Vempuluru Vijitha S, Raval Vishal, Kaliki Swathi
The Operation Eyesight Universal Institute for Eye Cancer.
Anant Bajaj Retina Institute, LV Prasad Eye Institute, Hyderabad, India.
Retina. 2025 Jul 3. doi: 10.1097/IAE.0000000000004601.
To report the outcomes and safety of 100 mcg/0.1cc intravitreal topotecan (IVitT100) as monotherapy for refractory/recurrent intraocular retinoblastoma (RB).
Retrospective single-center study of 7 RB patients who received IVitT100 as monotherapy between January 2024 and December 2024 with a minimum follow-up of 3 months after the last injection.
At presentation, six eyes were classified as Group D and one as Group C, according to the International Classification of Retinoblastoma. High-dose topotecan was administered for refractory and recurrent disease in 2 (29%) and 5 (71%) eyes, respectively. The active disease was in the form of a intraretinal tumor with vitreous seeds (n=3, 43%), intraretinal tumor with epiretinal seeds (n=1, 14%), isolated intraretinal tumor (n=1, 14%), isolated subretinal seeds (n=1, 14%), or isolated vitreous seeds (n=1, 14%). At a mean follow-up of 7 months (median, 6 months; range, 3 to 10) from the last injection of IVitT00, tumor control was achieved in 100% (4 of 4) eyes with vitreous seeds, 100% (1 of 1) with epiretinal seeds, 100% (1 of 1) with subretinal seeds, and 60% (3 of 5) with retinal tumor. Overall, tumor control was achieved in 5 eyes (71%) with IVitT100 monotherapy. Additional treatments were warranted in 2 eyes for complete tumor control. Globe salvage was achieved in all eyes (100%). The electroretinogram (n=4) showed no significant changes compared to the baseline.
IVitT100 is a safe option for refractory or recurrent intraocular RB and holds promise for controlling refractory vitreous seeds, subretinal seeds, and intraretinal tumors.
报告100微克/0.1毫升玻璃体内拓扑替康（IVitT100）作为难治性/复发性眼内视网膜母细胞瘤（RB）单一疗法的疗效和安全性。
对2024年1月至2024年12月期间接受IVitT100单一疗法的7例RB患者进行回顾性单中心研究，末次注射后至少随访3个月。
根据视网膜母细胞瘤国际分类，就诊时，6只眼分类为D组，1只眼分类为C组。高剂量拓扑替康分别用于2只（29%）难治性疾病眼和5只（71%）复发性疾病眼。活动性疾病表现为伴有玻璃体播散的视网膜内肿瘤（n = 3，43%）、伴有视网膜前播散的视网膜内肿瘤（n = 1，14%）、孤立性视网膜内肿瘤（n = 1，14%）、孤立性视网膜下播散（n = 1，14%）或孤立性玻璃体播散（n = 1，14%）。自末次注射IVitT100后平均随访7个月（中位数，6个月；范围，3至10个月），伴有玻璃体播散的4只眼中100%（4/4）实现肿瘤控制，伴有视网膜前播散的1只眼中100%（1/1）实现肿瘤控制，伴有视网膜下播散的1只眼中100%（1/1）实现肿瘤控制，伴有视网膜肿瘤的5只眼中60%（3/5）实现肿瘤控制。总体而言，IVitT100单一疗法使5只眼（71%）实现肿瘤控制。为实现完全肿瘤控制，2只眼需要额外治疗。所有眼（100%）均实现眼球挽救。与基线相比，视网膜电图（n = 4）无显著变化。
IVitT100是难治性或复发性眼内RB的一种安全选择，有望控制难治性玻璃体播散、视网膜下播散和视网膜内肿瘤。
