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与视网膜下基因治疗手术相关的医源性脉络膜新生血管形成。

Iatrogenic choroidal neovascularization associated with subretinal gene therapy surgery.

作者信息

Price Kenneth W, Pennesi Mark E, Lauer Andreas K, Bailey Steven T

机构信息

Casey Eye Institute, Oregon Health and Science University, Portland, OR, USA.

出版信息

Am J Ophthalmol Case Rep. 2022 Aug 6;27:101677. doi: 10.1016/j.ajoc.2022.101677. eCollection 2022 Sep.

DOI:10.1016/j.ajoc.2022.101677
PMID:36034763
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC9399261/
Abstract

PURPOSE

To report a case of iatrogenic choroidal neovascularization (CNV) developing one month after subretinal gene therapy surgery.

OBSERVATIONS

A 16-year-old male with biallelic RPE65 mutation associated retinal dystrophy was treated with subretinal voretigene neparvovec in the left eye. During initiation of a balanced salt solution pre-bleb, a faint and transient subretinal hemorrhage was observed at the retinotomy site. One month post-operatively, multi-modal imaging detected a CNV and a break in Bruch's membrane at the retinotomy site. The asymptomatic CNV was observed without treatment and resolved spontaneously.

CONCLUSIONS & IMPORTANCE: As subretinal gene therapy surgery becomes more common, clinicians should monitor for possible trauma induced CNV associated with retinotomy formation and subretinal injection.

摘要

目的

报告1例视网膜下基因治疗手术后1个月发生医源性脉络膜新生血管(CNV)的病例。

观察结果

一名16岁双等位基因RPE65突变相关视网膜营养不良男性患者接受了左眼视网膜下注射voretigene neparvovec治疗。在开始使用平衡盐溶液进行前房穿刺时,在视网膜切开部位观察到轻微且短暂的视网膜下出血。术后1个月,多模式成像检测到视网膜切开部位有CNV及 Bruch膜破裂。观察到无症状的CNV,未进行治疗,其自行消退。

结论与意义

随着视网膜下基因治疗手术越来越普遍,临床医生应监测与视网膜切开形成和视网膜下注射相关的可能创伤性CNV。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/52fc/9399261/722cdfc6ba4a/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/52fc/9399261/967d196c3297/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/52fc/9399261/3a039e191171/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/52fc/9399261/722cdfc6ba4a/gr3.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/52fc/9399261/967d196c3297/gr1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/52fc/9399261/3a039e191171/gr2.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/52fc/9399261/722cdfc6ba4a/gr3.jpg

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本文引用的文献

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N Engl J Med. 2015 May 14;372(20):1887-97. doi: 10.1056/NEJMoa1414221. Epub 2015 May 4.
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Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.RPE65基因疗法治疗莱伯先天性黑蒙的年龄依赖性效应:一项1期剂量递增试验。
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Voretigene neparvovec for inherited retinal dystrophy due to RPE65 mutations: a scoping review of eligibility and treatment challenges from clinical trials to real practice.用于 RPE65 突变引起的遗传性视网膜营养不良的 voretigene neparvovec:从临床试验到实际实践的资格和治疗挑战的范围审查。
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Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic -Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center.单一中心队列患者双等位基因介导遗传性视网膜营养不良接受 voretigene neparvovec 治疗的结局和不良反应。
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The Role of Subretinal Injection in Ophthalmic Surgery: Therapeutic Agent Delivery and Other Indications.视网膜下注射在眼科手术中的作用:治疗剂输送和其他适应证。
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N Engl J Med. 2008 May 22;358(21):2240-8. doi: 10.1056/NEJMoa0802315. Epub 2008 Apr 27.
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