Shields Carol L, Medina Robert, Evans Haley, Valdes-Perez Nicole, Acar Ahmet Burak, Bansal Rolika, Lally Sara E, Shields Jerry A
Ocular Oncology Service, Wills Eye Hospital, Thomas Jefferson University, Philadelphia, Pennsylvania.
Retina. 2025 Jan 1;45(1):1-6. doi: 10.1097/IAE.0000000000004283.
To evaluate the efficacy and safety of high-dose intravitreal topotecan (IvitTopo) for recurrent retinoblastoma.
There were 13 patients with recurrent retinoblastoma treated with high-dose IvitTopo (90 micrograms [ μ g]/0.18cc-100 µ g/0.20cc). The primary outcome measures were tumor control, globe salvage, and treatment complications.
At date first seen, median patient age was 9 months, and the affected eye was classified as International Classification of Retinoblastoma Group B (n = 2, 15%), Group C (n = 3, 23%), or Group D (n = 8, 62%) retinoblastoma with initial therapy of intravenous chemotherapy (n = 9, 69%) or intraarterial chemotherapy (n = 4, 31%). Recurrent tumor was detected at median 10 months as solid tumor (n = 3), subretinal seeds (n = 10), and/or vitreous seeds (n = 3) and high-dose IvitTopo (median three injections) delivered at monthly intervals. Additional chemotherapy was delivered by intraarterial (n = 8, 62%) or intravenous (n = 1, 8%) routes, and one eye received additional cryotherapy (n = 1, 8%). In three cases (23%), there was no additional therapy. At mean follow-up of 9 months, regression of solid tumor, subretinal seeds, and vitreous seeds was achieved in 12 cases (92%), and globe salvage was achieved in all cases (n = 13, 100%). Of those three eyes treated with high-dose IvitTopo alone, tumor control was initially achieved in all cases (100%), but one case that previously demonstrated massive vitreous seeding showed late recurrence of a solitary vitreous seed at 8 months. There were no complications.
High-dose IvitTopo is an effective and safe therapy for recurrent retinoblastoma, in conjunction with other therapy, and possibly as a stand-alone therapy.
评估高剂量玻璃体内注射拓扑替康（IvitTopo）治疗复发性视网膜母细胞瘤的疗效和安全性。
13例复发性视网膜母细胞瘤患者接受了高剂量IvitTopo治疗（90微克[μg]/0.18毫升 - 100μg/0.20毫升）。主要观察指标为肿瘤控制、眼球挽救及治疗并发症。
初次就诊时，患者中位年龄为9个月，患眼根据视网膜母细胞瘤国际分类法分为B组（n = 2，15%）、C组（n = 3，23%）或D组（n = 8，62%），初始治疗采用静脉化疗（n = 9，69%）或动脉内化疗（n = 4，31%）。复发性肿瘤中位在10个月时被检测到，表现为实体瘤（n = 3）、视网膜下播散灶（n = 10）和/或玻璃体播散灶（n = 3），并每月间隔给予高剂量IvitTopo（中位注射3次）。额外的化疗通过动脉内（n = 8，62%）或静脉内（n = 1，8%）途径进行，1只眼接受了额外的冷冻疗法（n = 1，8%）。3例（23%）未接受额外治疗。平均随访9个月时，12例（92%）的实体瘤、视网膜下播散灶和玻璃体播散灶实现了消退，所有病例（n = 13，100%）均实现了眼球挽救。在仅接受高剂量IvitTopo治疗的3只眼中，所有病例（100%）最初均实现了肿瘤控制，但1例先前有大量玻璃体播散的病例在8个月时出现了孤立玻璃体播散灶的晚期复发。未出现并发症。
高剂量IvitTopo联合其他治疗，可能作为单一治疗方法，是治疗复发性视网膜母细胞瘤的一种有效且安全的疗法。
