Schultz Neil M, Bhardwaj Shweta, Barclay Claudia, Gaspar Luis, Schwartz Jason
Astellas Pharma Inc, Northbrook, Illinois.
IQVIA, London, United Kingdom.
Clin Ther. 2021 Oct;43(10):1792-1818. doi: 10.1016/j.clinthera.2021.08.011. Epub 2021 Sep 20.
Age-related macular degeneration (AMD) is a leading cause of blindness, particularly in higher-income countries. Although dry AMD accounts for 85% to 90% of AMD cases, a comprehensive understanding of the global dry AMD burden is needed.
A targeted literature review was conducted in PubMed, MEDLINE, Embase, and the Cochrane Database of Systematic Reviews (1995-2019) to identify data on the epidemiology, management, and humanistic and economic burden of dry AMD in adults. A landscape analysis of patient-reported outcome (PRO) instruments in AMD was also conducted via searches in PubMed (1995-2019), ClinicalTrials.gov, PROQOLID, PROLABELS, and health technology assessment reports (2008-2018).
Thirty-seven of 4205 identified publications were included in the review. Dry AMD prevalence was 0.44% globally, varied across ethnic groups, and increased with age. Patients with dry AMD had higher risks of all-cause mortality (hazard ratio [HR] = 1.46; 95% CI, 0.99-2.16) and tobacco-related (HR = 2.86; 95% CI, 1.15-7.09) or cancer deaths (HR = 3.37; 95% CI, 1.56-7.29; P = 0.002) than those without dry AMD. Smoking, increasing age or cholesterol levels, and obesity are key risk factors for developing dry AMD. No treatment guidelines were identified for dry AMD specifically; management focuses on risk factor reduction and use of dietary supplements. In the United States and Italy, direct medical costs and health care resource utilization were lower in patients with dry versus wet AMD. Patients with dry AMD, particularly advanced disease, experienced significant visual function impairment. Dry AMD symptoms included reduced central vision, decreased ability to see at night, increased visual blurriness, distortion of straight lines and text, and faded color vision. Most PRO instruments used in AMD evaluations covered few, if any, of the identified symptoms reported by patients with dry AMD. Although the Quality of Life and Vision Function Questionnaire, 25-item National Eye Institute Vision Function Questionnaire, Low Vision Quality of Life, Impact of Vision Impairment-Very Low Vision, and Functional Reading Independence Index had strong content validity and psychometric properties in patients with dry AMD, they retained limited coverage of salient concepts.
Despite dry AMD accounting for most AMD cases, there are substantial gaps in the published literature, particularly the humanistic and economic burden of disease and the lack of differentiation among dry, wet, or unspecified dry AMD. The significant burden of illness alludes to a high unmet need for tolerable and effective treatment options, as well as PRO instruments with more coverage of dry AMD symptoms and salient concepts.
年龄相关性黄斑变性(AMD)是导致失明的主要原因,在高收入国家尤为如此。尽管干性AMD占AMD病例的85%至90%,但仍需要对全球干性AMD负担有全面的了解。
在PubMed、MEDLINE、Embase和Cochrane系统评价数据库(1995 - 2019年)中进行有针对性的文献综述,以确定关于成人干性AMD的流行病学、管理以及人文和经济负担的数据。还通过在PubMed(1995 - 2019年)、ClinicalTrials.gov、PROQOLID、PROLABELS和卫生技术评估报告(2008 - 2018年)中检索,对AMD患者报告结局(PRO)工具进行了全景分析。
在4205篇已识别的出版物中,有37篇被纳入综述。全球干性AMD患病率为0.44%,因种族而异,并随年龄增长而增加。与没有干性AMD的患者相比,干性AMD患者全因死亡风险更高(风险比[HR]=1.46;95%置信区间,0.99 - 2.16),与烟草相关的死亡风险(HR = 2.86;95%置信区间,1.15 - 7.09)或癌症死亡风险(HR = 3.37;95%置信区间,1.56 - 7.29;P = 0.002)更高。吸烟、年龄增长、胆固醇水平升高和肥胖是发生干性AMD的关键危险因素。未发现专门针对干性AMD的治疗指南;管理重点在于降低危险因素和使用膳食补充剂。在美国和意大利,干性AMD患者的直接医疗费用和卫生保健资源利用率低于湿性AMD患者。干性AMD患者,尤其是晚期疾病患者,存在明显的视觉功能损害。干性AMD症状包括中心视力下降、夜间视力下降、视物模糊增加、直线和文字变形以及色觉减退。AMD评估中使用的大多数PRO工具几乎未涵盖干性AMD患者报告的已识别症状。尽管生活质量与视觉功能问卷、25项国家眼科研究所视觉功能问卷、低视力生活质量、视力损害影响 - 极低视力和功能性阅读独立性指数在干性AMD患者中具有较强的内容效度和心理测量特性,但它们对显著概念的覆盖范围仍然有限。
尽管干性AMD占大多数AMD病例,但已发表的文献存在重大差距,特别是疾病的人文和经济负担以及干性、湿性或未明确的干性AMD之间缺乏区分。疾病的重大负担表明对可耐受且有效的治疗选择以及能更多覆盖干性AMD症状和显著概念的PRO工具存在高度未满足的需求。