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视网膜母细胞瘤患儿动脉内化疗中单药与多药治疗的比较。

Comparison of single- versus multi-drug treatment for intra-arterial chemotherapy (IAC) in children with retinoblastoma.

作者信息

Fulton Amy T, Maniar Arpita, Provenzano Alicia, Firosvi Tariq A, Rogers Julianne, Archambault Bridget, Liu Beiyu, Chow Shein-Chung, Landi Daniel B, Materin Miguel A, Hauck Erik F

机构信息

Department of Neurosurgery, Duke University, Durham, NC, USA.

Department of Ophthalmology, Duke University, Durham, NC, USA.

出版信息

Interv Neuroradiol. 2025 Mar 26:15910199251324028. doi: 10.1177/15910199251324028.

DOI:10.1177/15910199251324028
PMID:40135409
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11948239/
Abstract

ObjectiveIntra-arterial chemotherapy (IAC) is a well-established treatment for retinoblastoma (RB). However, there are no standardized recommendations regarding the choice of drugs. This study compares the outcomes of single- versus multi-drug therapy.MethodsClinical data was reviewed for RB children treated with IAC at our institution between 2018 and 2023. Patients were divided into single- and multi-drug treatment groups. Clinical parameters included total number of IAC treatments, treatment-related adverse events, duration of additional post-IAC treatments, residual disease, recurrence, and the need for enucleation.ObservationsA total of 101 IAC treatments were included. Multi-drug therapy showed improved outcomes as compared to single-drug therapy, particularly in RB group B and C patients. After multi-drug IAC, less secondary treatment time was required compared to single-drug treatment (2.1 months versus 4.6 months; p = 0.019). Group B and C patients required a median of 8.5 fewer months of secondary treatment after multi- vs. single-drug IAC. Patients treated with multi-drug IAC had an overall lower rate of residual disease or recurrence compared to single-drug IAC patients (26.3% vs. 35.7% recurrence; 52.6% vs. 71.4% residual). In group B and C patients, the difference was more pronounced (12.5% vs. 40% recurrence; 37.5% vs. 60% residual). The overall success rate in preventing enucleation was 90.9%.ConclusionsIAC treatment for RB is safe and effective. IAC prevented enucleation in >90% of our patients. Multi-drug IAC patients required less secondary treatment post-IAC, particularly group B and C patients.

摘要

目的

动脉内化疗(IAC)是视网膜母细胞瘤(RB)一种成熟的治疗方法。然而,关于药物选择尚无标准化建议。本研究比较了单药与多药治疗的效果。

方法

回顾了2018年至2023年在我院接受IAC治疗的RB患儿的临床资料。将患者分为单药和多药治疗组。临床参数包括IAC治疗的总次数、治疗相关不良事件、IAC治疗后额外治疗的持续时间、残留疾病、复发情况以及眼球摘除的必要性。

观察结果

共纳入101次IAC治疗。与单药治疗相比,多药治疗显示出更好的效果,尤其是在B组和C组RB患者中。多药IAC治疗后,与单药治疗相比,所需的二次治疗时间更短(2.1个月对4.6个月;p = 0.019)。B组和C组患者在接受多药IAC与单药IAC治疗后,二次治疗时间中位数少8.5个月。与单药IAC患者相比,接受多药IAC治疗的患者残留疾病或复发的总体发生率更低(复发率:26.3%对35.7%;残留率:52.6%对71.4%)。在B组和C组患者中,差异更为明显(复发率:12.5%对40%;残留率:37.5%对60%)。预防眼球摘除的总体成功率为90.9%。

结论

IAC治疗RB安全有效。IAC在我们超过90%的患者中预防了眼球摘除。多药IAC治疗的患者IAC治疗后所需的二次治疗更少,尤其是B组和C组患者。

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本文引用的文献

1
Single- Versus Triple-Agent Intra-Arterial Chemotherapy for Retinoblastoma.视网膜母细胞瘤的单药与三联动脉内化疗
Am J Ophthalmol. 2025 Jan;269:488-496. doi: 10.1016/j.ajo.2024.09.037. Epub 2024 Oct 10.
2
Current Indications of Secondary Enucleation in Retinoblastoma Management: A Position Paper on Behalf of the European Retinoblastoma Group (EURbG).视网膜母细胞瘤治疗中二次眼球摘除术的当前指征:代表欧洲视网膜母细胞瘤研究组(EURbG)发布的立场文件
Cancers (Basel). 2021 Jul 6;13(14):3392. doi: 10.3390/cancers13143392.
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Intra-arterial chemotherapy in retinoblastoma - A paradigm change.眼内动脉化疗治疗视网膜母细胞瘤——一种范式转变。
Indian J Ophthalmol. 2019 Jun;67(6):740-754. doi: 10.4103/ijo.IJO_866_19.
4
Ophthalmic Vascular Events after Primary Unilateral Intra-arterial Chemotherapy for Retinoblastoma in Early and Recent Eras.早期和近期时代原发性单侧眼内动脉化疗治疗视网膜母细胞瘤后的眼部血管事件。
Ophthalmology. 2018 Nov;125(11):1803-1811. doi: 10.1016/j.ophtha.2018.05.013. Epub 2018 Jun 18.
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Improved procedural safety following protocol changes for selective ophthalmic arterial infusion of chemotherapy for treatment of ocular retinoblastoma.针对眼部视网膜母细胞瘤的选择性眼动脉化疗,在方案变更后手术安全性得到改善。
Interv Neuroradiol. 2018 Jun;24(3):345-350. doi: 10.1177/1591019918755088. Epub 2018 Feb 13.
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Myoepithelial carcinoma with RB1 mutation: remarkable chemosensitivity to carcinoma of unknown origin therapy.伴有RB1突变的肌上皮癌:对原发灶不明癌治疗具有显著化疗敏感性。
BMC Cancer. 2017 Apr 8;17(1):250. doi: 10.1186/s12885-017-3249-x.
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Rev Invest Clin. 2016 May-Jun;68(3):137-42.
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Intra-arterial Chemotherapy for Retinoblastoma: A Systematic Review.视网膜母细胞瘤的动脉内化疗:一项系统评价
JAMA Ophthalmol. 2016 May 1;134(5):584-591. doi: 10.1001/jamaophthalmol.2016.0244.
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