Please use this identifier to cite or link to this item:
https://hdl.handle.net/2440/82291
Citations | ||
Scopus | Web of Science® | Altmetric |
---|---|---|
?
|
?
|
Type: | Journal article |
Title: | Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy |
Other Titles: | Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme(r)) therapy |
Author: | Braunlin, E. Rosenfeld, H. Kampmann, C. Johnson, J. Beck, M. Giugliani, R. Guffon, N. Ketteridge, D. Miranda, C. Scarpa, M. Schwartz, I. Teles, E. Wraith, J. Barrios, P. Dias da Silva, E. Kurio, G. Richardson, M. Gildengorin, G. Hopwood, J. Imperiale, M. et al. |
Citation: | Journal of Inherited Metabolic Disease, 2013; 36(2):385-394 |
Publisher: | Kluwer Academic Publ |
Issue Date: | 2013 |
ISSN: | 0141-8955 1573-2665 |
Statement of Responsibility: | E. Braunlin, H. Rosenfeld, C. Kampmann, J. Johnson, M. Beck, R. Giugliani, N. Guffon, D. Ketteridge, C. M. Sá Miranda, M. Scarpa, I. V. Schwartz, E. Leão Teles, J. E. Wraith, P. Barrios, E. Dias da Silva, G. Kurio, M. Richardson, G. Gildengorin, J. J. Hopwood, M. Imperiale, A. Schatz, C. Decker, P. Harmatz, MPS VI Study Group |
Abstract: | Characteristic cardiac valve abnormalities and left ventricular hypertrophy are present in untreated patients with mucopolysaccharidosis type VI (MPS VI). Cardiac ultrasound was performed to investigate these findings in subjects during long-term enzyme replacement therapy (ERT) with recombinant human arylsulfatase B (rhASB, rhN-acetylgalactosamine 4-sulfatase, galsulfase, Naglazyme®). Studies were conducted in 54 subjects before ERT was begun and at specific intervals for up to 96 weeks of weekly infusions of rhASB at 1 mg/kg during phase 1/2, phase 2, and phase 3 trials of rhASB. At baseline, mitral and aortic valve obstruction was present and was significantly greater in those ≥12 years of age. Mild mitral and trace aortic regurgitation were present, the former being significantly greater in those <12 years. Left ventricular hypertrophy, with averaged z-scores ranging from 1.6–1.9 SD greater than normal, was present for ages both <12 and ≥12 years. After 96 weeks of ERT, ventricular septal hypertrophy regressed in those <12 years. For those ≥12 years, septal hypertrophy was unchanged, and aortic regurgitation increased statistically but not physiologically. Obstructive gradients across mitral and aortic valves remained unchanged. The results suggest that long-term ERT is effective in reducing intraventricular septal hypertrophy and preventing progression of cardiac valve abnormalities when administered to those <12 years of age. |
Keywords: | MPS VI Study Group Heart Valves Humans Hypertrophy, Left Ventricular Mucopolysaccharidosis VI N-Acetylgalactosamine-4-Sulfatase Recombinant Proteins Treatment Outcome Adolescent Adult Child Female Male Clinical Trials as Topic Randomized Controlled Trials as Topic Young Adult Enzyme Replacement Therapy |
Rights: | © The Author(s) 2012. This article is published with open access at Springerlink.com |
DOI: | 10.1007/s10545-012-9481-2 |
Published version: | http://dx.doi.org/10.1007/s10545-012-9481-2 |
Appears in Collections: | Aurora harvest Paediatrics publications |
Files in This Item:
There are no files associated with this item.
Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.