Related Experiment Video
Updated: Jun 14, 2025
A Semi-Automated and Reproducible Biological-Based Method to Quantify Calcium Deposition In Vitro
Published on: June 2, 2022
Cyclical Etidronate Reduces the Progression of Arterial Calcifications in Patients with Pseudoxanthoma Elasticum: A
Iris M Harmsen1, Tim van den Beukel2, Madeleine Kok2,3
1Department of Vascular Medicine, University Medical Center Utrecht, Utrecht University, 3508 GA Utrecht, The Netherlands.
Insights
Cyclical etidronate significantly slowed arterial calcification progression in Pseudoxanthoma elasticum (PXE) patients over nearly three years. This treatment, a pyrophosphate analog, demonstrated safety and efficacy in reducing calcification in PXE arteries.
Area of Science:
- Cardiovascular Medicine
- Genetics
- Pharmacology
Background:
- Pseudoxanthoma elasticum (PXE) is a rare genetic disorder characterized by progressive arterial calcification.
- Mutations in the ABCC6 gene reduce pyrophosphate, a natural calcification inhibitor, exacerbating PXE.
- Previous studies indicated etidronate, a pyrophosphate analog, reduces arterial calcification in PXE patients.
Purpose of the Study:
- To assess the long-term efficacy and safety of cyclical etidronate in Pseudoxanthoma elasticum (PXE) patients.
- To evaluate etidronate's impact on arterial calcification progression beyond one year.
Main Methods:
- A prospective, single-center, observational cohort study.
- Seventy-three PXE patients served as their own controls, followed for a median of 3.6 years off-treatment and 2.8 years on cyclical etidronate.
- Arterial calcification was assessed using low-dose CT scans.
Main Results:
- The median absolute yearly progression of total calcification volume was significantly lower with etidronate (388 µL) compared to without (761 µL; p < 0.001).
- The adjusted rate of relative arterial calcification progression was 5.3% with etidronate versus 11.7% without.
- No serious adverse effects were reported during etidronate treatment.
Conclusions:
- Cyclical etidronate administration significantly reduces the progression rate of arterial calcification in PXE patients.
- Long-term treatment with etidronate is effective and safe for managing arterial calcification in PXE.
- Etidronate represents a promising therapeutic option for Pseudoxanthoma elasticum, particularly for patients with pre-existing calcifications.
Abstract:
Background: Pseudoxanthoma elasticum (PXE), a rare genetic disorder presenting with slowly progressing calcification of various tissues, including the arteries, is caused by mutations in the ABCC6 gene that lead to the reduction of pyrophosphate, a natural inhibitor of calcification. We showed that, compared to a placebo, the cyclical administration of etidronate, a stable pyrophosphate analog, significantly reduced arterial calcification assessed by low-dose CT scans after one year. The aim of the present prospective, single center, observational cohort study was the assessment of the efficacy and safety of cyclical etidronate in patients treated for periods longer than one year. Methods: Seventy-three patients were followed for a median of 3.6 years without etidronate and 2.8 years with etidronate, and each patient served as their own control. Results: The median absolute yearly progression of total calcification volume during the period with etidronate (388 [83-838] µL) was significantly lower than that without etidronate (761 [362-1415] µL; p < 0.001). The rates of the relative progression of arterial calcification were 11.7% (95% CI: 9.6-13.9) without etidronate compared to 5.3% (95% CI: 3.7-7.0) with etidronate, after adjustment for confounders. Conclusions: The cyclical administration of etidronate for nearly 3 years significantly reduced the progression rate of arterial calcification in patients with PXE with pre-existing calcifications without any serious adverse effects.
Related Concept Videos
01:20Imaging Studies for Cardiovascular System III: X-Ray
Definition and Purpose
An X-ray, or radiograph, is a non-invasive method that uses ionizing radiation to take images of internal structures. It is mainly used in cardiac imaging to examine the heart, lungs, and major blood vessels, aiming to identify abnormalities in the heart's size, shape, and position, such as heart failure, congenital defects, and vascular...
01:31Osteoclasts in Bone Remodeling