Tags

Type your tag names separated by a space and hit enter

Long-term changes in arterial structure and function and left ventricular geometry after enzyme replacement therapy in patients affected with Fabry disease.
Eur J Prev Cardiol. 2012 Feb; 19(1):43-54.EJ

Abstract

AIMS

Fabry disease is a lysosomal storage disorder due to deficient alpha-galactosidase A activity, characterised by glycosphingolipids deposition in tissues. Patients have a common arterial involvement and contract progressive renal and cardiac disease. Although short-term effects of enzyme replacement therapy (ERT) on target organs have been established, no data are available on the long-term outcome.

METHODS AND RESULTS

We studied the effects of ERT (agalsidase beta, 1 mg/kg/14 days) on arterial and cardiac structure and function during a longitudinal study beginning in 1999, with 4.5 ± 0.4 years follow-up (four visits) in 30 patients (age: 33 ± 12 years). In addition, we studied 16 untreated Fabry patients during 2.6 ± 1.6 years (two visits). Aortic stiffness was determined by carotid-femoral pulse wave velocity, central pulse pressure by aplanation tonometry, and carotid and radial intima-media thickness and diameter by high definition echotracking device. Left ventricular mass was determined by MRI. A significant decrease in aortic stiffness (-0.56 ± 0.13 m/s/yr, p = 0.0002) was observed after ERT whereas central pulse pressure did not change. Carotid intima-media thickness (IMT) increased (+18 ± 6 µm/yr; p < 0.005) whereas radial IMT remained stable. Radial artery diameter decreased (-50 ± 20 µm/years, p < 0.05) whereas carotid diameter did not change. Carotid circumferential wall stress was reduced (-1.7 ± 0.6 kPa/yrs, p < 0.01). Left ventricular mass index significantly decreased (-7.8 ± 2.3 g/m(2)/yr, p < 0.005).

CONCLUSION

A sustained reduction in aortic stiffness and left ventricular hypertrophy, and a limited radial artery wall thickening were observed after long-term enzyme replacement therapy. There was no significant benefit of treatment on carotid hypertrophy.

Authors+Show Affiliations

University of Versailles-Saint Quentin en Yvelines, Division of Medical Genetics, Hôpital Raymond Poincaré (AP-HP), Garches, France.No affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Journal Article
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

21450622

Citation

Collin, Cédric, et al. "Long-term Changes in Arterial Structure and Function and Left Ventricular Geometry After Enzyme Replacement Therapy in Patients Affected With Fabry Disease." European Journal of Preventive Cardiology, vol. 19, no. 1, 2012, pp. 43-54.
Collin C, Briet M, Tran TC, et al. Long-term changes in arterial structure and function and left ventricular geometry after enzyme replacement therapy in patients affected with Fabry disease. Eur J Prev Cardiol. 2012;19(1):43-54.
Collin, C., Briet, M., Tran, T. C., Beaussier, H., Benistan, K., Bensalah, M., Mousseaux, E., Froissart, M., Bozec, E., Laurent, S., Boutouyrie, P., & Germain, D. P. (2012). Long-term changes in arterial structure and function and left ventricular geometry after enzyme replacement therapy in patients affected with Fabry disease. European Journal of Preventive Cardiology, 19(1), 43-54. https://doi.org/10.1177/1741826710391118
Collin C, et al. Long-term Changes in Arterial Structure and Function and Left Ventricular Geometry After Enzyme Replacement Therapy in Patients Affected With Fabry Disease. Eur J Prev Cardiol. 2012;19(1):43-54. PubMed PMID: 21450622.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Long-term changes in arterial structure and function and left ventricular geometry after enzyme replacement therapy in patients affected with Fabry disease. AU - Collin,Cédric, AU - Briet,Marie, AU - Tran,Thi-Chien, AU - Beaussier,Hélène, AU - Benistan,Karelle, AU - Bensalah,Mourad, AU - Mousseaux,Elie, AU - Froissart,Marc, AU - Bozec,Erwan, AU - Laurent,Stéphane, AU - Boutouyrie,Pierre, AU - Germain,Dominique P, Y1 - 2011/03/04/ PY - 2011/4/1/entrez PY - 2011/4/1/pubmed PY - 2012/6/13/medline SP - 43 EP - 54 JF - European journal of preventive cardiology JO - Eur J Prev Cardiol VL - 19 IS - 1 N2 - AIMS: Fabry disease is a lysosomal storage disorder due to deficient alpha-galactosidase A activity, characterised by glycosphingolipids deposition in tissues. Patients have a common arterial involvement and contract progressive renal and cardiac disease. Although short-term effects of enzyme replacement therapy (ERT) on target organs have been established, no data are available on the long-term outcome. METHODS AND RESULTS: We studied the effects of ERT (agalsidase beta, 1 mg/kg/14 days) on arterial and cardiac structure and function during a longitudinal study beginning in 1999, with 4.5 ± 0.4 years follow-up (four visits) in 30 patients (age: 33 ± 12 years). In addition, we studied 16 untreated Fabry patients during 2.6 ± 1.6 years (two visits). Aortic stiffness was determined by carotid-femoral pulse wave velocity, central pulse pressure by aplanation tonometry, and carotid and radial intima-media thickness and diameter by high definition echotracking device. Left ventricular mass was determined by MRI. A significant decrease in aortic stiffness (-0.56 ± 0.13 m/s/yr, p = 0.0002) was observed after ERT whereas central pulse pressure did not change. Carotid intima-media thickness (IMT) increased (+18 ± 6 µm/yr; p < 0.005) whereas radial IMT remained stable. Radial artery diameter decreased (-50 ± 20 µm/years, p < 0.05) whereas carotid diameter did not change. Carotid circumferential wall stress was reduced (-1.7 ± 0.6 kPa/yrs, p < 0.01). Left ventricular mass index significantly decreased (-7.8 ± 2.3 g/m(2)/yr, p < 0.005). CONCLUSION: A sustained reduction in aortic stiffness and left ventricular hypertrophy, and a limited radial artery wall thickening were observed after long-term enzyme replacement therapy. There was no significant benefit of treatment on carotid hypertrophy. SN - 2047-4881 UR - https://www.unboundmedicine.com/medline/citation/21450622/Long_term_changes_in_arterial_structure_and_function_and_left_ventricular_geometry_after_enzyme_replacement_therapy_in_patients_affected_with_Fabry_disease_ L2 - http://journals.sagepub.com/doi/full/10.1177/1741826710391118?url_ver=Z39.88-2003&amp;rfr_id=ori:rid:crossref.org&amp;rfr_dat=cr_pub=pubmed DB - PRIME DP - Unbound Medicine ER -