London School of Hygiene and Tropical Medicine, London, UK.
Health Technol Assess. 2012;16(6):i-xii, 1-200. doi: 10.3310/hta16060.
The verteporfin photodynamic therapy (VPDT) cohort study aimed to answer five questions: (a) is VPDT in the NHS provided as in randomised trials?; (b) is 'outcome' the same in the nhs as in randomised trials?; (c) is 'outcome' the same for patients ineligible for randomised trials?; (d) is VPDT safe when provided in the NHS?; and (e) how effective and cost-effective is VPDT?
Treatment register.
All hospitals providing VPDT in the NHS.
All patients attending VPDT clinics.
Infusion of verteporfin followed by infrared laser exposure is called VPDT, and is used to treat neovascular age-related macular degeneration (nAMD). The VPDT cohort study advised clinicians to follow patients every 3 months during treatment or active observation, retreating based on criteria used in the previous commercial 'TAP' (Treatment of Age-related macular degeneration with Photodynamic therapy) trials of VPDT.
The primary outcome was logarithm of the minimum angle of resolution monocular best-corrected distance visual acuity (BCVA). Secondary outcomes were adverse reactions and events; morphological changes in treated nAMD (wet) lesions; and for a subset of patients, 6-monthly contrast sensitivity, generic and visual health-related quality of life (HRQoL) and resource use. Treated eyes were classified as eligible for the TAP trials (EFT), ineligible (IFT) or unclassifiable (UNC).
Forty-seven hospitals submitted data for 8323 treated eyes in 7748 patients; 4919 eyes in 4566 patients were treated more than 1 year before the last data submission or had completed treatment. Of 4043 eyes with nAMD in 4043 patients, 1227 were classified as EFT, 1187 as IFT and 1629 as UNC. HRQoL and resource use data were available for about 2000 patients. The mean number of treatments in years 1 and 2 was 2.3 and 0.4 respectively. About 50% of eyes completed treatment within 1 year. BCVA deterioration in year 1 did not differ between eligibility groups. EFT eyes lost 11.6 letters (95% confidence interval 10.1 to 13.0 letters) compared with 9.9 letters in VPDT-treated eyes in the TAP trials. EFT eyes had poorer BCVA at baseline than IFT and UNC eyes. Adverse reactions and events were reported for 1.4% of first visits - less frequently than those reported in the TAP trials. Associations between BCVA in the best-seeing eye with HRQoL and community health and social care resource use showed that the 11-letter difference in BCVA between VPDT and sham treatment in the TAP trials corresponded to differences in utility of 0.012 and health and social service costs of £60 and £92 in years 1 and 2, respectively. VPDT provided an incremental cost per quality-adjusted life-year (QALY) of £170,000 over 2 years.
VPDT was administered less frequently than in the TAP trials, with less than half of those treated followed up for > 1 year in routine clinical practice. Deterioration in BCVA over time in EFT eyes was similar to that in the TAP trials. The similar falls in BCVA after VPDT across the pre-defined TAP eligibility groups do not mean that the treatment is equally effective in these groups because deterioration in BCVA can be influenced by the parameters that determined group membership. Safety was no worse than in the TAP trials. The estimated cost per QALY was similar to the highest previous estimate. Although VPDT is no longer in use as monotherapy for neovascular AMD, its role as adjunctive treatment has not been fully explored. VPDT also has potential as monotherapy in the management of vascular malformations of the retina and choroid and with trials underway in neovascularisation due to myopia and polypoidal choroidopathy.
The National Institute for Health Research Health Technology Assessment programme.
光动力疗法（VPDT）队列研究旨在回答五个问题：（a）NHS 中提供的维替泊芬光动力疗法是否与随机试验相同？；（b）NHS 中的“结果”是否与随机试验相同？；（c）不符合随机试验条件的患者的“结果”是否相同？；（d）在 NHS 中提供光动力疗法是否安全？；（e）光动力疗法的有效性和成本效益如何？
治疗登记处。
在 NHS 中提供光动力疗法的所有医院。
所有接受光动力疗法诊所治疗的患者。
静脉注射维替泊芬，然后进行红外激光照射，称为光动力疗法，用于治疗新生血管性年龄相关性黄斑变性（nAMD）。VPDT 队列研究建议临床医生在治疗或主动观察期间每 3 个月对患者进行随访，根据以前的商业“TAP”（光动力疗法治疗年龄相关性黄斑变性）试验中使用的标准对患者进行重新治疗。
主要结局是最小角分辨率单眼最佳矫正距离视力（BCVA）的对数。次要结局是不良反应和事件；治疗 nAMD（湿性）病变的形态变化；以及对于一部分患者，6 个月的对比敏感度、通用和视觉健康相关生活质量（HRQoL）和资源使用。治疗眼被分为有资格参加 TAP 试验（EFT）、无资格（IFT）或无法分类（UNC）。
47 家医院提交了 7748 名患者的 8323 只治疗眼的数据；4566 名患者中有 4919 只眼的治疗时间超过最后一次数据提交前 1 年，或已完成治疗。在 4043 名患有 nAMD 的患者中，有 1227 只眼被归类为 EFT，1187 只眼为 IFT，1629 只眼为 UNC。大约有 2000 名患者提供了 HRQoL 和资源使用数据。第 1 年和第 2 年的平均治疗次数分别为 2.3 和 0.4。大约 50%的眼睛在一年内完成了治疗。第 1 年的 BCVA 恶化在各资格组之间没有差异。EFT 眼在 TAP 试验中与光动力疗法治疗的眼相比，损失了 11.6 个字母（95%置信区间为 10.1 至 13.0 个字母）。EFT 眼在基线时的 BCVA 比 IFT 和 UNC 眼差。首次就诊时报告了 1.4%的不良反应和事件，比 TAP 试验中报告的频率要低。最佳视力眼的 BCVA 与 HRQoL 和社区卫生及社会保健资源使用之间的相关性表明，TAP 试验中光动力疗法与假治疗之间 11 个字母的 BCVA 差异，分别对应于第 1 年和第 2 年效用的差异为 0.012 和健康和社会服务成本的差异为 60 英镑和 92 英镑。光动力疗法在 2 年内每增加一个质量调整生命年（QALY）的成本为 170000 英镑。
与 TAP 试验相比，光动力疗法的治疗频率较低，在常规临床实践中，不到一半的治疗患者随访时间超过 1 年。EFT 眼随时间推移 BCVA 的恶化与 TAP 试验相似。根据预先确定的 TAP 资格组，EFT 眼的 BCVA 下降相似，并不意味着治疗在这些组中同样有效，因为 BCVA 的下降可能受到确定组内成员的参数的影响。安全性并不比 TAP 试验差。估计的成本效益比是以前最高的估计值。尽管光动力疗法不再作为新生血管性 AMD 的单一疗法使用，但它作为辅助治疗的作用尚未得到充分探索。光动力疗法在治疗视网膜和脉络膜血管畸形以及由于近视和息肉样脉络膜血管病变引起的新生血管化方面也具有潜力，并且正在进行试验。
英国国家卫生研究院卫生技术评估计划。
