Abstract：
BACKGROUND: Access 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.
METHODS: The 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.
RESULTS: Thirty-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.
CONCLUSIONS: Higher dose carboplatin-based intravenous chemotherapy did not result in superior globe or vision salvage in group D or E retinoblastoma.
METHODS: The 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.
RESULTS: Thirty-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.
CONCLUSIONS: Higher dose carboplatin-based intravenous chemotherapy did not result in superior globe or vision salvage in group D or E retinoblastoma.
摘要：
背景：在低收入和中等收入国家（LMICs），视网膜母细胞瘤的动脉化疗途径有限。有必要优化全身化疗对晚期眼内视网膜母细胞瘤的疗效，特别是在低收入国家。目的是比较基于标准和高剂量卡铂的静脉化疗对D组和E组视网膜母细胞瘤的疗效。
方法：单中心，单盲,随机研究在2019-2021年期间进行。新诊断的D或E组视网膜母细胞瘤患者随机接受长春新碱，依托泊苷,和标准剂量与较高剂量（<36个月：18.6vs.28mg/kg；≥36个月：560vs.840mg/m2)卡铂。在诊断时和三个化疗周期后进行麻醉和超声检查。排除诊断时眼球/视力挽救可能性差的E组眼。
结果：分析了30例患者的32只眼：D组17只眼，E组15只眼。肿瘤对化疗的反应与回归模式有关（p=0.72），肿瘤缩小（直径：p=.11，高度：p=.96），视网膜下种子（p=.91），和玻璃体种子(p=.9)在两个处理臂之间是可比较的。全球打捞(D组[82%vs.67%；p=.58]；E组[12.5%vs.29%；p=.57])和有意义的视力恢复(D组[100%vs.75%；p=.13]；E组[100%vs.50％；p=.48]）在标准剂量和较高剂量组之间具有可比性。在较高剂量组中没有观察到过量的治疗相关毒性。
结论：在D组或E组视网膜母细胞瘤中，基于卡铂的高剂量静脉化疗未导致更好的眼球或视力恢复。
方法：单中心，单盲,随机研究在2019-2021年期间进行。新诊断的D或E组视网膜母细胞瘤患者随机接受长春新碱，依托泊苷,和标准剂量与较高剂量（<36个月：18.6vs.28mg/kg；≥36个月：560vs.840mg/m2)卡铂。在诊断时和三个化疗周期后进行麻醉和超声检查。排除诊断时眼球/视力挽救可能性差的E组眼。
结果：分析了30例患者的32只眼：D组17只眼，E组15只眼。肿瘤对化疗的反应与回归模式有关（p=0.72），肿瘤缩小（直径：p=.11，高度：p=.96），视网膜下种子（p=.91），和玻璃体种子(p=.9)在两个处理臂之间是可比较的。全球打捞(D组[82%vs.67%；p=.58]；E组[12.5%vs.29%；p=.57])和有意义的视力恢复(D组[100%vs.75%；p=.13]；E组[100%vs.50％；p=.48]）在标准剂量和较高剂量组之间具有可比性。在较高剂量组中没有观察到过量的治疗相关毒性。
结论：在D组或E组视网膜母细胞瘤中，基于卡铂的高剂量静脉化疗未导致更好的眼球或视力恢复。
