Published online before print September 2, 2003, doi: 10.1161/01.CIR.0000091253.71282.04
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
(Circulation. 2003;108:1299.)
© 2003 American Heart Association, Inc.
Brief Rapid Communications |
Improvement of Cardiac Function During Enzyme Replacement Therapy in Patients With Fabry Disease
A Prospective Strain Rate Imaging Study
From the Department of Medicine, Divisions of Cardiology and Nephrology (F.W., F.B., O.T., W.V., G.E., A.K., C.W., J.S.), and Department of Radiology (M.B., J.S.), University Hospital Würzburg, Germany.
*These authors contributed equally to this study.
Correspondence to Jörg M. Strotmann, Medizinische Klinik, Universitätsklinik Würzburg, Josef-Schneider Str. 2, Germany. E-mail strotmann_j{at}medizin.uni-wuerzburg.de
Received June 2, 2003; de novo received July 3, 2003; revision received July 28, 2003; accepted July 28, 2003.
Background— Enzyme replacement therapy (ERT) has been shown to enhance microvascular endothelial globotriaosylceramide clearance in the hearts of patients with Fabry disease. Whether these results can be translated into an improvement of myocardial function has yet to be demonstrated.
Methods and Results— Sixteen patients with Fabry disease who were treated in an open-label study with 1.0 mg/kg body weight of recombinant 
-Gal A (agalsidase ß, Fabrazyme) were followed up for 12 months. Myocardial function was quantified by ultrasonic strain rate imaging to assess radial and longitudinal myocardial deformation. End-diastolic thickness of the left ventricular posterior wall and myocardial mass (assessed by magnetic resonance imaging, n=10) was measured at baseline and after 12 months of ERT. Data were compared with 16 age-matched healthy controls. At baseline, both peak systolic strain rate and systolic strain were significantly reduced in the radial and longitudinal direction in patients compared with controls. Peak systolic strain rate increased significantly in the posterior wall (radial function) after one year of treatment (baseline, 2.8±0.2 s-1; 12 months, 3.7±0.3 s-1; P<0.05). In addition, end-systolic strain of the posterior wall increased significantly (baseline, 34±3%; 12 months, 45±4%; P<0.05). This enhancement in radial function was accompanied by an improvement in longitudinal function. End-diastolic thickness of the posterior wall decreased significantly after 12 months of treatment (baseline, 13.8±0.6 mm; 12 months, 11.8±0.6 mm; P<0.05). In parallel, myocardial mass decreased significantly from 201±18 to 180±21 g (P<0.05).
Conclusions— These results suggest that ERT can decrease left ventricular hypertrophy and improve regional myocardial function.
Key Words: Fabry disease • enzymes • hypertrophy • imaging
This article has been cited by other articles:
 |
|
 |
|
  C. A. Gange, M. S. Link, and M. S. Maron Utility of Cardiovascular Magnetic Resonance in the Diagnosis of Anderson-Fabry Disease Circulation, September 29, 2009; 120(13): e96 - e97. [Full Text] [PDF]
|
|
|
|
 |
|
 A Mehta, J T R Clarke, R Giugliani, P Elliott, A Linhart, M Beck, G Sunder-Plassmann, and on behalf of the FOS Investigators Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey J. Med. Genet., August 1, 2009; 46(8): 548 - 552. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 M Imbriaco, A Pisani, L Spinelli, A Cuocolo, G Messalli, E Capuano, M Marmo, R Liuzzi, B Visciano, B Cianciaruso, et al. Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study Heart, July 1, 2009; 95(13): 1103 - 1107. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 T. H. Marwick and J. Narula The Growth and Growth of Cardiac Ultrasound for the Evaluation of Myocardial Function J. Am. Coll. Cardiol. Img., June 1, 2009; 2(6): 790 - 792. [Full Text] [PDF]
|
|
|
|
|
|
B. L. Thurberg, J. T. Fallon, R. Mitchell, T. Aretz, R. E. Gordon, and M. W. O'Callaghan Cardiac Microvascular Pathology in Fabry Disease: Evaluation of Endomyocardial Biopsies Before and After Enzyme Replacement Therapy Circulation, May 19, 2009; 119(19): 2561 - 2567. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
F. Weidemann, M. Niemann, F. Breunig, S. Herrmann, M. Beer, S. Stork, W. Voelker, G. Ertl, C. Wanner, and J. Strotmann Long-Term Effects of Enzyme Replacement Therapy on Fabry Cardiomyopathy: Evidence for a Better Outcome With Early Treatment Circulation, February 3, 2009; 119(4): 524 - 529. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 S. Choi, H. Seo, M. Park, J. Kim, S. Hwang, K. Kwon, K. Her, and Y. Won Fabry disease with aortic regurgitation. Ann. Thorac. Surg., February 1, 2009; 87(2): 625 - 628. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 F. A. Flachskampf, J.-U. Voigt, and W. G. Daniel CHAPTER 4 Cardiac Ultrasound ESC Textbook of Cardiovascular Medicine, January 1, 2009; 2(1): med-9780199566990-chapter - med-9780199566990-chapter. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 T. Kovacevic-Preradovic, M. Zuber, C.H. A. Jost, U. Widmer, B. Seifert, G. Schulthess, A. Fischer, and R. Jenni Anderson-Fabry disease: long-term echocardiographic follow-up under enzyme replacement therapy Eur J Echocardiogr, November 1, 2008; 9(6): 729 - 735. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
F. Weidemann, C Wanner, and F. Breunig Nomen est Omen Eur J Echocardiogr, November 1, 2008; 9(6): 831 - 832. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
S. B. Joshi, W. Ahmar, G. Lee, and A. Aggarwal Fabry's disease presenting as ventricular tachycardia and Left Ventricular 'Hypertrophy' Eur J Echocardiogr, September 1, 2008; 9(5): 697 - 699. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 C. Chimenti, N. Hamdani, N. M. Boontje, F. DeCobelli, A. Esposito, J. G.F. Bronzwaer, G. J.M. Stienen, M. A. Russo, W. J. Paulus, A. Frustaci, et al. Myofilament Degradation and Dysfunction of Human Cardiomyocytes in Fabry Disease Am. J. Pathol., June 1, 2008; 172(6): 1482 - 1490. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 R. Mignani, S. Feriozzi, A. Pisani, A. Cioni, C. Comotti, M. Cossu, A. Foschi, A. Giudicissi, E. Gotti, V. A. Lozupone, et al. Agalsidase therapy in patients with Fabry disease on renal replacement therapy: a nationwide study in Italy Nephrol. Dial. Transplant., May 1, 2008; 23(5): 1628 - 1635. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
D H MacIver and M Townsend A novel mechanism of heart failure with normal ejection fraction Heart, April 1, 2008; 94(4): 446 - 449. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
A. Linhart Treatment of Anderson Fabry disease Heart, February 1, 2008; 94(2): 138 - 139. [Full Text] [PDF]
|
|
|
|
 |
|
 F. Weidemann, M. Niemann, S. Herrmann, M. Kung, S. Stork, C. Waller, M. Beer, F. Breunig, C. Wanner, W. Voelker, et al. A new echocardiographic approach for the detection of non-ischaemic fibrosis in hypertrophic myocardium Eur. Heart J., December 2, 2007; 28(24): 3020 - 3026. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
U Sechtem, H Mahrholdt, and H Vogelsberg Cardiac magnetic resonance in myocardial disease Heart, December 1, 2007; 93(12): 1520 - 1527. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
T. P. Abraham, V. L. Dimaano, and H.-Y. Liang Role of Tissue Doppler and Strain Echocardiography in Current Clinical Practice Circulation, November 27, 2007; 116(22): 2597 - 2609. [Full Text] [PDF]
|
|
|
|
 |
|
 M. Ries, J. T. Clarke, C. Whybra, A. Mehta, K. S. Loveday, R. O. Brady, M. Beck, and R. Schiffmann Enzyme Replacement in Fabry Disease: Pharmacokinetics and Pharmacodynamics of Agalsidase Alfa in Children and Adolescents J. Clin. Pharmacol., October 1, 2007; 47(10): 1222 - 1230. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
R. Lee and T. H. Marwick Assessment of subclinical left ventricular dysfunction in asymptomatic mitral regurgitation Eur J Echocardiogr, June 1, 2007; 8(3): 175 - 184. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
A. Marciniak, E. Eroglu, M. Marciniak, C. Sirbu, L. Herbots, W. Droogne, P. Claus, J. D'hooge, B. Bijnens, J. Vanhaecke, et al. The potential clinical role of ultrasonic strain and strain rate imaging in diagnosing acute rejection after heart transplantation Eur J Echocardiogr, June 1, 2007; 8(3): 213 - 221. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
A. Linhart and P. M Elliott The heart in Anderson-Fabry disease and other lysosomal storage disorders Heart, April 1, 2007; 93(4): 528 - 535. [Full Text] [PDF]
|
|
|
|
 |
|
 J. T.R. Clarke Narrative Review: Fabry Disease Ann Intern Med, March 20, 2007; 146(6): 425 - 433. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
M. Banikazemi, J. Bultas, S. Waldek, W. R. Wilcox, C. B. Whitley, M. McDonald, R. Finkel, S. Packman, D. G. Bichet, D. G. Warnock, et al. Agalsidase-Beta Therapy for Advanced Fabry Disease: A Randomized Trial Ann Intern Med, January 16, 2007; 146(2): 77 - 86. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 M. Ries, J. T.R. Clarke, C. Whybra, M. Timmons, C. Robinson, B. L. Schlaggar, G. Pastores, Y. H. Lien, C. Kampmann, R. O. Brady, et al. Enzyme-Replacement Therapy With Agalsidase Alfa in Children With Fabry Disease Pediatrics, September 1, 2006; 118(3): 924 - 932. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
F. Weidemann and J. M. Strotmann Detection of subclinical LV dysfunction by tissue Doppler imaging Eur. Heart J., August 1, 2006; 27(15): 1771 - 1772. [Full Text] [PDF]
|
|
|
|
|
|
H. Morita, M. G. Larson, S. C. Barr, R. S. Vasan, C. J. O'Donnell, J. N. Hirschhorn, D. Levy, D. Corey, C. E. Seidman, J.G. Seidman, et al. Single-Gene Mutations and Increased Left Ventricular Wall Thickness in the Community: The Framingham Heart Study Circulation, June 13, 2006; 113(23): 2697 - 2705. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 T. H. Marwick Measurement of Strain and Strain Rate by Echocardiography: Ready for Prime Time? J. Am. Coll. Cardiol., April 4, 2006; 47(7): 1313 - 1327. [Abstract] [Full Text] [PDF]
|
|
|
|
 |
|
 S.-i. Morimoto, A. Sugiura, M. Iwase, N. Kubo, S. Hiramitsu, A. Uemura, M. Ohtsuki, S. Kato, Y. Kato, and H. Hishida Relief of Left Ventricular Outflow Obstruction by Cibenzoline in a Patient With Fabry's Disease: A Case Report Angiology, March 1, 2006; 57(2): 241 - 245. [Abstract] [PDF]
|
|
|
|
|
|
P M Elliott, H Kindler, J S Shah, B Sachdev, O E Rimoldi, R Thaman, M T Tome, W J McKenna, P Lee, and P G Camici Coronary microvascular dysfunction in male patients with Anderson-Fabry disease and the effect of treatment with {alpha} galactosidase A Heart, March 1, 2006; 92(3): 357 - 360. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
F A Flachskampf and J-U Voigt The interventricular septum is functionally bilayered: a fresh look at a well known structure Heart, October 1, 2005; 91(10): 1260 - 1261. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
B. Cosyns, C. Weytjens, M. Vanderhoogstrate, C. Daniels, D. Schoors, and G. Van Camp Tissue Doppler imaging does not show infraclinical alteration of myocardial function after contrast echocardiography Eur J Echocardiogr, August 1, 2005; 6(4): 238 - 242. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
F. Weidemann, F. Breunig, M. Beer, J. Sandstede, S. Stork, W. Voelker, G. Ertl, A. Knoll, C. Wanner, and J. M. Strotmann The variation of morphological and functional cardiac manifestation in Fabry disease: potential implications for the time course of the disease Eur. Heart J., June 2, 2005; 26(12): 1221 - 1227. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
J S Shah, P Lee, D Hughes, R Thaman, B Sachdev, D Pellerin, A Mehta, and P M Elliott The natural history of left ventricular systolic function in Anderson-Fabry disease Heart, April 1, 2005; 91(4): 533 - 534. [Full Text] [PDF]
|
|
|
|
|
|
M. Ries, S. Gupta, D. F. Moore, V. Sachdev, J. M. Quirk, G. J. Murray, D. R. Rosing, C. Robinson, E. Schaefer, A. Gal, et al. Pediatric Fabry Disease Pediatrics, March 1, 2005; 115(3): e344 - e355. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
C. Chimenti, M. Pieroni, E. Morgante, D. Antuzzi, A. Russo, M. A. Russo, A. Maseri, and A. Frustaci Prevalence of Fabry Disease in Female Patients With Late-Onset Hypertrophic Cardiomyopathy Circulation, August 31, 2004; 110(9): 1047 - 1053. [Abstract] [Full Text] [PDF]
|
|
|
|
|
|
A. E. Weyman The year in echocardiography J. Am. Coll. Cardiol., January 7, 2004; 43(1): 140 - 148. [Full Text] [PDF]
|
|