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

Abstract

ABSTRACT 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.

Keywords

• high‐risk disease
• intraocular
• ocular survival
• retinoblastoma
• visual outcome