Comparison of single- versus multi-drug treatment for intra-arterial chemotherapy (IAC) in children with retinoblastoma

ObjectiveIntra-arterial chemotherapy (IAC) is a well-established treatment for retinoblastoma (RB). However, there are no standardized recommendations regarding the choice of drugs. This study compares the outcomes of single- versus multi-drug therapy.MethodsClinical data was reviewed for RB children treated with IAC at our institution between 2018 and 2023. Patients were divided into single- and multi-drug treatment groups. Clinical parameters included total number of IAC treatments, treatment-related adverse events, duration of additional post-IAC treatments, residual disease, recurrence, and the need for enucleation.ObservationsA total of 101 IAC treatments were included. Multi-drug therapy showed improved outcomes as compared to single-drug therapy, particularly in RB group B and C patients. After multi-drug IAC, less secondary treatment time was required compared to single-drug treatment (2.1 months versus 4.6 months; p = 0.019). Group B and C patients required a median of 8.5 fewer months of secondary treatment after multi- vs. single-drug IAC. Patients treated with multi-drug IAC had an overall lower rate of residual disease or recurrence compared to single-drug IAC patients (26.3% vs. 35.7% recurrence; 52.6% vs. 71.4% residual). In group B and C patients, the difference was more pronounced (12.5% vs. 40% recurrence; 37.5% vs. 60% residual). The overall success rate in preventing enucleation was 90.9%.ConclusionsIAC treatment for RB is safe and effective. IAC prevented enucleation in >90% of our patients. Multi-drug IAC patients required less secondary treatment post-IAC, particularly group B and C patients.