Sandra Liakopoulos, Leon von der Emde, Marvin L Biller, Thomas Ach, Frank G Holz
{"title":"Geographic Atrophy in Age-Related Macular Degeneration.","authors":"Sandra Liakopoulos, Leon von der Emde, Marvin L Biller, Thomas Ach, Frank G Holz","doi":"10.3238/arztebl.m2025.0003","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Age-related macular degeneration (AMD), a condition of multifactorial origin, is a major cause of irreversible vision loss in industrialized countries. The dry late stage of the disease, known as geographic atrophy (GA), is characterized by progressive loss of photoreceptor cells and retinal pigment epithelial cells in the central retina. An estimated 300 000 to 550 000 people in Germany suffer from GA.</p><p><strong>Methods: </strong>This review is based on pertinent literature retrieved by a selective search in the PubMed and Web of Science databases.</p><p><strong>Results: </strong>In 2023, the complement inhibitors pegcetacoplan and avacincaptad pegol were approved in the USA for repeated intravitreal injections and thereby became the first drugs ever approved for the treatment of GA. In Europe, the marketing authorization application for both drugs was withdrawn by the manufacturers after a negative judgment was expressed by the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP). The EMA stated that the significant slowing of atrophy progression that had been achieved in the approval trials did not lead to any clinically relevant functional benefit for the patients. Further treatment approaches, including gene therapy, are now being studied in clinical trials. There is evidence that micronutrients may slow the progression of atrophy.</p><p><strong>Conclusion: </strong>In Europe at present, there is no approved treatment for GA due to AMD. There is thus a continuing need for preventive and rehabilitative measures such as smoking cessation, a balanced diet, and magnifying visual aids for patients in the advanced stages of the disease.</p>","PeriodicalId":11258,"journal":{"name":"Deutsches Arzteblatt international","volume":" Forthcoming","pages":"82-88"},"PeriodicalIF":6.5000,"publicationDate":"2025-02-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Deutsches Arzteblatt international","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.3238/arztebl.m2025.0003","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"MEDICINE, GENERAL & INTERNAL","Score":null,"Total":0}
引用次数: 0
Abstract
Background: Age-related macular degeneration (AMD), a condition of multifactorial origin, is a major cause of irreversible vision loss in industrialized countries. The dry late stage of the disease, known as geographic atrophy (GA), is characterized by progressive loss of photoreceptor cells and retinal pigment epithelial cells in the central retina. An estimated 300 000 to 550 000 people in Germany suffer from GA.
Methods: This review is based on pertinent literature retrieved by a selective search in the PubMed and Web of Science databases.
Results: In 2023, the complement inhibitors pegcetacoplan and avacincaptad pegol were approved in the USA for repeated intravitreal injections and thereby became the first drugs ever approved for the treatment of GA. In Europe, the marketing authorization application for both drugs was withdrawn by the manufacturers after a negative judgment was expressed by the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP). The EMA stated that the significant slowing of atrophy progression that had been achieved in the approval trials did not lead to any clinically relevant functional benefit for the patients. Further treatment approaches, including gene therapy, are now being studied in clinical trials. There is evidence that micronutrients may slow the progression of atrophy.
Conclusion: In Europe at present, there is no approved treatment for GA due to AMD. There is thus a continuing need for preventive and rehabilitative measures such as smoking cessation, a balanced diet, and magnifying visual aids for patients in the advanced stages of the disease.
背景:年龄相关性黄斑变性(AMD)是一种多因素的疾病,是工业化国家不可逆视力丧失的主要原因。该疾病的干燥晚期,称为地理萎缩(GA),其特征是视网膜中央的光感受器细胞和视网膜色素上皮细胞的逐渐丧失。据估计,德国有30万至55万人患有GA。方法:本综述基于在PubMed和Web of Science数据库中选择性检索到的相关文献。结果:2023年,补体抑制剂pegcetacoplan和avacincaptad pegol在美国被批准用于反复玻璃体内注射,从而成为有史以来第一个被批准用于治疗GA的药物。在欧洲,这两种药物的上市许可申请在欧洲药品管理局(EMA)人用药品委员会做出负面判断后被制造商撤回。EMA表示,在批准试验中实现的显著减缓萎缩进展并未给患者带来任何临床相关的功能益处。包括基因疗法在内的进一步治疗方法目前正在临床试验中进行研究。有证据表明微量营养素可以减缓萎缩的进展。结论:在欧洲,目前还没有批准的治疗由AMD引起的GA的方法。因此,继续需要采取预防和康复措施,如戒烟、均衡饮食和为疾病晚期患者提供放大镜辅助器具。
期刊介绍:
Deutsches Ärzteblatt International is a bilingual (German and English) weekly online journal that focuses on clinical medicine and public health. It serves as the official publication for both the German Medical Association and the National Association of Statutory Health Insurance Physicians. The journal is dedicated to publishing independent, peer-reviewed articles that cover a wide range of clinical medicine disciplines. It also features editorials and a dedicated section for scientific discussion, known as correspondence.
The journal aims to provide valuable medical information to its international readership and offers insights into the German medical landscape. Since its launch in January 2008, Deutsches Ärzteblatt International has been recognized and included in several prestigious databases, which helps to ensure its content is accessible and credible to the global medical community. These databases include:
Carelit
CINAHL (Cumulative Index to Nursing and Allied Health Literature)
Compendex
DOAJ (Directory of Open Access Journals)
EMBASE (Excerpta Medica database)
EMNursing
GEOBASE (Geoscience & Environmental Data)
HINARI (Health InterNetwork Access to Research Initiative)
Index Copernicus
Medline (MEDLARS Online)
Medpilot
PsycINFO (Psychological Information Database)
Science Citation Index Expanded
Scopus
By being indexed in these databases, Deutsches Ärzteblatt International's articles are made available to researchers, clinicians, and healthcare professionals worldwide, contributing to the global exchange of medical knowledge and research.