Department of Ophthalmology of Créteil, University Paris XII, Créteil, France.
Eye (Lond). 2009 May;23(5):1150-4. doi: 10.1038/eye.2008.194. Epub 2008 Jul 18.
To assess the effects of pegaptanib in the treatment of subfoveal occult choroidal neovascularisation (CNV) associated with neovascular age-related macular degeneration (NV-AMD) in a compassionate use program in France.
Pegaptanib was authorized for patients with CNV-associated visual impairment and in whom usual care (thermal laser photocoagulation or photodynamic therapy with verteporfin) was not appropriate. Patients with occult CNV lesions received intravitreous pegaptanib (0.3 mg every 6 weeks) and were followed with repeated fluorescein angiography, scanning laser ophthalmoscopy-infracyanine green angiography, and ocular coherence tomography through 52 weeks.
Of 56 patients (predominantly occult, N=22; purely occult, N=8; occult with chorioretinal anastomosis, N=12; occult with pigment epithelial detachment, N=14), 30% had earlier treatment. All received eight pegaptanib injections. At week 52, 79% were responders (lost <15 letters of visual acuity), 43% gained >or=0 letters, and 9% gained >or=15 letters. The best functional results were obtained in the predominantly and pure occult subgroups (responders, 86 and 75%; gained >or=0 letters, 50 and 50%). Maximum visual outcomes that correlated with morphologic improvements on each diagnostic imaging tool were seen after at least three injections. No significant ocular or systemic adverse events occurred.
Treatment with pegaptanib was associated with objective functional improvements that can be correlated with objective clinical improvements on routine diagnostic imaging tools in patients with occult NV-AMD. Optimum treatment results appear after at least 4 months of therapy in the majority of cases.
评估培加他滨在法国同情用药计划中治疗与新生血管性年龄相关性黄斑变性（NV-AMD）相关的中心凹下隐匿性脉络膜新生血管（CNV）的疗效。
培加他滨被授权用于伴有 CNV 相关视力损害且常规治疗（热激光光凝或维替泊芬光动力疗法）不适当的患者。隐匿性 CNV 病变患者接受玻璃体内培加他滨（每 6 周 0.3mg）治疗，并通过重复荧光素血管造影、扫描激光检眼镜-亚甲蓝血管造影和眼部相干断层扫描进行随访，随访时间达 52 周。
56 例患者（主要为隐匿性，N=22；单纯隐匿性，N=8；隐匿性伴脉络膜视网膜吻合，N=12；隐匿性伴色素上皮脱离，N=14）中，30%的患者接受了更早的治疗。所有患者均接受了 8 次培加他滨注射。在第 52 周时，79%的患者为应答者（视力丧失<15 个字母），43%的患者增加了>或=0 个字母，9%的患者增加了>或=15 个字母。在主要和单纯隐匿性亚组中获得了最佳的功能结果（应答者分别为 86%和 75%；增加了>或=0 个字母分别为 50%和 50%）。在至少接受 3 次注射后，观察到与每种诊断成像工具的形态改善相关的最佳视力结果。未发生显著的眼部或全身不良事件。
在隐匿性 NV-AMD 患者中，培加他滨治疗与客观的功能改善相关，这些改善可与常规诊断成像工具上的客观临床改善相关。在大多数情况下，在至少 4 个月的治疗后，可获得最佳的治疗效果。
