Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease
Objectives - To investigate a 4-year prospective clinical trial of agalsidase alfa in children with Fabry disease, an X-linked metabolic disorder caused by a deficiency of the lysosomal enzyme α-galactosidase A. - Study design - Seventeen (16 boys, 1 girl; age range, 7.3 to 18.4 years) of the 24 chi...
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| Main Authors: | , |
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| Format: | Article (Journal) |
| Language: | English |
| Published: |
25 January 2010
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| In: |
The journal of pediatrics
Year: 2010, Volume: 156, Issue: 5, Pages: 832-837.e1 |
| ISSN: | 1097-6833 |
| DOI: | 10.1016/j.jpeds.2009.11.007 |
| Online Access: | Verlag, Volltext: https://doi.org/10.1016/j.jpeds.2009.11.007 Verlag, Volltext: http://www.sciencedirect.com/science/article/pii/S0022347609011147 
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| Author Notes: | Raphael Schiffmann, MD, Rick A. Martin, MD, Tyler Reimschisel, MD, Karen Johnson, MD, MPH, Victoria Castaneda, MD, Y. Howard Lien, MD, PhD, Gregory M. Pastores, MD, Christoph Kampmann, MD, Markus Ries, MD, PhD, MHSc, and Joe T.R. Clarke, MD, PhD |
MARC
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| 245 | 1 | 0 | |a Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease |c Raphael Schiffmann, MD, Rick A. Martin, MD, Tyler Reimschisel, MD, Karen Johnson, MD, MPH, Victoria Castaneda, MD, Y. Howard Lien, MD, PhD, Gregory M. Pastores, MD, Christoph Kampmann, MD, Markus Ries, MD, PhD, MHSc, and Joe T.R. Clarke, MD, PhD |
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| 520 | |a Objectives - To investigate a 4-year prospective clinical trial of agalsidase alfa in children with Fabry disease, an X-linked metabolic disorder caused by a deficiency of the lysosomal enzyme α-galactosidase A. - Study design - Seventeen (16 boys, 1 girl; age range, 7.3 to 18.4 years) of the 24 children who completed a 6-month, open-label agalsidase alfa study enrolled in a 3.5-year extension study that investigated the safety and potential efficacy of long-term treatment. All 17 patients completed the initial 6-month study, and 10 patients (9 boys) completed the extension study. - Results - Agalsidase alfa was well tolerated. In treated boys, there were sustained, statistically-significant improvements in the clinical features of Fabry disease, including reduced plasma globotriaosylceramide levels, reduced pain severity assessed by the Brief Pain Index, and improved heart rate variability. Mean urine globotriaosylceramide levels were reduced to normal range (P < .05 compared with baseline during 1.5 to 4 years). Kidney function and left ventricular mass indexed to height remained stable throughout. - Conclusions - This clinical trial demonstrates that treatment with agalsidase alfa was well tolerated and associated with improvement of Fabry disease-related features. | ||
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