Takeda A L, Colquitt J, Clegg A J, Jones J
Southampton Health Technology Assessments Centre, Wessex Institute for Health Research and Development, Mailpoint 728, Boldrewood, University of Southampton, Southampton, UK.
Br J Ophthalmol. 2007 Sep;91(9):1177-82. doi: 10.1136/bjo.2007.118562. Epub 2007 May 2.
To assess the clinical effectiveness of pegaptanib sodium and ranibizumab for neovascular age-related macular degeneration (AMD).
A systematic review of randomised controlled trials (RCTs) identified through searching 12 electronic databases, bibliographies and consultation with experts and manufacturers. RCTs were eligible if they assessed the effects of pegaptanib or ranibizumab with best supportive care, sham injection or photodynamic therapy (PDT) on patients with subfoveal choroidal neovascularisation associated with wet AMD and examined outcomes including visual acuity and adverse events.
Three RCTs of ranibizumab (MARINA, ANCHOR, FOCUS) and two of pegaptanib (VISION study) met the inclusion criteria. The RCTs included patients with different lesion types. The studies showed statistically significant benefit on different measures of visual acuity for patients receiving pegaptanib, ranibizumab or ranibizumab with PDT compared to control (sham injection, PDT or sham injection with PDT) after 12 months. These differences appeared to be clinically significant. Although adverse events were common among those receiving pegaptanib or ranibizumab, they were considered mild to moderate in nature. Meta-analysis of ranibizumab trials and indirect comparison of the two drugs were not possible due to differences in the study populations' lesion types. However, results from the RCTs of ranibizumab tended to show a greater effect on visual acuity than results from the RCT of pegaptanib.
Pegaptanib and ranibizumab appear to slow or stop the progression of neovascular AMD. Uncertainty remains over the relative benefits of pegaptanib compared with ranibizumab and other unlicensed drugs (eg, Avastin), due to the nature of the evidence. Head-to-head RCTs and economic evaluations comparing these alternatives are needed.
评估聚乙二醇化重组人血管内皮生长因子受体1拮抗剂（pegaptanib sodium）和雷珠单抗（ranibizumab）治疗新生血管性年龄相关性黄斑变性（AMD）的临床疗效。
通过检索12个电子数据库、参考文献以及咨询专家和生产商，对随机对照试验（RCT）进行系统评价。若RCT评估了聚乙二醇化重组人血管内皮生长因子受体1拮抗剂或雷珠单抗联合最佳支持治疗、假注射或光动力疗法（PDT）对伴有湿性AMD的黄斑中心凹下脉络膜新生血管患者的疗效，并检测了包括视力和不良事件在内的结局，则该RCT符合纳入标准。
三项雷珠单抗的RCT（MARINA、ANCHOR、FOCUS）和两项聚乙二醇化重组人血管内皮生长因子受体1拮抗剂的RCT（VISION研究）符合纳入标准。这些RCT纳入了不同病变类型的患者。研究表明，与对照组（假注射、PDT或PDT联合假注射）相比，接受聚乙二醇化重组人血管内皮生长因子受体1拮抗剂、雷珠单抗或雷珠单抗联合PDT治疗的患者在12个月后，不同视力测量指标上有统计学显著获益。这些差异似乎具有临床意义。尽管接受聚乙二醇化重组人血管内皮生长因子受体1拮抗剂或雷珠单抗治疗的患者中不良事件常见，但这些不良事件被认为性质为轻至中度。由于研究人群病变类型不同，无法对雷珠单抗试验进行荟萃分析以及对两种药物进行间接比较。然而，雷珠单抗RCT的结果倾向于显示其对视力的影响大于聚乙二醇化重组人血管内皮生长因子受体1拮抗剂RCT的结果。
聚乙二醇化重组人血管内皮生长因子受体1拮抗剂和雷珠单抗似乎能延缓或阻止新生血管性AMD的进展。由于证据的性质，聚乙二醇化重组人血管内皮生长因子受体1拮抗剂与雷珠单抗以及其他未获许可药物（如阿瓦斯汀）相比的相对获益仍不确定。需要进行直接比较这些药物的RCT和经济学评估。
