The Spectrum of Functional, Structural, and Patient-Reported Outcomes in Intermediate Age-Related Macular Degeneration: A MACUSTAR Study Report.

IF 2.1 4区 医学 Q2 OPHTHALMOLOGY
Ophthalmologica Pub Date : 2025-02-03 DOI:10.1159/000543231
Jan Henrik Terheyden, Frank G Holz, Charlotte Behning, Hannah M P Dunbar, Steffen Schmitz-Valckenberg, Adnan Tufail, Matthias Schmid, David P Crabb, Marlene Saßmannshausen, Alison Binns, Carel B Hoyng, Nadia Zakaria, Stephen Poor, Klaus-Peter Moll, Deborah Cosette, Cecília Martinho, Joana Batuca, José Cunha-Vaz, Ulrich F O Luhmann, Sergio Leal, Robert P Finger
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引用次数: 0

Abstract

Introduction: There is an unmet medical need for therapies in intermediate age-related macular degeneration (iAMD). The prospective European multicenter cohort study MACUSTAR validates structural, functional, and patient-reported iAMD endpoints for use in future trials. The multiplicity of assessments allows characterizing iAMD in more dimensions than previously available. We describe the heterogeneity of assessments in the iAMD baseline cohort of the MACUSTAR study.

Methods: A wide range of assessments was administered across 20 European study sites in accordance with established guidelines. These assessments encompassed multiple structural evaluations, such as color fundus photography, fundus autofluorescence, and optical coherence tomography. Additionally, functional tests were conducted, including assessments of best-corrected and low-luminance visual acuity (VA), Moorfields acuity, contrast sensitivity, reading speed, mesopic and scotopic microperimetry, and dark adaptometry. Moreover, patient-reported outcome assessments, specifically the Vision Impairment in Low Luminance questionnaire, were also incorporated into the evaluation process. Associations between variables were investigated using Phi coefficients, Pearson correlation coefficients and age-corrected regression models.

Results: Five-hundred eighty-five individuals with iAMD (66% women; mean (standard deviation) age: 72 ± 7 years) were included in the MACUSTAR study. Forty-nine percent had pigmentary abnormalities, 27% had reticular pseudodrusen; 10% and 9% had incomplete and complete retinal pigment epithelium and outer retinal atrophy at baseline, respectively. Mean best-corrected VA, low-luminance VA and mesopic average threshold on microperimetry at baseline were 0.03 ± 0.11 logMAR, 0.24 ± 0.16 logMAR, and 23.3 ± 3.7 dB. Mean Vision Impairment in Low Luminance (VILL) subscale scores at baseline were 2 ± 2 to 2 ± 3 logits. Phi coefficients between structural assessments ranged between 0.17 and 0.22 (median 0.21); correlation coefficients between function tests ranged between 0.07 and 0.69 (median 0.34) and between VILL scores ranged between 0.21 and 0.68 (median 0.23).

Conclusion: The findings from this broad and comprehensive spectrum of assessments of structure, function, and patient-reported outcomes in iAMD suggest that the disease spectrum is diverse and heterogeneous and that further efforts are necessary to fully understand and characterize iAMD in all its complexities. A further in-depth characterization will enable novel enrichment strategies for clinical trials in iAMD.

中度年龄相关性黄斑变性的功能、结构和患者报告的预后谱——一项MACUSTAR研究报告。
导论:对于中度年龄相关性黄斑变性(iAMD)的治疗有一个未满足的医学需求。欧洲前瞻性多中心队列研究MACUSTAR验证了未来试验中使用的结构、功能和患者报告的iAMD终点。评估的多样性允许在比以前更多的维度上描述iAMD。我们描述了MACUSTAR研究中iAMD基线队列评估的异质性。方法:根据既定指南,在20个欧洲研究地点进行了广泛的评估。这些评估包括多种结构评估,如彩色眼底摄影、眼底自身荧光和光学相干断层扫描。此外,还进行了功能测试,包括评估最佳矫正视力和低亮度视力(VA)、摩尔菲尔德视敏度、对比灵敏度、阅读速度、中视和暗视显微镜以及黑暗适应性。此外,患者报告的结果评估,特别是低亮度视力损害问卷,也被纳入评估过程。使用Phi系数、Pearson相关系数和年龄校正回归模型调查变量之间的关联。结果:585例iAMD患者(66%为女性;平均(标准差)年龄:72±7岁)纳入MACUSTAR研究。49%为色素异常,27%为网状假性肾小球;基线时视网膜色素上皮不完整和完全,视网膜外萎缩分别为10%和9%。基线时显微术最佳校正VA、低亮度VA和介观平均阈值分别为0.03±0.11 logMAR、0.24±0.16 logMAR和23.3±3.7 dB。基线时VILL亚量表平均得分为2±2至2±3 logits。结构评估之间的Phi系数范围为0.17至0.22(中位数0.21);功能测试之间的相关系数范围为0.07至0.69(中位数0.34),VILL评分之间的相关系数范围为0.21至0.68(中位数0.23)。结论:对iAMD的结构、功能和患者报告的结果进行了广泛而全面的评估,结果表明,疾病谱系是多样的,异质性的,需要进一步的努力来充分了解和表征iAMD的所有复杂性。进一步深入的表征将为iAMD的临床试验提供新的富集策略。试验注册:ClinicalTrials.gov, NCT03349801。2017年11月22日注册。
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来源期刊
Ophthalmologica
Ophthalmologica 医学-眼科学
CiteScore
5.10
自引率
3.80%
发文量
39
审稿时长
3 months
期刊介绍: Published since 1899, ''Ophthalmologica'' has become a frequently cited guide to international work in clinical and experimental ophthalmology. It contains a selection of patient-oriented contributions covering the etiology of eye diseases, diagnostic techniques, and advances in medical and surgical treatment. Straightforward, factual reporting provides both interesting and useful reading. In addition to original papers, ''Ophthalmologica'' features regularly timely reviews in an effort to keep the reader well informed and updated. The large international circulation of this journal reflects its importance.
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