Effects of etidronate on the Enpp1⁻/⁻ mouse model of generalized arterial calcification of infancy

Int J Mol Med. 2015 Jul;36(1):159-65. doi: 10.3892/ijmm.2015.2212. Epub 2015 May 15.

Abstract

Generalized arterial calcification of infancy (GACI) is an autosomal recessive disorder of spontaneous infantile arterial and periarticular calcification which is attributed to mutations in the ectonucleotide pyrophosphatase/phosphodiesterase 1 (Enpp1) gene. Whilst the bisphosphonate, etidronate, is currently used off-label for the treatment for GACI, recent studies have highlighted its detrimental effects on bone mineralisation. In the present study, we used the Enpp1-/- mouse model of GACI to examine the effects of etidronate treatment (100 µg/kg), on vascular and skeletal calcification. Micro-computed tomography (µCT) analysis revealed a significant decrease in trabecular bone mass, as reflected by the decrease in trabecular bone volume/tissue volume (BV/TV; %), trabecular thickness, trabecular separation, trabecular number and pattern factor (P<0.05) in the Enpp1-/- mice in comparison to the wild-type (WT) mice. Mechanical testing revealed that in the WT mice, treatment with etidronate significantly improved work to fracture and increased work post-failure (P<0.05, in comparison to the vehicle-treated WT mice). This significant increase, however, was not observed in the Enpp1-/- mice. Treatment with etidronate had no effect on bone parameters in the WT mice; however, the Enpp1-/- mice displayed an increased structural model index (SMI; P<0.05). We used a recently developed 3D µCT protocol to reconstruct and quantify the extensive aortic calcification in Enpp1-/- mice in comparison to the WT mice. However, treatment with etidronate did not prevent de novo calcification, and did not arrest the progression of established calcification of the aorta.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Aorta / pathology
  • Bone Density / drug effects
  • Bone Density Conservation Agents / adverse effects*
  • Bone Density Conservation Agents / therapeutic use
  • Calcification, Physiologic / drug effects*
  • Disease Models, Animal
  • Etidronic Acid / adverse effects*
  • Etidronic Acid / therapeutic use
  • Genetic Predisposition to Disease
  • Male
  • Mice
  • Mice, Knockout
  • Peptide Fragments / blood
  • Phosphoric Diester Hydrolases / genetics*
  • Procollagen / blood
  • Pyrophosphatases / genetics*
  • Vascular Calcification / drug therapy*
  • Vascular Calcification / genetics
  • X-Ray Microtomography

Substances

  • Bone Density Conservation Agents
  • Peptide Fragments
  • Procollagen
  • procollagen Type I N-terminal peptide
  • Phosphoric Diester Hydrolases
  • ectonucleotide pyrophosphatase phosphodiesterase 1
  • Pyrophosphatases
  • Etidronic Acid

Supplementary concepts

  • Arterial calcification of infancy