Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg.
Two different enzyme preparations, agalsidase alfa (Replagal(TM), Shire) and beta (Fabrazyme(TM), Genzyme), are registered for treatment of Fabry disease. We compared the efficacy of and tolerability towards the two agalsidase preparations administered at identical protein dose in a randomized contr...
Main Authors: | , , , , , , , , |
---|---|
Format: | Article |
Language: | English |
Published: |
Public Library of Science (PLoS)
2007-07-01
|
Series: | PLoS ONE |
Online Access: | http://europepmc.org/articles/PMC1913555?pdf=render |
id |
doaj-7637ac20457243d8ad529605a618aae4 |
---|---|
record_format |
Article |
spelling |
doaj-7637ac20457243d8ad529605a618aae42020-11-24T20:50:08ZengPublic Library of Science (PLoS)PLoS ONE1932-62032007-07-0127e59810.1371/journal.pone.0000598Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg.Anouk C VedderGabor E LinthorstGunnar HougeJohannna E M GroenerEls E OrmelBerto J BoumaJohannes M F G AertsAsle HirthCarla E M HollakTwo different enzyme preparations, agalsidase alfa (Replagal(TM), Shire) and beta (Fabrazyme(TM), Genzyme), are registered for treatment of Fabry disease. We compared the efficacy of and tolerability towards the two agalsidase preparations administered at identical protein dose in a randomized controlled open label trial.Thirty-four Fabry disease patients were treated with either agalsidase alfa or agalsidase beta at equal dose of 0.2 mg/kg biweekly. Primary endpoint was reduction in left ventricular mass after 12 and 24 months of treatment. Other endpoints included occurrence of treatment failure (defined as progression of cardiac, renal or cerebral disease), glomerular filtration rate, pain, anti-agalsidase antibodies, and globotriaosylceramide levels in plasma and urine. After 12 and 24 months of treatment no reduction in left ventricular mass was seen, which was not different between the two treatment groups. Also, no differences in glomerular filtration rate, pain and decline in globotriaosylceramide levels were found. Antibodies developed only in males (4/8 in the agalsidase alfa group and 6/8 in the agalsidase beta group). Treatment failure within 24 months of therapy was seen in 8/34 patients: 6 male patients (3 in each treatment group) and 2 female patients (both agalsidase alfa). The occurrence of treatment failures did not differ between the two treatment groups; chi(2) = 0.38 p = 0.54.Our study revealed no difference in reduction of left ventricular mass or other disease parameters after 12 and 24 months of treatment with either agalsidase alfa or beta at a dose of 0.2 mg/kg biweekly. Treatment failure occurred frequently in both groups and seems related to age and severe pre-treatment disease.International Standard Randomized Clinical Trial ISRCTN45178534 [http://www.controlled-trials.com/ISRCTN45178534].http://europepmc.org/articles/PMC1913555?pdf=render |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Anouk C Vedder Gabor E Linthorst Gunnar Houge Johannna E M Groener Els E Ormel Berto J Bouma Johannes M F G Aerts Asle Hirth Carla E M Hollak |
spellingShingle |
Anouk C Vedder Gabor E Linthorst Gunnar Houge Johannna E M Groener Els E Ormel Berto J Bouma Johannes M F G Aerts Asle Hirth Carla E M Hollak Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. PLoS ONE |
author_facet |
Anouk C Vedder Gabor E Linthorst Gunnar Houge Johannna E M Groener Els E Ormel Berto J Bouma Johannes M F G Aerts Asle Hirth Carla E M Hollak |
author_sort |
Anouk C Vedder |
title |
Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. |
title_short |
Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. |
title_full |
Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. |
title_fullStr |
Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. |
title_full_unstemmed |
Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. |
title_sort |
treatment of fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. |
publisher |
Public Library of Science (PLoS) |
series |
PLoS ONE |
issn |
1932-6203 |
publishDate |
2007-07-01 |
description |
Two different enzyme preparations, agalsidase alfa (Replagal(TM), Shire) and beta (Fabrazyme(TM), Genzyme), are registered for treatment of Fabry disease. We compared the efficacy of and tolerability towards the two agalsidase preparations administered at identical protein dose in a randomized controlled open label trial.Thirty-four Fabry disease patients were treated with either agalsidase alfa or agalsidase beta at equal dose of 0.2 mg/kg biweekly. Primary endpoint was reduction in left ventricular mass after 12 and 24 months of treatment. Other endpoints included occurrence of treatment failure (defined as progression of cardiac, renal or cerebral disease), glomerular filtration rate, pain, anti-agalsidase antibodies, and globotriaosylceramide levels in plasma and urine. After 12 and 24 months of treatment no reduction in left ventricular mass was seen, which was not different between the two treatment groups. Also, no differences in glomerular filtration rate, pain and decline in globotriaosylceramide levels were found. Antibodies developed only in males (4/8 in the agalsidase alfa group and 6/8 in the agalsidase beta group). Treatment failure within 24 months of therapy was seen in 8/34 patients: 6 male patients (3 in each treatment group) and 2 female patients (both agalsidase alfa). The occurrence of treatment failures did not differ between the two treatment groups; chi(2) = 0.38 p = 0.54.Our study revealed no difference in reduction of left ventricular mass or other disease parameters after 12 and 24 months of treatment with either agalsidase alfa or beta at a dose of 0.2 mg/kg biweekly. Treatment failure occurred frequently in both groups and seems related to age and severe pre-treatment disease.International Standard Randomized Clinical Trial ISRCTN45178534 [http://www.controlled-trials.com/ISRCTN45178534]. |
url |
http://europepmc.org/articles/PMC1913555?pdf=render |
work_keys_str_mv |
AT anoukcvedder treatmentoffabrydiseaseoutcomeofacomparativetrialwithagalsidasealfaorbetaatadoseof02mgkg AT gaborelinthorst treatmentoffabrydiseaseoutcomeofacomparativetrialwithagalsidasealfaorbetaatadoseof02mgkg AT gunnarhouge treatmentoffabrydiseaseoutcomeofacomparativetrialwithagalsidasealfaorbetaatadoseof02mgkg AT johannnaemgroener treatmentoffabrydiseaseoutcomeofacomparativetrialwithagalsidasealfaorbetaatadoseof02mgkg AT elseormel treatmentoffabrydiseaseoutcomeofacomparativetrialwithagalsidasealfaorbetaatadoseof02mgkg AT bertojbouma treatmentoffabrydiseaseoutcomeofacomparativetrialwithagalsidasealfaorbetaatadoseof02mgkg AT johannesmfgaerts treatmentoffabrydiseaseoutcomeofacomparativetrialwithagalsidasealfaorbetaatadoseof02mgkg AT aslehirth treatmentoffabrydiseaseoutcomeofacomparativetrialwithagalsidasealfaorbetaatadoseof02mgkg AT carlaemhollak treatmentoffabrydiseaseoutcomeofacomparativetrialwithagalsidasealfaorbetaatadoseof02mgkg |
_version_ |
1716804515007561728 |