Association of Innovation and Biomedical Research in Light and Image (AIBILI), Coimbra, Portugal.
Ophthalmology Department, Centro Hospitalar e Universitário de Coimbra (CHUC), Coimbra, Portugal.
Ophthalmologica. 2022;245(1):80-90. doi: 10.1159/000518235. Epub 2021 Jul 13.
Polypoidal choroidal vasculopathy (PCV) is far less common and studied in a Caucasian population than in an Asian population, and the optimal treatment approach remains to be confirmed.
A 52-week, double-masked, sham-controlled, phase 4, investigator-initiated randomized clinical trial (RCT) in naive symptomatic Caucasian patients with PCV treated with aflibercept in a treat-and-extend regimen (T&E) (intravitreal aflibercept injection [IVAI] T&E). Patients were randomized at week 16 to receive IVAI T&E plus either sham photodynamic therapy (PDT) or standard fluence PDT with verteporfin. The main outcome measures were changes in best-corrected visual acuity (BCVA) from baseline to 52 weeks and polyp occlusion at week 52. Data are presented as median (interquartile range [IQR]) for BCVA, number of IVAI, and change in central retinal thickness (CRT).
Of the 50 patients included in the study, 48 patients completed the 52 weeks of follow-up. During this period, a significant median (IQR) BCVA gain of 6 [2-12] Early Treatment Diabetic Retinopathy Study letters was observed for all patients (p < 0.001), after 8 (7-9) injections, with a significant reduction of -93.0 [-154.0, -44.0] µm in central macular thickness (p < 0.001). Using indocyanine green angiography, a complete occlusion of polypoidal lesions was documented in 72% of the cases. Still, no significant difference was detected between the sham PDT and the aflibercept PDT arms, at week 52, for BCVA change (6.5 [2-11] vs. 5 [2-13] letters (p = 0.98)), number of IVAIs (8.5 [7-9] vs. 8 [7-9] (p = 0.21)), change in CRT (-143 [-184; -47] vs. -89 [-123; -41.5] µm [p = 0.23]), and rates of complete polyp occlusion: 77 versus 68% (p = 0.53) or presence of fluid: 68 versus 57% (p = 0.56). No serious ocular adverse events were registered in the 2 arms.
To our knowledge, this is the first RCT to compare aflibercept T&E monotherapy with aflibercept T&E plus verteporfin PDT in a Caucasian population with PCV. Aflibercept monotherapy in a T&E showed to be effective and safe with a significant median BCVA improvement of 6 letters and a complete occlusion of polypoidal lesions in near 3 quarters of the eyes, at 1 year. As only 22% of the eyes underwent PDT treatment, the benefit of combined treatment for PCV in Caucasian patients could not be definitively elucidated from this study.
The clinical trial was registered in ClinicalTrials.gov Identifier NCT02495181 and the European Union Drug Regulating Authorities Clinical Trials Database EudraCT No. 2015-001368-20.
与亚洲人群相比， 白人人群中脉络膜息肉样血管病变（PCV）的发病率较低，研究也较少，最佳治疗方法仍有待证实。
这是一项为期 52 周的、双盲、假对照、四期、由研究者发起的随机临床试验（RCT），纳入了未经治疗的有症状的白人 PCV 患者，这些患者接受阿柏西普治疗，并采用治疗和扩展方案（T&E）（玻璃体内注射阿柏西普 T&E）。患者在第 16 周随机分为接受玻璃体内注射阿柏西普 T&E 加假光动力疗法（PDT）或标准维替泊芬荧光素 PDT。主要观察指标是从基线到 52 周时最佳矫正视力（BCVA）的变化和第 52 周时息肉闭塞情况。数据以 BCVA、玻璃体内注射次数和中央视网膜厚度（CRT）变化的中位数（四分位距[IQR]）表示。
在纳入研究的 50 例患者中，48 例患者完成了 52 周的随访。在此期间，所有患者的 BCVA 均有显著的中位数（IQR）改善，为 6 [2-12]个早期糖尿病视网膜病变研究字母（p < 0.001），经过 8（7-9）次注射后，中央黄斑厚度显著减少-93.0[-154.0，-44.0]µm（p < 0.001）。使用吲哚菁绿血管造影术，72%的病例记录到息肉样病变完全闭塞。然而，在第 52 周时，在 BCVA 变化（6.5[2-11]与 5[2-13]个字母（p = 0.98））、玻璃体内注射次数（8.5[7-9]与 8[7-9]（p = 0.21））、CRT 变化（-143[-184；-47]与-89[-123；-41.5]µm（p = 0.23））以及完全息肉闭塞率方面， sham PDT 组和阿柏西普 PDT 组之间没有发现显著差异：77%与 68%（p = 0.53）或存在液体：68%与 57%（p = 0.56）。在这 2 个组中都没有发现严重的眼部不良事件。
据我们所知，这是第一项在 PCV 白人人群中比较阿柏西普 T&E 单药治疗与阿柏西普 T&E 联合维替泊芬 PDT 的 RCT。阿柏西普 T&E 单药治疗在白人人群中显示出有效性和安全性，52 周时 BCVA 中位数改善 6 个字母，近四分之三的眼睛息肉样病变完全闭塞。由于只有 22%的眼睛接受 PDT 治疗，因此无法从这项研究中明确确定 PCV 白人患者联合治疗的益处。
该临床试验在 ClinicalTrials.gov 标识符 NCT02495181 和欧洲药品管理局临床试验数据库 EudraCT 注册，编号为 2015-001368-20。
