TOPLINE:

In children with retinoblastoma whose tumor had grown into the optic nerve, not receiving chemotherapy after removal of the eyeball was linked to higher risks for cancer recurrence in the eye socket, distant metastasis, and death than receiving the adjuvant therapy.

METHODOLOGY:

• This retrospective cohort study assessed whether chemotherapy after enucleation improved outcomes in children with retinoblastoma whose tumor had grown beyond the back of the eye into the optic nerve.
• Researchers included 292 children (mean age, 34 months; 57% boys) who underwent enucleation between 2011 and 2020 in nine countries across four continents.
• Children were grouped based on whether they received adjuvant therapy after enucleation: 95% received chemotherapy (mostly a standard three-drug regimen with or without orbital radiation), whereas 5% did not receive adjuvant chemotherapy.
• Researchers evaluated the recurrence of the tumor in the orbit, metastasis, and death over a mean follow-up period of 75.3 months after enucleation.

TAKEAWAY:

• Rates of events were higher in children who did not receive adjuvant chemotherapy than in those who received it: recurrence of tumor in the orbit (31% vs 2%; P < .001), metastasis (31% vs 6%; P = .001), and death (38% vs 9%; P = .007). Most events among children who did not receive chemotherapy occurred within 16 months after surgery.
• Children who did not receive adjuvant chemotherapy had a 19.31-fold higher risk for recurrence of tumor in the orbit (P < .001), a 5.39-fold higher risk for metastasis (P = .003), and a 4.74-fold higher risk for death (P = .002) than those who received chemotherapy.
• Among children with tumor invasion of the optic nerve but no other high-risk features, those who did not receive adjuvant chemotherapy had a 40.68-fold higher risk for metastasis over time than those who received chemotherapy (P < .001).
• All children who developed metastasis or had a recurrence of orbital tumor eventually died during the follow-up period.

IN PRACTICE:

“Overall, based on this large, multicenter study, there is evidence to support adjuvant chemotherapy after primary enucleation to improve clinical outcomes in this high-risk pediatric cancer patient population,” the researchers wrote.

SOURCE:

This study was led by Michelle Lin, of The Vision Center at Children’s Hospital Los Angeles in Los Angeles. It was published online on December 29, 2025, in Ophthalmology Retina.

LIMITATIONS:

This retrospective study involved different protocols for treatment. Some children received radiation with chemotherapy, which may have influenced the results. The small number of untreated patients and events made the risk estimates less certain.

DISCLOSURES:

One author reported receiving grants from multiple organizations, including The Wright Foundation, Children’s Oncology Group/St. Baldrick’s Foundation, and Alex’s Lemonade Stand Foundation. The article did not mention any conflicts of interest.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.