高危视网膜母细胞瘤化疗三周期与六周期的对比

JAMA Pub Date : 2024-10-21 DOI:10.1001/jama.2024.19981
Huijing Ye, Kang Xue, Ping Zhang, Rongxin Chen, Xiaowen Zhai, Li Ling, Wei Xiao, Lijuan Tang, Hongsheng Wang, Yuxiang Mao, Siming Ai, Yingwen Bi, Qing Liu, Yusha Zou, Jiang Qian, Huasheng Yang
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However, there is a lack of head-to-head clinical trials comparing 3 vs 6 cycles of CEV chemotherapy (carboplatin, etoposide, and vincristine) for enucleated unilateral retinoblastoma with high-risk pathological features.ObjectiveTo assess whether 3 cycles of CEV chemotherapy is noninferior to 6 cycles for enucleated unilateral retinoblastoma with high-risk pathological features.Design, Setting, and ParticipantsThis double-center, randomized, open-label, noninferiority trial was conducted at 2 premier eye centers in China and included 187 patients who had undergone enucleation for unilateral retinoblastoma with high-risk pathological features (massive choroidal infiltration, retrolaminar optic nerve invasion, or scleral infiltration) between August 2013 and March 2024. The final date of follow-up was March 21, 2024.InterventionsPatients were randomly assigned to receive either 3 (n = 94) or 6 (n = 93) cycles of CEV chemotherapy regimen after enucleation.Main Outcomes and MeasuresThe primary end point was disease-free survival, with a noninferiority margin of 12%. Secondary end points encompassed overall survival, safety, economic burden, and the quality of life of children.ResultsAll 187 patients (median [IQR] age, 25.0 [20.0-37.0] months; 83 [44.4%] female) completed the trial. Median (IQR) follow-up was 79.0 (65.5-102.5) months. Five-year disease-free survival was 90.4% for the 3-cycle group vs 89.2% for the 6-cycle group (difference, 1.2% [95% CI, −7.5% to 9.8%]), which met the noninferiority criterion (<jats:italic>P</jats:italic> = .003 for noninferiority). 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引用次数: 0

摘要

重要性辅助治疗是视网膜母细胞瘤重要而有效的治疗方法。然而,对于具有高危病理特征的单侧带核视网膜母细胞瘤,目前还缺乏头对头临床试验来比较 3 个周期与 6 个周期的 CEV 化疗(卡铂、依托泊苷和长春新碱)。目的评估对于具有高危病理特征的单侧带核视网膜母细胞瘤,3 个周期的 CEV 化疗是否不劣于 6 个周期的 CEV 化疗。设计、设置和参与者这项双中心、随机、开放标签、非劣效性试验在中国的两家顶级眼科中心进行,共纳入了187名在2013年8月至2024年3月期间因单侧视网膜母细胞瘤且具有高危病理特征(大量脉络膜浸润、视神经后膜浸润或巩膜浸润)而接受去核手术的患者。干预措施患者在去核后被随机分配接受3个周期(94例)或6个周期(93例)的CEV化疗方案。主要结果和测量主要终点为无病生存期,非劣效边际为12%。次要终点包括总生存期、安全性、经济负担和儿童生活质量。结果所有187名患者(中位数[IQR]年龄,25.0 [20.0-37.0]个月;83 [44.4%]名女性)均完成了试验。中位(IQR)随访时间为 79.0 (65.5-102.5) 个月。3周期组的5年无病生存率为90.4%,6周期组为89.2%(差异为1.2% [95% CI, -7.5%至9.8%]),符合非劣效性标准(非劣效性P = .003)。在单侧病理高危视网膜母细胞瘤患者中,3个周期CEV化疗的5年无病生存率不劣于6个周期CEV化疗:NCT01906814
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Three vs 6 Cycles of Chemotherapy for High-Risk Retinoblastoma
ImportanceAdjuvant therapy is an important and effective treatment for retinoblastoma. However, there is a lack of head-to-head clinical trials comparing 3 vs 6 cycles of CEV chemotherapy (carboplatin, etoposide, and vincristine) for enucleated unilateral retinoblastoma with high-risk pathological features.ObjectiveTo assess whether 3 cycles of CEV chemotherapy is noninferior to 6 cycles for enucleated unilateral retinoblastoma with high-risk pathological features.Design, Setting, and ParticipantsThis double-center, randomized, open-label, noninferiority trial was conducted at 2 premier eye centers in China and included 187 patients who had undergone enucleation for unilateral retinoblastoma with high-risk pathological features (massive choroidal infiltration, retrolaminar optic nerve invasion, or scleral infiltration) between August 2013 and March 2024. The final date of follow-up was March 21, 2024.InterventionsPatients were randomly assigned to receive either 3 (n = 94) or 6 (n = 93) cycles of CEV chemotherapy regimen after enucleation.Main Outcomes and MeasuresThe primary end point was disease-free survival, with a noninferiority margin of 12%. Secondary end points encompassed overall survival, safety, economic burden, and the quality of life of children.ResultsAll 187 patients (median [IQR] age, 25.0 [20.0-37.0] months; 83 [44.4%] female) completed the trial. Median (IQR) follow-up was 79.0 (65.5-102.5) months. Five-year disease-free survival was 90.4% for the 3-cycle group vs 89.2% for the 6-cycle group (difference, 1.2% [95% CI, −7.5% to 9.8%]), which met the noninferiority criterion (P = .003 for noninferiority). The 6-cycle group experienced a higher frequency of adverse events, greater reduction in quality of life scores, and increased costs compared with the 3-cycle group.Conclusions and RelevanceAmong patients with unilateral pathologic high-risk retinoblastoma, 3 cycles of CEV chemotherapy resulted in 5-year disease-free survival that was noninferior to 6 cycles of CEV chemotherapy.Trial RegistrationClinicalTrials.gov Identifier: NCT01906814
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