神经营养性角膜病变
Neurotrophic keratopathy
定义
神经营养性角膜病是一种罕见的角膜退行性疾病，其特征是角膜敏感性降低或丧失，可无症状或表现为红眼，在疾病的早期，视力轻微下降。它最终会导致失明。
别名
神经营养性角膜炎
基本事实
- 遗传方式
- 不适用
- 发病年龄
- 成年期、儿童期
- 患病率
- 1-5 / 10 000（Europe）
临床表型 15
极常见 99–80%2
- 第V颅神经形态异常 HP:0010824
- 角膜感觉减退 HP:0012155
常见 79–30%1
- 视力缓慢下降 HP:0007924
偶见 29–5%9
- 前葡萄膜炎 HP:0012122
- 散光 HP:0000483
- 视力模糊 HP:0000622
- 角膜瘢痕 HP:0000559
- 角膜基质水肿 HP:0012040
- 角膜溃疡 HP:0012804
- 糖尿病 HP:0000819
- 泪液分泌异常 HP:0000632
- 复发性角膜糜烂 HP:0000495
罕见 <4–1%3
- 痛觉过敏 HP:0012533
- 角膜穿孔 HP:0100583
- 感觉过敏 HP:0100963
近两年的全球研究 467L2
2024/08 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。
- 2026-08Comparative Effects of Topical Maresin-1 and Cyclosporine A in a Capsaicin-Induced Rat Model of Neurotrophic Keratopathy
- 2026-08Surgical interventions in herpes simplex virus keratitis: Outcomes in a large cohort
- 2026-08综述[Research status of topical insulin therapy for neurotrophic keratopathy]
- 2026-07Making Corneal Sensation Testing Accessible: Bench Validation of a Low-Cost Adjustable Monofilament Aesthesiometer
- 2026-07病例报告Combined use of insulin eye drops and cross-linked hyaluronic acid canalicular gel for the management of severe neurotrophic keratopathy
- 2026-07综述Sensory-secretory neuroimmune circuits of the ocular surface
- 2026-07Comparison of Nerve Growth Factor with Fetal Bovine Serum for Promoting Closure of Defects in Corneal Epithelial Cell Layers
- 2026-07A novel rat model of neurotrophic keratopathy complicated with limbal stem cell deficiency induced by trigeminal ophthalmic branch injury
- 2026-07Clinical Characteristics, Management, and Visual Outcomes of Spontaneous Corneal Perforation: A Single-Center Retrospective Study in South Korea
- 2026-07Neurotrophic Keratopathy: The Need for Better Diagnostic Criteria
- 2026-07综述Autograft Versus Allograft in Indirect Corneal Neurotization: A Systematic Review
- 2026-07Proteomic Profile Analysis of Plasma and Aqueous Humor from Glaucoma and Nonglaucomatous Patients
- 2026-07Topical Insulin for Corneal Persistent Epithelial Defects: A Multicenter Retrospective Study
- 2026-07综述病例报告Combining Corneal Neurotization With Limbal Dermoid Excision: Case Report and Literature Review
- 2026-06The ocular surface consequences of lacrimal gland loss are life-stage dependent
- 2026-06综述Corneal nerve regeneration: treatments and monitoring approaches
- 2026-06Corneal Neurotisation in Neurotrophic Keratopathy: A Retrospective Analysis of Functional Outcomes and Clinical Courses
- 2026-06Neurotrophic Keratitis Associated With Telisotuzumab Vedotin: A Case Report
- 2026-06病例报告Recovery of corneal sensation following eyelid surgery for neurotrophic keratopathy and preganglionic facial palsy
- 2026-06综述Current Concepts in Corneal Neurotization
境外已获批用于本病的药物 2L2
欧盟 1 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。
已获孤儿药资格、尚未获批的在研药物（10 项）
孤儿药资格只是一种监管身份——它意味着监管机构认可这是罕见病用药并给予研发激励，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。
- (1S,4R,5R,7S)-3,4-dibenzyl-2-oxo-6,8-dioxa-3-azabyciclo[3.2.1]octane-7欧盟2014-12-16Treatment of neurotrophic keratitis官方记录
- thymosin beta 4美国2013-12-31Treatment of patients with neurotrophic keratopathy官方记录
- Pigment Epithelium Derived Factor derived short peptide (Ac-Ser-Leu-Gl美国2022-11-28Treatment of Neurotrophic Keratitis官方记录
- exosomes purified from primed human bone marrow derived mesenchymal st美国2023-07-05Treatment of neurotrophic keratitis官方记录
- allogeneic human blood serum美国2024-09-30treatment of neurotrophic keratitis官方记录
- lacripep美国2024-12-18treatment of neurotrophic keratitis官方记录
- sodium (4Z,7Z,10R,11E,13E,15Z,17S,19Z) 10,17-Dihydroxy-docosa-4,7,11,1美国2025-01-03treatment of neurotrophic keratopathy官方记录
- a small-molecule serotonin 5-HT2A/2C receptor agonist美国2026-02-10treatment of neurotrophic keratopathy官方记录
- pathogen-inactivated allogeneic human growth factor美国2026-07-14treatment of neurotrophic keratitis官方记录
- tavilermide美国2026-07-14treatment of neurotrophic keratitis (NK)官方记录
数据来自欧洲药品管理局（EMA）的药品与孤儿药资格公开导出表，以及美国 FDA 孤儿药资格数据库。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 3L2
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NCT06975748A Phase II Study of STSP-0902 Ophthalmic Solution in Patients With Neurotrophic Keratitis中国研究中心 1 个：Jinan
其他状态的试验（2 项）
- 已完成NCT07120308To Evaluate the Efficacy and Safety of ZKY001 Eye Drops in the Treatment of Corneal Epithelial Defects Caused by Neurotrophic Keratitis中国研究中心 1 个：Xiamen
- 已完成NCT06597422A Study of STSP-0902 Ophthalmic Solution in Healthy Subjects中国研究中心 1 个：Beijing
中国境外的在招试验 14L2
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10意大利3墨西哥2波兰2西班牙2阿根廷1捷克1法国1德国1匈牙利1日本1荷兰1韩国1英国1另有 1 个国家/地区
CT.gov 报告命中 14 项，此处取回并展示最近的 13 项。
- 尚未开始招募NCT07694817Clinical Predictors of Visual Outcome in Patients Affected With Ocular Surface Diseases意大利
- 招募中NCT07519902An Efficacy and Safety Study of Cenegermin Ophthalmic Solution Compared With Vehicle in the Treatment of PCED阿根廷、捷克、法国、德国、匈牙利、意大利、日本、墨西哥 等 14 国
- 招募中NCT07502378Effect of Acoltremon Ophthalmic Solution 0.003% on Signs and Symptoms of Ocular Surface Disease in Stage I Neurotrophic Keratopathy Patients With Tear Deficiency美国
- 招募中NCT07566975Real-World Assessment of VEVYE® for Short-Term Symptom and Sign Improvement of Dry Eye Disease in a Neuro-Ophthalmology Clinic: A 6-Month Prospective Observational Study美国
- 招募中NCT07568730A Multicenter, Randomized, Vehicle-Controlled, Double-Masked to Open-Label Study to Evaluate the Safety and Efficacy of Lacripep in Subjects With Neurotrophic Keratitis美国
- 尚未开始招募NCT07073729Corneal Neurotization vs. Cenergermin for Neurotrophic Keratitis: A Pilot Study加拿大
- 招募中NCT06919081Efficacy of Topical Mesenchymal Stromal Cell Secretome for Ocular Surface Disease美国
- 招募中NCT06999733A Study Comparing KB801 Verse Placebo in Patients With Stage 2 or 3 Neurotrophic Keratitis美国
- 招募中NCT06964269Use of Acthar Gel Single-Dose Pre-Filled SelfJectTM Injector in Patients With Moderate-Severe Keratitis and Autoimmune Disease美国
- 招募中NCT05927428Assessment of the Initial Efficacy and Safety of BRM424 Ophthalmic Solutions in Patients With Neurotrophic Keratitis美国
- 招募中NCT05555589Assessment of the Safety and Efficacy of 0.1% RGN-259 Ophthalmic Solution for the Treatment of NK: SEER-2意大利、波兰、西班牙、美国
- 招募中NCT04700657The Prevalence of Hypoesthesia Related Keratitis in Ocular Graft Vs. Host Disease (GVHD) Patients美国
- 招募中NCT04604834Autologous Platelet-rich Plasma (APRP) in the Treatment of Neurotrophic Keratopathy墨西哥
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）