Switch to agalsidase alfa after shortage of agalsidase beta in Fabry disease: a systematic review and meta-analysis of the literature.
In 2009, the agalsidase beta shortage resulted in switching to agalsidase alfa treatment for many Fabry disease patients, offering the unique opportunity to compare the effects of the two drugs. Because single studies describing effects of switching on the disease course are limited and inconclusive, we performed a systematic review and meta-analysis of existing data.



Relevant studies were identified in the PubMed, Cochrane, ISI Web, and SCOPUS databases from July 2009 to September 2015. The following parameters were analyzed: clinical events, changes in organ function or structure, disease-related symptoms, lyso-Gb3 plasma levels, and adverse effects.



The nine studies (217 patients) included in our systematic review showed only marginal differences in most of the evaluated parameters. Seven of these studies were included in the meta-analysis (176 patients). The pooled incidence rate of major adverse events was reported for five studies (150 patients) and was equal to 0.04 events per person-year. No significant change was observed after the shift in glomerular filtration rate, whereas left ventricular mass index, left ventricular posterior wall dimension, and ejection fraction were significantly reduced over time. Our data showed that the switch to agalsidase alfa was well tolerated and associated with stable clinical conditions.Genet Med 19 3, 275-282.
Nephrology Section, Department of Public Health, University Federico II of Naples, Naples, Italy.
Antonio
A
Pisani
Statistics Section, Department of Public Health, University Federico II of Naples, Naples, Italy.
Dario
D
Bruzzese
Nephrology Section, Department of Public Health, University Federico II of Naples, Naples, Italy.
Massimo
0000-0003-1339-9228
M
Sabbatini
Department of Advanced Biomedical Sciences, Federico II University, Naples, Italy.
Letizia
0000-0002-9774-6268
L
Spinelli
Department of Radiology, Federico II University of Naples, Naples, Italy.
Massimo
0000-0001-5262-5430
M
Imbriaco
Department of Nephrology, Second University of Naples, Naples, Italy.
Eleonora
E
Riccio
D018450:Disease Progression; D056947:Enzyme Replacement Therapy / Q000379:methods; D000795:Fabry Disease / Q000188:drug therapy* / Q000378:metabolism; D005260:Female; D005919:Glomerular Filtration Rate / Q000187:drug effects; D006017:Glycolipids / Q000378:metabolism / Q000494:pharmacology; D006801:Humans; D007527:Isoenzymes / Q000235:genetics / Q000378:metabolism / Q000494:pharmacology*; D008297:Male; D011994:Recombinant Proteins; D013107:Sphingolipids / Q000378:metabolism / Q000494:pharmacology; D016896:Treatment Outcome; D000519:alpha-Galactosidase / Q000235:genetics / Q000378:metabolism / Q000494:pharmacology*
D006017:Glycolipids; D007527:Isoenzymes; D011994:Recombinant Proteins; D013107:Sphingolipids; C063288:globotriaosyl lysosphingolipid; C000627036:agalsidase alfa; D000519:alpha-Galactosidase; C459420:agalsidase beta