目的:为了评估发病情况,治疗频率,弹性假性黄瘤(PXE)患者继发性脉络膜新生血管(CNV)导致的抗血管内皮生长因子(抗VEGF)治疗的视觉结果。
方法:回顾性队列研究方法:对53例PXE患者的106只眼进行分析。CNV活性的评估依赖于眼底镜检查上可见的出血和光学相干断层扫描(OCT)上的视网膜内/下液体,单独定义治疗间隔的缩短或延长。基线最佳矫正视力(BCVA),开始抗VEGF治疗的年龄,并记录了渗出开始时的BCVA下降事件(2个或更多行的BCVA恶化)。Further,我们评估了第一年的注射次数和注射总数,同伴眼睛开始治疗的时间,和BCVA随着时间的推移。
结果:在77个月的中位观察期(IQR49;126)中,患者接受了28.0次抗VEGF注射(IQR9.8;43.5)。8例患者未接受注射(基线时中位年龄38.1岁),11例患者接受抗VEGF治疗一只眼(中位年龄47.2岁),34例患者双眼(中位年龄51.8岁)。首次抗VEGF治疗的中位年龄为52.80岁(IQR47.2-57.6)。应用Cox回归模型,治疗开始前,同侧眼的中位生存时间为16.8个月.在双侧接受治疗的患者组中,中位时差为9.6个月(IQR2.1-32.4,范围0-122)在治疗的第一年,中位注射次数为每只眼5.5次(IQR3-7次),并且与观察期间的总注射次数相关(2.33,CI1.22-3.44,p<0.001).最后一次随访时更好的BCVA与更好的基线BCVA(p<0.001,R2=0.318)相关,并且在渗出开始时没有BCVA下降(p=0.035,R2=0.339)。
结论:这项研究的结果表明,大多数年龄相对较小的PXE患者需要抗VEGF治疗。一旦开始一只眼睛的治疗,同眼治疗的时间相对较短。治疗开始前BCVA下降是导致视力结果恶化的危险因素,提示在渗出影响中央视网膜之前治疗是谨慎的。鉴于年轻的发病年龄和密集的治疗需求,PXE患者可能特别受益于长效抗VEGF治疗.
OBJECTIVE: To assess the onset, treatment frequency, and visual outcome of anti-vascular endothelial growth factor (anti-VEGF) treatment due to secondary choroidal neovascularization (CNV) in patients with pseudoxanthoma elasticum (PXE).
METHODS: Retrospective cohort study METHODS: One-hundred six eyes of 53 patients with PXE were analyzed. The assessment of CNV activity relied on hemorrhage visible on funduscopy and intra- / subretinal fluid on optical coherence tomography (OCT), individually defining a shortening or extension of treatment interval. Best-corrected visual acuity (BCVA) at baseline, age at anti-VEGF therapy initiation, and BCVA-drop events at exudation onset (worsening of BCVA of 2 or more lines) were documented. Further, we assessed the number of injections during the first year and the total number of injections, the time to treatment initiation of the fellow eye, and BCVA over time.
RESULTS: During a median observation period of 77 months (IQR 49; 126) patients received a median number of 28.0 anti-VEGF-injections (IQR 9.8; 43.5). Eight patients received no injection (median age at baseline 38.1 years), 11 patients underwent anti-VEGF treatment in one eye (median age 47.2 years) and 34 patients in both eyes (median age 51.8 years). The median age at the first anti-VEGF treatment was 52.80 years (IQR 47.2-57.6). Applying Cox regression models, the median \"survival\" time of fellow eye until treatment initiation was 16.8 months. In the group of bilateral treated patients, the median time difference was 9.6 months (IQR 2.1- 32.4, range 0-122) The median number of injections was 5.5 per eye in the first year of treatment (IQR 3-7) and was associated with the total number of injections in the observation period (2.33, CI 1.22-3.44, P < .001). A better BCVA at the last follow-up visit was associated with a better baseline BCVA (P < .001, R2 = 0.318) and with the absence of a BCVA drop at the onset of exudation (P = 0.035, R2 = 0.339).
CONCLUSIONS: The results of this study indicate that anti-VEGF treatment is required for most PXE patients at a relatively young age. Once treatment in one eye is initiated, the time to fellow eye treatment is relatively short. A BCVA drop before treatment initiation is a risk factor for worse visual outcomes, suggesting that treatment is prudent before exudation affects the central retina. Given the young age of onset and intensive treatment needs, patients with PXE might particularly benefit from longer-acting anti-VEGF therapeutics.