依替膦酸二钠可阻止假性黄色瘤弹性组织病的系统性动脉钙化。

Etidronate halts systemic arterial calcification in pseudoxanthoma elasticum.

机构信息

Department of Radiology, University Medical Center Utrecht, Utrecht University, the Netherlands.

Department of Vascular Medicine, University Medical Center Utrecht, Utrecht University, the Netherlands.

出版信息

Atherosclerosis. 2020 Jan;292:37-41. doi: 10.1016/j.atherosclerosis.2019.10.004. Epub 2019 Oct 11.

DOI:10.1016/j.atherosclerosis.2019.10.004

PMID:31756632

Abstract

BACKGROUND AND AIMS

In pseudoxanthoma elasticum (PXE), low levels of inorganic pyrophosphate result in extensive arterial calcification. Recently, the treatment of ectopic mineralization in the PXE (TEMP) trial showed that one year of treatment with etidronate halts progression of femoral artery calcification in PXE patients. The aim of this study was to test the efficacy of etidronate on calcification in different vascular beds.

METHODS

In this prespecified post-hoc analysis of the TEMP trial, arterial calcification mass was quantified in the carotid siphon, common carotid artery, thoracic and abdominal aorta, coronary arteries, iliac arteries, and the femoropopliteal and crural arteries using CT at baseline and after one year of etidronate treatment or placebo. In addition, a total arterial calcification score was calculated. The difference in calcification progression was compared between the etidronate and placebo group.

RESULTS

74 PXE patients were enrolled and randomized. Etidronate significantly halted progression of calcification in all vascular beds except for the coronary arteries. For the total arterial calcification score, the median absolute increase in mass score was -63.6 (-438.4-42.2) vs. 113.7 (9.4-377.1) (p < 0.01) and the median relative increase was -2.4% (-10.3-3.8) vs. 6.3% (0.2-15.8) (p < 0.01) in the etidronate and placebo arm, respectively.

CONCLUSIONS

Etidronate treatment halts systemic arterial calcification in PXE. Further research must assess the long term safety and efficacy of etidronate on clinical outcomes in PXE.

摘要

背景与目的

在弹性假黄瘤（PXE）中，无机焦磷酸盐水平降低导致广泛的动脉钙化。最近，PXE 异位矿化治疗（TEMP）试验的治疗表明，依替膦酸治疗一年可阻止 PXE 患者股动脉钙化进展。本研究旨在测试依替膦酸对不同血管床钙化的疗效。

方法

在 TEMP 试验的这项预设事后分析中，使用 CT 在基线时和依替膦酸治疗或安慰剂治疗一年后对颈动脉虹吸、颈总动脉、胸主动脉和腹主动脉、冠状动脉、髂动脉以及股腘和小腿动脉的动脉钙化质量进行定量。此外，还计算了总的动脉钙化评分。比较依替膦酸和安慰剂组之间钙化进展的差异。

结果

74 例 PXE 患者入组并随机分组。依替膦酸显著阻止了所有血管床（除冠状动脉外）的钙化进展。对于总的动脉钙化评分，质量评分的中位数绝对增加量为-63.6（-438.4-42.2）与 113.7（9.4-377.1）（p<0.01），中位数相对增加量为-2.4%（-10.3-3.8）与 6.3%（0.2-15.8）（p<0.01），分别在依替膦酸和安慰剂组。