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Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies.
PLoS One. 2017; 12(3):e0173358.Plos

Abstract

BACKGROUND

Anderson-Fabry disease (AFD) is an X-linked recessive inborn error of glycosphingolipid metabolism caused by a deficiency of alpha-galactosidase A. Renal failure, heart and cerebrovascular involvement reduce survival. A Cochrane review provided little evidence on the use of enzyme replacement therapy (ERT). We now complement this review through a linear regression and a pooled analysis of proportions from cohort studies.

OBJECTIVES

To evaluate the efficacy and safety of ERT for AFD.

MATERIALS AND METHODS

For the systematic review, a literature search was performed, from inception to March 2016, using Medline, EMBASE and LILACS. Inclusion criteria were cohort studies, patients with AFD on ERT or natural history, and at least one patient-important outcome (all-cause mortality, renal, cardiovascular or cerebrovascular events, and adverse events) reported. The pooled proportion and the confidence interval (CI) are shown for each outcome. Simple linear regressions for composite endpoints were performed.

RESULTS

77 cohort studies involving 15,305 participants proved eligible. The pooled proportions were as follows: a) for renal complications, agalsidase alfa 15.3% [95% CI 0.048, 0.303; I2 = 77.2%, p = 0.0005]; agalsidase beta 6% [95% CI 0.04, 0.07; I2 = not applicable]; and untreated patients 21.4% [95% CI 0.1522, 0.2835; I2 = 89.6%, p<0.0001]. Effect differences favored agalsidase beta compared to untreated patients; b) for cardiovascular complications, agalsidase alfa 28% [95% CI 0.07, 0.55; I2 = 96.7%, p<0.0001]; agalsidase beta 7% [95% CI 0.05, 0.08; I2 = not applicable]; and untreated patients 26.2% [95% CI 0.149, 0.394; I2 = 98.8%, p<0.0001]. Effect differences favored agalsidase beta compared to untreated patients; and c) for cerebrovascular complications, agalsidase alfa 11.1% [95% CI 0.058, 0.179; I2 = 70.5%, p = 0.0024]; agalsidase beta 3.5% [95% CI 0.024, 0.046; I2 = 0%, p = 0.4209]; and untreated patients 18.3% [95% CI 0.129, 0.245; I2 = 95% p < 0.0001]. Effect differences favored agalsidase beta over agalsidase alfa or untreated patients. A linear regression showed that Fabry patients receiving agalsidase alfa are more likely to have higher rates of composite endpoints compared to those receiving agalsidase beta.

CONCLUSIONS

Agalsidase beta is associated to a significantly lower incidence of renal, cardiovascular and cerebrovascular events than no ERT, and to a significantly lower incidence of cerebrovascular events than agalsidase alfa. In view of these results, the use of agalsidase beta for preventing major organ complications related to AFD can be recommended.

Authors+Show Affiliations

Institute of Science and Technology, Unesp - Univ Estadual Paulista, São José dos Campos, Brazil. McMaster Institute of Urology, McMaster University, Hamilton, Canada.Department of Pharmacy, Tanta Chest Hospital, Tanta, Egypt.IIS-Fundacion Jimenez Diaz, Universidad Autonoma Madrid, Madrid, Spain.Neurology Service, Dr Nestor Chamoles Laboratory of Neurochemistry, Buenos Aires, Argentina.McMaster Institute of Urology, McMaster University, Hamilton, Canada.Department of Internal Medicine, Nephrology Service, Federal University of Paraná, Curitiba, Brazil.

Pub Type(s)

Journal Article
Review
Systematic Review

Language

eng

PubMed ID

28296917

Citation

El Dib, Regina, et al. "Enzyme Replacement Therapy for Anderson-Fabry Disease: a Complementary Overview of a Cochrane Publication Through a Linear Regression and a Pooled Analysis of Proportions From Cohort Studies." PloS One, vol. 12, no. 3, 2017, pp. e0173358.
El Dib R, Gomaa H, Ortiz A, et al. Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies. PLoS ONE. 2017;12(3):e0173358.
El Dib, R., Gomaa, H., Ortiz, A., Politei, J., Kapoor, A., & Barreto, F. (2017). Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies. PloS One, 12(3), e0173358. https://doi.org/10.1371/journal.pone.0173358
El Dib R, et al. Enzyme Replacement Therapy for Anderson-Fabry Disease: a Complementary Overview of a Cochrane Publication Through a Linear Regression and a Pooled Analysis of Proportions From Cohort Studies. PLoS ONE. 2017;12(3):e0173358. PubMed PMID: 28296917.
* Article titles in AMA citation format should be in sentence-case
TY - JOUR T1 - Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies. AU - El Dib,Regina, AU - Gomaa,Huda, AU - Ortiz,Alberto, AU - Politei,Juan, AU - Kapoor,Anil, AU - Barreto,Fellype, Y1 - 2017/03/15/ PY - 2016/09/24/received PY - 2017/02/19/accepted PY - 2017/3/16/entrez PY - 2017/3/16/pubmed PY - 2017/9/9/medline SP - e0173358 EP - e0173358 JF - PloS one JO - PLoS ONE VL - 12 IS - 3 N2 - BACKGROUND: Anderson-Fabry disease (AFD) is an X-linked recessive inborn error of glycosphingolipid metabolism caused by a deficiency of alpha-galactosidase A. Renal failure, heart and cerebrovascular involvement reduce survival. A Cochrane review provided little evidence on the use of enzyme replacement therapy (ERT). We now complement this review through a linear regression and a pooled analysis of proportions from cohort studies. OBJECTIVES: To evaluate the efficacy and safety of ERT for AFD. MATERIALS AND METHODS: For the systematic review, a literature search was performed, from inception to March 2016, using Medline, EMBASE and LILACS. Inclusion criteria were cohort studies, patients with AFD on ERT or natural history, and at least one patient-important outcome (all-cause mortality, renal, cardiovascular or cerebrovascular events, and adverse events) reported. The pooled proportion and the confidence interval (CI) are shown for each outcome. Simple linear regressions for composite endpoints were performed. RESULTS: 77 cohort studies involving 15,305 participants proved eligible. The pooled proportions were as follows: a) for renal complications, agalsidase alfa 15.3% [95% CI 0.048, 0.303; I2 = 77.2%, p = 0.0005]; agalsidase beta 6% [95% CI 0.04, 0.07; I2 = not applicable]; and untreated patients 21.4% [95% CI 0.1522, 0.2835; I2 = 89.6%, p<0.0001]. Effect differences favored agalsidase beta compared to untreated patients; b) for cardiovascular complications, agalsidase alfa 28% [95% CI 0.07, 0.55; I2 = 96.7%, p<0.0001]; agalsidase beta 7% [95% CI 0.05, 0.08; I2 = not applicable]; and untreated patients 26.2% [95% CI 0.149, 0.394; I2 = 98.8%, p<0.0001]. Effect differences favored agalsidase beta compared to untreated patients; and c) for cerebrovascular complications, agalsidase alfa 11.1% [95% CI 0.058, 0.179; I2 = 70.5%, p = 0.0024]; agalsidase beta 3.5% [95% CI 0.024, 0.046; I2 = 0%, p = 0.4209]; and untreated patients 18.3% [95% CI 0.129, 0.245; I2 = 95% p < 0.0001]. Effect differences favored agalsidase beta over agalsidase alfa or untreated patients. A linear regression showed that Fabry patients receiving agalsidase alfa are more likely to have higher rates of composite endpoints compared to those receiving agalsidase beta. CONCLUSIONS: Agalsidase beta is associated to a significantly lower incidence of renal, cardiovascular and cerebrovascular events than no ERT, and to a significantly lower incidence of cerebrovascular events than agalsidase alfa. In view of these results, the use of agalsidase beta for preventing major organ complications related to AFD can be recommended. SN - 1932-6203 UR - https://www.unboundmedicine.com/medline/citation/28296917/Enzyme_replacement_therapy_for_Anderson_Fabry_disease:_A_complementary_overview_of_a_Cochrane_publication_through_a_linear_regression_and_a_pooled_analysis_of_proportions_from_cohort_studies_ L2 - http://dx.plos.org/10.1371/journal.pone.0173358 DB - PRIME DP - Unbound Medicine ER -