Dahr Sam S, Cusick Michael, Rodriguez-Coleman Hanna, Srivastava Sunil K, Thompson Darby J, Linehan W Marston, Ferris Frederick L, Chew Emily Y
Division of Epidemiology and Clinical Research, National Eye Institute, National Institutes of Health, Bethesda, Maryland 20892, USA.
Retina. 2007 Feb;27(2):150-8. doi: 10.1097/IAE.0b013e318030a290.
This pilot study was designed to provide preliminary data concerning the safety and efficacy of pegylated anti-vascular endothelial growth factor (VEGF) therapy, pegaptanib, for patients with juxtapapillary or large peripheral angiomas secondary to von Hippel-Lindau (VHL) disease.
This study was an open label, nonrandomized, prospective, pilot study of intravitreal injections of pegaptanib (3 mg/100 microL), given every 6 weeks for minimum of 6 injections. Five patients with severe ocular VHL lesions were enrolled in the study. The primary outcome of this study was a change of > or =15 letters (3 lines) in best-corrected visual acuity by 1 year. Secondary outcomes included changes in macular thickness, as determined by optical coherence tomography, and changes in fluorescein leakage.
Two of five patients completed the course of treatment and 1 year of follow-up. These two patients had progressive decrease in retinal hard exudate and reduction in central retinal thickness measured by optical coherence tomography. One of these two patients had improvement in visual acuity of 3 lines. No significant change in fluorescein leakage or tumor size was detected in either patient. Lesions in the other three patients continued to progress despite treatment, and these patients did not complete the entire treatment course. One patient developed a tractional retinal detachment. Additional serious adverse events included transient postinjection hypotony in two eyes.
Intravitreal injections of anti-VEGF therapy (pegaptanib) may decrease retinal thickening minimally and reduce retinal hard exudates in some patients with advanced VHL angiomas. This finding may be related to a reduction in vasopermeability, because there was no apparent effect of treatment on the size of the primary retinal angiomas in this small pilot study.
本初步研究旨在提供有关聚乙二醇化抗血管内皮生长因子（VEGF）疗法（培加替尼）治疗继发于冯·希佩尔-林道（VHL）病的紧邻视乳头或周边大血管瘤患者的安全性和有效性的初步数据。
本研究为一项开放标签、非随机、前瞻性的初步研究，玻璃体内注射培加替尼（3毫克/100微升），每6周注射一次，至少注射6次。5例患有严重眼部VHL病变的患者纳入本研究。本研究的主要结局是1年后最佳矫正视力提高≥15个字母（3行）。次要结局包括光学相干断层扫描测定的黄斑厚度变化以及荧光素渗漏变化。
5例患者中有2例完成了治疗疗程及1年的随访。这2例患者视网膜硬性渗出物逐渐减少，光学相干断层扫描测量的视网膜中心厚度降低。这2例患者中有1例视力提高了3行。2例患者的荧光素渗漏或肿瘤大小均未发现显著变化。尽管进行了治疗，其他3例患者的病变仍继续进展，且未完成整个治疗疗程。1例患者发生牵拉性视网膜脱离。其他严重不良事件包括2只眼注射后出现短暂性低眼压。
玻璃体内注射抗VEGF疗法（培加替尼）可能会使部分晚期VHL血管瘤患者的视网膜增厚略有减轻，并减少视网膜硬性渗出物。这一发现可能与血管通透性降低有关，因为在这项小型初步研究中，治疗对原发性视网膜血管瘤的大小没有明显影响。
