Real-world variability in ranibizumab treatment and associated clinical, quality of life, and safety outcomes over 24 months in patients with neovascular age-related macular degeneration: the HELIOS study
Real-world variability in ranibizumab treatment and associated clinical, quality of life, and safety outcomes over 24 months in patients with neovascular age-related macular degeneration: the HELIOS study
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DOI：
10.2147/opth.s49385
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发表时间：
2013-01-01
影响因子：
2.2
通讯作者：
Abraham, Ivo
中科院分区：
文献类型：
--
作者：
Rakic, Jean-Marie;Leys, Anita;Abraham, Ivo
本研究的目的是研究雷尼单抗在“现实世界”实践和临床环境中的治疗模式，以及评估24个月期间的生活质量结果。材料和方法：这是一项前瞻性、观察性、多中心、开放标签的研究，0.5 mg雷尼单抗通过玻璃体给药。患者随访超过24 +/- 3个月，中间数据点为6 +/- 2个月和12 +/- 2个月，有限数据点为2.5 +/- 1个月，与加载期结束相一致。结果包括视力（早期治疗糖尿病视网膜病变研究）、视觉功能（美国国家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III [HUI3]）和安全性。结果：共治疗湿性年龄相关性黄斑变性患者267例（平均+/-标准差[SD]年龄= 78.5 +/- 7.3岁，62.4%为女性，34.5%为双眼受累，74.9%为未治疗）（309只眼）。早期治疗糖尿病视网膜病变研究的平均+/- SD基线评分为56.3 +/- 14.3个字母。24个月平均注射+/- SD次数为7.6 +/- 4.1次，其中加载期和维持期分别为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。视力改善在2.5个月时达到基线水平，并在6个月时保持（均P < 0.0001）。12个月时平均视力较基线增加无统计学意义（P = 0.08）；与基线相比，24个月的下降具有统计学意义（P = 0.02）。总体而言，94.3%的患者在6个月时病情稳定或改善，81.5%的患者在24个月时病情稳定或改善。在6个月时，VFQ-25 （P = 0.03）和HUI3 （P = 0.02）较基线有显著改善，但在12个月和24个月时无显著改善。VFQ-25和HUI3的改善分别在38%和34%的患者中维持了24个月。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9例严重不良事件和9例非严重不良事件被怀疑与雷尼单抗治疗有关。结论：在不同的治疗条件下观察到，雷尼单抗的“真实世界”临床有效性可以通过视力和生活质量的初步改善以及24个月时这些结果在基线水平上的维持来证明。研究结果强调了个体化治疗和定期监测的必要性，以达到最佳的视力和生活质量结果。
Introduction: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.Materials and methods: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.Results: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-naive) 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.Conclusion: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.