Bessler Neil M
Baltimore, MD 21205, USA.
Retina. 2004 Aug;24(4):512-20. doi: 10.1097/00006982-200408000-00003.
To provide broad clinical experience and to gather safety data on photodynamic therapy with verteporfin (Visudyne, Novartis AG, Basel, Switzerland), also termed verteporfin therapy, in patients with predominantly classic subfoveal choroidal neovascularization (CNV) secondary to age-related macular degeneration (AMD). The Verteporfin in Age-related Macular Degeneration (VAM) Study was designed to provide expanded access to verteporfin therapy after beneficial results for these cases were reported but before regulatory approval in North America.
This open-label multicenter study from September 1999 through June 2000 enrolled among 222 centers patients 50 years or older in the United States, or 40 years or older in Canada, with age-related macular degeneration and subfoveal CNV with a lesion composition that was predominantly classic CNV on fluorescein angiography. Corrected visual acuity with habitual eyewear in the office setting was 20/40 to 20/200, inclusive. All patients received verteporfin therapy and returned for follow-up every 3 months. At those follow-up examinations, additional courses of treatment were recommended if any fluorescein leakage from CNV was identified. Safety information was collected from patient self-reporting, questioning (in person and by telephone), and physician evaluation. Safety was assessed by evaluating the effect of treatment on corrected distance visual acuity and by evaluating adverse events.
A total of 4,435 patients were enrolled of whom 4,051 (91%) completed the study after receiving 6,701 treatments. Most patients received only one treatment in VAM before regulatory approval of verteporfin in the United States and Canada. Three hundred patients (6.8%) experienced an adverse event considered by the treating ophthalmologist to be associated with treatment, including 115 (2.6%) with abnormal or decreased vision, of whom 25 (0.6%) experienced acute severe visual acuity decrease, and 14 (0.3%) with transient infusion-related back pain. Patients were advised to avoid exposure to direct sunlight for 24 hours; however, after verteporfin administration only 2 (0.05%) reported a photosensitivity reaction. An additional course of verteporfin therapy was administered to 1,739 of 2,314 patients (75.2%) who had a month 3 examination that was not their close-out visit and 177 of 266 (66.5%) who had a month 6 examination that was not their close-out visit.
Verteporfin therapy exhibited no additional or new safety concerns. The therapy associated with a low incidence of adverse events when expanded access was provided in a large, open-label, multicenter study, including a low incidence (0.05%) of reported photosensitivity reactions despite a short photosensitivity protection period (24 hours) following verteporfin administration.
在年龄相关性黄斑变性（AMD）继发的以典型性为主的黄斑中心凹下脉络膜新生血管（CNV）患者中，提供广泛的临床经验并收集维替泊芬（Visudyne，诺华公司，瑞士巴塞尔）光动力疗法（也称为维替泊芬疗法）的安全性数据。年龄相关性黄斑变性中的维替泊芬（VAM）研究旨在在北美监管批准之前，在这些病例取得有益结果后，提供更多使用维替泊芬疗法的机会。
这项1999年9月至2000年6月的开放标签多中心研究，在美国222个中心招募了50岁及以上的患者，在加拿大招募了40岁及以上的患者，这些患者患有年龄相关性黄斑变性和黄斑中心凹下CNV，其病变组成在荧光素血管造影上主要为典型性CNV。在诊室环境中使用习惯性眼镜矫正后的视力为20/40至20/200（含）。所有患者均接受维替泊芬治疗，并每3个月返回进行随访。在那些随访检查中，如果发现CNV有任何荧光素渗漏，则建议进行额外的治疗疗程。通过患者自我报告、询问（当面和通过电话）以及医生评估收集安全信息。通过评估治疗对矫正远视力的影响以及评估不良事件来评估安全性。
共招募了4435名患者，其中4051名（91%）在接受6701次治疗后完成了研究。在美国和加拿大维替泊芬获得监管批准之前，大多数患者在VAM研究中仅接受了一次治疗。300名患者（6.8%）发生了治疗眼科医生认为与治疗相关的不良事件，包括115名（2.6%）视力异常或下降，其中25名（0.6%）经历了急性严重视力下降，14名（0.3%）经历了短暂的输液相关背痛。建议患者在24小时内避免直接阳光照射；然而，在给予维替泊芬后，只有2名（0.05%）报告了光敏反应。在2314名进行了第3个月检查（非结束访视）的患者中，有1739名（75.2%）接受了额外的维替泊芬治疗疗程，在266名进行了第6个月检查（非结束访视）的患者中，有177名（66.5%）接受了额外的维替泊芬治疗疗程。
维替泊芬疗法未显示出额外的或新的安全问题。在一项大型、开放标签、多中心研究中提供扩大使用机会时，该疗法不良事件发生率较低，包括在维替泊芬给药后光敏保护期较短（24小时）的情况下，报告的光敏反应发生率较低（0.05%）。
