TY - JOUR
T1 - Enzyme replacement therapy for Fabry disease
T2 - A systematic review and meta-analysis
AU - Alegra, Taciane
AU - Vairo, Filippo
AU - de Souza, Monica V.
AU - Krug, Bárbara C.
AU - Schwartz, Ida V.D.
PY - 2012
Y1 - 2012
N2 - The specific treatment available for Fabry disease (FD) is enzyme replacement therapy (ERT) with agalsidase alfa or beta. A systematic review and meta-analysis was conducted to assess the efficacy and safety of ERT for FD. Only double-blind, randomized clinical trials (RCTs) comparing agalsidase alfa or beta and placebo were included. ERT with either agalsidase alfa or beta was considered similar for the purposes of analysis. Ten RCTs were identified, which showed improvements in neuropathic pain, in heart abnormalities and in globotriaosylceramide (GL-3) levels. A meta-analysis showed increased odds for fever, rigors, development of IgG antibodies to agalsidase, and no significant association with development of hypertension or reduction in the QRS complex duration on electrocardiogram. The RCTs included in this comparison enrolled few patients, were highly heterogeneous, and were focused mainly on surrogate endpoints, limiting any conclusions as to the real effect of ERT for FD. The available evidence suggests that response to ERT is variable across patient subgroups and that agalsidase may slow progression of FD, with slight improvement of existing changes. Nevertheless, many uncertainties remain, and further studies are necessary.
AB - The specific treatment available for Fabry disease (FD) is enzyme replacement therapy (ERT) with agalsidase alfa or beta. A systematic review and meta-analysis was conducted to assess the efficacy and safety of ERT for FD. Only double-blind, randomized clinical trials (RCTs) comparing agalsidase alfa or beta and placebo were included. ERT with either agalsidase alfa or beta was considered similar for the purposes of analysis. Ten RCTs were identified, which showed improvements in neuropathic pain, in heart abnormalities and in globotriaosylceramide (GL-3) levels. A meta-analysis showed increased odds for fever, rigors, development of IgG antibodies to agalsidase, and no significant association with development of hypertension or reduction in the QRS complex duration on electrocardiogram. The RCTs included in this comparison enrolled few patients, were highly heterogeneous, and were focused mainly on surrogate endpoints, limiting any conclusions as to the real effect of ERT for FD. The available evidence suggests that response to ERT is variable across patient subgroups and that agalsidase may slow progression of FD, with slight improvement of existing changes. Nevertheless, many uncertainties remain, and further studies are necessary.
KW - Alpha-Galactosidase
KW - Enzyme replacement therapy
KW - Fabry disease
KW - Fabry disease/therapy
UR - http://www.scopus.com/inward/record.url?scp=84872458046&partnerID=8YFLogxK
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U2 - 10.1590/S1415-47572012000600009
DO - 10.1590/S1415-47572012000600009
M3 - Review article
AN - SCOPUS:84872458046
SN - 1415-4757
VL - 35
SP - 947
EP - 954
JO - Genetics and Molecular Biology
JF - Genetics and Molecular Biology
IS - 4 SUPPL.
ER -