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弹性假黄瘤继发脉络膜新生血管的阿柏西普治疗:一项前瞻性研究。

Aflibercept for choroidal neovascularizations secondary to pseudoxanthoma elasticum: a prospective study.

机构信息

Department of Ophthalmology, University Hospital of Bonn, Bonn, Germany.

Centre for Rare Diseases Bonn (ZSEB), University of Bonn, Bonn, Germany.

出版信息

Graefes Arch Clin Exp Ophthalmol. 2020 Feb;258(2):311-318. doi: 10.1007/s00417-019-04551-4. Epub 2019 Dec 20.

Abstract

PURPOSE

To evaluate the use of 2 mg intravitreal aflibercept for treatment of choroidal neovascularization (CNV) secondary to angioid streaks in patients with pseudoxanthoma elasticum (PXE).

METHODS

In this 12-month prospective, open-label, uncontrolled, non-randomized interventional clinical trial, 15 PXE patients with CNV (mean age: 53 years, range 22-65) received one initial intravitreal injection of 2 mg aflibercept. Further injections were based on CNV activity at monthly examinations. The primary endpoint was change of best corrected visual acuity (BCVA) after 12 months. Secondary outcomes were change of central retinal thickness (CRT), leakage from CNV, retinal sensitivity, and vision-related quality of life.

RESULTS

BCVA improved from 75.0 ± 10.8 (± SD, Snellen equivalent 20/32) to 79.3 ± 7.3 ETDRS letters (20/32) at final visit (p = 0.083). CRT decreased from 317 ± 81 to 279 ± 51 μm (p = 0.004). Retinal sensitivity on microperimetry changed from 17.8 ± 4.5 to 18.5 ± 4.3 dB (p = 0.103) and vision-related quality of life from a VQF-25 score of 80.7 ± 10.4 to 83.5 ± 14.5 (p = 0.554). The mean number of injections was 6.7 ± 2.6, and 5 participants had persistent or reactivated CNV activity at final visit. The observed adverse events were comparable with studies on aflibercept for other indications.

CONCLUSION

The results of this study indicate that intravitreal aflibercept is a treatment option for CNV secondary to PXE.

摘要

目的

评估 2 毫克玻璃体内阿柏西普治疗假性黄色瘤弹性营养不良(PXE)患者伴发的脉络膜新生血管(CNV)的效果。

方法

在这项为期 12 个月的前瞻性、开放性、非对照、非随机干预性临床试验中,15 名 PXE 合并 CNV 患者(平均年龄 53 岁,范围 22-65 岁)接受了一次初始玻璃体内 2 毫克阿柏西普注射。根据每月检查 CNV 的活动情况进行进一步注射。主要终点是 12 个月后最佳矫正视力(BCVA)的变化。次要结局是中央视网膜厚度(CRT)、CNV 渗漏、视网膜敏感性和与视力相关的生活质量的变化。

结果

在最终随访时,BCVA 从 75.0±10.8(± SD,Snellen 等价物 20/32)提高到 79.3±7.3 ETDRS 字母(20/32)(p=0.083)。CRT 从 317±81 减少到 279±51μm(p=0.004)。微视野上的视网膜敏感性从 17.8±4.5 变为 18.5±4.3dB(p=0.103),与视力相关的生活质量从 VQF-25 评分 80.7±10.4 变为 83.5±14.5(p=0.554)。平均注射次数为 6.7±2.6,5 名患者在最终随访时仍有或重新出现 CNV 活动。观察到的不良事件与阿柏西普治疗其他适应证的研究相似。

结论

本研究结果表明,玻璃体内阿柏西普是治疗 PXE 继发 CNV 的一种治疗选择。

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