same group dropped from over 50 percent to 14 percent. This suggests that the new targeted treatments have been particularly effective at saving eyes that were previously considered too dangerous to keep. While the most severe cases still often required surgery, the ability to save eyes in the intermediate stages of the disease has improved dramatically.
Despite these encouraging findings for saving eyes, the study noted that the overall survival rate for the children remained very high in both time periods, with very few deaths recorded in the entire group of nearly 500 patients. Because so few children died, the researchers could not definitively say whether the newer treatments changed the survival rates, but the data confirmed that the shift in treatment did not come at the cost of lives. The researchers were careful to point out that because this was a review of past records and not a controlled experiment, they could not prove that the new drugs alone caused the improvement. It is possible that better imaging, earlier detection, and more experienced teams also played a role. However, the data strongly suggests that the era of targeted eye treatments has fundamentally changed the landscape for children with this cancer, offering a much higher chance of keeping their eyes than was possible just a decade ago.
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