Savastano Maria Cristina, Fossataro Claudia, Berni Alessandro, Savastano Alfonso, Cestrone Valentina, Giannuzzi Federico, Boselli Francesco, Carlà Matteo Mario, Cusato Mattia, Mottola Francesco, Pirolo Riccardo, D'Agostino Elena, Biagini Ilaria, Marcelli Sofia, Gravina Alessandro, Shen Mengxi, Rizzo Clara, Valentini Caterina Giovanna, Bianchi Maria, Teofili Luciana, Cheng Yuxuan, Wang Ruikang K, Rosenfeld Philip J, Rizzo Stanislao
Ophthalmology Unit, Fondazione Policlinico Universitario Agostino Gemelli, IRCCS, Rome, Italy.
Ophthalmology Unit, Catholic University "Sacro Cuore", Rome, Italy.
Ophthalmol Sci. 2025 Feb 4;5(4):100732. doi: 10.1016/j.xops.2025.100732. eCollection 2025 Jul-Aug.
Intravitreal injections (IVIs) of umbilical cord blood platelet-rich plasma (CB-PRP) were investigated to assess their safety and efficacy in slowing the progression of atrophy in eyes with late-stage dry age-related macular degeneration (AMD).
Randomized, controlled, prospective study.
Patients with AMD aged >65 years and diagnosed with bilateral geographic atrophy were enrolled.
One eye of each subject received the treatment of intravitreal CB-PRP 0.05 ml, while the fellow eye received a sham injection. Atrophic areas were identified as large choroidal hypertransmission defects (hyperTDs) on en face subretinal pigment epithelium slabs (64-400 μm beneath Bruch's membrane) obtained 0.321 from swept-source OCT angiography scans. The main outcome was the mean annualized growth rate of the square root transformed area measurements in both treated and nontreated eyes.
The mean ± standard deviation (SD) square root hyperTD area in the treated eyes was 3.30 ± 0.99 mm at baseline and 3.49 ± 0.98 mm after CB-PRP (IVI). In nontreated eyes, the mean square root hyperTD area was 2.96 ± 0.94 mm at baseline and 3.18 ± 0.94 mm after sham injections.
Twenty-six eyes of 13 patients were included. In treated eyes, the mean ± SD best-corrected visual acuity (BCVA) was 48.92 ± 16.33 letters at baseline and 51.46 ± 12.27 letters at last follow-up. In untreated eyes, BCVA was 67.69 ± 10.89 letters at baseline and 65.38 ± 10.34 letters at last follow-up. The mean follow-up was 258.46 ± 97.54 days. In both groups, no statistically significant difference was observed between the baseline and final BCVA. For treated eyes, the mean annualized growth rate (square root) was 0.275 mm and for nontreated eyes it was 0.321 mm. The annualized growth rate of the hyperTDs in treated eyes was 14.5% lower than that in nontreated eyes ( = 0.007). No adverse events were recorded.
These preliminary data suggest that intravitreal CB-PRP injections might be safe and effective in slowing the progression of atrophy in AMD. Extended follow-up and larger sample sizes are needed to confirm our findings and determine the optimal treatment regimen for this novel treatment option in late-stage dry AMD.
Proprietary or commercial disclosure may be found in the Footnotes and Disclosures at the end of this article.
研究玻璃体内注射脐带血富含血小板血浆（CB-PRP）在延缓晚期干性年龄相关性黄斑变性（AMD）患者眼部萎缩进展方面的安全性和有效性。
随机、对照、前瞻性研究。
纳入年龄大于65岁且诊断为双侧地图样萎缩的AMD患者。
每位受试者的一只眼睛接受0.05 ml玻璃体内CB-PRP治疗，另一只眼睛接受假注射。萎缩区域在扫频源光学相干断层扫描血管造影扫描获得的视网膜色素上皮层（位于布鲁赫膜下方64 - 400μm）的正面图像上被识别为大的脉络膜高透过性缺损（hyperTDs）。主要结局是治疗眼和未治疗眼平方根转换面积测量值的年均增长率。
治疗眼的平方根hyperTD面积在基线时平均为3.30±0.99 mm，CB-PRP（玻璃体内注射）后为3.49±0.98 mm。在未治疗眼，平方根hyperTD面积在基线时平均为2.96±0.94 mm，假注射后为3.18±0.94 mm。
纳入13例患者的26只眼。治疗眼的平均±标准差最佳矫正视力（BCVA）在基线时为48.92±16.33字母，末次随访时为51.46±12.27字母。未治疗眼的BCVA在基线时为67.69±10.89字母，末次随访时为65.38±10.34字母。平均随访时间为258.46±97.54天。两组在基线和最终BCVA之间均未观察到统计学上的显著差异。治疗眼年均增长率（平方根）为0.275 mm，未治疗眼为0.321 mm。治疗眼hyperTDs的年均增长率比未治疗眼低14.5%（P = 0.007）。未记录到不良事件。
这些初步数据表明，玻璃体内注射CB-PRP在延缓AMD萎缩进展方面可能是安全有效的。需要进行更长时间的随访和更大样本量的研究来证实我们的发现，并确定这种针对晚期干性AMD新治疗方案的最佳治疗方案。
本文末尾的脚注和披露中可能会有专有或商业披露信息。
