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Enzyme replacement therapy for Anderson-Fabry disease.

Abstract

BACKGROUND

Anderson-Fabry disease is an X-linked defect of glycosphingolipid metabolism. Progressive renal insufficiency is a major source of morbidity, additional complications result from cardio- and cerebro-vascular involvement. Survival is reduced among affected males and symptomatic female carriers.

OBJECTIVES

To evaluate the effectiveness and safety of enzyme replacement therapy compared to other interventions, placebo or no interventions, for treating Anderson-Fabry disease.

SEARCH STRATEGY

We searched 'Clinical Trials' on The Cochrane Library, MEDLINE, EMBASE, LILACS and the Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register (date of the most recent search: 07 April 2010).

SELECTION CRITERIA

Randomized controlled trials of agalsidase alfa or beta in participants diagnosed with Anderson-Fabry disease.

DATA COLLECTION AND ANALYSIS

Two authors selected relevant trials, assessed methodological quality and extracted data.

MAIN RESULTS

Five studies comparing either agalsidase alfa or beta in 187 participants fulfilled the selection criteria.Both trials comparing agalsidase alfa to placebo reported on globotriaosylceramide concentration in plasma and tissue; aggregate results were non-significant. One study reported pain scores, there was a statistically significant improvement for participants receiving treatment at up to three months, mean difference -2.10 (95% confidence interval (CI) -3.79 to -0.41); at up to five months, mean difference -1.90 (95% CI -3.65 to -0.15); and at up to six months, mean difference -2.00 (95% CI -3.66 to -0.34). There was a significant difference in pain-related quality of life at over five months and up to six months, mean difference -2.10 (95% CI -3.92 to -0.28) but not at other time-points. Neither study reported deaths.One of the three trials comparing agalsidase beta to placebo reported on globotriaosylceramide concentration in plasma and tissue and showed significant improvement: kidney, mean difference -1.70 (95% CI -2.09 to -1.31); heart, mean difference -0.90 (95% CI -1.18 to -0.62); and composite results (renal, cardiac, and cerebrovascular complications and death), mean difference -4.80 (95% CI -5.45 to -4.15). There was no significant difference between groups for death; no studies reported on pain.

AUTHORS' CONCLUSIONS

Five small, poor quality randomised controlled trials provide no robust evidence for use of either agalsidase alfa and beta to treat Anderson-Fabry disease.

Authors+Show Affiliations

Department of Surgery, St. Joseph's Healthcare Hamilton, McMaster University, 50 Charlton Avenue East, Hamilton, ON, Canada, L8N 4A6.No affiliation info available

Pub Type(s)

Journal Article
Meta-Analysis
Review
Systematic Review

Language

eng

PubMed ID

20464743

Citation

El Dib, Regina P., and Gregory M. Pastores. "Enzyme Replacement Therapy for Anderson-Fabry Disease." The Cochrane Database of Systematic Reviews, 2010, p. CD006663.
El Dib RP, Pastores GM. Enzyme replacement therapy for Anderson-Fabry disease. Cochrane Database Syst Rev. 2010.
El Dib, R. P., & Pastores, G. M. (2010). Enzyme replacement therapy for Anderson-Fabry disease. The Cochrane Database of Systematic Reviews, (5), CD006663. https://doi.org/10.1002/14651858.CD006663.pub2
El Dib RP, Pastores GM. Enzyme Replacement Therapy for Anderson-Fabry Disease. Cochrane Database Syst Rev. 2010 May 12;(5)CD006663. PubMed PMID: 20464743.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Enzyme replacement therapy for Anderson-Fabry disease. AU - El Dib,Regina P, AU - Pastores,Gregory M, Y1 - 2010/05/12/ PY - 2010/5/14/entrez PY - 2010/5/14/pubmed PY - 2010/6/17/medline SP - CD006663 EP - CD006663 JF - The Cochrane database of systematic reviews JO - Cochrane Database Syst Rev IS - 5 N2 - BACKGROUND: Anderson-Fabry disease is an X-linked defect of glycosphingolipid metabolism. Progressive renal insufficiency is a major source of morbidity, additional complications result from cardio- and cerebro-vascular involvement. Survival is reduced among affected males and symptomatic female carriers. OBJECTIVES: To evaluate the effectiveness and safety of enzyme replacement therapy compared to other interventions, placebo or no interventions, for treating Anderson-Fabry disease. SEARCH STRATEGY: We searched 'Clinical Trials' on The Cochrane Library, MEDLINE, EMBASE, LILACS and the Cystic Fibrosis and Genetic Disorders Group's Inborn Errors of Metabolism Trials Register (date of the most recent search: 07 April 2010). SELECTION CRITERIA: Randomized controlled trials of agalsidase alfa or beta in participants diagnosed with Anderson-Fabry disease. DATA COLLECTION AND ANALYSIS: Two authors selected relevant trials, assessed methodological quality and extracted data. MAIN RESULTS: Five studies comparing either agalsidase alfa or beta in 187 participants fulfilled the selection criteria.Both trials comparing agalsidase alfa to placebo reported on globotriaosylceramide concentration in plasma and tissue; aggregate results were non-significant. One study reported pain scores, there was a statistically significant improvement for participants receiving treatment at up to three months, mean difference -2.10 (95% confidence interval (CI) -3.79 to -0.41); at up to five months, mean difference -1.90 (95% CI -3.65 to -0.15); and at up to six months, mean difference -2.00 (95% CI -3.66 to -0.34). There was a significant difference in pain-related quality of life at over five months and up to six months, mean difference -2.10 (95% CI -3.92 to -0.28) but not at other time-points. Neither study reported deaths.One of the three trials comparing agalsidase beta to placebo reported on globotriaosylceramide concentration in plasma and tissue and showed significant improvement: kidney, mean difference -1.70 (95% CI -2.09 to -1.31); heart, mean difference -0.90 (95% CI -1.18 to -0.62); and composite results (renal, cardiac, and cerebrovascular complications and death), mean difference -4.80 (95% CI -5.45 to -4.15). There was no significant difference between groups for death; no studies reported on pain. AUTHORS' CONCLUSIONS: Five small, poor quality randomised controlled trials provide no robust evidence for use of either agalsidase alfa and beta to treat Anderson-Fabry disease. SN - 1469-493X UR - https://www.unboundmedicine.com/medline/citation/20464743/Enzyme_replacement_therapy_for_Anderson_Fabry_disease_ L2 - https://doi.org/10.1002/14651858.CD006663.pub2 DB - PRIME DP - Unbound Medicine ER -