The DOI matches its Crossref record; 14 of the 14 references it cites were found in a registry or an index.
Long-Term Effects of Enzyme Replacement Therapy on Fabry Cardiomyopathy: Evidence for a Better Outcome With Early Treatment
Open accessAbstract
Background— Enzyme replacement therapy with recombinant α-galactosidase A reduces left ventricular hypertrophy and improves regional myocardial function in patients with Fabry disease during short-term treatment. Whether enzyme replacement therapy is effective in all stages of Fabry cardiomyopathy during long-term follow-up is unknown. Methods and Results— We studied 32 Fabry patients over a period of 3 years regarding disease progression and clinical outcome under enzyme replacement therapy. Regional myocardial fibrosis was assessed by magnetic resonance imaging late-enhancement technique. Echocardiographic myocardial mass was calculated with the Devereux formula, and myocardial function was quantified by ultrasonic strain-rate imaging. In addition, exercise capacity was measured by bicycle stress test. All measurements were repeated at yearly intervals. At baseline, 9 patients demonstrated at least 2 fibrotic left ventricular segments (severe myocardial fibrosis), 11 had 1 left ventricular segment affected (mild fibrosis), and 12 were without fibrosis. In patients without fibrosis, enzyme replacement therapy resulted in a significant reduction in left ventricular mass (238±42 g at baseline, 202±46 g at 3 years; P for trend <0.001), an improvement in myocardial function (systolic radial strain rate, 2.3±0.4 and 2.9±0.6 seconds −1 , respectively; P for trend=0.045), and a higher exercise capacity obtained by bicycle stress exercise (106±14 and 122±26 W, respectively; P for trend=0.014). In contrast, patients with mild or severe fibrosis showed a minor reduction in left ventricular hypertrophy and no improvement in myocardial function or exercise capacity. Conclusions— These data suggest that treatment of Fabry cardiomyopathy with recombinant α-galactosidase A should best be started before myocardial fibrosis has developed to achieve long-term improvement in myocardial morphology and function and exercise capacity.
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Cites
American Heart Association Writing Group on Myocardial Segmentation and Registration for Cardiac Imaging:, Cerqueira, M. D., Weissman, N. J., Dilsizian, V., Jacobs, A. K., Kaul, S., Laskey, W. K., Pennell, D. J., Rumberger, J. A., Ryan, T., et al. (2002) · Circulation · doi:10.1161/hc0402.102975 · Cited by 6,990
Safety and Efficacy of Recombinant Human α-Galactosidase A Replacement Therapy in Fabry's Disease
Eng, C. M., Guffon, N., Wilcox, W. R., Germain, D. P., Lee, P., Waldek, S., Caplan, L., Linthorst, G. E., & Desnick, R. J. (2001) · New England Journal of Medicine · doi:10.1056/nejm200107053450102 · Cited by 1,559
Enzyme Replacement Therapy in Fabry Disease: A Randomized Controlled Trial
Schiffmann, R., Kopp, J. B., Austin III, H. A., Sabnis, S., Moore, D. F., Weibel, T., Balow, J. E., & Brady, R. O. (2001) · JAMA · doi:10.1001/jama.285.21.2743 · Cited by 1,302
Desnick, R. J., Brady, R., Barranger, J., Collins, A. J., Germain, D. P., Goldman, M., Grabowski, G., Packman, S., & Wilcox, W. R. (2003) · Annals of Internal Medicine · doi:10.7326/0003-4819-138-4-200302180-00014 · Cited by 766
Agalsidase-Beta Therapy for Advanced Fabry Disease: A Randomized Trial
Banikazemi, M., Bultas, J., Waldek, S., Wilcox, W. R., Whitley, C. B., McDonald, M., Finkel, R., Packman, S., Bichet, D. G., Warnock, D. G., et al. (2007) · Annals of Internal Medicine · doi:10.7326/0003-4819-146-2-200701160-00148 · Cited by 569
Moon, J. (2003) · European Heart Journal · doi:10.1016/j.ehj.2003.09.017 · Cited by 466
Fabry disease: Guidelines for the evaluation and management of multi-organ system involvement
Eng, C. M., Germain, D. P., Banikazemi, M., Warnock, D. G., Wanner, C., Hopkin, R. J., Bultas, J., Lee, P., Sims, K., Brodie, S. E., et al. (2006) · Genetics in Medicine · doi:10.1097/01.gim.0000237866.70357.c6 · Cited by 411
Self-citationWeidemann, F., Jamal, F., Sutherland, G. R., Claus, P., Kowalski, M., Hatle, L., De Scheerder, I., Bijnens, B., & Rademakers, F. E. (2002) · American Journal of Physiology-Heart and Circulatory Physiology · doi:10.1152/ajpheart.00025.2002 · Cited by 407
Self-citationWeidemann, F., Breunig, F., Beer, M., Sandstede, J., Turschner, O., Voelker, W., Ertl, G., Knoll, A., Wanner, C., & Strotmann, J. M. (2003) · Circulation · doi:10.1161/01.cir.0000091253.71282.04 · Cited by 389
Self-citationHughes, D. A., Elliott, P. M., Shah, J., Zuckerman, J., Coghlan, G., Brookes, J., & Mehta, A. B. (2008) · Heart · doi:10.1136/hrt.2006.104026 · Cited by 318
Cited by
Maron, B. J., & Maron, M. S. (2013) · The Lancet · doi:10.1016/s0140-6736(12)60397-3 · Cited by 1,231
Germain, D. P. (2010) · Orphanet Journal of Rare Diseases · doi:10.1186/1750-1172-5-30 · Cited by 1,198
Genetic Misdiagnoses and the Potential for Health Disparities
Manrai, A. K., Funke, B. H., Rehm, H. L., Olesen, M. S., Maron, B. A., Szolovits, P., Margulies, D. M., Loscalzo, J., & Kohane, I. S. (2016) · New England Journal of Medicine · doi:10.1056/nejmsa1507092 · Cited by 808
Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
Germain, D. P., Hughes, D. A., Nicholls, K., Bichet, D. G., Giugliani, R., Wilcox, W. R., Feliciani, C., Shankar, S. P., Ezgu, F., Amartino, H., et al. (2016) · New England Journal of Medicine · doi:10.1056/nejmoa1510198 · Cited by 555
Hughes, D. A., Nicholls, K., Shankar, S. P., Sunder-Plassmann, G., Koeller, D., Nedd, K., Vockley, G., Hamazaki, T., Lachmann, R., Ohashi, T., et al. (2017) · Journal of Medical Genetics · doi:10.1136/jmedgenet-2016-104178 · Cited by 386
Metadata
- Authors
- Weidemann, F., Niemann, M., Breunig, F., Herrmann, S., Beer, M., Störk, S., Voelker, W., Ertl, G., Wanner, C., & Strotmann, J.
- Venue
- Circulation
- Published
- 2009-02-03
- Volume
- 119
- Issue
- 4
- Pages
- 524-529
- ISSN
- 0009-7322 (ISSN-L)
- 1524-4539
- OpenAlex
- W2094976188
- Cited by
- 488
- References
- 14
- FWCI
- 23.82
- Type
- journal article
- Language
- en
- Open access
- bronze
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- CoChat. (2026). Citation check: Long-Term Effects of Enzyme Replacement Therapy on Fabry Cardiomyopathy: Evidence for a Better Outcome With Early Treatment. https://app.cochat.ai/papers/10.1161/circulationaha.108.794529
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- Weidemann, F., Niemann, M., Breunig, F., Herrmann, S., Beer, M., Störk, S., Voelker, W., Ertl, G., Wanner, C., & Strotmann, J. (2009). Long-Term Effects of Enzyme Replacement Therapy on Fabry Cardiomyopathy: Evidence for a Better Outcome With Early Treatment. Circulation. https://doi.org/10.1161/circulationaha.108.794529
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
