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Intervention Review

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Enzyme replacement therapy for Anderson-Fabry disease

  1. Regina P El Dib1,*,
  2. Paulo Nascimento2,
  3. Gregory M Pastores3

Editorial Group: Cochrane Cystic Fibrosis and Genetic Disorders Group

Published Online: 28 FEB 2013

Assessed as up-to-date: 12 DEC 2012

DOI: 10.1002/14651858.CD006663.pub3


How to Cite

El Dib RP, Nascimento P, Pastores GM. Enzyme replacement therapy for Anderson-Fabry disease. Cochrane Database of Systematic Reviews 2013, Issue 2. Art. No.: CD006663. DOI: 10.1002/14651858.CD006663.pub3.

Author Information

  1. 1

    Botucatu Medical School, Universidade Estadual Paulista (UNESP), Botucatu, São Paulo, Brazil

  2. 2

    UNESP - Univ Estadual Paulista, Botucatu, São Paulo, Brazil

  3. 3

    New York University School of Medicine, Neurogenetics Laboratory, New York, New York, USA

*Regina P El Dib, Botucatu Medical School, Universidade Estadual Paulista (UNESP), Distrito de Rubião Júnior, s/n, Botucatu, São Paulo, 18603-970, Brazil. eldib@fmb.unesp.br. re.lucci@terra.com.br.

Publication History

  1. Publication Status: New search for studies and content updated (no change to conclusions)
  2. Published Online: 28 FEB 2013

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This is not the most recent version of the article. View current version (25 JUL 2016)

[Analysis 1.1]
Analysis 1.1. Comparison 1 Agalsidase alfa versus placebo, Outcome 1 Plasma Gb3.
[Analysis 1.2]
Analysis 1.2. Comparison 1 Agalsidase alfa versus placebo, Outcome 2 Urine sediment Gb3.
[Analysis 1.3]
Analysis 1.3. Comparison 1 Agalsidase alfa versus placebo, Outcome 3 Kidney Gb3.
[Analysis 1.4]
Analysis 1.4. Comparison 1 Agalsidase alfa versus placebo, Outcome 4 Myocardial globotriaosylceramide levels.
[Analysis 1.5]
Analysis 1.5. Comparison 1 Agalsidase alfa versus placebo, Outcome 5 The Brief Pain Inventory Severity.
[Analysis 1.6]
Analysis 1.6. Comparison 1 Agalsidase alfa versus placebo, Outcome 6 The Brief Pain Inventory pain-related quality of life.
[Analysis 1.7]
Analysis 1.7. Comparison 1 Agalsidase alfa versus placebo, Outcome 7 Creatinine clearance.
[Analysis 1.8]
Analysis 1.8. Comparison 1 Agalsidase alfa versus placebo, Outcome 8 Insulin clearance.
[Analysis 1.9]
Analysis 1.9. Comparison 1 Agalsidase alfa versus placebo, Outcome 9 Glomeruli with mesangial widening.
[Analysis 1.10]
Analysis 1.10. Comparison 1 Agalsidase alfa versus placebo, Outcome 10 Glomeruli with segmental sclerosis.
[Analysis 1.11]
Analysis 1.11. Comparison 1 Agalsidase alfa versus placebo, Outcome 11 Obsolescent glomeruli.
[Analysis 1.12]
Analysis 1.12. Comparison 1 Agalsidase alfa versus placebo, Outcome 12 Echocardiographic data on left ventricular structure and function over three months and up to six months.
[Analysis 2.1]
Analysis 2.1. Comparison 2 Agalsidase beta versus placebo, Outcome 1 Microvascular endothelial deposits of globotriaosylceramide.
[Analysis 2.2]
Analysis 2.2. Comparison 2 Agalsidase beta versus placebo, Outcome 2 Death (intention-to-treat population).
[Analysis 2.3]
Analysis 2.3. Comparison 2 Agalsidase beta versus placebo, Outcome 3 Renal events (intention-to-treat population).
[Analysis 2.4]
Analysis 2.4. Comparison 2 Agalsidase beta versus placebo, Outcome 4 Cardiopulmonary exercise test.
[Analysis 2.5]
Analysis 2.5. Comparison 2 Agalsidase beta versus placebo, Outcome 5 Cardiopulmonary exercise test.
[Analysis 2.6]
Analysis 2.6. Comparison 2 Agalsidase beta versus placebo, Outcome 6 Cardiac events.
[Analysis 2.7]
Analysis 2.7. Comparison 2 Agalsidase beta versus placebo, Outcome 7 Cerebrovascular events (intention-to-treat population).
[Analysis 2.8]
Analysis 2.8. Comparison 2 Agalsidase beta versus placebo, Outcome 8 Participants achieving zero scores in skin over three months and up to six months.
[Analysis 2.9]
Analysis 2.9. Comparison 2 Agalsidase beta versus placebo, Outcome 9 Participants achieving zero score or reduction in skin over three months and up to six months.
[Analysis 2.10]
Analysis 2.10. Comparison 2 Agalsidase beta versus placebo, Outcome 10 Adverse event: Rigors.
[Analysis 2.11]
Analysis 2.11. Comparison 2 Agalsidase beta versus placebo, Outcome 11 Adverse event: Fever.
[Analysis 2.12]
Analysis 2.12. Comparison 2 Agalsidase beta versus placebo, Outcome 12 Adverse event: Temperature changed sensation.
[Analysis 2.13]
Analysis 2.13. Comparison 2 Agalsidase beta versus placebo, Outcome 13 Adverse event: Chills.
[Analysis 2.14]
Analysis 2.14. Comparison 2 Agalsidase beta versus placebo, Outcome 14 Adverse event: Hypertension.
[Analysis 2.15]
Analysis 2.15. Comparison 2 Agalsidase beta versus placebo, Outcome 15 Adverse event: Vomiting.
[Analysis 2.16]
Analysis 2.16. Comparison 2 Agalsidase beta versus placebo, Outcome 16 Adverse event: Chest pain.
[Analysis 2.17]
Analysis 2.17. Comparison 2 Agalsidase beta versus placebo, Outcome 17 Adverse event: Fatigue.
[Analysis 2.18]
Analysis 2.18. Comparison 2 Agalsidase beta versus placebo, Outcome 18 Adverse event: Headache.
[Analysis 2.19]
Analysis 2.19. Comparison 2 Agalsidase beta versus placebo, Outcome 19 Adverse event: Pain related to Fabry Disease.
[Analysis 3.1]
Analysis 3.1. Comparison 3 Agalsidase alfa versus agalsidase beta, Outcome 1 Death.
[Analysis 3.2]
Analysis 3.2. Comparison 3 Agalsidase alfa versus agalsidase beta, Outcome 2 Cardiac events.
[Analysis 3.3]
Analysis 3.3. Comparison 3 Agalsidase alfa versus agalsidase beta, Outcome 3 Any adverse event.
[Analysis 3.4]
Analysis 3.4. Comparison 3 Agalsidase alfa versus agalsidase beta, Outcome 4 Any serious adverse events.