A new study has found that three cycles of CEV chemotherapy (carboplatin, etoposide, and vincristine) are just as effective as six cycles in treating children with unilateral retinoblastoma and high-risk pathological features, such as choroidal or optic nerve invasion, after enucleation. The randomised, open-label trial, conducted at two leading eye centers in China, involved 187 patients and aimed to determine if fewer cycles of chemotherapy could maintain similar survival outcomes while reducing the treatment burden.
The trial, which followed patients for a median of 79 months, showed that five-year disease-free survival was 90.4% for those receiving three cycles of chemotherapy, compared to 89.2% for those receiving six cycles—meeting the predefined noninferiority margin of 12%. While the difference in survival rates was minimal, the study highlighted several advantages of the shorter treatment regimen.
The three-cycle group experienced fewer adverse events, less severe reductions in quality of life, and lower healthcare costs compared to the six-cycle group. The findings suggest that shorter chemotherapy regimens could provide similar clinical outcomes while improving the quality of life for young patients, making it a potentially more effective and cost-efficient treatment option.
This research represents a significant step forward in optimising retinoblastoma treatment, offering a less intensive alternative without compromising survival chances for children with high-risk features.
Reference
Ye H et al. Three vs 6 Cycles of Chemotherapy for High-Risk Retinoblastoma: A Randomized Clinical Trial. JAMA. 2024. DOI: 10.1001/jama.2024.19981.
