Rakic Jean-Marie, Leys Anita, Brié Heidi, Denhaerynck Kris, Pacheco Christy, Vancayzeele Stefaan, Hermans Christine, Macdonald Karen, Abraham Ivo
Department of Ophthalmology, Centre Hospitalier Universitaire de Liège, Site du Sart Tilman, Liège, Belgium.
Clin Ophthalmol. 2013;7:1849-58. doi: 10.2147/OPTH.S49385. Epub 2013 Sep 19.
The aim of this study was to examine ranibizumab treatment patterns in "real-world" practice and clinical settings, as well as to assess quality of life outcomes over a 24-month period.
This was a prospective, observational, multicenter, open-label study of 0.5 mg of ranibizumab administered intravitreally. Patients were followed over 24 ± 3 months with intermediate data points at 6 ± 2 months and 12 ± 2 months, and a limited data point at 2.5 ± 1 month that coincided with the end of the loading phase. Outcomes included visual acuity (Early Treatment Diabetic Retinopathy Study), visual function (National Eye Institute Visual Function Questionnaire-25 [NEI VFQ-25]), quality of life (Health Utilities Index Mark III [HUI3]), and safety.
A total of 267 patients with wet age-related macular degeneration (mean ± standard deviation [SD] age = 78.5 ± 7.3 years; 62.4% were female; 34.5% with dual eye involvement; 74.9% were treatment-naïve) were treated (309 eyes were treated). The mean ± SD Early Treatment Diabetic Retinopathy Study score at baseline was 56.3 ± 14.3 letters. The mean ± SD number of injections over 24 months was 7.6 ± 4.1, including 2.5 ± 0.7 and 5.9 ± 3.6 during the loading and maintenance phases, respectively, with corresponding treatment intervals of 4.8 ± 1.4 weeks and 11.5 ± 9.5 weeks, respectively. Improvements in visual acuity over baseline were reached at 2.5 months and maintained at 6 months (both P < 0.0001). The mean visual acuity increase over baseline at 12 months was not significant (P = 0.08); the decline over baseline at 24 months statistically significant (P = 0.02). Overall, 94.3% of patients showed stable or improved disease at 6 months and 81.5% of patients showed stable or improved disease at 24 months. At 6 months, improvements over baseline were significant for VFQ-25 (P = 0.03) and HUI3 (P = 0.02), but not at 12 months and 24 months. Improvements in VFQ-25 and HUI3 were maintained at 24 months in 38% and 34% of patients, respectively. In total 78 serious adverse events were reported in 40 patients and 77 nonserious adverse events in 34 patients. Nine serious adverse events and nine nonserious adverse events in 14 patients were suspected to be related to ranibizumab treatment.
The "real-world" clinical effectiveness of ranibizumab was evidenced by the initial improvements over baseline in visual acuity and quality of life, as well as the maintenance of these outcomes at baseline levels at 24 months, and this was observed under variable treatment conditions. The findings underscore the need for individualized treatment with regular monitoring to achieve optimal vision and quality of life outcomes.
本研究旨在探讨雷珠单抗在“真实世界”实践和临床环境中的治疗模式，并评估24个月期间的生活质量结果。
这是一项前瞻性、观察性、多中心、开放标签研究，对玻璃体内注射0.5mg雷珠单抗进行研究。患者随访24±3个月，中间数据点为6±2个月和12±2个月，以及在2.5±1个月时有一个有限的数据点，该点与负荷期结束时间一致。结果包括视力（早期治疗糖尿病性视网膜病变研究）、视觉功能（美国国立眼科研究所视觉功能问卷-25 [NEI VFQ-25]）、生活质量（健康效用指数Mark III [HUI3]）和安全性。
共有267例湿性年龄相关性黄斑变性患者（平均±标准差[SD]年龄=78.5±7.3岁；62.4%为女性；34.5%双眼受累；74.9%为初治患者）接受治疗（共治疗309只眼）。基线时早期治疗糖尿病性视网膜病变研究评分的平均±SD为56.3±14.3字母。24个月内注射次数的平均±SD为7.6±4.1次，其中负荷期和维持期分别为2.5±0.7次和5.9±3.6次，相应的治疗间隔分别为4.8±1.4周和11.5±9.5周。2.5个月时视力较基线有所改善，并在6个月时保持（均P<0.0001）。12个月时视力较基线的平均增加无统计学意义（P = 0.08）；24个月时较基线下降有统计学意义（P = 0.02）。总体而言，94.3%的患者在6个月时疾病稳定或改善，81.5%的患者在24个月时疾病稳定或改善。在6个月时，VFQ-（P = 0.03）和HUI3（P = 0.02）较基线有显著改善，但在12个月和24个月时无改善。在24个月时，分别有38%和34%的患者VFQ-25和HUI3保持改善。共有40例患者报告了78例严重不良事件，34例患者报告了77例非严重不良事件。14例患者中有9例严重不良事件和9例非严重不良事件被怀疑与雷珠单抗治疗有关。
雷珠单抗在“真实世界”中的临床有效性体现在视力和生活质量较基线的初始改善，以及在24个月时这些结果维持在基线水平，且这是在可变的治疗条件下观察到 的。研究结果强调需要进行个体化治疗并定期监测，以实现最佳视力和生活质量结果。
