Rosina Chiara, Bottoni Ferdinando, Staurenghi Giovanni
Luigi Sacco Hospital, University of Milano, Italy.
Clin Ophthalmol. 2008 Sep;2(3):485-8. doi: 10.2147/opth.s3399.
Pegaptanib sodium (Macugen((R))) blocks the extracellular vascular endothelial growth factor (VEGF) isoform VEGF(165), whose elevated levels are associated with the development of choroidal neovascularization (CNV). This selective inhibition prevents binding to the VEGF receptors and the development of the increased vascular permeability and the CNV associated with neovascular age-related degeneration (AMD). The VEGF Inhibition Study In Ocular Neovascularization (VISION) demonstrated that pegaptanib sodium confers clinically meaningful benefit in the treatment of all angiographic subtypes of neovascular AMD. It also has a favorable safety profile after 1 and 2 years of continuous treatment, and recent data suggest that the agent has a disease-modifying effect. Post hoc analysis of VISION suggests that treatment benefit may be greatest in patients with early lesions, in whom 80% achieved the primary endpoint of <15 letters lost, 47% maintained visual acuity (VA), and 20% gained >/=15 letters of vision. Similarly, our own clinical experience indicates that pegaptanib sodium achieves better outcomes in early lesions than in established lesions, particularly in patients with previously untreated minimally classic and occult lesions in whom VA improvement and lesion size stabilization has been recorded. Observations indicate that pegaptanib sodium has a slower mode of action than unselective VEGF inhibitors, resulting in an average of 3-4 injections being required to stabilize VA and lesion size. Pegaptanib sodium has good efficacy and safety profiles and represents a good treatment option for patients with early CNV membranes associated with neovascular AMD.
培加他尼钠（Macugen((R))）可阻断细胞外血管内皮生长因子（VEGF）异构体VEGF(165)，其水平升高与脉络膜新生血管（CNV）的发生有关。这种选择性抑制可防止与VEGF受体结合，以及与新生血管性年龄相关性黄斑变性（AMD）相关的血管通透性增加和CNV的发展。眼部新生血管的VEGF抑制研究（VISION）表明，培加他尼钠在治疗新生血管性AMD的所有血管造影亚型方面具有临床意义的益处。在连续治疗1年和2年后，它还具有良好的安全性，最近的数据表明该药物具有疾病修饰作用。VISION的事后分析表明，治疗益处可能在早期病变患者中最大，其中80%达到了主要终点，即视力下降<15个字母，47%维持了视力（VA），20%视力提高>/=15个字母。同样，我们自己的临床经验表明，培加他尼钠在早期病变中比在已形成的病变中取得更好的效果，特别是在以前未经治疗的轻度典型和隐匿性病变患者中，已记录到VA改善和病变大小稳定。观察表明，培加他尼钠的作用方式比非选择性VEGF抑制剂慢，平均需要3-4次注射才能稳定VA和病变大小。培加他尼钠具有良好的疗效和安全性，是与新生血管性AMD相关的早期CNV膜患者的良好治疗选择。
