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Enzyme replacement therapy with agalsidase alfa in children with Fabry disease.
Acta Paediatr. 2007 Jan; 96(1):122-7.AP

Abstract

AIM

To assess the effects of enzyme replacement therapy (ERT) in children with Fabry disease.

METHODS

Safety and efficacy of ERT with agalsidase alfa, 0.2 mg/kg infused over 40 minutes every 2 weeks for 23 weeks, were studied in a multicentre open-label trial in nine boys and four girls. Median age at the start of the study was 11.0 years (range 3.5-18 years).

RESULTS

Fifty-four adverse events were reported in 11 patients. No serious adverse events related to ERT were reported. Twelve of the 54 adverse events were considered possibly or probably related to ERT. Infusion reactions (8 mild, 3 moderate) occurred in four boys, in seven infusions. One boy developed IgG antibodies, although he continued to make good clinical progress. At the end of the study, two of the four boys and the one girl on regular pain medication at baseline had stopped taking analgesics. Brief Pain Inventory (BPI) scores decreased in most patients by week 12 and were sustained until the end of the study. This change was greater in the boys, who had higher (worse) BPI scores at baseline. Pain-related quality of life (QoL) scores also decreased during the study. Plasma globotriaosylceramide concentrations and urinary globotriaosylceramide:sphingomyelin ratios decreased after 12 and 23 weeks of therapy, particularly in the boys. Increases in sweat volume were recorded in three out of five of the boys and in one of two girls tested after 23 weeks of treatment.

CONCLUSION

ERT with agalsidase alfa in children with Fabry disease is well tolerated and, in the short term, appears to decrease pain and to improve pain-related QoL.

Authors+Show Affiliations

Department of Paediatric Endocrinology, Diabetes and Metabolism, Addenbrooke's University Teaching Hospital, Cambridge, UK. uma.ramaswami@addenbrookes.nhs.ukNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info availableNo affiliation info available

Pub Type(s)

Clinical Trial
Journal Article
Multicenter Study
Research Support, Non-U.S. Gov't

Language

eng

PubMed ID

17187618

Citation

Ramaswami, U, et al. "Enzyme Replacement Therapy With Agalsidase Alfa in Children With Fabry Disease." Acta Paediatrica (Oslo, Norway : 1992), vol. 96, no. 1, 2007, pp. 122-7.
Ramaswami U, Wendt S, Pintos-Morell G, et al. Enzyme replacement therapy with agalsidase alfa in children with Fabry disease. Acta Paediatr. 2007;96(1):122-7.
Ramaswami, U., Wendt, S., Pintos-Morell, G., Parini, R., Whybra, C., Leon Leal, J. A., Santus, F., & Beck, M. (2007). Enzyme replacement therapy with agalsidase alfa in children with Fabry disease. Acta Paediatrica (Oslo, Norway : 1992), 96(1), 122-7.
Ramaswami U, et al. Enzyme Replacement Therapy With Agalsidase Alfa in Children With Fabry Disease. Acta Paediatr. 2007;96(1):122-7. PubMed PMID: 17187618.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Enzyme replacement therapy with agalsidase alfa in children with Fabry disease. AU - Ramaswami,U, AU - Wendt,S, AU - Pintos-Morell,G, AU - Parini,R, AU - Whybra,C, AU - Leon Leal,J A, AU - Santus,F, AU - Beck,M, PY - 2006/12/26/pubmed PY - 2007/4/14/medline PY - 2006/12/26/entrez SP - 122 EP - 7 JF - Acta paediatrica (Oslo, Norway : 1992) JO - Acta Paediatr VL - 96 IS - 1 N2 - AIM: To assess the effects of enzyme replacement therapy (ERT) in children with Fabry disease. METHODS: Safety and efficacy of ERT with agalsidase alfa, 0.2 mg/kg infused over 40 minutes every 2 weeks for 23 weeks, were studied in a multicentre open-label trial in nine boys and four girls. Median age at the start of the study was 11.0 years (range 3.5-18 years). RESULTS: Fifty-four adverse events were reported in 11 patients. No serious adverse events related to ERT were reported. Twelve of the 54 adverse events were considered possibly or probably related to ERT. Infusion reactions (8 mild, 3 moderate) occurred in four boys, in seven infusions. One boy developed IgG antibodies, although he continued to make good clinical progress. At the end of the study, two of the four boys and the one girl on regular pain medication at baseline had stopped taking analgesics. Brief Pain Inventory (BPI) scores decreased in most patients by week 12 and were sustained until the end of the study. This change was greater in the boys, who had higher (worse) BPI scores at baseline. Pain-related quality of life (QoL) scores also decreased during the study. Plasma globotriaosylceramide concentrations and urinary globotriaosylceramide:sphingomyelin ratios decreased after 12 and 23 weeks of therapy, particularly in the boys. Increases in sweat volume were recorded in three out of five of the boys and in one of two girls tested after 23 weeks of treatment. CONCLUSION: ERT with agalsidase alfa in children with Fabry disease is well tolerated and, in the short term, appears to decrease pain and to improve pain-related QoL. SN - 0803-5253 UR - https://www.unboundmedicine.com/medline/citation/17187618/Enzyme_replacement_therapy_with_agalsidase_alfa_in_children_with_Fabry_disease_ L2 - https://doi.org/10.1111/j.1651-2227.2007.00029.x DB - PRIME DP - Unbound Medicine ER -