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Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease.
J Pediatr. 2010 May; 156(5):832-7, 837.e1.JPed

Abstract

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 alpha-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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Authors+Show Affiliations

Schiffmann R
Institute of Metabolic Disease, Baylor Research Institute, Dallas, TX 75226, USA. Raphael.Schiffmann@baylorhealth.edu
Martin RA
No affiliation info available
Reimschisel T
No affiliation info available
Johnson K
No affiliation info available
Castaneda V
No affiliation info available
Lien YH
No affiliation info available
Pastores GM
No affiliation info available
Kampmann C
No affiliation info available
Ries M
No affiliation info available
Clarke JT
No affiliation info available

MeSH

AdolescentBody SizeChildFabry DiseaseFemaleHumansIsoenzymesKidneyMalePain MeasurementRecombinant ProteinsSweatTrihexosylceramidesVentricular Function, Leftalpha-Galactosidase

Pub Type(s)

Clinical Trial, Phase III
Journal Article
Multicenter Study

Language

eng

PubMed ID

20097359

Citation

Schiffmann, Raphael, et al. "Four-year Prospective Clinical Trial of Agalsidase Alfa in Children With Fabry Disease." The Journal of Pediatrics, vol. 156, no. 5, 2010, pp. 832-7, 837.e1.
Schiffmann R, Martin RA, Reimschisel T, et al. Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease. J Pediatr. 2010;156(5):832-7, 837.e1.
Schiffmann, R., Martin, R. A., Reimschisel, T., Johnson, K., Castaneda, V., Lien, Y. H., Pastores, G. M., Kampmann, C., Ries, M., & Clarke, J. T. (2010). Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease. The Journal of Pediatrics, 156(5), 832-7, e1. https://doi.org/10.1016/j.jpeds.2009.11.007
Schiffmann R, et al. Four-year Prospective Clinical Trial of Agalsidase Alfa in Children With Fabry Disease. J Pediatr. 2010;156(5):832-7, 837.e1. PubMed PMID: 20097359.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Four-year prospective clinical trial of agalsidase alfa in children with Fabry disease. AU - Schiffmann,Raphael, AU - Martin,Rick A, AU - Reimschisel,Tyler, AU - Johnson,Karen, AU - Castaneda,Victoria, AU - Lien,Y Howard, AU - Pastores,Gregory M, AU - Kampmann,Christoph, AU - Ries,Markus, AU - Clarke,Joe T R, Y1 - 2010/01/25/ PY - 2009/06/15/received PY - 2009/09/03/revised PY - 2009/11/04/accepted PY - 2010/1/26/entrez PY - 2010/1/26/pubmed PY - 2010/5/4/medline SP - 832-7, 837.e1 JF - The Journal of pediatrics JO - J Pediatr VL - 156 IS - 5 N2 - 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 alpha-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. SN - 1097-6833 UR - https://www.unboundmedicine.com/medline/citation/20097359/Four_year_prospective_clinical_trial_of_agalsidase_alfa_in_children_with_Fabry_disease_ L2 - https://linkinghub.elsevier.com/retrieve/pii/S0022-3476(09)01114-7 DB - PRIME DP - Unbound Medicine ER -