Eye Salvage and Vision Preservation in High-Risk Intraocular Retinoblastoma Patients: Long-Term Results From the Prospective Phase II AIEOP RTB 012 Study.

Introduction: This study presents the outcomes of high-risk group retinoblastoma (Rb) patients enrolled in the AIEOP RTB 012 Protocol.

Methods: Patients with intraocular unilateral Rb classified as group C/D according to "International Intraocular Retinoblastoma Classification" (IIRC), as well as those with bilateral Rb with at least one eye group C/D/E-IIRC, were treated with four cycles of carboplatin/etoposide combined with focal treatments. The primary endpoint was to evaluate ocular event-free survival (EFS) and overall survival (OS), where events were defined as the need for radiotherapy, eye enucleation, and second-line treatment. Visual acuity (VA) was assessed in all available cases.

Results: Between February 2012 and September 2017, 60 patients were enrolled (88 eyes), 32 unilateral Rb, 28 bilateral. Twelve eyes were classified as group A/B, 15 group C, 40 group D, and 21 group E. At a median follow-up of 8.71 years, 42/88 eyes were preserved. The 2- and 5-year eye EFS rates were 31.1% (95% CI: 24.4-44.0) and 29.5% (95% CI: 20.4-39.2), respectively. The corresponding 2- and 5-year eye OS rates were 63.3% (95% CI: 52.7-72.7) and 48.9% (95% CI: 38.1-58.8). Ocular survival significantly differed across IIRC groups, with 2- and 5-year survival rates of 100% for group A/B, 86.7% and 73.3% for group C, 65% and 42.5% for group D, and 23.8% and 14.3% for group E, respectively (p < 0.05). No severe toxicity was reported. Among 28 bilateral Rb patients, 12 had VA of at least 20/30 according to the Snellen chart.

Conclusion: The AIEOP RTB 012 protocol has proven to be both safe and effective, ensuring a favorable final VA.