A single‐blinded, randomized controlled trial of standard versus higher dose carboplatin‐based intravenous chemotherapy for group D and E retinoblastoma

Abstract

AbstractBackgroundAccess to intra‐arterial chemotherapy for retinoblastoma in low‐ and middle‐income countries (LMICs) is limited. There is a need to optimize the efficacy of systemic chemotherapy for advanced intraocular retinoblastoma, particularly in LMICs. The aim was to compare the efficacy of standard versus higher dose carboplatin‐based intravenous chemotherapy for group D and E retinoblastoma.MethodsThe single‐center, single‐blinded, randomized study was conducted during 2019–2021. Patients with newly diagnosed group D or E retinoblastoma were randomized to receive vincristine, etoposide, and standard versus higher dose (<36 months: 18.6 vs. 28 mg/kg; ≥36 months: 560 vs. 840 mg/m2) carboplatin. Examination under anesthesia and ultrasonography was performed at diagnosis and following three cycles of chemotherapy. Group E eyes with poor likelihood of globe/vision salvage at diagnosis were excluded.ResultsThirty‐two eyes of 30 patients were analyzed: 17 group D and 15 group E eyes. The tumor response to chemotherapy with regards to regression pattern (p = .72), tumor shrinkage (diameter: p = .11, height: p = .96), subretinal seeds (p = .91), and vitreous seeds (p = .9) were comparable between the two treatment arms. The globe salvage (group D [82% vs. 67%; p = .58]; group E [12.5% vs. 29%; p = .57]) and salvage of meaningful vision (group D [100% vs. 75%; p = .13]; group E [100% vs. 50%; p = .48]) were comparable between standard and higher dose arms. No excess treatment‐related toxicity was observed in the higher dose arm.ConclusionsHigher dose carboplatin‐based intravenous chemotherapy did not result in superior globe or vision salvage in group D or E retinoblastoma.