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本文引用的文献

1
An AAV Dual Vector Strategy Ameliorates the Stargardt Phenotype in Adult Mice.AAV 双载体策略可改善成年小鼠的斯塔加特表型。
Hum Gene Ther. 2019 May;30(5):590-600. doi: 10.1089/hum.2018.156. Epub 2018 Dec 24.
2
Stargardt macular dystrophy and evolving therapies.斯特格黄斑营养不良与不断发展的治疗方法。
Expert Opin Biol Ther. 2018 Oct;18(10):1049-1059. doi: 10.1080/14712598.2018.1513486. Epub 2018 Sep 10.
3
Phase 1 clinical study of an embryonic stem cell-derived retinal pigment epithelium patch in age-related macular degeneration.胚胎干细胞源性视网膜色素上皮贴片中年龄相关性黄斑变性的 1 期临床研究。
Nat Biotechnol. 2018 Apr;36(4):328-337. doi: 10.1038/nbt.4114. Epub 2018 Mar 19.
4
Novel therapeutics for Stargardt disease.治疗斯塔加特病的新型疗法。
Graefes Arch Clin Exp Ophthalmol. 2017 Jun;255(6):1057-1062. doi: 10.1007/s00417-017-3619-8. Epub 2017 Mar 11.
5
Stargardt disease: clinical features, molecular genetics, animal models and therapeutic options.斯塔加特病:临床特征、分子遗传学、动物模型及治疗选择
Br J Ophthalmol. 2017 Jan;101(1):25-30. doi: 10.1136/bjophthalmol-2016-308823. Epub 2016 Aug 4.
6
Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4 mouse and bipolar cells in the rd1 mouse and human retina ex vivo.单残基腺相关病毒衣壳突变可改善Abca4小鼠中光感受器以及rd1小鼠和离体人视网膜中双极细胞的转导。
Gene Ther. 2016 Nov;23(11):767-774. doi: 10.1038/gt.2016.54. Epub 2016 Jul 14.
7
Subretinal Transplantation of Embryonic Stem Cell-Derived Retinal Pigment Epithelium for the Treatment of Macular Degeneration: An Assessment at 4 Years.胚胎干细胞源性视网膜色素上皮细胞的视网膜下移植治疗黄斑变性:4年评估
Invest Ophthalmol Vis Sci. 2016 Apr 1;57(5):ORSFc1-9. doi: 10.1167/iovs.15-18681.
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Treatment of macular degeneration using embryonic stem cell-derived retinal pigment epithelium: preliminary results in Asian patients.使用胚胎干细胞衍生的视网膜色素上皮细胞治疗黄斑变性:亚洲患者的初步结果。
Stem Cell Reports. 2015 May 12;4(5):860-72. doi: 10.1016/j.stemcr.2015.04.005. Epub 2015 Apr 30.
9
Gene Therapy of ABCA4-Associated Diseases.ABCA4相关疾病的基因治疗。
Cold Spring Harb Perspect Med. 2015 Jan 8;5(5):a017301. doi: 10.1101/cshperspect.a017301.
10
Human embryonic stem cell-derived retinal pigment epithelium in patients with age-related macular degeneration and Stargardt's macular dystrophy: follow-up of two open-label phase 1/2 studies.人胚胎干细胞源性视网膜色素上皮细胞治疗年龄相关性黄斑变性和斯塔加特型黄斑营养不良患者:两项开放性、1/2 期研究的随访。
Lancet. 2015 Feb 7;385(9967):509-16. doi: 10.1016/S0140-6736(14)61376-3. Epub 2014 Oct 15.

《斯塔加特病的直接医疗成本:基于索赔的分析》。

The Direct Healthcare Cost of Stargardt Disease: A Claims-Based Analysis.

机构信息

Wilmer Eye Institute, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA.

Department of Epidemiology, Johns Hopkins Bloomberg School of Public Health, Baltimore, Maryland, USA.

出版信息

Ophthalmic Epidemiol. 2021 Dec;28(6):533-539. doi: 10.1080/09286586.2021.1883675. Epub 2021 Feb 21.

DOI:10.1080/09286586.2021.1883675
PMID:33615979
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11207193/
Abstract

: Stargardt disease (SD) is the most common juvenile macular degeneration and a leading cause of uncorrectable childhood blindness. The progressive and incurable nature of this chronic condition entails a long-term financial burden on affected individuals. The economic costs of SD have not been characterized in detail, so we aimed to estimate the direct healthcare cost of SD.: Outpatient administrative claims data (2010-2014) for patients with SD were analyzed from the IBM® MarketScan® Commercial Claims and Encounters Database. Two comparison groups were selected: nonexudative age-related macular degeneration (AMD) and bilateral sensorineural hearing loss (SHL). Gross median payments per year of insurance coverage were calculated.: A total of 472,428 patients were analyzed (5,015 SD, 369,750 SHL and 97,663 AMD patients respectively). The payment per year of insurance coverage for SD (median: 105.58 USD, IQR: 50.53 USD-218.71 USD) was higher than that of SHL (median: 51.01 USD, IQR: 25.66 USD-121.66 USD, < .001) and AMD (median: 76.20 USD, IQR: 38.00 USD-164.86 USD, < .001). When adjusted for age, sex, year of first service, and type of benefit plan, the annual payment for SD was 47.83 USD higher than SHL ( < .001) and 17.34 USD higher than AMD ( < .001).: There is a significant direct healthcare cost associated with SD. The annual per-patient cost of SD was higher than SHL, another condition that causes sensory impairment in people of all ages, and nonexudative AMD which causes a similar pattern of visual loss that typically begins later in life. The total lifetime per-patient cost of SD may exceed that of nonexudative AMD.

摘要

: 斯塔加特病(SD)是最常见的青少年黄斑变性,也是儿童不可矫正失明的主要原因。这种慢性疾病的进行性和不可治愈性质给患者带来了长期的经济负担。SD 的经济成本尚未详细描述,因此我们旨在估计 SD 的直接医疗保健成本。: 从 IBM® MarketScan®商业索赔和遭遇数据库中分析了 2010-2014 年 SD 患者的门诊行政索赔数据。选择了两个对照组:非渗出性年龄相关性黄斑变性(AMD)和双侧感觉神经性听力损失(SHL)。计算了每年保险覆盖的总中位数支付额。: 共分析了 472428 名患者(分别为 5015 名 SD、369750 名 SHL 和 97663 名 AMD 患者)。SD 每年的保险覆盖支付额(中位数:105.58 美元,IQR:50.53 美元-218.71 美元)高于 SHL(中位数:51.01 美元,IQR:25.66 美元-121.66 美元,<0.001)和 AMD(中位数:76.20 美元,IQR:38.00 美元-164.86 美元,<0.001)。在调整年龄、性别、首次服务年度和福利计划类型后,SD 的年支付额比 SHL 高 47.83 美元(<0.001),比 AMD 高 17.34 美元(<0.001)。: SD 与显著的直接医疗保健成本相关。SD 每位患者的年治疗费用高于 SHL,后者是一种导致所有年龄段人群感觉障碍的疾病,也高于非渗出性 AMD,后者导致类似的视力丧失模式,通常在生命后期开始。SD 的每位患者终生总治疗费用可能超过非渗出性 AMD。