Publication

Etidronate for Prevention of Ectopic Mineralization in Patients With Pseudoxanthoma Elasticum

Kranenburg, G., de Jong, P. A., Bartsta, JW., Lagerweij, SJ., Lam, M. G. E. H., Ossewaarde-Van Norel, J., Risseeuw, S., Van Leeuwen, R., Imhof, S. M., Verhaar, H., de Vries, JJ., Slart, R., Luurtsema, G., den Harder, A. M., Visseren, F. L. J., Mali, WP. & Spiering, W., 13-Mar-2018, In : Journal of the American College of Cardiology. 71, 10, p. 1117-1126 10 p.

Research output: Contribution to journalArticleAcademicpeer-review

APA

Kranenburg, G., de Jong, P. A., Bartsta, JW., Lagerweij, SJ., Lam, M. G. E. H., Ossewaarde-Van Norel, J., Risseeuw, S., Van Leeuwen, R., Imhof, S. M., Verhaar, H., de Vries, JJ., Slart, R., Luurtsema, G., den Harder, A. M., Visseren, F. L. J., Mali, WP., & Spiering, W. (2018). Etidronate for Prevention of Ectopic Mineralization in Patients With Pseudoxanthoma Elasticum. Journal of the American College of Cardiology, 71(10), 1117-1126. https://doi.org/10.1016/j.jacc.2017.12.062

Author

Kranenburg, G. ; de Jong, P.A. ; Bartsta, JW ; Lagerweij, SJ ; Lam, Marnix G. E. H. ; Ossewaarde-Van Norel, Jeannette ; Risseeuw, S ; Van Leeuwen, R ; Imhof, S.M. ; Verhaar, H. ; de Vries, JJ ; Slart, Riemer ; Luurtsema, Geert ; den Harder, Annemarie M ; Visseren, F. L. J. ; Mali, WP ; Spiering, Wilko. / Etidronate for Prevention of Ectopic Mineralization in Patients With Pseudoxanthoma Elasticum. In: Journal of the American College of Cardiology. 2018 ; Vol. 71, No. 10. pp. 1117-1126.

Harvard

Kranenburg, G, de Jong, PA, Bartsta, JW, Lagerweij, SJ, Lam, MGEH, Ossewaarde-Van Norel, J, Risseeuw, S, Van Leeuwen, R, Imhof, SM, Verhaar, H, de Vries, JJ, Slart, R, Luurtsema, G, den Harder, AM, Visseren, FLJ, Mali, WP & Spiering, W 2018, 'Etidronate for Prevention of Ectopic Mineralization in Patients With Pseudoxanthoma Elasticum', Journal of the American College of Cardiology, vol. 71, no. 10, pp. 1117-1126. https://doi.org/10.1016/j.jacc.2017.12.062

Standard

Etidronate for Prevention of Ectopic Mineralization in Patients With Pseudoxanthoma Elasticum. / Kranenburg, G.; de Jong, P.A.; Bartsta, JW; Lagerweij, SJ; Lam, Marnix G. E. H.; Ossewaarde-Van Norel, Jeannette; Risseeuw, S; Van Leeuwen, R; Imhof, S.M.; Verhaar, H.; de Vries, JJ; Slart, Riemer; Luurtsema, Geert; den Harder, Annemarie M; Visseren, F. L. J.; Mali, WP; Spiering, Wilko.

In: Journal of the American College of Cardiology, Vol. 71, No. 10, 13.03.2018, p. 1117-1126.

Research output: Contribution to journalArticleAcademicpeer-review

Vancouver

Kranenburg G, de Jong PA, Bartsta JW, Lagerweij SJ, Lam MGEH, Ossewaarde-Van Norel J et al. Etidronate for Prevention of Ectopic Mineralization in Patients With Pseudoxanthoma Elasticum. Journal of the American College of Cardiology. 2018 Mar 13;71(10):1117-1126. https://doi.org/10.1016/j.jacc.2017.12.062


BibTeX

@article{2e282b547dd44574b196e78cef01c346,
title = "Etidronate for Prevention of Ectopic Mineralization in Patients With Pseudoxanthoma Elasticum",
abstract = "BACKGROUND In pseudoxanthoma elasticum (PXE), low pyrophosphate levels may cause ectopic mineralization, leading to skin changes, visual impairment, and peripheral arterial disease.OBJECTIVES The authors hypothesized that etidronate, a pyrophosphate analog, might reduce ectopic mineralization in PXE.METHODS In the Treatment of Ectopic Mineralization in Pseudoxanthoma Elasticum trial, adults with PXE and leg arterial calcifications (n = 74) were randomly assigned to etidronate or placebo (cyclical 20 mg/kg for 2 weeks every 12 weeks). The primary outcome was ectopic mineralization, quantified with (18)fluoride positron emission tomography scans as femoral arterial wall target-to-background ratios (TBRfemoral). Secondary outcomes were computed tomography arterial calcification and ophthalmological changes. Safety outcomes were bone density, serum calcium, and phosphate.RESULTS During 12 months of follow-up, the TBRfemoral increased 6% (interquartile range [IQR]: -12% to 25%) in the etidronate group and 7% (IQR: -9% to 32%) in the placebo group (p = 0.465). Arterial calcification decreased 4% (IQR: -11% to 7%) in the etidronate group and increased 8% (IQR: -1% to 20%) in the placebo group (p = 0.001). Etidronate treatment was associated with significantly fewer subretinal neovascularization events (1 vs. 9, p = 0.007). Bone density decreased 4% -12% in the etidronate group and 6% -9% in the placebo group (p = 0.374). Hypocalcemia (<2.20mmol/l) occurred in 3 versus 1 patient (8.1% vs. 2.7%, p = 0.304). Eighteen patients (48.6%) treated with etidronate, compared with 0 patients treated with placebo (p <0.001), experienced hyperphosphatemia (>1.5 mmol/l) and recovered spontaneously.CONCLUSIONS In patients with PXE, etidronate reduced arterial calcification and subretinal neovascularization events but did not lower femoral 18fluoride sodium positron emission tomography activity compared with placebo, without important safety issues. (Treatment of Ectopic Mineralization in Pseudoxanthoma elasticum; NTR5180) (c) 2018 by the American College of Cardiology Foundation.",
keywords = "arterial calcification, bisphosphonates, etidronate, PXE, GENERALIZED ARTERIAL CALCIFICATION, VASCULAR CALCIFICATION, CARDIOVASCULAR RISK, AORTIC CALCIFICATION, ABCC6 GENE, BISPHOSPHONATES, PYROPHOSPHATE, MUTATIONS, INFANCY, DISEASE",
author = "G. Kranenburg and {de Jong}, P.A. and JW Bartsta and SJ Lagerweij and Lam, {Marnix G. E. H.} and {Ossewaarde-Van Norel}, Jeannette and S Risseeuw and {Van Leeuwen}, R and S.M. Imhof and H. Verhaar and {de Vries}, JJ and Riemer Slart and Geert Luurtsema and {den Harder}, {Annemarie M} and Visseren, {F. L. J.} and WP Mali and Wilko Spiering",
year = "2018",
month = mar,
day = "13",
doi = "10.1016/j.jacc.2017.12.062",
language = "English",
volume = "71",
pages = "1117--1126",
journal = "Journal of the American College of Cardiology",
issn = "0735-1097",
publisher = "ELSEVIER SCIENCE INC",
number = "10",

}

RIS

TY - JOUR

T1 - Etidronate for Prevention of Ectopic Mineralization in Patients With Pseudoxanthoma Elasticum

AU - Kranenburg, G.

AU - de Jong, P.A.

AU - Bartsta, JW

AU - Lagerweij, SJ

AU - Lam, Marnix G. E. H.

AU - Ossewaarde-Van Norel, Jeannette

AU - Risseeuw, S

AU - Van Leeuwen, R

AU - Imhof, S.M.

AU - Verhaar, H.

AU - de Vries, JJ

AU - Slart, Riemer

AU - Luurtsema, Geert

AU - den Harder, Annemarie M

AU - Visseren, F. L. J.

AU - Mali, WP

AU - Spiering, Wilko

PY - 2018/3/13

Y1 - 2018/3/13

N2 - BACKGROUND In pseudoxanthoma elasticum (PXE), low pyrophosphate levels may cause ectopic mineralization, leading to skin changes, visual impairment, and peripheral arterial disease.OBJECTIVES The authors hypothesized that etidronate, a pyrophosphate analog, might reduce ectopic mineralization in PXE.METHODS In the Treatment of Ectopic Mineralization in Pseudoxanthoma Elasticum trial, adults with PXE and leg arterial calcifications (n = 74) were randomly assigned to etidronate or placebo (cyclical 20 mg/kg for 2 weeks every 12 weeks). The primary outcome was ectopic mineralization, quantified with (18)fluoride positron emission tomography scans as femoral arterial wall target-to-background ratios (TBRfemoral). Secondary outcomes were computed tomography arterial calcification and ophthalmological changes. Safety outcomes were bone density, serum calcium, and phosphate.RESULTS During 12 months of follow-up, the TBRfemoral increased 6% (interquartile range [IQR]: -12% to 25%) in the etidronate group and 7% (IQR: -9% to 32%) in the placebo group (p = 0.465). Arterial calcification decreased 4% (IQR: -11% to 7%) in the etidronate group and increased 8% (IQR: -1% to 20%) in the placebo group (p = 0.001). Etidronate treatment was associated with significantly fewer subretinal neovascularization events (1 vs. 9, p = 0.007). Bone density decreased 4% -12% in the etidronate group and 6% -9% in the placebo group (p = 0.374). Hypocalcemia (<2.20mmol/l) occurred in 3 versus 1 patient (8.1% vs. 2.7%, p = 0.304). Eighteen patients (48.6%) treated with etidronate, compared with 0 patients treated with placebo (p <0.001), experienced hyperphosphatemia (>1.5 mmol/l) and recovered spontaneously.CONCLUSIONS In patients with PXE, etidronate reduced arterial calcification and subretinal neovascularization events but did not lower femoral 18fluoride sodium positron emission tomography activity compared with placebo, without important safety issues. (Treatment of Ectopic Mineralization in Pseudoxanthoma elasticum; NTR5180) (c) 2018 by the American College of Cardiology Foundation.

AB - BACKGROUND In pseudoxanthoma elasticum (PXE), low pyrophosphate levels may cause ectopic mineralization, leading to skin changes, visual impairment, and peripheral arterial disease.OBJECTIVES The authors hypothesized that etidronate, a pyrophosphate analog, might reduce ectopic mineralization in PXE.METHODS In the Treatment of Ectopic Mineralization in Pseudoxanthoma Elasticum trial, adults with PXE and leg arterial calcifications (n = 74) were randomly assigned to etidronate or placebo (cyclical 20 mg/kg for 2 weeks every 12 weeks). The primary outcome was ectopic mineralization, quantified with (18)fluoride positron emission tomography scans as femoral arterial wall target-to-background ratios (TBRfemoral). Secondary outcomes were computed tomography arterial calcification and ophthalmological changes. Safety outcomes were bone density, serum calcium, and phosphate.RESULTS During 12 months of follow-up, the TBRfemoral increased 6% (interquartile range [IQR]: -12% to 25%) in the etidronate group and 7% (IQR: -9% to 32%) in the placebo group (p = 0.465). Arterial calcification decreased 4% (IQR: -11% to 7%) in the etidronate group and increased 8% (IQR: -1% to 20%) in the placebo group (p = 0.001). Etidronate treatment was associated with significantly fewer subretinal neovascularization events (1 vs. 9, p = 0.007). Bone density decreased 4% -12% in the etidronate group and 6% -9% in the placebo group (p = 0.374). Hypocalcemia (<2.20mmol/l) occurred in 3 versus 1 patient (8.1% vs. 2.7%, p = 0.304). Eighteen patients (48.6%) treated with etidronate, compared with 0 patients treated with placebo (p <0.001), experienced hyperphosphatemia (>1.5 mmol/l) and recovered spontaneously.CONCLUSIONS In patients with PXE, etidronate reduced arterial calcification and subretinal neovascularization events but did not lower femoral 18fluoride sodium positron emission tomography activity compared with placebo, without important safety issues. (Treatment of Ectopic Mineralization in Pseudoxanthoma elasticum; NTR5180) (c) 2018 by the American College of Cardiology Foundation.

KW - arterial calcification

KW - bisphosphonates

KW - etidronate

KW - PXE

KW - GENERALIZED ARTERIAL CALCIFICATION

KW - VASCULAR CALCIFICATION

KW - CARDIOVASCULAR RISK

KW - AORTIC CALCIFICATION

KW - ABCC6 GENE

KW - BISPHOSPHONATES

KW - PYROPHOSPHATE

KW - MUTATIONS

KW - INFANCY

KW - DISEASE

U2 - 10.1016/j.jacc.2017.12.062

DO - 10.1016/j.jacc.2017.12.062

M3 - Article

VL - 71

SP - 1117

EP - 1126

JO - Journal of the American College of Cardiology

JF - Journal of the American College of Cardiology

SN - 0735-1097

IS - 10

ER -

ID: 55382247