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Long-Term Use of Intravitreal Bevacizumab (Avastin) for the Treatment of Von Hippel-Lindau Associated Retinal Hemangioblastomas

机译:长期使用玻璃体内贝伐单抗(Avastin)治疗Von Hippel-Lindau相关性视网膜血管母细胞瘤

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Retinal hemangioblastomas are the most common manifestation of Von Hippel-Lindau (VHL) disease [1-3].While peripheral retinal hemangioblastomas may be treated by thermal laser treatment or cryotherapy, optic nerve andmacular lesions are more difficult to treat [4, 5]. Based on the theoretical benefit of administering anti-VEGF treatment,intra-vitreally administered bevacizumab (Avastin, a general pan-VEGF inhibitor) is attractive [6, 7]. Several short-term case series using ranibizumab (Lucentis, mAb fragment of bevacizumab with stronger affinity forVEGF-A) have shown it has promising but minimal success on most VHL-related hemangioblastomas [8, 9]. Acomprehensive study by Wong et al. examined 5 patients over a period up to 61 weeks (47 ± 14 weeks) while Michels et al. examined one patient over a period of 4 months. Due to the short-term nature of these studies, we attempted long-termbevacizumab treatment over 60 months in a monocular subject with progressive visual loss due to a VHL associatedmacular and optic nerve hemangioblastoma. Over the treatment regimen of 15 injections, visual acuity improved 25letters, OCT thickness improved from 646 um to 424 um, and structural lesions stabilized while exudates and edemaresolved.
机译:视网膜血管母细胞瘤是Von Hippel-Lindau(VHL)病的最常见表现[1-3]。虽然外周视网膜血管母细胞瘤可以通过热激光治疗或冷冻疗法治疗,但视神经和黄斑病变更难以治疗[4,5] 。基于给予抗VEGF治疗的理论益处,玻璃体内给予贝伐单抗(Avastin,一种一般的泛VEGF抑制剂)具有吸引力[6,7]。使用兰尼单抗(Lucentis,贝伐单抗的mAb片段,对VEGF-A具有更强的亲和力)的几个短期病例系列研究表明,它在大多数与VHL相关的成血管细胞瘤上具有成功的希望,但收效甚微[8,9]。 Wong等人的综合研究。在长达61周(47±14周)的时间内检查了5例患者,而Michels等(2005年)的研究则是这样。在4个月内检查了一名患者。由于这些研究的短期性质,我们尝试在单眼受试者中进行60个月的长期贝伐单抗治疗,该受试者由于VHL相关性黄斑部和视神经血管母细胞瘤而出现进行性视力丧失。在15次注射的治疗方案中,视敏度提高了25字母,OCT厚度从646 um提高到424 um,并且结构性病变在渗出液和水肿得以解决的同时得以稳定。

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