Department of Ophthalmology, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
Department of Radiology and Nuclear Medicine, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
PLoS One. 2020 Oct 20;15(10):e0240970. doi: 10.1371/journal.pone.0240970. eCollection 2020.
To assess the effect of the bisphosphonate etidronate on choroidal neovascular (CNV) activity in patients with pseudoxanthoma elasticum (PXE).
This is an ancillary study in a single center, randomized, double-blind placebo-controlled trial (RCT) in which 74 patients with PXE were assigned to either one-year etidronate or placebo treatment. Spectral domain optical coherence tomography (SD-OCT) imaging and color fundus photography were performed every three months for one year and were systematically assessed on signs of CNV activity.
In the etidronate group, 11 (30%) of the patients had CNV activity at baseline, compared to 25 (67%) of the patients in the placebo group (P = 0.005). The proportion of eyes with CNV activity during the study ranged from 18-33% in the etidronate group and 42-56% in the placebo group and no significant difference in improvement or worsening of CNV activity was found (P = 0.168). Using a generalized mixed model for repeated measures, there was a protective effect of etidronate in crude analysis (RR 0.86, 95% CI 0.75-0.98) that disappeared when adjusting for baseline CNV activity (RR 0.97, 95% CI 0.84-1.13).
In this post-hoc RCT analysis we did not observe a protecting or deteriorating effect of etidronate on CNV activity in patients with PXE after adjustment for baseline CNV.
评估双膦酸盐依替膦酸对弹性假黄瘤（PXE）患者脉络膜新生血管（CNV）活动的影响。
这是一项单中心、随机、双盲安慰剂对照试验（RCT）的辅助研究，其中 74 例 PXE 患者被分配至依替膦酸或安慰剂治疗组，接受为期 1 年的治疗。在基线、第 3、6 和 12 个月时进行频域光学相干断层扫描（SD-OCT）成像和眼底彩色照相，并进行系统评估 CNV 活动的迹象。
在依替膦酸组中，有 11 例（30%）患者在基线时存在 CNV 活动，而安慰剂组中有 25 例（67%）患者存在 CNV 活动（P = 0.005）。在研究期间，依替膦酸组中 CNV 活动的眼比例为 18-33%，安慰剂组为 42-56%，CNV 活动的改善或恶化无显著差异（P = 0.168）。使用重复测量广义混合模型进行分析，依替膦酸在粗分析中有保护作用（RR 0.86，95%CI 0.75-0.98），但在调整基线 CNV 活动后消失（RR 0.97，95%CI 0.84-1.13）。
在这项事后 RCT 分析中，我们没有观察到依替膦酸对 PXE 患者 CNV 活动的保护或恶化作用，调整基线 CNV 活动后也是如此。
