早产儿视网膜病
Retinopathy of prematurity
定义 英文原文（暂无中文）
A rare retinal vasoproliferative disease affecting preterm infants characterized initially by a delay in physiologic retinal vascular development and compromised physiologic vascularity, and subsequently by aberrant angiogenesis in the form of intravitreal neovascularization.
别名
晶体后纤维增生症
基本事实
- 遗传方式
- 不适用
- 发病年龄
- 婴儿期、新生儿期
- 患病率
- 1-5 / 10 000（Europe）
相关基因 3
| 基因 | 名称 | 关联类型 |
|---|---|---|
| FZD4 | frizzled class receptor 4 | Major susceptibility factor in |
| LRP5 | LDL receptor related protein 5 | Major susceptibility factor in |
| NDP | norrin cystine knot growth factor NDP | Major susceptibility factor in |
临床表型 15
极常见 99–80%4
- 视网膜脉管形态异常 HP:0008046
- 早产 HP:0001622
- 早产儿视网膜病 HP:0500049
- 小于胎龄儿 HP:0001518
偶见 29–5%11
- 黄斑形态异常 HP:0001103
- 弱视 HP:0000646
- 失明 HP:0000618
- 白内障 HP:0000518
- 青光眼 HP:0000501
- 近视 HP:0000545
- 视力下降 HP:0007663
- 视网膜小动脉迂曲 HP:0001136
- 斜视 HP:0000486
- 牵拉性视网膜脱离 HP:0007917
- 玻璃体出血 HP:0007902
近两年的全球研究 3,715L2
2024/10 起在 Europe PMC 检索所得，按发表时间倒序显示最近 20 篇。标题未译成中文——自动翻译需要接入 LLM 服务，尚未引入。
- 2026-11Retinopathy of prematurity training should be judged by competence, not exposure alone
- 2026-10Cost-effectiveness of community-based artificial intelligence screening for retinopathy of prematurity
- 2026-10Zone II stage 2 retinopathy of prematurity without plus disease: long-term follow-up and predictors of persistent avascular retina
- 2026-10Effect of Prematurity on Foveal Development During Infancy: Increased Foveal Inner and Outer Retina Layer Thicknesses in Extremely Premature Infants
- 2026-10开放获取Müller Glia-Vasculature Interactions in the Developing Retina
- 2026-09Exploring a potential healthy survivor bias in retinopathy of prematurity
- 2026-09综述开放获取Recent trends in encapsulated bioactives for improving the delivery of infant milk fortifiers
- 2026-09Late Exudative Retinal Detachment in Prior Treated Retinoblastoma
- 2026-09Ultrawidefield OCT angiography in laser-treated retinopathy of prematurity
- 2026-09Intratracheal budesonide combined with surfactant for respiratory distress syndrome in preterm infants: A randomized clinical trial
- 2026-09综述Current Trends in AI and Eye Disease Diagnostics
- 2026-09随机对照试验Nine-year ocular developmental outcomes after ranibizumab or laser treatment for zone II retinopathy of prematurity: follow-up of a randomized trial cohort
- 2026-09Electroretinographic assessment of retinal function in preterm infants using the RETeval™ device: a proposal for reference data
- 2026-09Early neurobehavior and general movements in high-risk preterm infants: Interrelationships and associations with medical and social factors
- 2026-09An unusual segmental retinal venous abnormality detected during retinopathy of prematurity screening in an infant with a PAX2 variant
- 2026-09Advances in diagnostic training for retinopathy of prematurity with the introduction of the Eyesi Indirect Ophthalmoscope ROP Simulator: an exploratory study to assess its construct and face validity
- 2026-09Validation of the DIGIROP 2.0 Models in predicting the risk of treatment-requiring retinopathy of prematurity in a Taiwanese cohort
- 2026-09Airway management and postoperative respiratory recovery after intravitreal injection in preterm infants
- 2026-09The predictive role of early and late-stage inflammatory markers in the development of retinopathy of prematurity in discordant twins
- 2026-09综述Retinopathy of prematurity in the anti-VEGF era: Screening systems and practical treatment decision-making across anti-VEGF, laser, and surgery
境外已获批用于本病的药物 1L2
欧盟 0 项、美国 1 项。同一药物在两地各批一次的，会分别列出。
「境外已获批」不等于「在中国能用」。中间隔着进口注册、临床急需境外新药通道、海南博鳌乐城国际医疗旅游先行区等几条路径，各有各的条件与费用。这一节能确定地告诉你的只有一件事：这个病在世界范围内已经有获得批准的药物，它叫什么名字。拿这个名字去问主治医生，是下一步最省力的做法。
药名一律保留英文原文，不作翻译——中国的药品通用名与英文名的音译经常不一致，译错会让人去找一个不存在的药。
- Eylea美国2023-02-08aflibercept官方记录
尚未获批的在研药物（18 项）
这些药已被欧盟或美国的监管机构认定为罕见病用药（英文 orphan drug designation，中文行业里通称「孤儿药资格」——「孤儿」说的是这类药市场太小、没有厂商愿意认领，不是在说病人）。但这只是一种监管身份：意味着监管机构给予研发上的激励，不代表这个药已被证明有效，也不代表将来一定能上市，绝大多数最终不会成药。列在这里是为了看清有哪些方向正在被尝试。
- antisense oligonucleotide (TATCCGGAGGGCTCGCCATGCTGCT)欧盟2003-10-02Treatment of retinopathy of prematurity官方记录
- mecasermin rinfabate欧盟2006-08-28Prevention of retinopathy of prematurity in neonates of less than 32 weeks of gestational age官方记录
- retinol欧盟2017-07-17Prevention of retinopathy of prematurity官方记录
- propranolol hydrochloride欧盟2019-10-17Treatment of retinopathy of prematurity官方记录
- melatonin欧盟2021-06-21Prevention of retinopathy of prematurity官方记录
- insulin human欧盟2022-01-14Prevention of retinopathy of prematurity官方记录
- sodium (4Z,7Z,10R,11E,13E,15Z,17S,19Z)10,17-dihydroxy-docosa-4,7,11,13欧盟2022-06-21Prevention of retinopathy of prematurity官方记录
- myo-inositol美国2005-04-07Prevention of retinopathy of prematurity in preterm infants at risk for developing retinopathy of prematurity官方记录
- mecasermin rinfabate美国2012-09-20Prevention of retinopathy of prematurity in premature infants born at risk for the disease官方记录
- 3-[3-(6-guanidino-1-oxoisoindolin-2yl) propanamido]-3-(pyridine-3yl) p美国2017-01-17Treatment of retinopathy of prematurity官方记录
- retinol palmitate (vitamin A palmitate)美国2017-06-28Prevention of retinopathy of prematurity (ROP)官方记录
- Propranolol美国2019-08-23Treatment of retinopathy of prematurity官方记录
- Sodium (4Z,7Z,10R,11E,13E,15Z,17S,19Z) 10,17-dihydroxy-docosa-4,7,11,1美国2020-03-30Prevention of retinopathy of prematurity官方记录
- Melatonin美国2020-04-20Treatment of retinopathy of prematurity官方记录
- Human Insulin (rDNA)美国2022-02-28Prevention of retinopathy of prematurity (ROP)官方记录
- Arginyl-Glutamine Dipeptide美国2022-09-20Prevention of Retinopathy of Prematurity官方记录
- methyldopa美国2024-02-27Prevention of retinopathy of prematurity官方记录
- 31-amino acid TREM-1 inhibitory peptide美国2026-03-22Treatment of retinopathy of prematurity官方记录
数据来自欧洲药品管理局（EMA）的药品与罕见病用药资格公开导出表，以及美国 FDA 的罕见病用药资格数据库（Orphan Drug Product Designation Database）。两边口径不同：欧盟一侧取的是当前状态仍为「已授权」的药品；美国一侧记录的是「曾获批准」这一事实，FDA 的公开表不追踪药物此后是否退市（例如 Relyvrio 于 2024 年撤市，表中仍记为已获批）。请以官方记录页为准。
在中国开展的临床试验 7L2
按病名在 ClinicalTrials.gov 检索、并校验研究中心含中国大陆而来。登记状态不等于现在真的能入组——务必按 NCT 号到原站核实，并与主治医生商量。
登记为可入组 1
- 招募中NCT06717412Efficacy and Safety of Low-dose Conbercept for Retinopathy of Prematurity Therapy中国研究中心 3 个：Xi'an、Xianyang
其他状态的试验（6 项）
- 已完成NCT03154723Effects of Early Vitamin A Supplementation on the Risk for Retinopathy of Prematurity in Extremely Preterm Infants中国研究中心 1 个：Zhengzhou
- 状态未知NCT02977000Oral Propranolol Improve Retinopathy of Prematurity Outcomes in Very Preterm Infants中国研究中心 1 个：Zhengzhou
- 已完成NCT03115255Serum Vascular Endothelial Growth Factor in Infants With Intravitreal Ranibizumab中国研究中心 1 个：Shanghai
- 状态未知NCT04985448Real World Study of the Effectiveness and Safety of Conbercept Ophthalmic Injection in the Treatment of Retinopathy of Prematurity - Multicenter, Retrospective and Observational Study Based on Real World Data中国研究中心 2 个：Beijing、Chengdu
- 已完成NCT05576792A 24-week Study Evaluating the Effectiveness and Safety of Lucentis® 0.2mg in Retinopathy of Prematurity Participants in China中国研究中心 1 个：Guangzhou
- 已完成NCT06729333A Multicenter and Prospective Study of Screening Retinopathy of Prematurity in China中国研究中心 1 个：Shanghai
中国境外的在招试验 23L2
这些试验在中国没有研究中心，通常无法直接报名——入组一般要求在当地居住并接受随访。列在这里是因为它另有用处：看清楚全世界正在试哪些药、做到了哪一期、由谁在做。把药名和 NCT 编号记下来去问主治医生，或据此进一步查该药是否已在境外获批、是否有拓展性用药（expanded access）通道。
美国6土耳其5西班牙2德国2瑞士2法国2加拿大1挪威1沙特阿拉伯1克罗地亚1荷兰1英国1中国台湾1以色列1另有 7 个国家/地区
CT.gov 报告命中 23 项，此处取回并展示最近的 15 项。
- 尚未开始招募NCT07502781Heterologous Cord Blood-Derived Red Blood Cell for Transfusion in Extremely Preterm Infants西班牙
- 招募中NCT07738874Analyzing Retinal Microanatomy in Retinopathy of Prematurity to Improve Care美国
- 尚未开始招募NCT07112430Fentanyl Intranasal for Retinopathy of Prematurity Screening in Preterm Infants加拿大
- 尚未开始招募NCT07661654HBM-Based Education for Retinopathy of Prematurity Screening in Parents of Premature Infants
- 招募中NCT07093255Safety, PK and PD of FLQ-101 in Premature Neonates美国
- 尚未开始招募NCT07427719Retinopathy of Prematurity - Visual Function and Retinal Structure挪威
- 招募中NCT07701759Incidence of Hypoxia During Intravitreal Anti-VEGF (Vascular Endothelial Growth Factor) Injections for Retinopathy of Prematurity (ROP) Under Topical Anesthesia沙特阿拉伯
- 尚未开始招募NCT07123948The Correlation Between Red Cell Transfusion and Complications of Prematurity克罗地亚
- 招募中NCT06612541Point-of-Care Ophthalmic Diagnostic Imaging of Retinopathy of Prematurity美国
- 尚未开始招募NCT06649513Long-term Ophthalmic Outcomes in Ex-premature Infants荷兰
- 招募中NCT05558059Imaging Retinal Vasculature in Infant Eyes美国
- 招募中NCT06672913Impact of Standardized Skin-to-Skin Care on Clinical Outcomes in Infants Born ≤ 32 Weeks: A Multicenter Study土耳其
- 招募中NCT06315556An Observational Study to Collect Data on How Aflibercept (Eylea) Given Using a Paediatric Dosing Device is Used in Preterm Babies With Retinopathy of Prematurity in the United Kingdom (UK)英国
- 招募中NCT06520410Safety and Efficacy of 18 mm Short Vitrectomy Probe for Pediatric Vitreoretinal Surgeries中国台湾
- 招募中NCT06694103The Effect of Two Non-Pharmacological Methods on Pain During Retinopathy Examination土耳其
外部标识与链接
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本页数据来源
- 疾病定义、同义词、基因、表型、流行病学：Orphanet（CC BY 4.0）
- 中文病名：Orphanet 中文包，冻结于 2020-06-01
- 表型中文标签：HPO 简体中文翻译（CHPO 上游成果）